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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

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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.clinicalstudydatarequest.com/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05386680