Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 125

Inclusion Criteria

- Diagnostic confirmation during screening period of 5q SMA
- The patient must be treatment naive (historical or current use) for all SMN-targeting therapies (e.g., risdiplam (Evrysdi) and nusinersen (Spinraza)).
- = 2 years and < 18 years of age at screening visit 1
- Onset of clinical signs and symptoms at = 6 months of age
- Patient must have a complete HFMSE assessment with an available total score, as administered by qualified clinical evaluator during the screening period for trial eligibility
- Able to sit independently at screening, but has never had the ability to walk independently.
- Estimated life expectancy > 2 years from screening, in the opinion of the Investigator
- Meets age-appropriate institutional criteria for use of anesthesia/sedation as assessed by the physician responsible for administering anesthesia/sedation

(Full list of inclusion criteria can be found in the clinical protocol Section 5.1)
Are the trial subjects under 18? yes
Number of subjects for this age range: 125
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Excluding SMA, any medical condition considered clinically significant by the Investigator, including cardiomyopathy, hepatic dysfunction, kidney disorder, endocrine disorder including diabetes
mellitus, gastrointestinal disorders, metabolic disorders, untreated B6 deficiency, severe respiratory compromise and significant brain abnormalities at either Screening or Baseline that, in the opinion of the investigator, would interfere with the overall interpretation of safety or efficacy of the study.
- Anti-AAV9 antibody titers >1:50 at Screening as determined by ligand binding immunoassay. NOTE: A negative anti-AAV9 antibody titer is defined as =1:50.
- Clinically significant abnormalities in test results during screening period and/or during baseline period as determined by the Investigator
- Participants will be excluded from the trial for inpatient surgery hospitalization, hospitalization for a pulmonary event, or hospitalization for nutritional support within 2 months prior to Screening and up to Day 1. In addition, patients will not be eligible for the trial if at Screening, inpatient major surgery is planned at any time during the 64-week study. Any other surgeries must not interfere with the ability of the patient to perform the study assessments.
- Prior injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the participant's ability to perform any of the outcome measure testing required in the protocol and from which the participant has not fully recovered or achieved a stable baseline upon entry into screening period
- Contraindications for lumbar puncture procedure, (including but not limited to cutaneous infection at the treatment site and signs or symptoms of increased intracranial pressure), active administration of any intrathecal therapy, presence of an implanted shunt for the drainage of CSF, presence of an implanted central nervous system (CNS) catheter, or any impediment to CSF access.

(Full list of exclusion criteria can be found in the clinical protocol Section
5.2)

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials

Randomized, placebo-controlled, double-blind trial of intrathecal (IT) OAV101 administration in patients with later onset Type 2 spinal muscular atrophy (SMA), to evaluate the efficacy and safety.

Phase 3

ovartis Healthcare Pvt Ltd

Updated 1/22/2024