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Antisense Oligonucleotide

High-Dose Nusinersen for Spinal Muscular Atrophy (DEVOTE Trial)

Q: Where is this research being conducted?

<p>Children's hospitals in Vancouver, <a href="https://www.withpower.com/clinical-trials/colorado">Colorado</a>, and Memphis are some of the locations where this study is enrolling patients. Including these 10 other sites, a total of 13 medical centres are participating.</p>

Phase 3

Waitlist Available

Research Sponsored by Biogen

Eligibility Criteria Checklist

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

Must have

Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote)

Participants ≥18 years of age at Screening (can be ambulatory or nonambulatory) with HFMSE total score ≥4 points at Screening and RULM entry item A score ≥3 points at Screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 to day 302

Awards & highlights

DEVOTE Trial Summary

This trial is testing a higher dose of nusinersen, a drug used to treat spinal muscular atrophy (SMA). The objectives are to see if the higher dose is more effective and safe than the current approved dose, as measured by the CHOP-INTEND score.

Who is the study for?

This trial is for individuals with spinal muscular atrophy (SMA). Eligible participants include those aged 2-10 years who can sit but not walk, have a specific score on the HFMSE test, and genetic confirmation of SMA. Infants up to 7 months old with symptom onset by 6 months are also eligible. Participants must be currently treated with nusinersen for at least one year prior to screening.Check my eligibility

What is being tested?

The study tests higher doses of Nusinersen given through spinal injection in people with SMA. It aims to see if these doses improve muscle function more than the standard dose, using a scale called CHOP-INTEND as a measure for children's motor skills.See study design

What are the potential side effects?

Potential side effects from higher doses of Nusinersen may include back pain or headache due to the injection method, respiratory issues like shortness of breath or coughing, constipation or nausea, and potential risk of kidney damage.

DEVOTE Trial Eligibility Criteria

Inclusion Criteria

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

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My condition is confirmed by genetic testing for 5q SMA.

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I am 18 or older and can move with or without help, with specific movement scores.

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I am between 2 and 9 years old.

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I have been on nusinersen treatment for over a year.

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I can sit by myself but I've never been able to walk on my own.

DEVOTE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 to day 302

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 to day 302

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 to day 302 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Part A and C Later-onset SMA: Change from Baseline in Ulnar Length

Part A and C: Change from Baseline in Activated Partial Thromboplastin Time (aPTT)

Part A and C: Change from Baseline in Body Length/Height

+17 more

Secondary outcome measures

Neuromuscular Diseases

Part A and B Later-onset SMA: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) Score

Part A and B Later-onset SMA: Change from Baseline in Pediatric Quality of Life Inventory™ (PedsQL)

+39 more

Side effects data

From 2017 Phase 3 trial • 126 Patients • NCT02292537

43%

Pyrexia

30%

Upper respiratory tract infection

29%

Headache

29%

Vomiting

25%

Cough

25%

Back pain

24%

Nasopharyngitis

11%

Gastroenteritis

10%

Gastroenteritis viral

10%

Influenza

10%

Diarrhoea

Ear infection

Bronchitis

Conjunctivitis

Rhinorrhoea

Epistaxis

Upper respiratory tract congestion

Otitis media

Constipation

Joint contracture

Pharyngitis streptococcal

Pneumonia

Arthralgia

Pain in extremity

Pneumonia viral

Scoliosis

Respiratory distress

Respiratory syncytial virus bronchitis

Post lumbar puncture syndrome

Abdominal distension

Pain

Bacteraemia

Parainfluenzae virus infection

Pneumonia aspiration

100%

80%

60%

40%

20%

Study treatment Arm

Nusinersen

Sham Procedure

DEVOTE Trial Design

4Treatment groups

Experimental Treatment

Active Control

Group I: 50/28 mg Randomized Treatment GroupExperimental Treatment1 Intervention

Part B: Participants with infantile- or later-onset SMA will receive loading doses of 50 mg of nusinersen intrathecally on Days 1 and 15 followed by maintenance doses of 28 mg on Days 135 and 279. Sham procedure will be administered on Days 29, 64 and 183.

Group II: 28/28 Milligram (mg) Safety GroupExperimental Treatment1 Intervention

Part A: Participants with later-onset SMA will receive loading doses of 28 mg of nusinersen intrathecally on Days 1, 15 and 29 followed by maintenance doses of 28 mg on Days 149 and 269.

Group III: 12/50/28 mg Titration GroupExperimental Treatment1 Intervention

Part C: Participants who have been receiving the approved dose of 12 mg for at least 1 year prior to entry, will receive a single bolus dose of 50 mg of nusinersen intrathecally on Day 1 (4 months after their most recent maintenance dose of 12 mg) followed by maintenance doses of 28 mg on Days 121 and 241.

Group IV: 12/12 mg Randomized Control GroupActive Control1 Intervention

Part B: Participants with infantile- or later-onset SMA will receive loading doses of 12 mg of nusinersen intrathecally on Days 1, 15, 29, and 64 followed by maintenance doses of 12 mg on Days 183 and 279. Sham procedure will be administered on Day 135.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Nusinersen

2014

Completed Phase 3

~200

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor

639 Previous Clinical Trials

467,291 Total Patients Enrolled

Medical DirectorStudy DirectorBiogen

2,777 Previous Clinical Trials

8,063,335 Total Patients Enrolled

Media Library

Nusinersen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04089566 — Phase 3

Spinal Muscular Atrophy Research Study Groups: 50/28 mg Randomized Treatment Group, 28/28 Milligram (mg) Safety Group, 12/12 mg Randomized Control Group, 12/50/28 mg Titration Group

Spinal Muscular Atrophy Clinical Trial 2023: Nusinersen Highlights & Side Effects. Trial Name: NCT04089566 — Phase 3

Nusinersen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04089566 — Phase 3

Frequently Asked Questions

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

Where is this research being conducted?

"Children's hospitals in Vancouver, Colorado, and Memphis are some of the locations where this study is enrolling patients. Including these 10 other sites, a total of 13 medical centres are participating."

Answered by AI

Are there any available openings for willing participants in this experiment?

"Yes, this information is accurate. The clinical trial was posted on March 26th, 2020 and has been actively recruiting patients since. The most recent update was on June 29th, 2022."

Answered by AI

Have medical professionals tried anything like this before?

"Nusinersen has been under investigation since 2015 when the first study, sponsored by Biogen, began. In 2015 there were 25 participants. After the Phase 2 drug approval, 7 clinical trials with Nusinersen are currently ongoing in 70 cities and 31 countries."

Answered by AI

Can you please provide me with a list of prior clinical trials that have used Nusinersen?

"Nusinersen was first studied in 2015. To date, 9 clinical trials have been completed with 7 more underway as of this writing. A large number of these live studies are located in Vancouver, BC."

Answered by AI