ClinicalTrials.Veeva

Menu

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam (STRENGTH)

Novartis logo

Novartis

Status and phase

Active, not recruiting
Phase 3

Conditions

Spinal Muscular Atrophy

Treatments

Genetic: OAV101

Study type

Interventional

Funder types

Industry

Identifiers

NCT05386680
COAV101B12302

Details and patient eligibility

About

This is an open-label, single arm, multi-center study. Approximately 28 participants aged 2 to <18 years will be enrolled stratified as 2 to 5 years and 6 to < 18 years. The study is comprised of 3 periods, Screening (up to 45 days), Treatment (1 day), and Follow-up (52 weeks).

Full description

During the Screening period and on Day -1 (Baseline), eligibility will be assessed, including confirmation that nusinersen (Spinraza®) or risdiplam (Evrysdi®) have not been used for the defined period (4 month and 15 days prior to Day -1 respectively). On Day - 1 (Baseline) participants will be admitted to the hospital for pre-treatment baseline procedures. Prednisolone (or equivalent) will be given and continued as per the study protocol.

Participants who meet eligibility criteria at Screening and Baseline will receive a single dose of OAV101 (1.2 x 10^14 vector genomes) by lumbar intrathecal injection on Day 1 (Treatment) and will then undergo in-patient safety monitoring atleast the next 48 hours, after which the participant may be discharged according to Investigator judgement.

During Follow-up, safety monitoring will continue as per the visits defined in the Assessment Schedule. Safety for participants enrolled will be evaluated by the study team together with the Data Monitoring Committee (DMC).

Final analysis will be performed after all participants have completed Week 52 or discontinued prior to Week 52. At the end of study, participants will be invited to enroll in a Novartis-sponsored long-term follow-up study to monitor long-term safety and efficacy.

Enrollment

27 patients

Sex

All

Ages

2 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

  • SMA diagnosis
  • Aged 2 to < 18 years
  • Have had at least four loading doses of nusinersen (Spinraza®) or at least 3 months of treatment with risdiplam (Evrysdi®) at Screening
  • Must have symptoms of SMA as defined in the protocol

Exclusion Criteria:

  • Anti Adeno Associated Virus Serotype 9 (AAV9) antibody titer using an immunoassay is reported as elevated

  • Clinically significant abnormalities in test results during screening

  • Contraindications for lumbar puncture procedure

  • At Baseline, participants are excluded if they received:

    • nusinersen (Spinraza®) or
    • risdiplam (Evrysdi®) within a defined timeframe
  • Vaccinations 2 weeks prior to administration of OAV101

  • Hospitalization for a pulmonary event, or for nutritional support within 2 months prior to Screening or inpatient major surgery planned.

  • Presence of an infection or febrile illness up to 30 days prior to administration of OAV101

  • Requiring invasive ventilation

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

27 participants in 1 patient group

OAV-101
Experimental group
Description:
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose
Treatment:
Genetic: OAV101

Trial contacts and locations

13

Loading...

Central trial contact

Novartis Pharmaceuticals; Novartis Pharmaceuticals

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location
© Copyright 2024 Veeva Systems