Second-Chance treatment for babies whose SMA gene therapy stopped working

NCT ID NCT05861999

Recruiting now Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

Summary

This study is testing whether the drug risdiplam can help babies and toddlers with spinal muscular atrophy (SMA) who stopped improving or got worse after receiving gene therapy. It will involve 28 children under 2 years old who have two copies of the SMN2 gene. Researchers will measure changes in motor skills over 72 weeks to see if risdiplam can restart their development.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie
RECRUITING

Berlin, 13353, Germany
Children'S Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITING

Atlanta, Georgia, 30329-2309, United States
Children's Hospital of Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital of the King's Daughter
RECRUITING

Norfolk, Virginia, 23510, United States
Great Ormond Street Hospital For Children
RECRUITING

London, WC1N 3JH, United Kingdom
Helen DeVos Children's Hospital at Spectrum Health
RECRUITING

Grand Rapids, Michigan, 49503, United States
Instytut Pomnik Centrum Zdrowia Dziecka
RECRUITING

Warsaw, 04-730, Poland
Schneider Children's Medical Center of Israel
RECRUITING

Petah Tikva, 4920235, Israel
Sidra Medicine
RECRUITING

Al Rayyan, Qatar
Soroka Medical Center
RECRUITING

Beersheba, 8410101, Israel
Sourasky MC, Dana-Dwek Children's Hospital
RECRUITING

Tel Aviv, 6423906, Israel
Stanford Univ Medical Center
RECRUITING

Palo Alto, California, 94304, United States
UKGM Standort Gießen
RECRUITING

Giessen, 35392, Germany
University of Arkansas for Medical Sciences
RECRUITING

Little Rock, Arkansas, 72103, United States
University of Florida Pediatrics
RECRUITING

Gainesville, Florida, 32610, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
Uniwersyteckie Centrum Kliniczne
RECRUITING

Gdansk, 80-952, Poland
Valley Children's Hospital
RECRUITING

Madera, California, 93636, United States

Conditions

Explore the condition pages connected to this study.

MUSCULAR ATROPHY, SPINAL