Scientists at the medical school are conducting hundreds of clinical trials daily. Learn more about all our work via the Feinberg Office of Research Clinical Trials page.
Our current active protocols are listed below, searchable by disease or condition. If you have any questions about the particular study protocol, please contact the study coordinator. For more information about the research or participation in general, please call 312-695-8643.
The Parkinson’s Progression Markers Initiative (PPMI)
This is a observational, multi-center study to assess progression of clinical features, imaging and biologic biomarkers in Parkinson disease (PD) patients compared to healthy controls (HC) and in PD patient subtypes. …
This is a observational, multi-center study to assess progression of clinical features, imaging and biologic biomarkers in Parkinson disease (PD) patients compared to healthy controls (HC) and in PD patient subtypes. The primary objective of this study is to identify clinical, imaging and biologic markers of PD progression for use in clinical trials of disease-modifying therapies.
Genetic Cohort-PD Subjects
• Have at least two of the following: resting tremor, slowness of movement, muscle rigidity
• Parkinson disease diagnosis for 7 years or less
• Male or female 18 years or older
• Confirmation of LRRK2, GBA, or SNCA genetic mutation
• Willing to undergo genetic testing
Genetic Cohort-Unaffected Subjects
• 50 years or older with LRRK2/GBA mutation or first degree relative with LRRK2/GBA mutation
• Willing to undergo genetic testing
• 30 years or older with SNCA mutation or first degree relative with SNCA mutation
• Willing to undergo genetic testing
Peripheral Neuropathy Research Registry (PNRR)
National Peripheral Neuropathy Research Registry, a collection of different types of information, such as patient medical, family, and social histories and blood samples. The information is carefully maintained so that it can be studied repeatedly in t…
National Peripheral Neuropathy Research Registry, a collection of different types of information, such as patient medical, family, and social histories and blood samples. The information is carefully maintained so that it can be studied repeatedly in the future. The registry aims to help researchers’ access large amounts of information about people with PN. By using this registry, researchers will facilitate both basic and clinical research studies that will bring improved understandings of the etiology (origination) and pathogenesis (development) of PN. They will specifically ask why some patients with peripheral neuropathy develop neuropathic pain and others do not, and what the characteristics of patients with painful peripheral neuropathy are in terms of their symptoms, examination findings, and blood tests. Ultimately this research may result in improved diagnosis, more effective treatments, and possibly prevention.
Inclusion criteria: 1. Diabetic Peripheral Neuropathy 2. Chemo-therapy Induced Peripheral Neuropathy 3. HIV-induced Peripheral Neuropathy 4. Idiopathic Peripheral Neuropathy; Exclusion criteria: Any other type of Peripheral Neuropathy
Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD)
The purpose of this study is to observe mothers with and without epilepsy, along with their children, from pregnancy until age 6 and assess the developmental and neurocognitive influence of anti-epileptic medications.
Non-Invasive Marker of Ovarian Reserve in Women with Epilepsy
The purpose of this study is to assess and compare AMH hormone levels in women with epilepsy vs. healthy controls, utilizing medical history questionnaires and a finger stick.
NUDB 13C03: Northwestern Brain Tumor Institute Research Database
The Northwestern Brain Tumor Institute (NBTI) currently uses an electronic database to collect and store information about patients who come to the NBTI for evaluations, including diagnosis, treatment, follow-up, and/or to obtain add…
The Northwestern Brain Tumor Institute (NBTI) currently uses an electronic database to collect and store information about patients who come to the NBTI for evaluations, including diagnosis, treatment, follow-up, and/or to obtain additional opinions. This database is called the Northwestern Brain Tumor Institute Database or NBTIDB, and it was developed to replace older paper methods for collecting and storing information.
The purpose of this study is to allow researchers involved with the NBTIDB to use data stored in it for future research studies and projects. The NBTIDB also allows researchers to track whether or not patients have agreed to allow their information to be linked to their leftover tissue samples, which are kept in the hospital’s pathology department, for future research studies.
You may be eligible to take part in the research component of the NBTIDB if you are either a new or returning patient, over the age of 18, who is being seen by one of the clinicians at the NBTI and are or will be entered into the NBTIDB, or a patient who is not coming to the NBTI for evaluation, but would still like to participate in the NBTIDB.
Parkinson’s Disease and Movement Disorders Center Biorepository
This is a registry aimed to collect biologic and clinical information, such as blood and tissue samples, and family and medical histories from patients diagnosed with a movement disorder. The purpose of studying materials from the regi…
This is a registry aimed to collect biologic and clinical information, such as blood and tissue samples, and family and medical histories from patients diagnosed with a movement disorder. The purpose of studying materials from the registry is to identify factors that either cause these neurologic conditions or increase one’s risk for developing them. Samples collected for this biorepository include a blood sample (or a saliva sample) and a skin biopsy. Participants may choose to donate one or both samples.
• Diagnosis of a movement disorder
• Male or female 5 years of age or older when diagnosed
• Genetic mutation related to a movement disorder
• Family members of patients with movement disorders
rTMS: A Treatment to Restore Function after Severe TBI
The purpose of this study is to address the need for targeted treatments that induce functional and structural changes in the brain, ultimately improving neurobehavioral functioning, the investigators propose examining the therapeutic effectiven…
The purpose of this study is to address the need for targeted treatments that induce functional and structural changes in the brain, ultimately improving neurobehavioral functioning, the investigators propose examining the therapeutic effectiveness of repetitive Transcranial Magnetic Stimulation (rTMS). The objective is to improve functional recovery for persons remaining in vegetative (VS) and minimally conscious (MCS) states 3 to 12 months after severe TBI. The approach is to determine the neurobehavioral effect of rTMS, the relationship between neurobehavioral changes and net neural effects, and to identify and define the neural mechanisms related to neurobehavioral improvements by providing 30 active or placebo rTMS sessions.
-Veteran of any combat era
-History of Post Traumatic Amnesia < 1 day for mild TBI; 1 day> x < 7days for moderate TBI))
-Ability to obtain a Motor Threshold (MT) will be determined during the screening process.
-If on a psychotropic medication regimen, that regimen will be stable for at least 4 weeks prior to entry to the study and patient will be willing to remain on a stable regimen during the acute treatment phase.
-Has an adequately stable condition and environment to enable attendance at scheduled clinic visits.
-For female participants, agrees to use one of the following acceptable methods of birth control: abstinence, oral contraceptive; Norplant
-Able to read, verbalize understanding, and voluntarily sign the Informed Consent Form prior to participating in any study-specific procedures or assessments.
-Pregnant or lactating female.
-Unable to be safely withdraw, at least two-weeks prior to treatment commencement, from medications that substantially increase the risk of having seizures
-Have a cardiac pacemaker or a cochlear implant
-Have an implanted device (deep brain stimulation) or metal in the brain (see standard MRI exclusion criteria including metal screening section in telephone screen, Appendix A).
-Have a mass lesion, cerebral infarct or other active central nervous system (CNS) disease, including a seizure disorder.
-Known current psychosis as determined by DSM-IV coding in chart (Axis I, psychotic disorder, schizophrenia) or a history of a non-mood psychotic disorder.
-Diagnosis of Bipolar Affective Disorder (as determined by chart review and intake interview)
-Current amnesic disorders, dementia, mini mental state examination (MMSE) 24 or delirium.
-Current substance abuse (not including caffeine or nicotine) as determined by positive toxicology screen, or by history via AUDIT, within 3 months prior to screening
-Prior history of seizures
-Severe TBI or open head injury
-TBI within last two months or in acute stage
-Participation in another concurrent clinical trial
-Patients with prior exposure to rTMS/ECT
-Active current suicidal intent or plan. Patient at risk for suicide will be required to establish a written safety plan involving their primary psychiatrist and the treatment team before entering the clinical trial
Carotid Revascularization and Medical Management for Asymptomatic Carotid Stenosis Trial (CREST-2) P
Carotid revascularization for primary prevention of stroke (CREST-2) is a multicenter, randomized controlled trials of carotid revascularization and intensive medical management versus medical managem…
Carotid revascularization for primary prevention of stroke (CREST-2) is a multicenter, randomized controlled trials of carotid revascularization and intensive medical management versus medical management alone in patients with asymptomatic high-grade carotid stenosis. One trial will randomize patients in a 1:1 ratio to endarterectomy versus no endarterectomy and another will randomize patients in a 1:1 ratio to carotid stenting with embolic protection versus no stenting. Medical management will be uniform for all randomized treatment groups and will be centrally directed.
Alliance A071401: Phase II Trial Of SMO/AKT/NF2 Inhibitors in Progressive Meningiomas with SMO/AKT/NF2 Mutations
This phase II trial studies how well two drugs, vismodegib and GSK2256098, work in treating patients with meningiomas that may have gotten bigger or grew back after treatment. Vismodegi…
This phase II trial studies how well two drugs, vismodegib and GSK2256098, work in treating patients with meningiomas that may have gotten bigger or grew back after treatment. Vismodegib and GSK2256098 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
The purpose of this study is to test good and bad effects of these two different drugs against meningioma tumors with altered (or mutated) genes. Altered genes can cause a tumor to grow. The study drugs, vismodegib and GSK2256098, target these genes. The study drugs could shrink the cancer, or the cancer could stay the same size or grow. They may cause side effects. Researchers hope to learn if the study drugs will shrink the cancer by at least one-half compared to its present size.
Today, therapy for meningioma is the same for all patients, and is not based on tumor genetic testing. This trial is trying to see if tumor genetic testing would be helpful at guiding treatment in meningioma patients.
Evaluating the Circadian Response to Light in Delayed Sleep-Wake Phase Disorder
This study is looking at factors that contribute to the timing of sleep. Interested subjects will undergo screening to determine their sleep-wake habits, then will have an eye test and a blood draw. Participation will i…
This study is looking at factors that contribute to the timing of sleep. Interested subjects will undergo screening to determine their sleep-wake habits, then will have an eye test and a blood draw. Participation will involve 2 outpatient visits, separated by up to 3 weeks of sleep-wake activity monitoring.
Individuals with delayed sleep-wake phase disorder and healthy controls
A wearable myoelectric-computer interface to reduce muscle co-activation in acute and chronic stroke
We are conducting a study investigating the use of small wearable devices, called myoelectric computer interfaces, to reduce abnormal arm muscle coordination in individuals with impaired arm movement …
We are conducting a study investigating the use of small wearable devices, called myoelectric computer interfaces, to reduce abnormal arm muscle coordination in individuals with impaired arm movement from a stroke. Training will take place predominantly at home, with some sessions in the lab as well. This study could potentially lead to improved arm function for stroke survivors who have abnormal arm coordination.
At least 18 years of age
- Had a stroke more than 6 months ago
- No large impairment in vision (glasses), memory, language or concentration
- Not currently participating in another research study on the arm
Enroll-HD: A Prospective Registry Study in a Global Huntington’s Disease Cohort
The purpose of this research study is to collect clinical information about patients and their health. We will also collect biological samples, such as blood and DNA (the genetic material in your blood). Researchers wil…
The purpose of this research study is to collect clinical information about patients and their health. We will also collect biological samples, such as blood and DNA (the genetic material in your blood). Researchers will use this information and samples to learn more about HD and to try to find new treatments for the disease. People from many countries contribute to Enroll-HD.
Individuals 18 yrs or older affected by Huntington's Disease (HD) or from a HD family or are a "community control" (a person who does not carry the HD genetic mutation that causes Huntington's disease and is not part of an HD family, but would like to participate in the study). Research visits are conducted yearly and will consists of a collection of medical and family history and biological samples.
The Role of Circadian Dysfunction in Hepatic Encephalopathy in Patients with Cirrhosis
Individuals with advanced liver disease (cirrhosis) often report new or worsening sleep problems.
1) Diagnosis of end-stage liver disease or cirrhosis; 2) being evaluated for liver transplant; 3) Age >=18yo; 4) no severe kidney disease (for example, patients currently on dialysis are not eligible)
Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) to Study Natural History and Genetic Modifiers in Spinocerebellar Ataxia (SCA)
The purpose of this study is to collect natural history data among ataxia patients in a cohort of SCA 1, 2, 3, 6 patients using clinic-based neurolo…
The purpose of this study is to collect natural history data among ataxia patients in a cohort of SCA 1, 2, 3, 6 patients using clinic-based neurological rating scales and performance measures. Another study goal is to identify new measures of ataxia, cognition, and neurobehavior in comparison to traditional clinician rating methods. The gene analysis is expected to establish a relationship, if any, between age at onset of disease and disease progression rates.
• Age 18 and older
• Presence of symptoms and signs of ataxia
• Molecular diagnosis of SCA 1, 2, 3, 6 either in the participant or an affected family member
• Willingness to participate in the study and ability to give informed consent.
A Randomized, Double-Blind, Placebo-Controlled, 52-Week Phase II Study to Evaluate the Efficacy of Intravenous RO7046015/Prasinezumab (PRX002) in Participants with Early Parkinson’s Disease with a 6-Year All-Participants-On-Treatment Extension (PASADENA)
This is a multicenter, Phase II study to eva…
This is a multicenter, Phase II study to evaluate the effect of IV administration of RO7046015 in participants with early stage Parkinson's Disease (PD). Participants will be eligible if they have PD with bradykinesia plus one of the other cardinal signs of PD (resting tremor, rigidity) being present, without any other known or suspected cause of PD and are either untreated or treated with Azilect or Selegiline. The study will consist of two parts: a 52-week, treatment period of the study medication vs placebo (Part 1) after which eligible participants will continue into an all-participants-on-treatment (RO7046015) blinded to dose extension for an additional 52 weeks (Part 2).
*Men and women, aged 40 to 80 years inclusive, early PD , who were recently (< 2 years) diagnosed, and either untreated or treated with Azilect or Selegiline
Genetic causes and pathogenic mechanisms of adult epilepsies
The purpose of this study is to look at genetic markers of epilepsy in patients and their families using blood, saliva, skin, and brain tissue analysis.
cIRB: Topiramate as a disease altering therapy for Cryptogenic Sensory Peripheral Neuropathy (CSPN)
This is a 96-week placebo-controlled trial of topiramate at a target dose of 100 mg daily (50 mg twice daily) as a potentially disease altering therapy for Cryptogenic Sensory Peripheral Neuropathy
INCLUSION: 1. Age 18-75; 2. Diagnosis of confirmed cryptogenic symptomatic distal symmetric peripheral polyneuropathy; 3. Prediabetes based on American Diabetes Association; 4. No history of prior therapy with topiramate; 5. Waist circumference >102 cm for men, >88 cm for women
Microburst VNS Therapy Feasibility Study in Subjects with Refractory Epilepsy
The purpose of this study is for patients with first-time implants of a Vagus Nerve Stimulator (VNS) to have the new Microburst stimulation settings optimized using fMRI scans.
cIRB: AtRial Cardiopathy and Antithrombotic Drugs In prevention After Crypotgenic Stroke
ARCADIA is a multicenter, biomarker-driven, randomized, double-blind, active-control, phase 3 clinical trial of apixaban versus aspirin in patients who have evidence of atrial cardiopathy and a recent stroke of u…
ARCADIA is a multicenter, biomarker-driven, randomized, double-blind, active-control, phase 3 clinical trial of apixaban versus aspirin in patients who have evidence of atrial cardiopathy and a recent stroke of unknown cause. Eleven hundred subjects will be recruited over 2.5 years at 120 sites in the NINDS StrokeNet consortium. Subjects will be followed for a minimum of 1.5 years and a maximum of 4 years for the primary efficacy outcome of recurrent stroke and the primary safety outcomes of symptomatic intracranial hemorrhage and major hemorrhage other than intracranial hemorrhage.
1) Age ≥ 45 years.
2) Clinical diagnosis of ischemic stroke + brain imaging to rule out hemorrhagic stroke.
3) Modified Rankin Scale (MRS) score ≤ 4.
4) Ability to be randomized within 3 to 120 days after stroke onset.
5) ESUS, defined as all of the following:
Stroke detected by CT or MRI that is not lacunar. Lacunar is defined as a subcortical (this includes pons and midbrain) infarct in the distribution of the small, penetrating cerebral arteries whose largest dimension is ≤1.5 cm on CT or ≤2.0 cm on MRI diffusion images/
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study, with an Active-Treatment Dose-Blinded Period, to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of BIIB054 in Subjects with Parkinson’s Disease (Protocol #228PD201)
The primary objective of the study is to eva…
The primary objective of the study is to evaluate the clinical efficacy of BIIB054 via dose response using the change from baseline in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score.
Diagnosed with Parkinson's disease (PD) within a maximum of 3 years prior to Screening.
Score of ≤2.5 on the Modified Hoehn and Yahr Scale.
Has not received any medication for the treatment of the motor symptoms of PD for at least 12 weeks prior to Day 1 and, in the opinion of the Investigator, is not expected to require PD treatment for at least 6 months following Day 1.
Clinical Trial Readiness for SCA1 and SCA3
Subjects must be 18-65 years of age with symptoms of ataxia or a diagnosis of SCA1 or SCA3 or have an affected family member. Subjects with a previous diagnosis of early stage SCA1 or SCA 3 may also be eligible. Subject must be able to undergo a MRI (brain…
Subjects must be 18-65 years of age with symptoms of ataxia or a diagnosis of SCA1 or SCA3 or have an affected family member. Subjects with a previous diagnosis of early stage SCA1 or SCA 3 may also be eligible. Subject must be able to undergo a MRI (brain scan) and not weigh over 300 lbs.
Subjects aged 18 to 65 with presence of symptomatic ataxic disease or asymptomatic mutation carrier or subjects with definite molecular diagnosis of SCA1 or SCA3 or another affected family member or Subjects of age >18 with previous diagnosis of early stage SCA1 and SCA3. Subjects must not make changes in physical/occupational therapy status within two months prior to enrollment. Subjects must not weigh over 300 lbs.
5) 6) No changes in sical/occupational therapy status within two months prior to enrolment
A Phase 3, Randomized, Rater-Blinded, Multi-Center Study to Evaluate the Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Patients with Wilson Disease Aged 12 Years and Older with an Extension Period of Up to 60 Months
The primary objective of this study is to eval…
The primary objective of this study is to evaluate the efficacy of the drug WTX101 administered for 48 weeks, compared to standard of care (SoC), on copper (Cu) control in subjects with Wilson's disease aged 18 and older.
Diagnosis of Wilson's Disease, Treatment for >28 days with chelation therapy, Zn therapy or a combination of chelator and Zn; willing to avoid the use of vitamins and/or minerals containing CU, Zn or Mo throughout the study, willing to undergo > 48-hour washout from current WD treatment
Gut Microbial remodeling with Resistant Maltodextrin for motor and non-motor symptoms in Parkinson's disease: safety and tolerability study.
The objective of this study is to examine thesafety and tolerability of resistant maltodextrin (RM), a prebioticnon-digestible fiber, and its effect on the gut …
The objective of this study is to examine thesafety and tolerability of resistant maltodextrin (RM), a prebioticnon-digestible fiber, and its effect on the gut microbiome, as well as motor and non-motorsymptoms, in Parkinson's Disease.
We will conduct a randomized, parallel-groupdouble-blinded controlled trial assessing resistant maltodextrin to placebo. You will be randomized (like a coin flip) toreceive either resistant maltodextrin or maltodextrin to take once a day in themorning for 4 weeks.
Participation in this research study lasts 6-7 weeks and includes 3 in-person visits and 4 phone assessments.
You may be eligible if you:
Radicava® (Edaravone) Biomarker Study in Participants with Amyotrophic Lateral Sclerosis
REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and p…
REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and protein biomarkers will also be investigated.
Location of study: Les Turner ALS Center at Northwestern Medicine, 259 E. Erie St., Lavin 19, Chicago, IL 60611.
A Randomized Controlled Study to Compare the Safety and Efficacy of IPX203 with Immediate-Release Carbidopa-Levodopa in Parkinson's Disease Patients with Motor Fluctuations (Protocol #: IPX203-B16-02)
The study will consist of a 3-week, open-label IR CD-LD dose adjustment period; a 4-week, open-label…
The study will consist of a 3-week, open-label IR CD-LD dose adjustment period; a 4-week, open-label period for conversion to IPX203; followed by a 13-week double-blind treatment period with subjects randomized in a 1:1 ratio, to receive either IPX203 (with matching IR CD-LD placebo) or IR CD-LD (with matching IPX203 placebo). The purpose is to evaluate the safety and efficacy of IPX203 (carbidopa and levodopa) extended-release capsules (IPX203 ER CD-LD) in comparison to immediate release (IR) CD-LD in the treatment of CD-LD-experienced subjects with Parkinson's disease (PD) who have motor fluctuations.
Subjects with Parkinson's disease (PD) who have motor fluctuations and experience with carbidopa-levodopa medication.
Natural history study of ALS and other motor neuron disorders
Location of study: Les Turner ALS Center at Northwestern Medicine, 259 E. Erie St., Lavin 19, Chicago, IL 60611.
A Phase 1 Single- and Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB094 Administered Intrathecally to Adults With Parkinson’s Disease (Protocol# 254PD101)
The purpose of study is to determine whether BIIB094 may improve PD symptoms in subjects with or…
The purpose of study is to determine whether BIIB094 may improve PD symptoms in subjects with or without changes in the LRRK2 gene. The study medication will be given as an injection into your back near the spinal cord. This iscalled an “intrathecal” injection.
2. Diagnosis of PD w/in 7yrs without motor fluctuationsor dyskinesia.
3. Not on any medication for PD or on stable therapy for 8weeks prior to screening.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated with Huntington Disease
The purpose of this research study is to evaluate the effectiveness of valbenazine to reduce chorea associated w…
The purpose of this research study is to evaluate the effectiveness of valbenazine to reduce chorea associated with Huntington disease (HD) and the safety and tolerability of valbenazine.
• Age 18 to 75 years of age.
• Diagnosis of motor manifest HD
• Genetic diagnosis of HD with CAG repeat ≥ 37
An Open-Label Extension Study of the Safety and Clinical Utility of IPX203 in Parkinson’s Disease Patients with Motor Fluctuations (IPX203-B16-03)
This is a 9-month, multicenter open-label safety extension study. The purpose of this study is to evaluate the long-term safety and clinical utility…
This is a 9-month, multicenter open-label safety extension study. The purpose of this study is to evaluate the long-term safety and clinical utility of IPX203 in the treatment of subjects with advanced Parkinson's disease (PD) who have motor fluctuations.
Subjects who have successfully completed Study IPX203-B16-02 [A Randomized Controlled Study to Compare the Safety and Efficacy of IPX203 with Immediate-Release (IR) Carbidopa-Levodopa (CD-LD) in Parkinson's Disease Patients with Motor Fluctuations] may have the opportunity to enroll in this open-label study.
Oxidative Markers and Efficacy in ALS/MND Phenotypes Treated with Edaravone
We are only recruiting patients who have not started their edaravone treatment.
Location of study: Les Turner ALS Center at Northwestern Medicine, 259 E. Erie St., Lavin 19, Chicago, IL 60611.
Either possible, probable, or definite ALS,predominantly lower motor neuron disease Predominantly upper motor neuron disease, orbulbar
With or without cognitive involvement
Willing to participate
On no experimental treatment
Ages 18 - 85
No prior exposure to Radicava
On a stable dose of riluzole for 30 days or offriluzole
Male or female
Females of childbearing age must usecontraception
Unstable medical illness
Abnormal liver function (>2x ULN)
Unlikely to survive for at least 26 weeks
A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of PR001A in Patients with Parkinson’s Disease with at Least One GBA1 Mutation (PROPEL, Protocol #: PRV-PD101)
PR001A is an investigational gene therapy product that is being developed for the treatment of PD in patient…
PR001A is an investigational gene therapy product that is being developed for the treatment of PD in patients with GBA1 mutations. The purpose of this study is to find out what effects PR001A has on Parkinson’s disease patients. Participants will be assigned to receive one dose of PR001A by injection into the cisterna magna (a large space at the base of the brain).
1. 40-75 years of age.
2. Diagnosis of PD with H&Y 3-4.
3. On stable PD therapy for 8 weeks prior to baseline.
4. At least 1 GBA gene mutation.
Platform Trial for the Treatment of Amyotrophic Lateral Sclerosis (ALS): A perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
In this trial, multiple investigational products for ALS will be tested simul…
In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen.
The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.
Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo.
The following regimens are active in the trial:
Regimen A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8
New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.
Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 36 Weeks of Treatment with NLY01 in Early-stage Parkinson’s Disease
The purpose of study is to determine if NLY01 helps lessen symptoms and signs of early Parkinson’s disease and …
The purpose of study is to determine if NLY01 helps lessen symptoms and signs of early Parkinson’s disease and whether it slows the progression of Parkinson’s disease.
1. Subjects aged 30-80 yrs of age
2. Lessthan 5 yrs of PD symptom onset
3. Must not be taking any medication for PD treatment
A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation
This is a randomized, double-blind, placebo-controlled, 3-part dose escalation stud…
This is a randomized, double-blind, placebo-controlled, 3-part dose escalation study to examine the efficacy, safety, tolerability of BIIB067, administered by intrathecal bolus injection to up to approximately 183 adult subjects with ALS and confirmed SOD1 mutation.Northwestern is only participating in Part C of this study.The primary objective of Part C of this study is to evaluate the clinical efficacy of BIIB067 administered to adult subjects with ALS, who are fast-progressing, and confirmed SOD1 mutation.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients with Early Stage of Huntington Disease
The purpose of this study is to evaluate the safety and effectiveness of the study drug, pridopidine,on everyda…
The purpose of this study is to evaluate the safety and effectiveness of the study drug, pridopidine,on everyday functioning and daily activities, as well as movement and behaviorin participants with early stage Huntington Disease (HD)
1. >25yrs of age.
2. Diagnosis of HD with CAG repeat > 36
A Randomized, Double-blind, Placebo-Controlled, 2-Period Crossover, Phase 2 Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Oral TAK-071 in Parkinson Disease Patients With Cognitive Impairment and an Elevated Risk of Falls
TAK-071 is an investigational …
TAK-071 is an investigational drug that is being studied as a possible treatment for people withParkinson’s Disease who have a history of falls.
1.Subjects aged 40 to 75years who have a clinical diagnosis of PD.
2.Study partner supervisingand assisting with medication administration.
3.On stable medication for atleast 4 weeks.
4. At least 2 falls in the last 6 monthsprior to screening.
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects with Wilson Disease
Given the limited information on the frequency and spectrum of disease manifestations and clinical course of Wilson disease, this study aims to assess the utility and feasibility of various assessments…
Given the limited information on the frequency and spectrum of disease manifestations and clinical course of Wilson disease, this study aims to assess the utility and feasibility of various assessments and biomarkers to inform endpoint selection for future clinical studies, better understand the relationship between biomarkers and potential clinical outcomes, and characterize the clinical presentation of Wilson disease.
UX701-CL001 is a clinical survey study for subjects with a confirmed diagnosis of Wilson disease (Figure 1). Subjects will complete assessments at the study site and at home to evaluate the clinical manifestations of Wilson disease in clinical and real-world environments. The study includes general assessments; patient- and clinician-reported outcome (PRO and CRO)