AveXis Managed Access Program Cohort for Access to AVXS-101

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03955679
• Date of registration: 15/05/2019
• Prospective Registration: Yes
• Primary sponsor: United BioSource, LLC
• Public title: AveXis Managed Access Program Cohort for Access to AVXS-101
• Scientific 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
• Date of first enrolment: August 7, 2019
• Recruitment status: Approved for marketing
• URL: https://clinicaltrials.gov/show/NCT03955679
• Study type: Expanded Access

Countries of recruitment

United States

Key inclusion & exclusion 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.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Spinal Muscular Atrophy

Intervention(s)

Genetic: AVXS-101

Secondary ID(s)

AVXS-101-MAP-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

AveXis, Inc.