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Pivotal Study of N-acetyl-L-leucine for CACNA1A

NCT07221292 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2026-03-27

No results posted yet for this study

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.

Conditions

  • CACNA1A
  • Spinocerebellar Ataxia Type 6
  • Episodic Ataxia Type 2
  • Familial Hemiplegic Migraine-1

Interventions

DRUG

N-Acetyl-L-Leucine

N-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)

OTHER

Placebo

Matching Placebo Sachet

Sponsors & Collaborators

  • IntraBio Inc

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-01
Primary Completion
2027-08-01
Completion
2028-11-01
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Switzerland

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07221292 on ClinicalTrials.gov