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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05386680




Registration number
NCT05386680
Ethics application status
Date submitted
18/05/2022
Date registered
23/05/2022
Date last updated
19/01/2024

Titles & IDs
Public title
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
Scientific title
Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)
Secondary ID [1] 0 0
COAV101B12302
Universal Trial Number (UTN)
Trial acronym
STRENGTH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - OAV101

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


Treatment: Other: OAV101
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number and percentage of participants reporting AEs, related AEs, SAEs, and AESIs
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
Change from baseline to Week 52 visit in the HFMSE total score
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
Change from baseline to Week 52 visit in the RULM total Score
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
Change from baseline to Week 52 visit in Assessment of Caregiver Experience in ACEND instrument score
Timepoint [3] 0 0
52 weeks

Eligibility
Key inclusion 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
Minimum age
2 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/01/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
3/12/2024
Actual
Sample size
Target
Accrual to date
Final
27
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Virginia
Country [3] 0 0
United States of America
State/province [3] 0 0
Wisconsin
Country [4] 0 0
Belgium
State/province [4] 0 0
Leuven
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec
Country [6] 0 0
France
State/province [6] 0 0
Bron Cedex
Country [7] 0 0
France
State/province [7] 0 0
Toulouse Cedex
Country [8] 0 0
Italy
State/province [8] 0 0
RM
Country [9] 0 0
Japan
State/province [9] 0 0
Fukuoka
Country [10] 0 0
Japan
State/province [10] 0 0
Tokyo
Country [11] 0 0
Netherlands
State/province [11] 0 0
Utrecht
Country [12] 0 0
Spain
State/province [12] 0 0
Catalunya

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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).
Trial website
https://clinicaltrials.gov/study/NCT05386680
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05386680