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

Part of paid clinical trials in Baltimore, Maryland.

Sponsor
IntraBio Inc
Study ID
NCT07221292
Phase
PHASE3
Status
Not Yet Recruiting

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Conditions

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

Eligibility Criteria

Sex
ALL
Age
4 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • N-Acetyl-L-Leucine — DRUG
    N-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)
  • Placebo — OTHER
    Matching Placebo Sachet

Study Details

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.

Key Dates

Start date
Sep 1, 2026
Status verified
Jun 2026
Primary completion
Aug 1, 2027
Completion
Nov 1, 2028

Study Design

Enrollment
60 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: N-acetyl-L-leucine (IB1001)
    Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients will receive a total daily dose of 2-4 g/day based on weight-tiered doses.
  • Placebo Comparator: Placebo
    Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients will receive a total daily dose of 2-4 g/day based on weight-tiered doses.

Primary Outcome Measure

Scale for the Assessment and Rating of Ataxia [ Time Frame: End of Period I (week 12) vs. End of Period 2 (week 24) ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Johns Hopkins UniversityBaltimoreMaryland21287
Kristin Baranano, MD
[email protected]
Kristin Baranano, MD (PRINCIPAL_INVESTIGATOR)
Massachusetts General HospitalBostonMassachusetts02114-
The University of Texas Health (UT Health)HoustonTexas77030-

Find similar trials in Baltimore, MD

By research site
Johns Hopkins University· Baltimore, MDMassachusetts General Hospital· Boston, MAThe University of Texas Health (UT Health)· Houston, TX

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