A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia

Part of paid clinical trials in Chicago, Illinois.

Sponsor
Larimar Therapeutics, Inc.
Study ID
NCT07778836
Phase
PHASE3
Status
Recruiting
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Conditions

  • Friedreich Ataxia
  • Friedreich's Ataxia

Eligibility Criteria

Sex
ALL
Age
12 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • Nomlabofusp — DRUG
    Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia.
  • Placebo — DRUG
    The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.

Study Details

To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia

Key Dates

First listed
Aug 21, 2026
Start date
Aug 5, 2026
Status verified
Aug 2026
Primary completion
Jul 31, 2029
Completion
Dec 31, 2029

Study Design

Enrollment
150 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Nomlabofusp
    Daily subcutaneous injection of nomlabofusp for 72 weeks
  • Placebo Comparator: Placebo
    Daily subcutaneous injection of placebo for 72 weeks

Primary Outcome Measure

Change from baseline in Upright Stability Score (USS) subscale E of modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific) [ Time Frame: Baseline, Weeks 12, 24, 36, 48, 72 ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Northwestern Medical Group, Department of NeurologyChicagoIllinois60611
Rizwan S Akhtar, MD
Cynthia Poon, PhD
[email protected]
(312) 503-8216
ClinilabsEatontownNew Jersey07724
Magdy Shenouda, MD
[email protected]
(646) 215-6400
University of Texas Southwestern Medical CenterDallasTexas75390
Vikram Shakkottai, MD, PhD
Sharon Primeaux
[email protected]
(214) 645-0563

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By research site
Northwestern Medical Group, Department of Neurology· Chicago, ILClinilabs· Eatontown, NJUniversity of Texas Southwestern Medical Center· Dallas, TX

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