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AveXis Managed Access Program Cohort for Access to AVXS-101

NCT03955679 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2020-08-28

No results posted yet for this study

Summary

The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.

Conditions

Interventions

GENETIC

AVXS-101

AVXS-101 is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken β-actin-hybrid promoter (CB). AVXS-101 will be administered as a one-time intravenous infusion over approximately 60 minutes. Dosage will be determined by the participants weight.

Sponsors & Collaborators

  • AveXis, Inc.

    collaborator INDUSTRY

  • United BioSource, LLC

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03955679 on ClinicalTrials.gov