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Small Molecule

Risdiplam for Spinal Muscular Atrophy (PUPFISH Trial)

Phase 2
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
Male or female newborn infant aged <20 days at first dose
Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing
Must not have
Presence of clinical symptoms or signs consistent with SMA Type 0
Concurrent or previous administration of nusinersen or onasemnogene abeparvovec
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

"This trial will look at how the drug risdiplam is processed in the bodies of infants under 20 days old with spinal muscular atrophy (SMA), as well as how safe it is

Who is the study for?
This trial is for newborn infants under 20 days old diagnosed with spinal muscular atrophy (SMA) or identified as SMA-positive through screening. They must be born after a full-term pregnancy, well-nourished, hydrated, and recovered from any acute illness. Parents should be open to tube feeding if recommended.
What is being tested?
The study tests Risdiplam's pharmacokinetics—how the drug moves into, through, and out of the body—and safety in infants with SMA. It aims to understand how young patients process this medication.
What are the potential side effects?
While specific side effects for Risdiplam in this age group are not detailed here, common ones may include fever, diarrhea, rash or other allergic reactions. Close monitoring will ensure infant safety during the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My newborn is less than 20 days old.
Select...
My newborn has been diagnosed with 5q-autosomal recessive SMA.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I show symptoms of SMA Type 0.
Select...
I have been treated with nusinersen or onasemnogene abeparvovec.

Timeline

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
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%
Atelectasis
1%
Nephrolithiasis
1%
Encephalitis
1%
Brain contusion
1%
Ocular hyperaemia
1%
Neck pain
1%
Dysmenorrhoea
1%
Infective thrombosis
1%
Amenorrhoea
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.

Trial Design

1Treatment groups
Experimental Treatment
Group I: RisdiplamExperimental Treatment1 Intervention
Participants will receive risdiplam once daily for 28 days.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Risdiplam
2016
Completed Phase 2
~420

Find a Location

Who is running the clinical trial?

Hoffmann-La RocheLead Sponsor
2,458 Previous Clinical Trials
1,097,925 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,227 Previous Clinical Trials
896,412 Total Patients Enrolled
~6 spots leftby Aug 2025
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