Onasemnogene abeparvovec for Spinal muscular atrophy

Inclusion criteria

• {"criterion_text":"- Any patient with SMA who received AVXS-101 gene replacement therapy in a Novartis Pharma AG -sponsored clinical study"}
• {"criterion_text":"- Patient/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule"}

Exclusion criteria

• {"criterion_text":"- Patient/parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study"}

Primary endpoints

• {"endpoint_text":"- Number of participants who reach developmental milestones","definition_or_measurement_approach":"Count of participants who achieve pre-specified developmental milestones (assessed during visits)."}
• {"endpoint_text":"- Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score","definition_or_measurement_approach":"Change from baseline measured using the HFMSE scale."}
• {"endpoint_text":"- Change from baseline in Revised Upper Limb Module (RULM)","definition_or_measurement_approach":"Change from baseline measured using the RULM assessment."}
• {"endpoint_text":"- Change from baseline in Bayley Scales of Infant and Toddler Development (Bayley-III)","definition_or_measurement_approach":"Change from baseline measured using the Bayley-III developmental scale."}
• {"endpoint_text":"- Change from baseline in Cogstate Computerized Cognitive Battery","definition_or_measurement_approach":"Change from baseline measured using the Cogstate computerized cognitive battery."}
• {"endpoint_text":"- Change from baseline in Clinical Evaluation of Language Fundamentals (CELF-5)","definition_or_measurement_approach":"Change from baseline measured using the CELF-5 language assessment."}
• {"endpoint_text":"- Change from baseline in Assessment of Caregiver Experience with Neuromuscular Disease (ACEND)","definition_or_measurement_approach":"Change from baseline in caregiver-reported experience as measured by ACEND questionnaire."}
• {"endpoint_text":"- Number of participants who experience a clinically significant change from baseline in pulmonary assessment results and require ventilatory support","definition_or_measurement_approach":"Count of participants with clinically significant pulmonary assessment changes leading to ventilatory support requirement."}
• {"endpoint_text":"- Number of participants who experience swallowing dysfunction and require nutritional support","definition_or_measurement_approach":"Count of participants developing swallowing dysfunction requiring nutritional support."}
• {"endpoint_text":"- Number of participants who experience a clinically significant change from baseline in physical examination findings","definition_or_measurement_approach":"Count of participants with clinically significant changes on physical exam compared to baseline."}
• {"endpoint_text":"- Number of participants who experience a clinically significant change from baseline in vital signs measurements","definition_or_measurement_approach":"Count of participants with clinically significant changes in vital signs versus baseline."}
• {"endpoint_text":"- Change from baseline in height measurements","definition_or_measurement_approach":"Change from baseline in measured height."}
• {"endpoint_text":"- Change from baseline in weight measurements","definition_or_measurement_approach":"Change from baseline in measured weight."}
• {"endpoint_text":"- Number of participants who experience a clinically significant change from baseline in clinical laboratory assessments","definition_or_measurement_approach":"Count of participants with clinically significant laboratory changes compared to baseline."}
• {"endpoint_text":"- Number of participants who experience a clinically significant change from baseline in cardiac assessments","definition_or_measurement_approach":"Count of participants with clinically significant changes on cardiac assessments versus baseline (ECG/echo etc.)."}
• {"endpoint_text":"- Number of participants who experience a clinically significant change from baseline in observational phase questionnaire results","definition_or_measurement_approach":"Count of participants with clinically significant changes on observational phase questionnaires compared to baseline."}
• {"endpoint_text":"- Concomitant Medications","definition_or_measurement_approach":"Recording and summary of concomitant medications used during the observational phase."}
• {"endpoint_text":"- Any other treatment for SMA for the observational phase","definition_or_measurement_approach":"Recording of any other SMA-specific treatments received during the observational phase."}
• {"endpoint_text":"- Number of participants who experience at least one serious adverse event (SAE)","definition_or_measurement_approach":"Count of participants experiencing ≥1 SAE during follow-up."}
• {"endpoint_text":"- Number of participants who experience at least one adverse event of special interest (AESI)","definition_or_measurement_approach":"Count of participants experiencing ≥1 AESI during follow-up."}

France

Earliest CTIS Part Ii Submission Date

16-04-2024

Latest Decision Or Authorization Date

25-07-2024

Processing Time Days

100

Number Of Sites

1

Number Of Participants

3

Belgium

Earliest CTIS Part Ii Submission Date

18-04-2024

Latest Decision Or Authorization Date

26-06-2024

Processing Time Days

69

Number Of Sites

2

Number Of Participants

4

Italy

Earliest CTIS Part Ii Submission Date

18-04-2024

Latest Decision Or Authorization Date

02-07-2024

Processing Time Days

75

Number Of Sites

3

Number Of Participants

15

Primary sponsor

Full Name

Novartis Pharma AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Clinical trial management, monitoring, safety reporting (SUSAR reporting to CEC + investigational sites) and other sponsor duties (codes 1,12,15,2,8)

Name

Medidata Solutions Inc.

Responsibilities

Electronic data capture / data management (duties codes 6 and 7)

Third parties

• {"country":"United States","full_name":"EPL Pathology Archives LLC","duties_or_roles":"Biomarkers Samples Storage","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Chillibean Limited","duties_or_roles":"Editing and quality assurance of videos, video archiving","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"SGS France","duties_or_roles":"Biomarkers (SMN protein/Blood) Analysis","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Sponsor duties include codes 1,12,15 (SUSAR reporting to CEC + investigational sites), 2,8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Duties codes 6 and 7 (electronic data capture/data management functions)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis/review, Echo CG and ECG analysis/review","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labconnect LLC","duties_or_roles":"Clinical chemistry, Clinical haematology, Troponin I testing and Exploratory Biomarkers","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Travel coordination and expense management","organisation_type":"Hospital/Clinic/Other health care facility"}