Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study
IntraBio Inc
Summary
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Description
This is a multinational, randomized, placebo-controlled, double-blinded, cross-over Phase III study that will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) versus Placebo for the treatment of CACNA1A Disorders. Patients will be assessed during three study periods: a baseline period (approximately 2-weeks), after which they will be randomized (1:1) to receive treatment with IB1001 or Placebo for approximately 12-weeks during the first intervention period ("Period I"). Following Period I, patients will crossover to receive the opposite treatment (IB1001 or Placebo) for approxima…
Eligibility
- Age range
- 4+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Individuals who meet all of the following criteria are eligible to participate in the study: 1. Written informed consent signed by the patient and/or their legal representative / parent/ impartial witness. 2. Male or female aged ≥4 years with a genetically-confirmed diagnosis of a CACNA1A Disorder (including patients with loss-of-function and fain-of-function mutations, e.g. Episodic Ataxia Type 2 (EA2), Familial Hemiplegic Migraine Type 1, Spinocerebellar Ataxia type C (SCA6), Developmental and Epileptic encephalopathy 42 (DEE42), Congenital ataxia or cerebellar hypoplasi…
Interventions
- DrugN-Acetyl-L-Leucine
N-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)
- OtherPlacebo
Matching Placebo Sachet
Locations (5)
- Massachusetts General HospitalBoston, Massachusetts
- The University of Texas Health (UT Health)Houston, Texas
- University of CologneCologne
- University of GiessenGiessen
- University Hospital Bern InselspitalBern