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SMN2 Splicing Modifier

Risdiplam for Spinal Muscular Atrophy (HINALEA 1 Trial)

Phase 4

Recruiting

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 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Drug Has Already Been Approved

Summary

This trial aims to test the effectiveness and safety of risdiplam as an early treatment for young children with spinal muscular atrophy who have a specific genetic makeup.

Who is the study for?

This trial is for children under 2 years old with spinal muscular atrophy (SMA) who have two SMN2 gene copies and received onasemnogene abeparvovec. They should not have had a significant decline in function since receiving the gene therapy between 3 to 7 months before joining.

What is being tested?

The study tests risdiplam's effectiveness and safety as an early treatment for SMA in kids who've already had gene therapy. It's an open-label, single-arm trial, meaning all participants receive risdiplam without being compared to a placebo or other treatment.

What are the potential side effects?

While specific side effects are not listed here, risdiplam may cause reactions that can vary from child to child. These could include issues related to how the body functions due to SMA progression or responses to medication.

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

Atelectasis

Nephrolithiasis

Encephalitis

Brain contusion

Ocular hyperaemia

Neck pain

Dysmenorrhoea

Infective thrombosis

Amenorrhoea

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.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups

Experimental Treatment

Group I: RisdiplamExperimental Treatment1 Intervention

Participants will receive risdiplam orally once daily for 72 weeks (Treatment Period). The Treatment Period will be followed by a 1-year Treatment Extension Period for a total study duration of 120 weeks (approximately 2.5 years) for each participant enrolled.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

risdiplam

2021

Completed Phase 1

~140

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