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

Part of paid clinical trials in Chicago, Illinois.

Sponsor
Hoffmann-La Roche
Study ID
NCT05808764
Phase
PHASE2
Status
Recruiting
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Conditions

  • Muscular Atrophy, Spinal

Eligibility Criteria

Sex
ALL
Age
N/A - 19 Days
Healthy Volunteers
Not accepted

Interventions

  • Risdiplam — DRUG
    Participants will receive 0.15 mg/kg risdiplam orally once daily for 28 days.

Study Details

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.

Key Dates

Start date
Apr 26, 2024
Status verified
May 2026
Primary completion
Nov 30, 2026
Completion
Nov 30, 2026

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Risdiplam
    Participants will receive risdiplam once daily for 28 days.

Primary Outcome Measure

Plasma Concentrations of Risdiplam [ Time Frame: From Day 1 through Day 28 ]

Central Contacts

  • Reference Study ID Number: BN44619 https://forpatients.roche.com/
    888-662-6728 (U.S. Only)
    [email protected]

Locations (3)

FacilityCityStateZIPSite coordinators
Ann and Robert H. Lurie Children Hospital of ChicagoChicagoIllinois60611-
University Of MichiganAnn ArborMichigan48109-
Clinic for Special Children.GordonvillePennsylvania17529-

Find similar trials in Chicago, IL

By research site
Ann and Robert H. Lurie Children Hospital of Chicago· Chicago, ILUniversity Of Michigan· Ann Arbor, MIClinic for Special Children.· Gordonville, PA

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