CURRENT RECRUITING TRIALS

ATAXIAS

No recruiting studies at this time.

HUNTINGTON'S DISEASE

HOFFMAN LAROCHE- WE45491

Frequency of Selected Single Nucleotide Polymorphisms in Phase With the Mutant and Wild-Type HTT Alleles in Huntington Disease Gene Expansion Carriers

For participation in this epidemiological study, a single-day visit at the study site is required. Participants will be recruited from Huntington Disease clinics, and they will be asked to answer questions regarding their demographics, including sex, age, race and ethnicity, and their medical and medication history. At the end of the visit, a blood sample will be drawn to allow testing with a sequencing assay that is specifically designed for phasing single nucleotide polymorphisms (SNPs) on the wild-type Huntington (wtHTT) and mutant Huntington (mHTT) alleles.

 

PARKINSON'S DISEASE

 REGENERON ALN-SNCA-PD-2482

First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson’s Disease (PD)

This study is researching an experimental drug called ALN-SNCA (called “study drug”). The study is focused on people with early Parkinson’s disease, a disorder of the nervous system that affects movement. Parkinson’s disease is caused by a gradual loss of nerve cells in the brain, especially those due to the harmful build-up of a protein called α-synuclein.

The aim of the study is to see if the study drug is safe and tolerated well enough to continue testing it in future studies and what side effects may happen from taking the study drug.

The study is looking at several other research questions, including:

  • Whether the study drug can lower the level of α-synuclein protein in the Cerebrospinal Fluid (also referred to as “CSF”, the fluid that surrounds the brain and spinal cord)
  • How much study drug is in the blood, urine, and CSF at different times
  • Compatible research to better understand the study drug (ALN-SNCA) and Parkinson’s disease, including (but not limited to), whether the study drug can slow down the progression of Parkinson’s disease symptoms

ROCHE PARAISO

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of prasinezumab compared with placebo in participants with early-stage Parkinson’s disease (PD) on stable symptomatic monotherapy with levodopa.

PARKINSON’S PROGRESSION MARKERS INITIATIVE (PPMI-2)

The Parkinson’s Progression Markers Initiative (PPMI) 2.0 Clinical – Establishing a Deeply Phenotyped PD Cohort

The purpose of this study is to obtain information from people with and without Parkinson disease (PD) so that researchers may better understand how Parkinson disease progresses, in order to inform better treatments.

This major international study was funded by the Michael J Fox Foundation. For more information on PPMI, click here.

PRION DISEASES

No recruiting studies at this time.

UPCOMING TRIALS

ATAXIAS

ACT DCS (Friedreich’s Ataxia)

Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia  

Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been demonstrated to modulate cerebellar excitability and improve symptoms in patients with cerebellar ataxias. In this randomized, double-blind, placebo-controlled study, the investigators will evaluate whether a two-weeks’ treatment with cerebellar anodal tDCS and spinal cathodal tDCS can improve symptoms in patients with neurodegenerative cerebellar ataxia and can modulate cerebello-motor connectivity, at short and long term.

HUNTINGTON'S DISEASE

HOFFMAN LAROCHE BP45378 POINT-HD

A Phase I, 2-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-ascending Doses of Intrathecally Administered RG6496 in a Randomized, Placebo-controlled, Investigator/Participant-blind Study With an Open-label Extension in Huntington’s Disease Gene Expansion Carriers 

This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington’s disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 [single-ascending dose] followed by Part 2 [open-label extension (OLE)].
 
PARKINSON'S DISEASE

PARKINSON’S FOUNDATION – PD GENEration PF

Development of a central repository for PD-related genomic data for future research.

  • PD GENEration: Powered by the Parkinson’s Foundation is a global research initiative offering free genetic testing and counseling for people living with Parkinson’s disease (PD). 

  • Participants can enroll online or in-person. 

  • Participants receive their genetic results and contribute to advancing understanding of the biological and genetic factors behind Parkinson’s. 

  • PD GENEration helps identify genetic variants linked to PD, accelerates clinical trial access and supports the development of personalized treatments. 

REM SLEEP BEHAVIOUR DISORDER

YALE UNIVERSITY – PRISMS

An Exploratory Study of the Potential for Rational Immune System Manipulation to Prevent Emergence of Synucleinopathy Manifestations in Persons With REM Sleep Behaviour Disorder (RBD) (PRISMS)  

This is a phase 2 study to assess the ability of adalimumab as compared to placebo to reduce or prevent progression of synuclein-related neurodegeneration in persons with idiopathic REM Sleep Behavior Disorder (RBD). The Primary Endpoint will be change from baseline in expression of the Parkinson Disease Related Pattern (PDRP) will be assessed using change in 18-flurodeoxyglucose (FDG) Positron Emission Tomography (PET) imaging.
 

ACTIVE (NOT RECRUITING) TRIALS

ATAXIAS

ARROWHEAD ARO-ATXN2

A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2

Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.

PARKINSON'S DISEASE

BIOGEN LUMA

A Study to Assess the Safety of BIIB122 Tablets and if it Can Slow the Worsening of Early-Stage Parkinson’s Disease in Participants Between the Ages of 30 and 80 (LUMA)

In this study, researchers will learn more about a study drug called BIIB122 in participants with early-stage Parkinson’s disease (PD).  The main question researchers want to answer is: How long does it take for PD symptoms to worsen during BIIB122 treatment? Researchers will answer this and other questions by measuring the symptoms of PD over time using a variety of scoring tools.

The study will include adults aged 30 to 80 who were diagnosed with PD within 2 years of starting the study.

Patients must not be on or have previously taken Levadopa (or similar) treatment.

HOFFMAN LAROCHE – PADOVA 

A Phase IIb, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Intravenous Prasinezumab in Participants with Early Parkinson’s Disease

Currently, there is no approved medication to modify the progression of Parkinson’s disease (PD).

The PADOVA (Roche BN42358) clinical trial is studying the safety and efficacy of a drug for individuals with early Parkinson’s disease.

  • Aged 50 to 85, diagnosed with PD for at least 6 months and up to 3 years
  • Currently taking no more than 1 medication for PD (maximum daily dose of 450 mg of levodopa, 1 mg of rasagiline, or 10 mg of selegiline)
  • Able to undergo medical procedures such as MRI and DaTScans
  • Willing to use a smartphone application and smartwatch for the duration of the study
  • The study will last at least  76 weeks
  • There is no compensation for this study. However, costs related to participation in the study, such as travel and meals during visit days, will be reimbursed.

 

The Team – ALS

THE TEAM

Dr. R. Postuma

Dr. R. Postuma

Principal Investigator

Dr. M Pandolfo

Dr. M Pandolfo

Principal Investigator

Nadine Zablith

Nadine Zablith

Clinical Research Coordinator

Sara Elatris

Sara Elatris

Clinical Research Coordinator

The Team – ALS

The Team 

Dr. A-L Lafontaine

Dr. A-L Lafontaine

Principal Investigator

Yara Nasr

Yara Nasr

Team Lead

Salwa Regragui

Salwa Regragui

Clinical Research Coordinator