← Back to Search

Small Molecule

risdiplam for Spinal Muscular Atrophy

Phase 1
Waitlist Available
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up part 1: up to day 20 in periods 1-2, up to day 14 in periods 3-5; parts 2 and 3: up to day 11 in periods 1-4
Awards & highlights

Study Summary

This trial is testing whether two different formulations of risdiplam are absorbed the same and have the same effect in healthy people. It will also look at whether food or omeprazole affect how well the drug is absorbed.

Eligible Conditions
  • Spinal Muscular Atrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~part 1: up to day 20 in periods 1-2, up to day 14 in periods 3-5; parts 2 and 3: up to day 11 in periods 1-4
This trial's timeline: 3 weeks for screening, Varies for treatment, and part 1: up to day 20 in periods 1-2, up to day 14 in periods 3-5; parts 2 and 3: up to day 11 in periods 1-4 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part 1: Plasma Concentration of Risdiplam in Cohorts A and B
Part 1: Plasma Concentration of Risdiplam in Cohorts C and D
Part 1: Plasma Concentration of Risdiplam in Fed and Fasted States in Cohort E
+1 more
Secondary outcome measures
Percentage of Participants with Adverse Events and Serious Adverse Events

Trial Design

3Treatment groups
Experimental Treatment
Group I: Part 3 (optional)Experimental Treatment1 Intervention
Group 1: participants will receive, in four-period crossover design, a single oral dose of risdiplam oral solution 5 mg in both fed and fasted states and the selected dispersible tablet (F22) as swallowed tablet in both fed and fasted states. Group 2: participants will receive, in four-period crossover design, a single oral dose of risdiplam oral solution 5 mg in both fed and fasted states and the selected dispersible tablet (F22) as tablet dispersed in water in both fed and fasted states.
Group II: Part 2 (optional)Experimental Treatment1 Intervention
Group 1: participants will receive, in four-period crossover design, a single oral dose of risdiplam oral solution 5 mg in both fed and fasted states and the selected dispersible tablet (F21) as swallowed tablet in both fed and fasted states. Group 2: participants will receive, in four-period crossover design, a single oral dose of risdiplam oral solution 5 mg in both fed and fasted states and the selected dispersible tablet (F21) as tablet dispersed in water in both fed and fasted states.
Group III: Part 1Experimental Treatment2 Interventions
Cohort A+B: participants will receive, in a five-period crossover way, a single oral dose of risdiplam oral solution 5 mg in fasted state and thereafter risdiplam/F21 or F22 dispersible tablet 5 mg as tablet in fasted and fed states; tablet dispersed in water in fasted and fed states, with a 14-day wash-out period in between the single-dose administrations. Cohort C+D: participants will receive, in a two-period fixed sequence design, a single dose of risdiplam/F21 or F22 dispersible tablet 5 mg in fasted state and omeprazole 40 mg once daily for 7 days + a single dose of risdiplam/F21 or F22 dispersible tablet 5 mg in fasted state, on the 7th day of omeprazole. There will be a 14-day wash-out between the two treatment periods. Cohort E: participants will receive, in a two-period crossover design, a single oral dose of risdiplam oral solution 5 mg in fasted and fed states, with a 14-day wash-out period in between the single-dose administrations.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
risdiplam
2021
Completed Phase 1
~140
omeprazole
2011
Completed Phase 4
~4239590

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,428 Previous Clinical Trials
1,088,923 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,199 Previous Clinical Trials
888,371 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this study open to those aged 50 and above?

"In order to be a participant in this research trial, one must fall within the 18-55 year old age range."

Answered by AI

Is this medical trial still accepting participants?

"Clinicaltrials.gov shows that this medical trial is now in its recruitment phase, with the first post being made on February 1st 2021 and updated most recently on November 22nd 2022."

Answered by AI

Are there any additional research initiatives concerning risdiplam?

"Currently, there are 15 active trials researching the efficacy of risdiplam with 3 being in Phase 3. While most experiments take place in Boston, Massachusetts, 191 other locations across the world have been conducting research for this drug."

Answered by AI

Is risdiplam a viable option for managing patient safety?

"While there is only limited evidence in favour of risdiplam's efficacy and safety, the Power team have rated it a 1 on their scale."

Answered by AI

What is the uppermost capacity of participants for this clinical experiment?

"Affirmative. A glance at clinicaltrials.gov exposes that this medical trial is actively seeking participants. This research was initially posted on February 1st 2021 and underwent its most recent update on November 22nd 2022. In total, 268 patients need to be recruited from 2 different institutions."

Answered by AI

Are there any specific demographics that would be ideal for enrollment in this experiment?

"This clinical trial seeks 268 individuals, aged 18 to 55 years old, affected by muscular dystrophy and spinal atrophy. In addition to this essential criterion, participants must have a Body Mass Index (BMI) between 18.0 – 32.0 kg/m2; male participants whose partners are women of childbearing potential or pregnant should remain abstinent with the use of adequate contraception from the treatment period until 4 months after their last dose of risdiplam for non-pregnant WOCBPs and 28 days post final dose in case if pregnancy; males must refrain from donating sperm during said timeframe as well; willing"

Answered by AI

To what maladies is risdiplam a potential remedy?

"Risdiplam is commonly prescribed to address hypersecretory ailments, and can provide relief for individuals suffering with gastroesophageal reflux disease (GERD), heartburn, or Zollinger-Ellison Syndrome."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Daytona Beach Clinical Rsch Unit
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
2021. "Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04718181.
~31 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top