An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

Part of paid clinical trials in Los Angeles, California.

Sponsor
Larimar Therapeutics, Inc.
Study ID
NCT06447025
Phase
PHASE2
Status
Recruiting
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Conditions

  • Friedreich Ataxia

Eligibility Criteria

Sex
ALL
Age
2 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • CTI-1601 — DRUG
    CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia

Study Details

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.

Key Dates

Start date
Jan 25, 2024
Status verified
May 2026
Primary completion
Jan 31, 2027
Completion
Jan 31, 2027

Study Design

Enrollment
85 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CTI-1601
    Once daily subcutaneous injection of 50 mg CTI-1601 in subjects ≥ 18 years of age or a weight-based dose of 0.8 mg/kg up to a maximum of 50 mg in subjects ≥ 2 to 17 years of age.

Primary Outcome Measure

Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity [ Time Frame: Up to 24 months ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
University of California Los AngelesLos AngelesCalifornia90095-
Fixel Institute for Neurological Disease, University of Florida HealthGainesvilleFlorida32608-
Morsani Center for Advanced Health Care, University of South Florida HealthTampaFlorida33612
Kayla Zayas
[email protected]
813-974-5909
Lucretia Campbell
[email protected]
813-974-5909
University of IowaIowa CityIowa52242-
Uncommon CuresChevy ChaseMaryland20815
Rachael Batabyal, MD
[email protected]
240-858-4938
Tam Roshan Lal, MB, ChB
[email protected]
240-858-4912
Clinilabs Drug Development, Corp.EatontownNew Jersey07724-
Ohio State University United StatesColumbusOhio43210-
Children's Hospital of the University of Pennsylvania (CHOP)PhiladelphiaPennsylvania19104
David Lynch, MD, PhD
[email protected]
215-590-2242
McKenzie Wells, MS
[email protected]
267-426-9608

Find similar trials in Los Angeles, CA

By research site
University of California Los Angeles· Los Angeles, CAFixel Institute for Neurological Disease, University of Florida Health· Gainesville, FLMorsani Center for Advanced Health Care, University of South Florida Health· Tampa, FLUniversity of Iowa· Iowa City, IAUncommon Cures· Chevy Chase, MDClinilabs Drug Development, Corp.· Eatontown, NJ

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