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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
Treatment Details
Side effects data
From 2023 Phase 2 trial • 231 Patients • NCT0290868522%
Nasopharyngitis
15%
Upper respiratory tract infection
13%
Vomiting
13%
Pyrexia
10%
Headache
10%
Cough
9%
Diarrhoea
8%
Gastroenteritis
7%
Pneumonia
6%
Respiratory tract infection
6%
Abdominal pain
5%
Bronchitis
5%
Pharyngitis
5%
Rash
4%
Ear pain
3%
Nausea
3%
Back pain
3%
Arthralgia
3%
Influenza
3%
Limb injury
3%
Oropharyngeal pain
3%
Rhinorrhoea
3%
Eczema
3%
Constipation
3%
Influenza like illness
3%
Ear infection
2%
Contusion
2%
Erythema
2%
Urinary tract infection
2%
Sinusitis
2%
Arthropod bite
2%
Gastritis
2%
Decreased appetite
2%
Pain in extremity
2%
Dry skin
2%
Pruritus
2%
Gastrointestinal infection
1%
Ocular hyperaemia
1%
Infective thrombosis
1%
Encephalitis
1%
Brain contusion
1%
Nephrolithiasis
1%
Atelectasis
1%
Neck pain
1%
Amenorrhoea
1%
Dysmenorrhoea
1%
Pneumonia mycoplasmal
1%
Rhinitis
1%
Scarlet fever
1%
Tonsillitis
1%
Dehydration
1%
Device related infection
1%
Herpes zoster
1%
Post procedural infection
1%
Pyelonephritis
1%
Viral upper respiratory tract infection
1%
Partial seizures
1%
Haematuria
1%
Pneumonitis aspiration
1%
Pneumothorax
1%
Dizziness
1%
Nasal congestion
1%
Rhinitis allergic
1%
Tachycardia
1%
Asthma
1%
Epistaxis
1%
Productive cough
1%
Acne
1%
Alopecia
1%
Blister
1%
Dermatitis
1%
Abdominal pain upper
1%
Aphthous ulcer
1%
Asthenia
1%
Seborrhoeic dermatitis
1%
Hypersensitivity
1%
Conjunctivitis
1%
Cystitis
1%
Groin infection
100%
80%
60%
40%
20%
0%
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
Find a Location
Who is running the clinical trial?
Hoffmann-La RocheLead Sponsor
2,463 Previous Clinical Trials
1,103,066 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,233 Previous Clinical Trials
902,195 Total Patients Enrolled