NOMLABOFUSP for Friedreich's ataxia

Inclusion criteria

• {"criterion_text":"- • Individuals (children and adults) aged 12 to 40, male or female, with a diagnosis of FA\n- • Individuals capable of giving informed consent and completing physical function tests (see Objectives)\n- • Individuals able to administer subcutaneous injections at home (with the help of a caregiver if needed)\n- • Individuals using contraception, if able to conceive"}

Exclusion criteria

• {"criterion_text":"- • Previous treatment with nomlabofusp\n- • Allergy to nomlabofusp ingredients, cetirizine, or famotidine\n- • Pregnancy during the study\n- • Severe diabetes, liver disease, or heart disease\n- • Health conditions that could pose an increased risk to the participant or interfere with the understanding of the study results\n- • Drug or alcohol use disorder"}

Primary endpoints

• {"endpoint_text":"- PRIMARY: • Difference between participant groups (see Study Design Section) on the Modified Friedreich’s Ataxia Rating Scale (mFARS) total score at study Week 72 compared with baseline","definition_or_measurement_approach":"Measured as difference in mFARS total score between nomlabofusp and placebo groups at Week 72 versus baseline (mFARS total score change at Week 72 compared to baseline)."}

Secondary endpoints

• {"endpoint_text":"- SECONDARY: • Difference between participant groups in Clinical Global Impression of Severity (CGI-S) score at study Week 72 compared with baseline","definition_or_measurement_approach":"Change in CGI-S score at Week 72 versus baseline between treatment groups."}
• {"endpoint_text":"- SECONDARY: • Differences between participant groups in scores on physical function tests (Upright Stability Score (USS) Subscale E of the mFARS, 9-hole peg test, and 25-foot walk test) at study Week 72 compared with baseline","definition_or_measurement_approach":"Between-group differences in specified physical function test scores (USS Subscale E of mFARS, 9-hole peg test, 25-foot walk test) at Week 72 versus baseline."}
• {"endpoint_text":"- SECONDARY: • Differences between participant groups in scores on 1) the Friedreich’s Ataxia Rating Scale – Activities of Daily Living and 2) the fatigue numeric rating scale at study Week 72 compared with baseline","definition_or_measurement_approach":"Between-group differences in FA-ADL and fatigue numeric rating scale scores at Week 72 versus baseline."}
• {"endpoint_text":"- SECONDARY: • Differences between participant groups in left ventricular mass index, measured on echocardiogram, at study Week 72 compared with baseline","definition_or_measurement_approach":"Between-group differences in left ventricular mass index (echocardiogram) at Week 72 versus baseline."}
• {"endpoint_text":"- SECONDARY: • Differences between participant groups in changes over time in frataxin amount measured in the skin and in the inside of the cheek","definition_or_measurement_approach":"Between-group differences in longitudinal changes of frataxin levels measured in skin and buccal (inside cheek) samples."}
• {"endpoint_text":"- SAFETY: • Incidence of side effects and serious side effects happening after receiving nomlabofusp, monitored for the entire study duration","definition_or_measurement_approach":"Incidence and characterization of adverse events and serious adverse events throughout study follow-up."}
• {"endpoint_text":"- IMMUNOGENICITY: • Incidence of antidrug antibodies (proteins that cause the immune system to fight the drug) throughout the study","definition_or_measurement_approach":"Incidence of anti-drug antibodies measured during the study period."}

Methods

• Recruitment and retention provided by Jumo Health USA Inc. (role: Recruitment and retention) — no country-specific channels detailed in CTIS record.
• Patient travel services provided by Block Clinical Inc. (role: Patient travel services) to support participant attendance.
• Home health services provided by Accellacare Limited (role: Home health) to support at-home administration/visits.
• Centralized lab services (Icon (Lr) Limited) and specialty labs (Altasciences Compagnie Inc., PPD Laboratories, IRCCS Foundation Istituto Neurologico Carlo Besta) supporting PK/FXN/genetic/ADA testing that may be part of recruitment/assessment pathway.
• Clinical Research Organization support by Premier Research Group S.L. for overall study conduct.

France

Earliest CTIS Part Ii Submission Date

11-02-2026

Latest Decision Or Authorization Date

16-04-2026

Processing Time Days

64

Number Of Sites

1

Number Of Participants

18

Primary sponsor

Full Name

Larimar Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Premier Research Group S.L.

Responsibilities

Clinical Research Organization

Third parties

• {"country":"Canada","full_name":"Altasciences Compagnie Inc.","duties_or_roles":"Specialty lab (PK, FXN - buccal and skin)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Clinchoice Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Block Clinical Inc.","duties_or_roles":"Patient travel services","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"PharmaLex GmbH","duties_or_roles":"Regulatory expertise and submissions","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pci San Diego Inc.","duties_or_roles":"Storage and distribution of investigational drug","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Sherpa Clinical Packaging LLC","duties_or_roles":"Storage and distribution of investigational drug","organisation_type":"Pharmaceutical company"}
• {"country":"Italy","full_name":"IRCCS Foundation Istituto Neurologico Carlo Besta","duties_or_roles":"Specialty lab (genetic testing)","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Llx Solutions LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Recruitment and retention","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Ireland","full_name":"Icon (Lr) Limited","duties_or_roles":"Centralized labs","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Laboratories","duties_or_roles":"Specialty lab (ADA)","organisation_type":"Industry"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"IRT, eDiary","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Spain","full_name":"Premier Research Group S.L.","duties_or_roles":"Clinical Research Organization","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Clario","duties_or_roles":"Cardiac safety and imaging","organisation_type":"Industry"}
• {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"Home health","organisation_type":"Pharmaceutical company"}