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Splicing Modifier

Risdiplam for Spinal Muscular Atrophy (Jewelfish 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

For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures and agreement to refrain from donating sperm

Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previous treatment with Nusinersen, Olesoxime, or AVXS-101

Must not have

History of malignancy if not considered cured

Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is exploring the safety and effectiveness of a new drug for Spinal Muscular Atrophy, a rare disease that causes muscle weakness.

Who is the study for?

This trial is for adults, children, and infants with Spinal Muscular Atrophy who have been part of a previous study or treated with specific drugs. Women must not be pregnant and agree to contraception; men also need to use birth control. Participants should be well enough in the investigator's opinion, and young children may need feeding tubes or ventilation as recommended.

What is being tested?

The trial tests Risdiplam's safety, tolerability, how it moves through and affects the body (PK/PD) in those with SMA previously enrolled in a related study or treated with certain other medications. It's an exploratory open-label study without comparison groups.

What are the potential side effects?

Risdiplam can potentially cause side effects such as sensitivity reactions due to its ingredients, issues affecting vision based on past ophthalmological diseases history exclusion criteria, and possibly gastrointestinal disturbances since tube placement might be needed.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I agree to not have sex or use birth control and not donate sperm.

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I have been part of the Moonfish study or treated with Nusinersen, Olesoxime, or AVXS-101.

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I agree to either not have sex or use birth control, and not donate sperm.

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I have been diagnosed with 5q-autosomal recessive SMA.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of cancer that is not considered cured.

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I haven't had a serious illness or fever in the last month.

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I have heart issues shown on an ECG or have cardiovascular disease.

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I have previously used chloroquine, hydroxychloroquine, retigabin, vigabatrin, or thioridazine.

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My child under 2 was hospitalized for a lung problem recently and hasn’t fully recovered.

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I am over 6 years old and at high risk for suicidal behavior.

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I haven't taken any drugs affecting FMO1 or FMO3 enzymes in the last 2 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2023 Phase 2 trial • 231 Patients • NCT02908685

22%

Nasopharyngitis

15%

Upper respiratory tract infection

13%

Vomiting

13%

Pyrexia

10%

Headache

10%

Cough

Diarrhoea

Gastroenteritis

Pneumonia

Respiratory tract infection

Abdominal pain

Bronchitis

Pharyngitis

Rash

Ear pain

Nausea

Back pain

Arthralgia

Influenza

Limb injury

Oropharyngeal pain

Rhinorrhoea

Eczema

Constipation

Influenza like illness

Ear infection

Contusion

Erythema

Urinary tract infection

Sinusitis

Arthropod bite

Gastritis

Decreased appetite

Pain in extremity

Dry skin

Pruritus

Gastrointestinal infection

Ocular hyperaemia

Infective thrombosis

Encephalitis

Brain contusion

Nephrolithiasis

Atelectasis

Neck pain

Amenorrhoea

Dysmenorrhoea

Pneumonia mycoplasmal

Rhinitis

Scarlet fever

Tonsillitis

Dehydration

Device related infection

Herpes zoster

Post procedural infection

Pyelonephritis

Viral upper respiratory tract infection

Partial seizures

Haematuria

Pneumonitis aspiration

Pneumothorax

Dizziness

Nasal congestion

Rhinitis allergic

Tachycardia

Asthma

Epistaxis

Productive cough

Acne

Alopecia

Blister

Dermatitis

Abdominal pain upper

Aphthous ulcer

Asthenia

Seborrhoeic dermatitis

Hypersensitivity

Conjunctivitis

Cystitis

Groin infection

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: RisdiplamExperimental Treatment1 Intervention

Participants will receive multiple doses of risdiplam orally once daily for 24 months. After 24-month treatment, participants will be offered the opportunity to enter the open-label extension (OLE) phase for 3 years.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Risdiplam

2016

Completed Phase 2

~420

0 / 11Eligibility criteria met