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Antisense Oligonucleotide

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

Phase 3

Recruiting

Research Sponsored by Biogen

Eligibility Criteria Checklist

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

Must have

RULM entry item A score ≥3

RULM total score ≥5 and ≤30 at Screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to day 1695

Awards & highlights

ASCEND Trial Summary

This trial is testing a new drug for spinal muscular atrophy and its effects on motor function. The safety and tolerability of the drug will also be monitored.

Who is the study for?

This trial is for individuals aged 15-50 with a confirmed genetic diagnosis of spinal muscular atrophy (SMA), weighing over 20 kg, and able to perform certain functional assessments. Participants must have been previously treated with risdiplam but are willing to stop it and start high-dose nusinersen treatment. They should not be able to walk independently for 15 feet, not have severe illnesses or conditions that could affect the study, nor be pregnant or breastfeeding.Check my eligibility

What is being tested?

The trial is testing whether a higher dose of nusinersen can improve motor function in patients who have SMA and were previously treated with risdiplam. It will also assess the safety and tolerability of this increased dosage regimen.See study design

What are the potential side effects?

While specific side effects for high-dose nusinersen are being studied, common ones may include back pain from injections, respiratory infections due to weakened muscles, headache, constipation or nausea. Serious side effects might involve kidney damage or bleeding complications.

ASCEND Trial Eligibility Criteria

Inclusion Criteria

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

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My upper limb mobility score is 3 or higher.

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My RULM score is between 5 and 30.

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I am willing to stop taking risdiplam.

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I have been taking risdiplam as prescribed or through a special program.

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I have been treated with risdiplam for at least 6 months and have not used nusinersen.

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My SMA symptoms started after I was 6 months old.

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I weigh more than 20 kilograms.

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

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I stopped taking nusinersen over 16 months ago and have been on risdiplam for more than 12 months.

Select...

I cannot walk 15 feet on my own without help.

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I am between 15 and 50 years old.

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I am ready and can start treatment with HD nusinersen.

ASCEND Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to day 1695

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to day 1695

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 1695 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 in Total Revised Upper Limb Module (RULM) Score

Secondary outcome measures

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Electrocardiogram

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

ASCEND Trial Design

1Treatment groups

Experimental Treatment

Group I: Higher Dose NusinersenExperimental Treatment1 Intervention

All participants in the core study period, previously treated with risdiplam (nusinersen-naive participants and nusinersen-experienced participants), will receive HD nusinersen, administered as 2 loading doses of 50 milligrams (mg) each, approximately 2 weeks apart, followed by maintenance doses of 28 mg approximately every 4 months. Following the core study period, participants may be given the opportunity to receive maintenance doses of 28 mg nusinersen administered approximately every 4 months up to 2 years during the optional long-term extension (LTE) period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Nusinersen

2014

Completed Phase 3

~200

0 / 12Eligibility criteria met

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor

639 Previous Clinical Trials

467,277 Total Patients Enrolled

Medical DirectorStudy DirectorBiogen

2,777 Previous Clinical Trials

8,063,819 Total Patients Enrolled

Media Library

Nusinersen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT05067790 — Phase 3

Spinal Muscular Atrophy Research Study Groups: Higher Dose Nusinersen

Spinal Muscular Atrophy Clinical Trial 2023: Nusinersen Highlights & Side Effects. Trial Name: NCT05067790 — Phase 3

Nusinersen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05067790 — Phase 3

Frequently Asked Questions

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

What other scientific research has been conducted on Nusinersen?

"Nusinersen was first researched in 2015. To date, there have been 9 completed clinical trials with 7 active ones currently underway; a majority of these are based in Chicago, Illinois."

Answered by AI

Does this research only include individuals 80 and older?

"The age requirements for this clinical trial are 15-50. If you don't meet that age bracket, there are still options; there are 55 studies for people younger and 156 studies for those who are older."

Answered by AI

Are there any participating hospitals in Canada or the United States?

"There are a dozen sites conducting this clinical trial, with locations in Chicago, Iowa City and New york. If you enroll in the study, aim to choose a site near your home to cut down on travel time and costs."

Answered by AI

Are there any available positions for patients in this study?

"That is correct. The latest information on clinicaltrials.gov suggests that this trial began searching for patients on 1/21/2022 and is still actively recruiting. They are looking to enroll 135 total participants across 12 different sites."

Answered by AI

How many test subjects are a part of this experiment?

"As 135 patients that meet the clinical trial's requirements are needed to run this study, sponsor Biogen will be opening up different sites across America. For example, Ann & Robert H. Lurie Children's Hospital of Chicago in Illinois and University of Iowa Children's Hospital in Iowa City, Iowa."

Answered by AI

Has this type of clinical trial been conducted before?

"Nusinersen has been researched since 2015, when the first trial was conducted by Biogen. After the initial study with 25 participants, Nusinersen received Phase 2 approval. As of today, there are 7 active studies being performed in 70 cities and 31 countries."

Answered by AI

Are there any recorded cases of Nusinersen being harmful to humans?

"Nusinersen has received a score of 3, due to the fact that it is in Phase 3 trials. This means that there is efficacy data as well as multiple rounds of safety data supporting its use."

Answered by AI

Who would be an ideal candidate for this research project?

"This clinical trial is looking for 135 people who have atrophy and meet the following additional criteria: they must be between the ages of 15 and 50, they must have genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation, they must be diagnosed with later-onset SMA with symptom onset at age >6 months, they must weigh more than 20 kg, they must have received oral risdiplam per the approved label or per the managed access program as follows: Nusinersen-naive participants must have had prior"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam

2022. "A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05067790.

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