Patient Guide: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Why Use Quri.ai for This Trial?

  • ✓ Simplified eligibility checker - know if you qualify in minutes
  • ✓ Direct contact with study coordinators at 7 locations
  • ✓ Plain language explanations of medical terms and procedures
  • ✓ Personalized enrollment support throughout your journey
  • ✓ Free service - no hidden fees or obligations
Trial ID: NCTNCT03779334
Status: ACTIVE_NOT_RECRUITING
Condition: Muscular Atrophy, Spinal
Phase: PHASE2

Where You Can Participate

This study is available at 7 locations across the country.

Top locations include:
  • • Orlando, Florida
  • • Randwick, New South Wales
  • • Liège,
  • • And 4 more locations

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A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - Join Clinical Trial NCTNCT03779334

How to Join This Clinical Trial - NCTNCT03779334

Learn how to participate in this PHASE2 trial studying an investigational therapy for Muscular Atrophy, Spinal. Current status: ACTIVE_NOT_RECRUITING.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with Muscular Atrophy, Spinal. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied
Muscular Atrophy, Spinal
Treatment Being Tested
Investigational treatment
Study Phase
PHASE2 - Safety and effectiveness study
Enrollment Status
ACTIVE_NOT_RECRUITING
Study Identifier
NCTNCT03779334 - ClinicalTrials.gov Identifier
Sponsored By
Study sponsor

What to Expect as a Participant

Participating in this clinical trial involves:

  • Initial screening to determine eligibility
  • Regular study visits and health assessments
  • Receiving the study treatment or placebo
  • Medical monitoring and follow-up care
  • Contributing to medical research that may help others
  • Potential access to new treatments before they're widely available

Clinical Trial Benefits and Compensation

Participants in this clinical research study may receive:

  • Close medical monitoring by healthcare professionals
  • Access to potential new treatments
  • Compensation for time and travel (varies by study)
  • No-cost study-related medical care
  • The opportunity to help advance medical knowledge

Where Is This Clinical Trial Located?

This study is enrolling participants at 7 locations. Find a study site near you:

Clinical Research Site

Orlando, Florida 32837 - United States

Status: Contact for availability

Clinical Research Site

Randwick, New South Wales 2031 - Australia

Status: Contact for availability

Clinical Research Site

Liège, 4000 - Belgium

Status: Contact for availability

Clinical Research Site

Sao Paulo, São Paulo 05403-000 - Brazil

Status: Contact for availability

Clinical Research Site

Gda?sk, 80-952 - Poland

Status: Contact for availability

Clinical Research Site

Moscow, Moskovskaja Oblast 125412 - Russian Federation

Status: Contact for availability

Clinical Research Site

Kaohsiung, 807 - Taiwan

Status: Contact for availability

How to Enroll in This Study

To learn more about participating in this PHASE2 clinical trial for Muscular Atrophy, Spinal:

  1. Review the eligibility criteria with your healthcare provider
  2. Contact the study team for a pre-screening interview
  3. Schedule an in-person screening visit if eligible
  4. Review and sign the informed consent form
  5. Begin participation in the clinical trial

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