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AveXis Managed Access Program Cohort for Access to AVXS-101

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ClinicalTrials.gov Identifier: NCT03955679
Expanded Access Status : Approved for marketing
First Posted : May 20, 2019
Last Update Posted : August 28, 2020
Sponsor:
Collaborator:
AveXis, Inc.
Information provided by (Responsible Party):
United BioSource, LLC

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

Condition or disease Intervention/treatment
Spinal Muscular Atrophy Genetic: AVXS-101

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 Design
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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

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • 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.
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  1. Patients with SMA must satisfy both a. and b. of the following specified criteria:

    1. Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 1, 2, or 3 copies of SMN2.
    2. Either patients with onset of symptoms prior to < 6 months (< 180 days) of age or pre-symptomatic patients, less than 6 months of age, with 1, 2, or 3 copies of SMN2 who are not excluded for other reasons.
  2. Weight ≥ 2.6 kg to ≤ 13.5 kg at dosing.
  3. Patients must have a pre-treatment swallowing evaluation test performed prior to administration of AVXS-101.
  4. Patients must have a formal pulmonary evaluation including documentation of non-invasive ventilatory use prior to administration of AVXS-101. Ventilation should be actively managed by an appropriately trained specialist per the published standard of care.
  5. Up-to-date on childhood vaccinations. Seasonal vaccinations and palivizumab prophylaxis (also known as Synagis) to prevent respiratory syncytial virus (RSV) infections have been administered as recommended by the American Academy of Pediatrics.
  6. Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule.

Exclusion Criteria:

  1. Tracheostomy.
  2. Contraindication to receiving glucocorticosteroids or their excipients.
  3. Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer > 1:50 (or any value reported as elevated for the laboratory) as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay. Should a potential patient demonstrate AntiAAV9 antibody titer > 1:50, he or she may be retested and will be eligible to participate if the AntiAAV9 antibody titer upon retesting is ≤ 1:50.
  4. Clinically significant abnormal laboratory values for troponin-I, and platelets. ALT, AST, bilirubin or gamma glutamyl transferase (GGT) > 2 x the upper limit of normal (ULN) prior to gene replacement therapy that in the judgment of the Treating Physician or AveXis would create too great a risk for the patient to be treated with AVXS-101 or prophylactic prednisolone. Note: Elevated bilirubin > 2 x ULN if associated with neonatal jaundice is not considered exclusionary.
  5. Medical conditions, diagnoses (especially cardiac), or on concurrent medications prior to gene replacement therapy that in the judgment of the treating physician or sponsor would create too great a risk for the patient to be treated with AVXS-101 or prophylactic prednisolone.
  6. Participation or expected participation in current treatment clinical study (with the exception of observational cohort studies or non-interventional studies) for an unapproved investigational agent.
  7. Parent(s)/legal guardian(s) unwilling to keep study results/observations confidential or to refrain from posting confidential study results/observations on social media sites.
  8. Parent(s)/legal guardian(s) refuses to sign consent form.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03955679


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
United BioSource, LLC
AveXis, Inc.
More Information
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Additional Information:

Publications:

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Responsible Party: United BioSource, LLC
ClinicalTrials.gov Identifier: NCT03955679    
Other Study ID Numbers: AVXS-101-MAP-001
First Posted: May 20, 2019    Key Record Dates
Last Update Posted: August 28, 2020
Last Verified: August 2020
Keywords provided by United BioSource, LLC:
Spinal Muscular Atrophy
SMA
Gene replacement therapy
AVXS-101
 Compassionate Use
Managed Access Program
MAP
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
 Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases