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Virus Therapy
OAV101 for Spinal Muscular Atrophy (SPECTRUM Trial)
Phase 3
Recruiting
Research Sponsored by Novartis Pharmaceuticals
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 year 5
Awards & highlights
All Individual Drugs Already Approved
Approved for 5 Other Conditions
No Placebo-Only Group
Pivotal Trial
Summary
This trial will study the long-term safety and effectiveness of OAV101 in patients who were part of a previous clinical trial. The study will last for 15 years from the date the patients received O
Who is the study for?
This trial is for patients who have previously taken part in a clinical study involving OAV101, a gene therapy for spinal muscular atrophy. Participants must provide written consent and be able to follow the study's procedures as directed by their doctor or legal guardian.
What is being tested?
The long-term safety and effectiveness of onasemnogene abeparvovec (OAV101) are being studied over a period of 15 years in individuals with spinal muscular atrophy who received this treatment during earlier trials.
What are the potential side effects?
While specific side effects are not listed here, they will monitor any long-term effects from the gene therapy OAV101 given in previous studies. This may include tracking any new health issues or changes related to the treatment.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to year 5
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to year 5
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
Therapeutic procedure
Secondary study objectives
Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
Change from Baseline in the Revised Upper Limb Module (RULM) total score
Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).
+2 moreAwards & Highlights
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
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: Intravenous (IV) & Intrathecal (IT) Onasemnogene AbeparvovecExperimental Treatment1 Intervention
Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)
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Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,921 Previous Clinical Trials
4,254,137 Total Patients Enrolled