Condition or disease | Intervention/treatment |
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Spinal Muscular Atrophy | Genetic: AVXS-101 |
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.
Study Type : | Expanded Access |
Expanded Access Type : | Treatment IND/Protocol |
See clinical trials of the intervention/treatment in this expanded access record. | |
Official Title: | 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 |
Tracking Information | |
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First Submitted Date | May 15, 2019 |
First Posted Date | May 20, 2019 |
Last Update Posted Date | August 28, 2020 |
Descriptive Information | |
Brief Title | AveXis Managed Access Program Cohort for Access to AVXS-101 |
Brief 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. |
Study Type | Expanded Access |
Expanded Access Type | Treatment IND/Protocol |
Intervention | 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.
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |
Expanded Access Status | Approved for marketing |
Contacts | Contact information is only displayed when the study is recruiting subjects |
Listed Location Countries | United States |
Removed Location Countries | |
Administrative Information | |
NCT Number | NCT03955679 |
Responsible Party | United BioSource, LLC |
Study Sponsor | United BioSource, LLC |
Collaborators | AveXis, Inc. |
Investigators | Not Provided |
PRS Account | United BioSource, LLC |
Verification Date | August 2020 |