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Adult SMA Research and Clinical Hub

NCT06978985 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 600

Last updated 2025-05-18

No results posted yet for this study

Summary

Adult SMA REACH is a data collection study aiming to gain a better understanding of the impact of standards of care and new treatments on the natural history of Spinal Muscular Atrophy (SMA). This study is sponsored by The Newcastle upon Tyne Hospitals NHS Foundation Trust. Adult SMA REACH is funded by Biogen and Roche.

Currently, there are three drug treatments available for SMA in the UK: Zolgensma, Nusinersen and Risdiplam. Zolgensma is the only approved drug - Nusinersen and Risdiplam are currently available as part of Managed Access Agreements (MAA).

Conditions

  • Spinal Muscular Atrophy (SMA)

Interventions

DRUG

Risdiplam

Risdiplam

DRUG

Nusinersen Injectable Product

Nusinersen

Sponsors & Collaborators

  • Newcastle University

    collaborator OTHER

  • John Walton Muscular Dystrophy Research Centre

    collaborator UNKNOWN

  • Biogen

    collaborator INDUSTRY

  • Roche Products Limited

    collaborator UNKNOWN

  • Newcastle-upon-Tyne Hospitals NHS Trust

    lead OTHER

Principal Investigators

  • Prof Chiara Marini-Bettolo, MD, PhD · Newcastle-upon-Tyne Hospitals NHS Trust

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-29
Primary Completion
2027-04-30
Completion
2027-04-30

Countries

  • United Kingdom

Study Locations

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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 NCT06978985 on ClinicalTrials.gov