Clinical trial NCT05335876: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Official title: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Spinal Muscular Atrophy (SMA)
Keywords: Zolgensma
OAV101
AVXS 101
gene therapy
Muscle atrophy
SBMA
spinal and bulbar muscular atrophy
spinal muscular atrophy
bulbar muscular atrophy
muscle function
myopathy
muscle wasting
atrophied muscle
loss of muscle strength
Spinal Muscular Atrophy (SMA)
survival motor neuron 1 gene (SMN1)
SMN protein depletion
survival motor neuron 2 gene (SMN2)
chromosome 5q13
neurogenetic disorder
onasemnogene abeparvovec
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-04-20
Enrollment: 175
Sex: ALL
Minimum age (years): 0
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.

Detailed description: The study is comprised of a Baseline Visit and 2 Follow-up Periods. For Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. For the first 2 years (Follow-up Period 1), visits will occur every 6 months. For Years 3 to 5 (Follow-up Period 2) follow-up visits will be conducted annually. All patients will enter the study at the baseline visit and continue for 5 years.

Eligibility criteria: 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.

Exclusion Criteria:

There are no exclusion criteria for this study.

Primary outcomes:
measure: Number of participants with treatment-emergent serious adverse events (SAEs)
timeFrame: Up to Year 5
measure: Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
timeFrame: Up to Year 5
Secondary outcomes:
measure: The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist
timeFrame: Up to Year 5
measure: The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist
timeFrame: Up to Year 5
measure: Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
timeFrame: Up to Year 5
measure: Change from Baseline in the Revised Upper Limb Module (RULM) total score
timeFrame: Up to Year 5
measure: Systolic and diastolic blood pressure (mmHg)
timeFrame: Up to Year 5
measure: Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)
timeFrame: Up to Year 5
measure: Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)
timeFrame: Up to Year 5
measure: Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)
timeFrame: Up to Year 5
measure: Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).
timeFrame: Up to Year 5
Other outcomes:
Locations:
city: Norfolk
country: United States
facility: Child Hosp Of The Kings Daughters
state: Virginia
city: Randwick
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Leuven
country: Belgium
facility: Novartis Investigative Site
city: Montreal
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Chongqing
country: China
facility: Novartis Investigative Site
state: Chongqing
city: Guangzhou
country: China
facility: Novartis Investigative Site
state: Guangdong
city: Chengdu
country: China
facility: Novartis Investigative Site
state: Sichuan
city: Beijing
country: China
facility: Novartis Investigative Site
city: Beijing
country: China
facility: Novartis Investigative Site
city: Copenhagen
country: Denmark
facility: Novartis Investigative Site
city: Bron Cedex
country: France
facility: Novartis Investigative Site
city: Garches
country: France
facility: Novartis Investigative Site
city: Strasbourg
country: France
facility: Novartis Investigative Site
city: Toulouse Cedex
country: France
facility: Novartis Investigative Site
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Kurume city
country: Japan
facility: Novartis Investigative Site
state: Fukuoka
city: Shinjuku ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Kuala Lumpur
country: Malaysia
facility: Novartis Investigative Site
city: Kuala Lumpur
country: Malaysia
facility: Novartis Investigative Site
city: Utrecht
country: Netherlands
facility: Novartis Investigative Site
city: Riyadh
country: Saudi Arabia
facility: Novartis Investigative Site
city: Singapore
country: Singapore
facility: Novartis Investigative Site
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Kaohsiung
country: Taiwan
facility: Novartis Investigative Site
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
city: Bangkok
country: Thailand
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
city: Newcastle Upon Tyne
country: United Kingdom
facility: Novartis Investigative Site
city: Hanoi
country: Vietnam
facility: Novartis Investigative Site
Study officials:
References: