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SMN2 Splicing Modifier

Risdiplam for Spinal Muscular Atrophy (HINALEA 2 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 postevent (up to week 120)

Awards & highlights

HINALEA 2 Trial Summary

This trial aims to test the effectiveness and safety of risdiplam in young children with spinal muscular atrophy (SMA) who have not responded well to a previous treatment. The study will focus

Who is the study for?

This trial is for children under 2 years old with a genetic condition called SMA and have two SMN2 gene copies. They must have had gene therapy before but are not improving or getting worse. The child should be able to swallow and show changes in breathing, moving, or other abilities.Check my eligibility

What is being tested?

The study tests Risdiplam's effectiveness and safety in young kids with SMA who stopped getting better after receiving gene therapy. It's an open-label trial, meaning everyone knows they're getting Risdiplam, without comparing it to another treatment.See study design

What are the potential side effects?

While the specific side effects of Risdiplam in this context aren't listed here, common ones may include fever, diarrhea, rash, joint pain, urinary tract infections; however individual experiences can vary.

HINALEA 2 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

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

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~as per respiratory event on day 10 and day 20 postevent (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 postevent (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

Dry skin

Pruritus

Erythema

Pain in extremity

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

Post procedural infection

Neck pain

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 2 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.

Has the drug Risdiplam received approval from the FDA?

"Our team at Power has evaluated the safety of Risdiplam to be 3 on our scale, as this trial is categorized as Phase 4 indicating that the treatment has gained approval."

Answered by AI

Are there any available positions for patients in this ongoing clinical trial?

"As per clinicaltrials.gov, this investigation is actively seeking participants. The trial was first listed on 4/20/2024 and its most recent edit was made on 3/12/2024."

Answered by AI

What is the current number of individuals receiving medical care as part of this research project?

"Indeed, the information available on clinicaltrials.gov highlights that this investigation is presently seeking suitable candidates. The trial was initially publicized on April 20th, 2024 and its latest update occurred on March 12th, 2024. This study aims to enroll a total of 28 participants from only one designated location."

Answered by AI

Is it feasible for me to participate in this clinical trial?

"This clinical trial is enrolling 28 infants diagnosed with spinal muscular atrophy aged between 3 months and 24 months. Notably, candidates must fulfill the subsequent conditions: younger than two years old when providing informed consent, confirmed diagnosis of 5q-autosomal recessive SMA, presence of two SMN2 gene copies confirmed, treatment with onasemnogene abeparvovec either before symptoms appear or after they manifest, initiation of onasemnogene abeparvovec therapy for SMA no less than three months prior to enrollment. Furthermore, according to the investigator's judgment should showcase a stagnation or decrease in"

Answered by AI