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Small Molecule

Risdiplam for Spinal Muscular Atrophy (FIREFISH Trial)

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 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months (up to the ccod of 12 november 2020)

Awards & highlights

FIREFISH Trial Summary

This trial is testing a new drug for safety, side effects, and effectiveness in treating Infants with Spinal Muscular Dystrophy. The study will last for 2 years and will test different doses of the drug to find the best one.

Who is the study for?

This trial is for infants with Type 1 spinal muscular atrophy (SMA) who are between 37 to 42 weeks gestational age, have symptoms of SMA starting after 28 days but before three months old, and weigh in the third percentile or higher. They must not have severe respiratory issues, recent major illnesses, invasive ventilation needs, or a history of certain drug treatments.Check my eligibility

What is being tested?

The study tests Risdiplam's safety and effectiveness in treating Type 1 SMA over a period of two parts: initial dose finding followed by a confirmatory phase using the selected dose for up to two years. It aims to understand how the body processes the drug and its impact on disease progression.See study design

What are the potential side effects?

While specific side effects for Risdiplam are not listed here, common concerns may include potential reactions at varying degrees such as digestive disturbances, liver function changes, sensitivity reactions or impacts on growth and development due to medication intake.

FIREFISH Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from first dose of risdiplam up to a minimum of 24 months (ccod of 12 november 2020)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from first dose of risdiplam up to a minimum of 24 months (ccod of 12 november 2020)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from first dose of risdiplam up to a minimum of 24 months (ccod of 12 november 2020) 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

Part 2: Percentage of Infants Who Are Sitting Without Support for at Least 5 Seconds as Assessed by Item 22 of the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) at Month 12

Secondary outcome measures

Part 1 and Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Part 2: Anthropometric Examination of Height, Head Circumference and Chest Circumference Measured in Centimeters

Part 2: Anthropometric Examination of Weight Measured in Kilograms

+29 more

Side effects data

From 2023 Phase 2 trial • 231 Patients • NCT02908685

100%

Pyrexia

57%

Cough

43%

Bronchitis

43%

Upper respiratory tract infection

43%

Nasopharyngitis

43%

Diarrhoea

43%

Gastroenteritis

29%

Ligament sprain

29%

Ear pain

29%

Pain in extremity

29%

Vomiting

29%

Respiratory tract infection

29%

Headache

14%

Abdominal pain

14%

Chronic respiratory failure

14%

Gastrointestinal infection

14%

Myalgia

14%

Malaise

14%

Influenza

14%

Urinary tract infection

14%

Oropharyngeal pain

14%

Rhinorrhoea

14%

Eczema

14%

Ear infection

14%

Respiratory tract inflammation

14%

Palmar erythema

14%

Skin exfoliation

14%

Hand-foot-and-mouth disease

14%

Pollakiuria

14%

Decreased appetite

14%

Tonsillitis

14%

Nausea

14%

Rash

14%

Upper respiratory tract inflammation

14%

Dehydration

14%

Dizziness

14%

Dry skin

14%

Nasal dryness

14%

Hypoglycaemia

14%

Fatigue

14%

Abdominal pain upper

14%

Contusion

14%

Allergy to arthropod bite

14%

Femur fracture

14%

Constipation

14%

Erythema

14%

Musculoskeletal chest pain

14%

Eczema eyelids

14%

Limb injury

14%

Erythema infectiosum

100%

80%

60%

40%

20%

Study treatment Arm

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: Risdiplam

Part 2: Placebo

FIREFISH Trial Design

2Treatment groups

Experimental Treatment

Group I: Part 2 (Confirmatory): Risdiplam (RO7034067)Experimental Treatment1 Intervention

Participants will receive risdiplam (RO7034067), administered orally once daily at the dose defined in Part 1 of the study, for a duration of 24 months. They will thereafter enter a 3-year open-label extension phase and continue to receive risdiplam at the same dose.

Group II: Part 1 (Dose Finding): Risdiplam (RO7034067)Experimental Treatment1 Intervention

Participants will receive multiple ascending doses of risdiplam (RO7034067), administered orally once daily for a minimum of 4 weeks to select the dose for Part 2. During the first year of treatment, most participants will switch to the Part 2 dose. During the second year of treatment, all Part 1 participants will be receiving the Part 2 dose. They will thereafter enter a 3-year open-label extension phase and continue to receive risdiplam at the same dose.

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

2,428 Previous Clinical Trials

1,088,992 Total Patients Enrolled

Clinical TrialsStudy DirectorHoffmann-La Roche

2,199 Previous Clinical Trials

888,440 Total Patients Enrolled

Media Library

Risdiplam (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT02913482 — Phase 2

Spinal Muscular Atrophy Research Study Groups: Part 2 (Confirmatory): Risdiplam (RO7034067), Part 1 (Dose Finding): Risdiplam (RO7034067)

Spinal Muscular Atrophy Clinical Trial 2023: Risdiplam Highlights & Side Effects. Trial Name: NCT02913482 — Phase 2

Risdiplam (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02913482 — Phase 2

Spinal Muscular Atrophy Patient Testimony for trial: Trial Name: NCT02913482 — Phase 2

Frequently Asked Questions

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

Does this study have an age limit?

"This trial is available for infants that are at least 1 month old but no older than 7 months."

Answered by AI

Who would be most likely to benefit from taking part in this trial?

"This study is looking for 62 infant patients, who currently have muscular atrophy of the spine. The ideal candidate also meets the following requirements: a clinical history or symptoms attributable to Type 1 SMA that began after 28 days but before 3 months old, a gestational age between 37 and 42 weeks, confirmation of 5q-autosomal recessive SMA diagnosis, two survival motor neuron 2 (SMN2) gene copies as confirmed by central testing, body weight at or above the third percentile for age according to appropriate country-specific guidelines."

Answered by AI

How many sites are authorized to run this trial?

"Presently, this clinical trial is running at 4 sites. The sites are based in Palo Alto, Chicago and New york along with other locations 4 other locations. To minimize travel requirements, patients should enroll at the nearest location to them."

Answered by AI

How many people are being given this medication to test its effects?

"This study is not currently enrolling patients. The listing for this clinical trial was first posted on December 23rd, 2016 and was last edited on December 24th, 2021. There are 207 other trials recruiting patients with muscular atrophy and 6 studies actively looking for participants that specifically test Risdiplam."

Answered by AI

Can people with the qualifying condition participate in this research right now?

"This particular clinical trial is not looking for participants at this moment in time. It was initially posted on December 23rd, 2016 but has not been updated since December 24th, 2021. There are 213 other trials that are actively recruiting patients."

Answered by AI

Is this a groundbreaking clinical trial?

"Risdiplam has been under investigation since 2016 when the first clinical trial was conducted by Hoffmann-La Roche. After the successful Phase 1 study with 231 people, Risdiplam received approval for Phases 2 & 3. There are currently 6 active trials involving this drug that span 22 cities and 22 countries."

Answered by AI

What are some other ways that Risdiplam has been studied in the past?

"Presently, there are six ongoing clinical trials researching Risdiplam. Three of these active studies are in their third phase. The majority of investigations concerning Risdiplam's efficacy are based in Basel and Lombardia; however, 144 different locations worldwide are running similar research projects."

Answered by AI