A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA) (OPAL)

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-520130-29-00

Acronym

SRK-015-005

Enrollment

Registered

2025-10-21

Start date

2025-12-17

Completion date

Unknown

Last updated

2025-11-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal Muscular Atrophy

Brief summary

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

Detailed description

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

Interventions

DRUGAPITEGROMAB

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

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

Secondary

Measure	Time frame
Relationship of apitegromab concentrations and total latent myostatin concentration in serum, Population PK/PD model-based parameters such as Imax, IC50, and Kout, Incidence of TEAEs and SAEs by severity	—

Countries

Belgium, France, Italy, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026