A Study to Determine if Nomlabofusp is Effective and Safe in Adults and Children with Friedreich’s Ataxia

2025-521628-31-00 Protocol CLIN-1601-301 Therapeutic confirmatory (Phase III) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 1 sites · Protocol CLIN-1601-301

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Authorised, recruitment pending
Participants planned 150
Countries 1
Sites 1

Friedreich’s ataxia is the most common inherited ataxia in humans and results from a deficiency of the mitochondrial protein, FXN. Friedreich’s ataxia is a rare, progressive, multisystem disease with an incidence that is estimated to be 1:29,000, and a carrier frequency of ~1:85.

PRIMARY: • To learn if nomlabofusp improves movement ability in study participants (adults and children with FA) compared with placebo

Key facts

Sponsor
Larimar Therapeutics Inc.
Participant type
Pediatric, Patients
Age range
0-17 years, 18-64 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10]
Decision date (initial)
2026-04-16
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
Yes
Funding sources
Larimar Therapeutics, Inc., United States

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

PRIMARY:
• To learn if nomlabofusp improves movement ability in study participants (adults and children with FA) compared with placebo

Secondary objectives 7

  1. SECONDARY: • To learn if nomlabofusp improves severity of FA disease in study participants compared with placebo
  2. SECONDARY: • To learn if nomlabofusp improves physical abilities in study participants compared with placebo
  3. SECONDARY: • To assess the impact of nomlabofusp on quality of life in study participants compared with placebo
  4. SECONDARY: • To learn if nomlabofusp improves heart wall structure in study participants compared with placebo
  5. SECONDARY: • To learn if nomlabofusp increases frataxin amount in the body in study participants compared with placebo
  6. SAFETY: • To measure the safety of nomlabofusp in study participants
  7. IMMUNOGENICITY: • To measure immune system responses to nomlabofusp in study participants

Conditions and MedDRA coding

Friedreich’s ataxia is the most common inherited ataxia in humans and results from a deficiency of the mitochondrial protein, FXN. Friedreich’s ataxia is a rare, progressive, multisystem disease with an incidence that is estimated to be 1:29,000, and a carrier frequency of ~1:85.

VersionLevelCodeTermSystem organ class
20.0 PT 10017374 Friedreich's ataxia 100000004850
20.0 SOC 10010331 Congenital familial and genetic disorders 21

Study design 1 period

#TitleAllocationBlindingRoles blindedArms
1 Double-blind Treatment Period
Subjects who meet all the inclusion criteria and none of the exclusion criteria will enroll into the study and be randomized to receive study drug (nomlabofusp or placebo). Subjects will receive SC study drug QD throughout the Treatment Period.
 Randomised Controlled Double [{"id":181979,"code":5,"name":"Carer"},{"id":181981,"code":2,"name":"Investigator"},{"id":181980,"code":1,"name":"Subject"}]

Regulatory references

Scientific advice from competent authorities
European Medicines Agency
EMA paediatric investigation plan (PIP)
EMEA-003022-PIP01-21
Plan to share IPD
No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

  1. • Individuals (children and adults) aged 12 to 40, male or female, with a diagnosis of FA
  2. • Individuals capable of giving informed consent and completing physical function tests (see Objectives)
  3. • Individuals able to administer subcutaneous injections at home (with the help of a caregiver if needed)
  4. • Individuals using contraception, if able to conceive

Exclusion criteria 6

  1. • Previous treatment with nomlabofusp
  2. • Allergy to nomlabofusp ingredients, cetirizine, or famotidine
  3. • Pregnancy during the study
  4. • Severe diabetes, liver disease, or heart disease
  5. • Health conditions that could pose an increased risk to the participant or interfere with the understanding of the study results
  6. • Drug or alcohol use disorder

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. 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

Secondary endpoints 7

  1. SECONDARY: • Difference between participant groups in Clinical Global Impression of Severity (CGI-S) score at study Week 72 compared with baseline
  2. 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
  3. 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
  4. SECONDARY: • Differences between participant groups in left ventricular mass index, measured on echocardiogram, at study Week 72 compared with baseline
  5. SECONDARY: • Differences between participant groups in changes over time in frataxin amount measured in the skin and in the inside of the cheek
  6. SAFETY: • Incidence of side effects and serious side effects happening after receiving nomlabofusp, monitored for the entire study duration
  7. IMMUNOGENICITY: • Incidence of antidrug antibodies (proteins that cause the immune system to fight the drug) throughout the study

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Nomlabofusp

PRD12342666 · Product

Active substance
Nomlabofusp
Substance synonyms
TAT-frataxin fusion protein, Human frataxin fused to TAT cell-penetrating peptide, CTI-1601
Other product name
CTI-1601
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
SUBCUTANEOUS INJECTION
Max daily dose
50 mg/ml milligram(s)/millilitre
Max total dose
25200 mg/ml milligram(s)/millilitre
Max treatment duration
72 Week(s)
Authorisation status
Not Authorised
MA holder
LARIMAR THERAPEUTICS, INC.
Paediatric formulation
No
Orphan designation
Yes
Orphan designation number
EU/3/20/2328

Placebo 1

Placebo to Nomlabofusp

N/A · Product

Other product name
N/A
Pharmaceutical form
N/A
ATC code
N/A — N/A
Marketing authorisation
N/A
MA holder
N/A
MA country
Iceland
Paediatric formulation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Larimar Therapeutics Inc.

Sponsor organisation
Larimar Therapeutics Inc.
Address
3 Bala Plaza East Suite 506
City
Bala Cynwyd
Postcode
19004-3481
Country
United States

Scientific contact point

Organisation
Larimar Therapeutics Inc.
Contact name
Larimar Therapeutics

Public contact point

Organisation
Larimar Therapeutics Inc.
Contact name
Larimar Therapeutics

Third parties 16

OrganisationCity, countryDuties
Altasciences Compagnie Inc.
ORG-100037610
 Laval, Canada Other
Clinchoice Inc.
ORG-100027185
 Horsham, United States Data management
Block Clinical Inc.
ORG-100048643
 San Diego, United States Other
PharmaLex GmbH
ORG-100001378
 Bad Homburg, Germany Other
Pci San Diego Inc.
ORG-100011937
 San Diego, United States Other
Sherpa Clinical Packaging LLC
ORG-100042876
 San Diego, United States Other
IRCCS Foundation Istituto Neurologico Carlo Besta
ORG-100006637
 Milan, Italy Other
Fortrea Inc.
ORG-100012602
 Durham, United States Other
Llx Solutions LLC
ORG-100046614
 Waltham, United States Code 10
Jumo Health USA Inc.
ORG-100044054
 New Haven, United States Other
Icon (Lr) Limited
ORG-100042612
 Dublin 18, Ireland Other
PPD Laboratories
ORL-000001474
 Richmond, VA, United States Other
Suvoda LLC
ORG-100043523
 Conshohocken, United States Other
Premier Research Group S.L.
ORG-100013963
 Madrid, Spain Other
Clario
ORL-000002742
 Philadelphia, United States Other
Accellacare Limited
ORG-100044508
 Dublin 18, Ireland Other

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
France Authorised, recruitment pending 18 1
Rest of world
Canada, United Kingdom, United States, Australia
 — 132 —

Investigational sites

France

1 site · Authorised, recruitment pending
Assistance Publique Hopitaux De Paris
Neurology, 47 Boulevard De L Hopital, 75651, Paris Cedex 13

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 28 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol_2025-521628-31-00_Redacted 03a
Protocol (for publication) D4_Diary-Daily-Dosing_eCOA SR_HH_2025-521628-31-00 1.0
Protocol (for publication) D4_Diary-Daily-Dosing_eCOA SR_HH_2025-521628-31-00 1.0
Protocol (for publication) D4_Diary-Past-Dose-Entry_eCOA SR_HH_2025-521628-31-00 1.0
Protocol (for publication) D4_Diary-Past-Dose-Entry_eCOA SR_HH_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-EQ-5D-5L-Interview-Admin_2025-521628-31-00 1.2
Protocol (for publication) D4_Q-EQ-5D-5L-Interview-Admin_2025-521628-31-00 1.2
Protocol (for publication) D4_Q-EQ-5D-5L-Proxy1_2025-521628-31-00 1.1
Protocol (for publication) D4_Q-EQ-5D-5L-Proxy1_2025-521628-31-00 1.3
Protocol (for publication) D4_Q-EQ-5D-5L-Self-Complete_2025-521628-31-00 1.2
Protocol (for publication) D4_Q-EQ-5D-5L-Self-Complete_2025-521628-31-00 1.2
Protocol (for publication) D4_Q-EQ-5D-Y-5L-Interview-Admin_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-EQ-5D-Y-5L-Interview-Admin_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-EQ-5D-Y-5L-Proxy1_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-EQ-5D-Y-5L-Proxy1_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-EQ-5D-Y-5L-Self-Complete_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-EQ-5D-Y-5L-Self-Complete_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-FA-ADL_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-FA-ADL_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-Fatigue-NRS_2025-521628-31-00 1.0
Protocol (for publication) D4_Q-Fatigue-NRS_2025-521628-31-00 1.0
Recruitment arrangements (for publication) K1_Informed Consent and Patient Recruitment Procedure 1.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Assent 12-17_redacted 2.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main Adult_redacted 3.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Main Parents_redacted 3.0
Subject information and informed consent form (for publication) L1_SIS and ICF_Pregnant Partner_redacted 2.0
Synopsis of the protocol (for publication) D1_Protocol-synopsis_2025-521628-31-00 02a
Synopsis of the protocol (for publication) D1_Protocol-synopsis_2025-521628-31-00 02a

Application history

2 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-12-19 France Acceptable
2026-04-13
 2026-04-16
2 NON SUBSTANTIAL MODIFICATION NSM-1 2026-04-28 France Acceptable
2026-04-13
 2026-04-28

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