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Virus Therapy

Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec 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 15

Awards & highlights

SPECTRUM 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.Check my eligibility

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.See study design

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.

SPECTRUM Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to year 15

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to year 15

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to year 15 for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Number of participants with treatment emergent Adverse Events of Special Interest (AESI)

Therapeutic procedure

Secondary outcome measures

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 - Respiratory Rate (breaths/min)

+6 more

SPECTRUM 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)

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor

2,863 Previous Clinical Trials

4,199,075 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 researchers currently enrolling participants for this medical study?

"Indeed, details on clinicaltrials.gov affirm the ongoing recruitment for this investigation. The trial was initially disclosed on December 19th, 2022 and most recently revised on April 2nd, 2024. There is a need to enroll 260 participants distributed among 15 research locations."

Answered by AI

Do several medical facilities within the city conduct this particular research study?

"At the moment, patient recruitment is ongoing at 15 diverse locations. These include Copenhagen, Norfolk, and Garches among others. Opting for a nearby site could reduce travel demands when participating in this study."

Answered by AI

For what medical purpose is Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec usually prescribed?

"Intravenous (IV) and Intrathecal (IT) administration of Onasemnogene Abeparvovec can address conditions related to antibiotics, infections, and disinfection."

Answered by AI

What are the risks associated with Intravenous (IV) & Intrathecal (IT) administrations of Onasemnogene Abeparvovec for individuals undergoing treatment?

"According to our assessment at Power, the safety rating for Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec is graded as 3 due to its Phase 3 trial status, signifying existing efficacy data and multiple sets of safety data."

Answered by AI

What additional research has been carried out on the use of IV and IT Onasemnogene Abeparvovec?

"At this moment, there are 14 ongoing research projects investigating the use of Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec. Among these trials, four fall under Phase 3 evaluation. While a significant number of investigation sites for Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec can be found in Kunming, Yunnan, a total of 561 locations globally are conducting studies on this therapeutic approach."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

2022. "Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05335876.

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