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

Muscular Atrophy, Spinal Clinical Trial

Official title:
A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05808764
Study type Interventional
Source Hoffmann-La Roche
Contact Reference Study ID Number: BN44619 https://forpatients.roche.com
Phone 888-662-6728 (U.S. Only)
Email global-roche-genentech-trials@gene.com
Status Recruiting
Phase Phase 2
Start date April 26, 2024
Completion date August 31, 2025
View Details
See also
  Status Clinical Trial Phase
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Completed NCT02908685 - A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants Phase 2
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Completed NCT05073133 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) Phase 4
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Recruiting NCT05042921 - Pediatric Spinal Muscular Atrophy (SMA) China Registry
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Completed NCT00466349 - International SMA Patient Registry
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Recruiting NCT05861999 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy Phase 4
Recruiting NCT05755451 - Natural History of SMA
Active, not recruiting NCT01233817 - Progressive Strength Training in Spinal Muscular Atrophy N/A
Recruiting NCT04317794 - Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
Active, not recruiting NCT05618379 - Adult Spinal Muscular Atrophy (SMA) China Registry
Active, not recruiting NCT04488133 - A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec Phase 4
Completed NCT03781479 - Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients Phase 2
Completed NCT00568802 - A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients Phase 1/Phase 2