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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05335876




Registration number
NCT05335876
Ethics application status
Date submitted
12/04/2022
Date registered
20/04/2022

Titles & IDs
Public title
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Scientific title
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Secondary ID [1] 0 0
COAV101A12308
Universal Trial Number (UTN)
Trial acronym
SPECTRUM
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy (SMA) 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 - onasemnogene abeparvovec

Experimental: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec - Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)


Treatment: Other: onasemnogene abeparvovec
Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken ß-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment-emergent serious adverse events (SAEs)
Timepoint [1] 0 0
Up to Year 5
Primary outcome [2] 0 0
Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
Timepoint [2] 0 0
Up to Year 5
Secondary outcome [1] 0 0
The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist
Timepoint [1] 0 0
Up to Year 5
Secondary outcome [2] 0 0
The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist
Timepoint [2] 0 0
Up to Year 5
Secondary outcome [3] 0 0
Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
Timepoint [3] 0 0
Up to Year 5
Secondary outcome [4] 0 0
Change from Baseline in the Revised Upper Limb Module (RULM) total score
Timepoint [4] 0 0
Up to Year 5
Secondary outcome [5] 0 0
Systolic and diastolic blood pressure (mmHg)
Timepoint [5] 0 0
Up to Year 5
Secondary outcome [6] 0 0
Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)
Timepoint [6] 0 0
Up to Year 5
Secondary outcome [7] 0 0
Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)
Timepoint [7] 0 0
Up to Year 5
Secondary outcome [8] 0 0
Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)
Timepoint [8] 0 0
Up to Year 5
Secondary outcome [9] 0 0
Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).
Timepoint [9] 0 0
Up to Year 5

Eligibility
Key inclusion criteria
1. Participated in an OAV101 clinical trial.
2. Written informed consent must be obtained before any assessment is performed.
3. Patient/Parent/legal guardian willing and able to comply with study procedures.
Minimum age
0 Years
Maximum age
100 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
There are no exclusion criteria for this study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
26/06/2030
Actual
Sample size
Target
175
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Novartis Investigative Site - Randwick
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Virginia
Country [2] 0 0
Belgium
State/province [2] 0 0
Leuven
Country [3] 0 0
Canada
State/province [3] 0 0
Quebec
Country [4] 0 0
China
State/province [4] 0 0
Chongqing
Country [5] 0 0
China
State/province [5] 0 0
Guangdong
Country [6] 0 0
China
State/province [6] 0 0
Sichuan
Country [7] 0 0
China
State/province [7] 0 0
Beijing
Country [8] 0 0
Denmark
State/province [8] 0 0
Copenhagen
Country [9] 0 0
France
State/province [9] 0 0
Bron Cedex
Country [10] 0 0
France
State/province [10] 0 0
Garches
Country [11] 0 0
France
State/province [11] 0 0
Strasbourg
Country [12] 0 0
France
State/province [12] 0 0
Toulouse Cedex
Country [13] 0 0
Italy
State/province [13] 0 0
RM
Country [14] 0 0
Japan
State/province [14] 0 0
Fukuoka
Country [15] 0 0
Japan
State/province [15] 0 0
Tokyo
Country [16] 0 0
Malaysia
State/province [16] 0 0
Kuala Lumpur
Country [17] 0 0
Netherlands
State/province [17] 0 0
Utrecht
Country [18] 0 0
Saudi Arabia
State/province [18] 0 0
Riyadh
Country [19] 0 0
Singapore
State/province [19] 0 0
Singapore
Country [20] 0 0
Spain
State/province [20] 0 0
Catalunya
Country [21] 0 0
Taiwan
State/province [21] 0 0
Kaohsiung
Country [22] 0 0
Taiwan
State/province [22] 0 0
Taipei
Country [23] 0 0
Thailand
State/province [23] 0 0
Bangkok
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Newcastle Upon Tyne
Country [26] 0 0
Vietnam
State/province [26] 0 0
Hanoi

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
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
medinfo.gtx@novartis.com
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/NCT05335876