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Antisense Oligonucleotide

nusinersen for Spinal Muscular Atrophy (SHINE Trial)

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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to day 1814
Awards & highlights

SHINE Trial Summary

This trial is looking at the long-term effects of a drug given to people with Spinal Muscular Atrophy. The drug is given by injection into the spine, and the researchers are looking at safety and how well the drug works.

Eligible Conditions
  • Spinal Muscular Atrophy

SHINE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day 1814
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 1814 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 concomitant medications
Number of participants experiencing Adverse events (AEs) and/or Serious Adverse Events (SAEs)
Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
+5 more
Secondary outcome measures
Change from Baseline in 6-Minute Walk Test (6MWT)
Change from Baseline in Cobb-Angle on X-Ray of the thoracolumbar spine
Change from Baseline in Compound Muscular Action Potential (CMAP)
+21 more

Side effects data

From 2017 Phase 2 trial • 21 Patients • NCT01839656
75%
Pyrexia
75%
Respiratory tract infection
75%
Joint contracture
50%
Acute respiratory failure
50%
Respiratory distress
50%
Diarrhoea
50%
Teething
50%
Vomiting
50%
Oedema peripheral
50%
Torticollis
50%
Cough
50%
Dermatitis diaper
50%
Chronic respiratory failure
50%
Scoliosis
50%
Kyphosis
25%
Speech disorder developmental
25%
Blister
25%
Dehydration
25%
High arched palate
25%
Plagiocephaly
25%
Haematemesis
25%
Discomfort
25%
Arthropod bite
25%
Deformity thorax
25%
Nasal congestion
25%
Ecchymosis
25%
Corona virus infection
25%
Pneumonia bacterial
25%
Lymphadenopathy
25%
Ventricular tachycardia
25%
Cryptorchism
25%
Cerumen impaction
25%
Astigmatism
25%
Abdominal distension
25%
Constipation
25%
Flatulence
25%
Gastrointestinal hypomotility
25%
Tooth crowding
25%
Seasonal allergy
25%
Candida nappy rash
25%
Nasopharyngitis
25%
Parainfluenzae virus infection
25%
Pharyngitis streptococcal
25%
Pneumonia
25%
Pneumonia pseudomonas aeruginosa
25%
Pneumonia viral
25%
Respiratory syncytial virus bronchiolitis
25%
Excoriation
25%
Joint dislocation
25%
Procedural pain
25%
Soft tissue injury
25%
Sunburn
25%
Hyperkalaemia
25%
Hypophagia
25%
Malnutrition
25%
Weight gain poor
25%
Trismus
25%
Dyspnoea
25%
Rhinitis allergic
25%
Dermatitis contact
25%
Eczema
25%
Erythema
25%
Heat rash
25%
Asphyxia
25%
Bradycardia
25%
Tachycardia
25%
Upper respiratory tract infection
25%
Thermal burn
25%
Urinary retention postoperative
25%
Bacterial test positive
25%
Weight decreased
25%
Abdominal pain
25%
Otitis media
25%
Bronchial secretion retention
25%
Osteopenia
25%
Haemangioma
25%
Headache
25%
Muscle contractions involuntary
25%
Nystagmus
25%
Rash
25%
Skin disorder
25%
Skin irritation
25%
Essential hypertension
100%
80%
60%
40%
20%
0%
Study treatment Arm
Nusinersen 6 mg
Nusinersen 12 mg

SHINE Trial Design

5Treatment groups
Experimental Treatment
Group I: Group 5Experimental Treatment1 Intervention
Participants transitioned from 232SM202 (NCT02462759)
Group II: Group 4Experimental Treatment1 Intervention
Participants transitioned from ISIS 396443-CS3A (NCT01839656)
Group III: Group 3Experimental Treatment1 Intervention
Participants transitioned from ISIS 396443-CS12 (NCT02052791)
Group IV: Group 2Experimental Treatment1 Intervention
Participants transitioned from ISIS 396443-CS4 (NCT02292537)
Group V: Group 1Experimental Treatment1 Intervention
Participants transitioned from ISIS 396443-CS3B (NCT02193074)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
nusinersen
2014
Completed Phase 3
~440

Find a Location

Who is running the clinical trial?

BiogenLead Sponsor
639 Previous Clinical Trials
467,144 Total Patients Enrolled
Medical DirectorStudy DirectorBiogen
2,777 Previous Clinical Trials
8,063,188 Total Patients Enrolled

Frequently Asked Questions

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

Are there comparable medical studies to nusinersen?

"Research Site was the first to study nusinersen back in 2015. So far, there have been 9 completed studies with 7 more active clinical trials. A majority of these trials take place in Aurora, Colorado."

Answered by AI

Are participants currently being sought for this trial?

"This study is not actively recruiting patients at the moment, according to the latest update on November 7th, 2021. However, if you are looking for other trials, there are 206 trials for muscular atrophy and 7 trials for nusinersen that are currently looking for participants."

Answered by AI

What makes this experiment novel in the medical field?

"First researched in 2015, nusinersen has had a long journey to approval. Biogen sponsored the first study in 2015, which only involved 25 patients. After this Phase 1 trial, nusinersen received drug approval for Phase 2. Presently, there are 7 live studies being conducted in 70 different cities and 31 nations."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Research site
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
2015. "A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02594124.
~31 spots leftby Apr 2025
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