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 to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
- First Posted Date
- 2019-04-19
- Last Posted Date
- 2021-02-21
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 26
- Registration Number
- NCT03920865
- Locations
- 🇺🇸
Clinical Pharmacology of Miami, Inc., Miami, Florida, United States
🇺🇸Orlando Clinical Research Center, Orlando, Florida, United States
🇺🇸American Research Corporation Inc., San Antonio, Texas, United States
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
- First Posted Date
- 2018-12-19
- Last Posted Date
- 2024-12-06
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 26
- Registration Number
- NCT03779334
- Locations
- 🇵🇱
Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology, Gda?sk, Poland
🇨🇳Kaohsiung Medical University Chung-Ho Hospital; Pediatric Neurology, Kaohsiung, Taiwan
🇺🇸Nemours Children's Hospital, Orlando, Florida, United States
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants
- First Posted Date
- 2017-02-02
- Last Posted Date
- 2018-10-04
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 24
- Registration Number
- NCT03040635
- Locations
- 🇺🇸
Collaborative Neuroscience Network, Inc., Garden Grove, California, United States
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
- First Posted Date
- 2017-01-26
- Last Posted Date
- 2024-12-12
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 174
- Registration Number
- NCT03032172
- Locations
- 🇧🇪
UZ Leuven Gasthuisberg, Leuven, Belgium
🇮🇹IRCCS Ospedale Pediatrico Bambino Gesù; U.O. Malattie Neuromuscolari e Neurodegenerative, Roma, Lazio, Italy
🇮🇹Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile, Roma, Lazio, Italy
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
- First Posted Date
- 2016-09-23
- Last Posted Date
- 2024-08-07
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 62
- Registration Number
- NCT02913482
- Locations
- 🇺🇸
Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
🇵🇱Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology, Gda?sk, Poland
🇺🇦Ams of Ukraine; Inst. of Neurology, Psychiatry & Narcology, Kharkov, Ukraine
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
- First Posted Date
- 2016-09-21
- Last Posted Date
- 2024-04-24
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 231
- Registration Number
- NCT02908685
- Locations
- 🇯🇵
Minami Kyushu National Hospital, Kagoshima, Japan
🇯🇵Miyagi Children's Hospital, Miyagi, Japan
🇯🇵Shiga Medical Center for Children, Shiga, Japan
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
- Conditions
- Interventions
- First Posted Date
- 2015-12-17
- Last Posted Date
- 2018-10-04
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 33
- Registration Number
- NCT02633709
- Locations
- 🇳🇱
Pra International Group B.V, Groningen, Netherlands
Drug Development Updates
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