A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Scholar Rock Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Phenomena and Processes [G] - Musculoskeletal and Neural Physiological Phenomena [G11]

Trial duration

17 Dec 2025 → ongoing

Decision date (initial)

2025-10-21

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Scholar Rock, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Efficacy

Evaluate the PK and PD of aepitegromab in subjects <2 years old with SMA.

Evaluate the motor function outcomes (ie, efficacy) due to apitegromab treatment

Secondary objectives 2

1. Characterize the PK/PD relationship of apitegromab administration in relation to total latent myostatin.
2. Assess the safety and toleratibily of apitegromab administered to subjects receiving an SMN therapy.

Conditions and MedDRA coding

Spinal Muscular Atrophy

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002951-PIP02-21

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Is <2 years old at the time of the informed consent
2. Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
3. Has confirmed diagnosis of 5q autosomal recessive SMA
4. Has confirmed presence of SMN2 gene copy(ies)
5. Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)
6. Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit
7. Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score <55.

Exclusion criteria 3

1. Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route, as assessed by the Investigator and/or an Oral and Swallowing Abilities Tool score of 0.
2. Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study.
3. Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Population PK model-based parameters such as CL, Vd, Vc and Vp
2. Apitegromab and total latent myostatin concentrations in serum
3. Change from baseline in the raw score of the BSID-4 GMS at 48 weeks

Secondary endpoints 3

1. Relationship of apitegromab concentrations and total latent myostatin concentration in serum
2. Population PK/PD model-based parameters such as Imax, IC50, and Kout
3. Incidence of TEAEs and SAEs by severity

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Scholar Rock Inc.

Sponsor organisation

Scholar Rock Inc.

Address

301 Binney Street

City

Cambridge

Postcode

02142-1071

Country

United States

Scientific contact point

Organisation

Scholar Rock Inc.

Contact name

Kelley Capocelli

Public contact point

Organisation

Scholar Rock Inc.

Contact name

Kelley Capocelli

Third parties 15

Locations

5 EU/EEA countries · 11 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 66 files

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

Application history

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