Trials / Approved For Marketing
Approved For MarketingNCT03955679
AveXis Managed Access Program Cohort for Access to AVXS-101
A Managed Access Program (MAP) Cohort Treatment Protocol to Provide AVXS-101 to Patients With a Genetic Diagnosis of Spinal Muscular Atrophy (SMA) With 1, 2 or 3 Copies of SMN2
- Status
- Approved For Marketing
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- United BioSource, LLC · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- —
Summary
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.
Detailed description
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA. The requesting Physician submits a request for access to drug (often referred to as Compassionate Use) to AveXis which is reviewed and approved by the medical team experienced with the drug and indication. The requesting Physician should refer to the latest Investigator's Brochure (IB) or approved label for overview of drug including: nonclinical and clinical experience, risk and benefits.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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. |
Timeline
- First posted
- 2019-05-20
- Last updated
- 2020-08-28
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT03955679. Inclusion in this directory is not an endorsement.