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A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT05808764 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-05-04

No results posted yet for this study

Summary

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Risdiplam

Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
19 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-26
Primary Completion
2026-11-30
Completion
2026-11-30
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Germany
  • Italy
  • Netherlands
  • Norway
  • Poland

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05808764 on ClinicalTrials.gov