Clinical research and trials

Clinical research is medical research involving people. There are two different types of research: 

  • Clinical trials are research studies performed on people focusing on a medical, surgical, or behavioral intervention. These help researchers determine if a new treatment, such as a new drug, diet, or medical device (eg. implants, etc.), is safe and effective in people. Often, a clinical trial is used to figure out if a new treatment is more effective and/or less harmful than the current standard of care.

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  • Non-interventional research is very important to pave the way for future clinical trials. These studies involve gathering information and collecting physiological and biomarker data, which are all essential to help understand the disease mechanism and to develop new treatments.

Clinical Trials

Biohaven 206A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects with Spinocerebellar Ataxia​

Who is this study for? 

Ages 18-75; diagnosed with genetically confirmed SCA 1, 2, 3, 7, or 10; ability to ambulate 8 meters without human assistance; fluent in English; adequate hearing, vision, and language abilities to complete testing

How long will I need to participate? 

2 Years

What is the treatment being studied? 

Troriluzole or Placebo Tablet, taken by mouth

What is the objective of the study? 

The overall objective is to evaluate the long-term efficacy and safety of Troriluzole in adult patients with Spinocerebellar Ataxia (SCA). Troriluzole potentially may provide therapeutic effects for SCA via neuroprotective mechanisms.

Who should I contact? 

Nadia Amokrane, na2855@cumc.columbia.edu

Biohaven 201A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects with Spinocerebellar Ataxia​

Who is this study for?

Ages 18-75; diagnosed with genetically confirmed SCA 1, 2, 3, 6, 7, 8, or 10; ability to ambulate 8 meters without human assistance; fluent in English; adequate hearing, vision, and language abilities to complete testing

How long will I need to participate? 

1 Year

What is the treatment being studied? 

BHV-4157 or Placebo Tablet, taken by mouth

What is the objective of the study? 

The overall objective is to evaluate the efficacy and safety of BHV-4157 in patients with Spinocerebellar Ataxia (SCA) after 8 weeks of treatment. BHV-4157 potentially may provide therapeutic effects for SCA via neuroprotective mechanisms.

Who should I contact?

Nadia Amokrane, na2855@cumc.columbia.edu

Biohaven 301 - A Phase III, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy​

Who is this study for?

Ages 40-75; diagnosed with MSA-P or MSA-C; ability to ambulate 10 steps without human assistance; consent of caregiver

How long will I need to participate?

1 Year

What is the treatment being studied? 

BHV-3241 or Placebo Tablet, taken orally

What is the objective of the study? 

The objective is to evaluate the efficacy and safety of BHV-3241 in patients with multiple system atrophy (MSA). BHV-3241 potentially may provide therapeutic effects for MSA by reducing neuroinflammation.

Who should I contact? 

Anisha Viswanathan, av2935@cumc.columbia.edu

Non-Interventional Studies

READISCAClinical Trial Readiness for Rare Neurological and Neuromuscular Diseases​

Who is this study for? 

Ages 18-65; diagnosed with SCA 1 or 3

How long will I need to participate? 

5 Years

What is the objective of the study? 

The objective is to establish the world's largest cohorts of pre-manifest and early-stage SCA1 and SCA3 individuals by combining and expanding existing cohorts, COA data, and biofluid samples (blood and cerebrospinal fluid) from the US and Europe.

Who should I contact? 

Nadia Amokrane, na2855@cumc.columbia.edu

CRC-SCANatural History of Spinocerebellar Ataxias

Who is this study for? 

Ages 6+; diagnosed with SCA 1, 2, 3, 6, 7, 8, or 10

How long will I need to participate? 

5 Years

What is the objective of the study? 

This observational study focuses on assessing the progression rate of SCA 1, 2, 3, 6, 7, 8, and 10 using validated neurological rating scales, timed performance measures, and patient-reported outcome measures.

Who should I contact? 

Nadia Amokrane, na2855@cumc.columbia.edu

Cerebellar Impulsivity Study 

Who is this study for? 

Ages 18+; diagnosed with SCA, essential tremor, or Parkinson's Disease

How long will I need to participate? 

30 minutes

What is the objective of the study? 

The purpose of this observational study is to evaluate decision making, thinking patterns, motor function, emotions and behaviors in patients with ataxia and healthy controls. 

Who should I contact? 

Nadia Amokrane, na2855@cumc.columbia.edu

For more information or questions about clinical research and ongoing trials,

please call us at (212) 305-5779.