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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 as per respiratory event on day 10 and day 20 post-event (up to week 120)

Awards & highlights

HINALEA 1 Trial 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.Check my eligibility

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.See study design

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.

HINALEA 1 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ as per respiratory event on day 10 and day 20 post-event (up to week 120)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~as per respiratory event on day 10 and day 20 post-event (up to week 120)

This trial's timeline: 3 weeks for screening, Varies for treatment, and as per respiratory event on day 10 and day 20 post-event (up to week 120) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment

Secondary outcome measures

Percentage of Participants With Adverse Events

Percentage of Participants With Serious Adverse Events

Percentage of Participants With Treatment Discontinuation Due to Adverse Events

Other outcome measures

Change from Baseline in Bulbar/Swallowing Function Assessment as Measured by the Oral and Swallowing Abilities Tool (OrSAT) at 72 Weeks of Risdiplam Treatment and Over Time

Change from Baseline in the Raw Score of BSID-III Gross Motor Score Over Time Under Risdiplam Treatment

Change in Swallowing Function Assessment as Measured by the Pediatric Functional Oral Intake Scale (p-FOIS) at 72 Weeks of Risdiplam Treatment and Over Time

+8 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

Rhinorrhoea

Eczema

Contusion

Gastrointestinal infection

Urinary tract infection

Gastritis

Sinusitis

Arthropod bite

Decreased appetite

Pain in extremity

Dry skin

Pruritus

Erythema

Brain contusion

Rhinitis allergic

Herpes zoster

Pneumonia mycoplasmal

Dehydration

Asthenia

Hypersensitivity

Conjunctivitis

Cystitis

Groin infection

Rhinitis

Scarlet fever

Tonsillitis

Device related infection

Encephalitis

Infective thrombosis

Pyelonephritis

Viral upper respiratory tract infection

Partial seizures

Haematuria

Nephrolithiasis

Atelectasis

Pneumonitis aspiration

Pneumothorax

Tachycardia

Ocular hyperaemia

Abdominal pain upper

Aphthous ulcer

Dizziness

Amenorrhoea

Dysmenorrhoea

Nasal congestion

Asthma

Epistaxis

Productive cough

Acne

Alopecia

Blister

Dermatitis

Seborrhoeic dermatitis

Neck pain

Post procedural 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

HINALEA 1 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

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

1,090,133 Total Patients Enrolled

Clinical TrialsStudy DirectorHoffmann-La Roche

2,201 Previous Clinical Trials

888,850 Total Patients Enrolled

Frequently Asked Questions

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

Are there opportunities for patient enrollment in the ongoing recruitment phase of this clinical trial?

"Affirmative. As per clinicaltrials.gov, this ongoing trial commenced on February 28th, 2024 and was most recently updated on February 14th of the same year. This study aims to enroll a total of 28 participants at one designated site."

Answered by AI

What is the current number of individuals actively involved in this medical study?

"Indeed, clinicaltrials.gov data confirms the ongoing patient recruitment for this trial. Initially posted on February 28th, 2024, and last updated on February 14th of the same year, the study aims to recruit a total of 28 patients from one designated site."

Answered by AI

Who would be considered the ideal candidates to participate in this research investigation?

"This clinical study aims to enroll 28 infants diagnosed with spinal muscular atrophy between the ages of 3 and 24 months. Key inclusion criteria involve being under two years old at the time consent is given, a confirmed diagnosis of 5q-autosomal recessive SMA, presence of two SMN2 gene copies, administration of onasemnogene abeparvovec either before or after symptoms appear, prior treatment with onasemnogene abeparvovec for SMA lasting between 3 to 7 months before enrollment, and no substantial functional decline post-treatment according to the investigator's assessment."

Answered by AI

What are the potential risks associated with Risdiplam when used by patients?

"Our research team at Power has assessed the safety of Risdiplam as 3 on a scale of 1 to 3. This rating is due to the Phase 4 trial status, indicating that this therapy has gained regulatory approval."

Answered by AI

Is the research trial open to individuals aged 65 and above?

"Participants eligible for this research must be between 3 and 24 months old. Among the trials available, there are 49 studies tailored to those under 18 years old and 130 designed specifically for individuals above the age of retirement."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

2024. "A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05861986.

All > Spinal Muscular Atrophy