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Small Molecule

Risdiplam for Spinal Muscular Atrophy (SUNFISH Trial)

Q: Does this research project include geriatric patients in its sample size?

Children aged 2 to 25 can participate in this clinical trial. In total, there are 53 clinical trials underway for patients under 18 years old and 151 for patients over 65.

Q: Are recruitment efforts ongoing for this clinical trial?

The trial, which was first posted on 10/20/2016, is not recruiting patients at this time according to clinicaltrials.gov. However, there are 211 other trials that are currently looking for participants.

Q: Are there a lot of hospitals running this clinical trial in Canada?

This particular trial is being conducted at Columbia University Medical Center, The Neurological Institute of <a href="https://www.withpower.com/clinical-trials/new-york">New york</a>, Alberta Children's Hospital Division of Pediatric Neurology, and McGill University Health Centre - Glen Site. In addition, there are 5 other undisclosed locations.

Q: What is the patient volume for this research?

This study is not recruiting patients presently. The trial was initially posted on October 20th, 2016 and was last updated on August 31st, 2022. If the reader is looking for other trials, there are 205 trials actively recruiting participants with muscular atrophy and 6 studies for Risdiplam actively recruiting patients.

Q: What makes this research project unique?

Risdiplam has been under research since 2016 when the first study, sponsored by Hoffmann-La Roche, was completed with 231 participants. After the successful Phase 2 &#x26; 3 drug approval, 6 more active studies involving Risdiplam have popped up in 22 different cities and countries.

Q: Can you give some examples of other investigations that have used Risdiplam?

Risdiplam was first studied in the year 2016 at Jichi Medical University Hospital. So far, there have been 18243 completed trials. Currently, there are 6 live studies, a significant amount of which are based in <a href="https://www.withpower.com/clinical-trials/new-york">New york</a> City.

Phase 2

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 younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up year 5 visit pre-dose, 1, 2, 4, 6, 24 hours post-dose

Awards & highlights

SUNFISH Trial Summary

This trial is testing a new drug for people with Type 2 or Type 3 SMA. The first part is to see if it's safe and tolerable, and the second part is to see if it works.

Eligible Conditions

Spinal Muscular Atrophy

SUNFISH Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ year 5 visit pre-dose, 1, 2, 4, 6, 24 hours post-dose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~year 5 visit pre-dose, 1, 2, 4, 6, 24 hours post-dose

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 5 visit pre-dose, 1, 2, 4, 6, 24 hours post-dose 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: Selected Part 2 Dose of Risdiplam for Participants With BW of <20kg

Part 1: Selected Part 2 Dose of Risdiplam for Participants With a Body Weight (BW) of >/=20kg

Part 2: Change From Baseline in the Total Motor Function Measure 32 (MFM-32) Total Score at Month 12

Secondary outcome measures

Median Fold Change From Baseline in Survival of Motor Neuron (SMN) Protein Levels in Blood

Part 1 and 2: Area Under the Curve (AUC) From 0 to 24 Hours of Risdiplam at Year 5 Visit

Part 1 and 2: Concentration at the End of a Dosing Interval (Ctrough) of Risdiplam at Year 5

+27 more

Side effects data

From 2023 Phase 2 trial • 231 Patients • NCT02908685

22%

Nasopharyngitis

15%

Upper respiratory tract infection

13%

Pyrexia

13%

Vomiting

10%

Cough

10%

Headache

Diarrhoea

Gastroenteritis

Pneumonia

Respiratory tract infection

Abdominal pain

Bronchitis

Pharyngitis

Rash

Ear pain

Nausea

Influenza like illness

Ear infection

Influenza

Constipation

Limb injury

Arthralgia

Back pain

Oropharyngeal pain

Eczema

Rhinorrhoea

Contusion

Gastrointestinal infection

Urinary tract infection

Gastritis

Sinusitis

Arthropod bite

Pruritus

Erythema

Decreased appetite

Dry skin

Pain in extremity

Encephalitis

Brain contusion

Herpes zoster

Pneumonia mycoplasmal

Dehydration

Hypersensitivity

Conjunctivitis

Cystitis

Groin infection

Rhinitis

Scarlet fever

Tonsillitis

Device related infection

Infective thrombosis

Pyelonephritis

Viral upper respiratory tract infection

Partial seizures

Haematuria

Nephrolithiasis

Atelectasis

Pneumonitis aspiration

Pneumothorax

Tachycardia

Ocular hyperaemia

Abdominal pain upper

Dizziness

Amenorrhoea

Dysmenorrhoea

Nasal congestion

Asthma

Productive cough

Acne

Alopecia

Blister

Dermatitis

Seborrhoeic dermatitis

Aphthous ulcer

Rhinitis allergic

Epistaxis

Post procedural infection

Asthenia

Neck pain

100%

80%

60%

40%

20%

Study treatment Arm

Part 2 OLT: Risdiplam/Risdiplam

Part 2 OLT: Placebo/Risdiplam

Part 2 OLE: Risdiplam

Part 1 Group B: Children (0.25 mg/kg Risdiplam)

Part 1 Group A: Adolescents and Adults (3 mg Risdiplam)

Part 1 Group A: Adolescents and Adults (5 mg Risdiplam)

Part 1 Group B: Children (Placebo-Control Period Pooled)

Part 1 Group A: Adolescents and Adults (Placebo-Control Period Pooled)

Part 1 Group B: Children (0.02 mg/kg Risdiplam)

Part 1 Group B: Children (0.05 mg/kg Risdiplam)

Part 1 Group B: Children (0.15 mg/kg Risdiplam)

Part 1 Group A: OLE

Part 1 Group B: OLE

Part 2 Placebo-Controlled: Risdiplam

Part 2 Placebo-Controlled: Placebo

SUNFISH Trial Design

6Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2: RisdiplamExperimental Treatment1 Intervention

Participants aged 2-25 years will receive risdiplam at the dose selected based on the results from Part 1 of the study (5 mg once daily for participants with a body weight (BW) >/=20kg or 0.25 mg/kg for participants with a BW <20 kg), for 24 months. After 24-month treatment, participants will be offered the opportunity to enter the open-label phase.

Group II: Part 1 Group B: Children (Risdiplam)Experimental Treatment1 Intervention

Children aged 2-11 years will receive risdiplam for at least 12 weeks. Once the placebo-controlled period is completed and Part 2 dose is selected, participants will be switched to Part 2 dose and will be treated in an open-label phase.

Group III: Part 1 Group A: Adolescents and Adults (Risdiplam)Experimental Treatment1 Intervention

Adolescent and adult participants aged 12-25 years will receive risdiplam for at least 12 weeks. Once the placebo-controlled period is completed and Part 2 dose is selected, participants will be switched to Part 2 dose and will be treated in an open-label phase.

Group IV: Part 1 Group A: Adolescents and Adults (Placebo)Placebo Group2 Interventions

Adolescent and adult participants aged 12-25 years will receive placebo matching to risdiplam for at least 12 weeks. Once placebo-controlled period is completed, participants will be first switched to their cohort risdiplam dose. After the Part 2 dose is selected, participants will be switched to Part 2 dose and will be treated in an open-label phase.

Group V: Part 1 Group B: Children (Placebo)Placebo Group2 Interventions

Children aged 2-11 years will receive placebo matching to risdiplam for at least 12 weeks. Once placebo-controlled period is completed, participants will be first switched to their cohort risdiplam dose. After the Part 2 dose is selected, participants will be switched to Part 2 dose and will be treated in an open-label phase.

Group VI: Part 2: PlaceboPlacebo Group2 Interventions

Participants aged 2-25 years will receive placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants will be switched to risdiplam (5 mg once daily for participants with a body weight (BW) >/=20kg or 0.25 mg/kg for participants with a BW <20) in a blinded manner and participants will continue with treatment until Month 24. After Month 24, participants will be offered the opportunity to enter the open-label phase.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Risdiplam

2016

Completed Phase 2

~420

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor

2,432 Previous Clinical Trials

1,089,655 Total Patients Enrolled

Clinical TrialsStudy DirectorHoffmann-La Roche

2,201 Previous Clinical Trials

888,372 Total Patients Enrolled

Frequently Asked Questions

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

Does this research project include geriatric patients in its sample size?

"Children aged 2 to 25 can participate in this clinical trial. In total, there are 53 clinical trials underway for patients under 18 years old and 151 for patients over 65."

Answered by AI

Are recruitment efforts ongoing for this clinical trial?

"The trial, which was first posted on 10/20/2016, is not recruiting patients at this time according to clinicaltrials.gov. However, there are 211 other trials that are currently looking for participants."

Answered by AI

Are there a lot of hospitals running this clinical trial in Canada?

"This particular trial is being conducted at Columbia University Medical Center, The Neurological Institute of New york, Alberta Children's Hospital Division of Pediatric Neurology, and McGill University Health Centre - Glen Site. In addition, there are 5 other undisclosed locations."

Answered by AI

What is the patient volume for this research?

"This study is not recruiting patients presently. The trial was initially posted on October 20th, 2016 and was last updated on August 31st, 2022. If the reader is looking for other trials, there are 205 trials actively recruiting participants with muscular atrophy and 6 studies for Risdiplam actively recruiting patients."

Answered by AI

What makes this research project unique?

"Risdiplam has been under research since 2016 when the first study, sponsored by Hoffmann-La Roche, was completed with 231 participants. After the successful Phase 2 & 3 drug approval, 6 more active studies involving Risdiplam have popped up in 22 different cities and countries."

Answered by AI

Can you give some examples of other investigations that have used Risdiplam?

"Risdiplam was first studied in the year 2016 at Jichi Medical University Hospital. So far, there have been 18243 completed trials. Currently, there are 6 live studies, a significant amount of which are based in New york City."

Answered by AI

Recent research and studies

The Lancet NeurologyJournal

Safety and Efficacy of Once-daily Risdiplam in Type 2 and Non-ambulant Type 3 Spinal Muscular Atrophy (SUNFISH Part 2): A Phase 3, Double-blind, Randomised, Placebo-controlled Trial

Mercuri, Eugenio, Nicolas Deconinck, Elena S Mazzone, Andres Nascimento, Maryam Oskoui, Kayoko Saito, Carole Vuillerot, et al.. 2022. “Safety and Efficacy of Once-daily Risdiplam in Type 2 and Non-ambulant Type 3 Spinal Muscular Atrophy (SUNFISH Part 2): A Phase 3, Double-blind, Randomised, Placebo-controlled Trial”. The Lancet Neurology. Elsevier BV. doi:10.1016/s1474-4422(21)00367-7.

The Lancet NeurologyJournal

Safety and Efficacy of Once-daily Risdiplam in Type 2 and Non-ambulant Type 3 Spinal Muscular Atrophy (SUNFISH Part 2): A Phase 3, Double-blind, Randomised, Placebo-controlled Trial

The Lancet NeurologyJournal

Safety and Efficacy of Once-daily Risdiplam in Type 2 and Non-ambulant Type 3 Spinal Muscular Atrophy (SUNFISH Part 2): A Phase 3, Double-blind, Randomised, Placebo-controlled Trial

clinicaltrials.govStudy

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

2016. "A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02908685.