Risdiplam
- Generic Name
- Risdiplam
- Brand Names
- Evrysdi
- Drug Type
- Small Molecule
- Chemical Formula
- C22H23N7O
- CAS Number
- 1825352-65-5
- Unique Ingredient Identifier
- 76RS4S2ET1
- Background
Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA). It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor nusinersen, the biggest difference being their route of administration: nusinersen r...
- Indication
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA).
- Associated Conditions
- Spinal Muscular Atrophy (SMA)
- Associated Therapies
- -
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
- First Posted Date
- 2023-05-17
- Last Posted Date
- 2024-12-02
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 28
- Registration Number
- NCT05861986
- Locations
- 🇺🇸
Columbia University Medical Center, New York, New York, United States
🇺🇸The University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, United States
🇺🇸Cook Children's Jane and John Justin Neurosciences Center, Fort Worth, Texas, United States
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
- First Posted Date
- 2023-05-17
- Last Posted Date
- 2024-11-22
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 28
- Registration Number
- NCT05861999
- Locations
- 🇺🇸
Children's Healthcare of Atlanta Center for Advanced Pediatrics, Atlanta, Georgia, United States
🇺🇸University of Texas Southwestern Medical Center, Dallas, Texas, United States
🇺🇸Children's Hospital of the King's Daughter, Norfolk, Virginia, United States
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
- First Posted Date
- 2023-04-11
- Last Posted Date
- 2024-12-03
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 10
- Registration Number
- NCT05808764
- Locations
- 🇨🇦
Children'S Hospital of Eastern Ontario, Ottawa, Ontario, Canada
🇺🇸Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
🇳🇱UMC Utrecht, Utrecht, Netherlands
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
- First Posted Date
- 2022-08-31
- Last Posted Date
- 2024-02-07
- Lead Sponsor
- Clinic for Special Children
- Target Recruit Count
- 10
- Registration Number
- NCT05522361
- Locations
- 🇺🇸
Clinic for Special Children, Strasburg, Pennsylvania, United States
Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- First Posted Date
- 2022-02-10
- Last Posted Date
- 2024-10-21
- Lead Sponsor
- Genentech, Inc.
- Target Recruit Count
- 500
- Registration Number
- NCT05232929
- Locations
- 🇺🇸
Barrow Neurological Institute, Phoenix, Arizona, United States
🇺🇸Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
- Conditions
- First Posted Date
- 2022-02-02
- Last Posted Date
- 2024-03-05
- Lead Sponsor
- Bakri Elsheikh
- Target Recruit Count
- 30
- Registration Number
- NCT05219487
- Locations
- 🇺🇸
The Ohio State University Wexner Medical Center, Columbus, Ohio, United States
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
- Conditions
- Interventions
- First Posted Date
- 2021-11-10
- Last Posted Date
- 2024-12-19
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 259
- Registration Number
- NCT05115110
- Locations
- 🇺🇸
Nemours Children's Hospital, Orlando, Florida, United States
🇺🇸Boston Childrens Hospital, Boston, Massachusetts, United States
🇺🇸Columbia University Medical Center, New York, New York, United States
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
- First Posted Date
- 2021-01-22
- Last Posted Date
- 2023-03-13
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 131
- Registration Number
- NCT04718181
- Locations
- 🇺🇸
Dallas Clinical Research Unit, Dallas, Texas, United States
🇺🇸Daytona Beach Clinical Rsch Unit, Daytona Beach, Florida, United States
🇺🇸Covance Clinical Research Unit, Inc, Madison, Wisconsin, United States
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- Conditions
- First Posted Date
- 2020-02-05
- Last Posted Date
- 2020-10-05
- Lead Sponsor
- Genentech, Inc.
- Registration Number
- NCT04256265
- Locations
- 🇺🇸
Arkansas Children's Hospital; Pediatrics, Little Rock, Arkansas, United States
🇺🇸Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
- First Posted Date
- 2019-06-18
- Last Posted Date
- 2020-10-19
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 35
- Registration Number
- NCT03988907
- Locations
- 🇺🇸
Covance Research Unit - Dallas, Dallas, Texas, United States
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.