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Antisense Oligonucleotide

Nusinersen for SMA (RESPOND Trial)

Phase 4

Waitlist Available

Research Sponsored by Biogen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must have received IV onasemnogene abeparvovec after SMA symptom onset

SMN2 copy number of ≥1

Must not have

Ongoing severe or serious AEs related to onasemnogene abeparvovec

Prior exposure to Nusinersen

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to day 778

Awards & highlights

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial is evaluating nusinersen as a treatment for SMA in patients who have previously received onasemnogene abeparvovec. The objectives are to assess the safety and efficacy of nusinersen in this population.

Who is the study for?

This trial is for children under 36 months with Spinal Muscular Atrophy who have a specific genetic mutation and at least one copy of the SMN2 gene. They must have previously received onasemnogene abeparvovec, not been exposed to Nusinersen before, and can't be suffering from severe side effects related to onasemnogene abeparvovec.

What is being tested?

The study tests the effectiveness of Nusinersen in young patients with SMA who've already been treated with onasemnogene abeparvovec. It aims to see if additional treatment improves clinical outcomes and assesses safety and tolerability.

What are the potential side effects?

While specific side effects for this trial are not listed, common ones associated with Nusinersen include respiratory issues, constipation, headache, back pain, and potential post-lumbar puncture syndrome.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I received IV onasemnogene abeparvovec after showing symptoms of SMA.

Select...

My genetic test shows I have at least one copy of the SMN2 gene.

Select...

I have received onasemnogene abeparvovec treatment before.

Select...

I am 3 years old or younger when I first received Nusinersen.

Select...

I have a confirmed genetic form of SMA due to SMN1 changes.

Select...

I received onasemnogene abeparvovec treatment over 2 months ago.

Select...

I received IV onasemnogene abeparvovec when I was 6 weeks old or younger.

Select...

My SMA symptoms started before I was 4 months old.

Select...

My child is less than 9 months old.

Select...

My genetic test shows I have 2 copies of the SMN2 gene.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am experiencing severe side effects from onasemnogene abeparvovec treatment.

Select...

I have previously been treated with Nusinersen.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to day 778

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to day 778

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 778 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score

Secondary study objectives

Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score

Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score

Change from Baseline in Revised Upper Limb Module (RULM) Score

+3 more

Side effects data

From 2017 Phase 3 trial • 126 Patients • NCT02292537

45%

Upper respiratory tract infection

36%

Pyrexia

36%

Nasopharyngitis

21%

Cough

19%

Gastroenteritis

17%

Rhinorrhoea

17%

Joint contracture

14%

Pneumonia

12%

Vomiting

12%

Ear infection

10%

Constipation

10%

Bronchitis

10%

Gastroenteritis viral

10%

Otitis media

10%

Arthralgia

Diarrhoea

Pharyngitis streptococcal

Pain in extremity

Scoliosis

Headache

Faecaloma

Influenza

Dehydration

Respiratory distress

Conjunctivitis

Upper respiratory tract congestion

Pneumonia adenoviral

Respiratory syncytial virus infection

Respiratory failure

Pneumonia parainfluenzae viral

100%

80%

60%

40%

20%

Study treatment Arm

Sham Procedure

Nusinersen

Awards & Highlights

Drug Has Already Been Approved

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

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.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Nusinersen 12 mgExperimental Treatment1 Intervention

Participants will receive Nusinersen 12 milligrams (mg) via intrathecal (IT) injection as loading doses on Days 1, 15, 29, and 64 followed by maintenance doses, every 4 months, on Days 183, 302, 421, 540 and 659.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Nusinersen

2014

Completed Phase 3

~340

0 / 12Eligibility criteria met