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Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)

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ClinicalTrials.gov Identifier: NCT05335876
Recruitment Status : Recruiting
First Posted : April 20, 2022
Last Update Posted : April 4, 2024
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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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 15 years from the date of OAV101 administration in the previous clinical trial.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy (SMA) Biological: onasemnogene abeparvovec Phase 3

Detailed Description:
The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1 and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101 administration), participants/caregivers will be contacted using tele-visits annually for remote assessments. All patients will enter the study at the baseline visit and continue until 15 years since OAV101 administration is reached. Total duration of participation in the study will be dependent upon time of enrollment relative to OAV101 administration and will vary by participant.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 260 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Actual Study Start Date : December 19, 2022
Estimated Primary Completion Date : October 18, 2039
Estimated Study Completion Date : October 18, 2039

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec
Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)
 Biological: 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 β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
Other Name: Zolgensma


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with treatment-emergent serious adverse events (SAEs) [ Time Frame: Up to Year 15 ]

    An SAE is defined as any adverse event [appearance of (or worsening of any pre-existing)] undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:

    • fatal
    • life-threatening
    • results in persistent or significant disability/incapacity
    • constitutes a congenital anomaly/birth defect, fetal death or congenital abnormality or birth defect
    • requires in-patient hospitalization or prolongation of existing hospitalization, unless hospitalization is for routine treatment or monitoring of the studied indication, not associated with any deterioration in condition
    • is medically significant, e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above

  2. Number of participants with treatment emergent Adverse Events of Special Interest (AESI) [ Time Frame: Up to Year 15 ]
    The following are important identified and important potential risks (AESI) associated with OAV101: Hepatotoxicity, Transient Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These will be assessed by the investigator.


Secondary Outcome Measures :
  1. The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist [ Time Frame: Up to Year 5 ]
    The Developmental Milestone Checklist is a sponsor created list of items using relevant definitions obtained from World Health Organization Multicentre Growth Reference Study (WHO-MGRS). These will be assessed via the milestone checklist, formed of 6 yes/no questions. The developmental milestones are: sitting with support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.

  2. The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist [ Time Frame: Up to Year 5 ]
    The Developmental Milestone Checklist is a sponsor created list of items using relevant definitions obtained from World Health Organization Multicentre Growth Reference Study (WHO-MGRS). These will be assessed via the milestone checklist, formed of 6 yes/no questions. The developmental milestones are: sitting with support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone.

  3. Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score [ Time Frame: Up to Year 5 ]
    The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.

  4. Change from Baseline in the Revised Upper Limb Module (RULM) total score [ Time Frame: Up to Year 5 ]
    The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability.

  5. Systolic and diastolic blood pressure (mmHg) [ Time Frame: Up to Year 15 ]
  6. Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min) [ Time Frame: Up to Year 15 ]
  7. Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min) [ Time Frame: Up to Year 15 ]
  8. Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius) [ Time Frame: Up to Year 15 ]
  9. Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%). [ Time Frame: Up to Year 15 ]
    Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05335876


Contacts
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Contact: Novartis Gene Therapies Med Info (US, Latin America, Canada) +1-833-828-3947 medinfo.gtx@novartis.com
Contact: Novartis Gene Therapies Med Info (Europe, Middle East, Africa, Asia-Pacific) +353 (1) 566-2364 medinfoemea.gtx@novartis.com

Locations
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United States, Virginia
Child Hosp of the Kings Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Erin Smith    757-668-9026    erin.smith@chkd.org   
Principal Investigator: Crystal Proud         
Australia, New South Wales
Novartis Investigative Site Recruiting
Randwick, New South Wales, Australia, 2031
Belgium
Novartis Investigative Site Recruiting
Leuven, Belgium, 3000
Canada, Quebec
Novartis Investigative Site Recruiting
Montreal, Quebec, Canada, H4A 3J1
Denmark
Novartis Investigative Site Recruiting
Copenhagen, Denmark, 2100 O
France
Novartis Investigative Site Recruiting
Garches, France, 92380
Novartis Investigative Site Recruiting
Strasbourg, France, 67000
Italy
Novartis Investigative Site Recruiting
Roma, RM, Italy, 00168
Japan
Novartis Investigative Site Recruiting
Kurume city, Fukuoka, Japan, 830-0011
Singapore
Novartis Investigative Site Recruiting
Singapore, Singapore, 119074
Spain
Novartis Investigative Site Recruiting
Barcelona, Catalunya, Spain, 08035
Taiwan
Novartis Investigative Site Recruiting
Kaohsiung, Taiwan, 80756
Novartis Investigative Site Recruiting
Taipei, Taiwan, 10002
United Kingdom
Novartis Investigative Site Recruiting
London, United Kingdom, WC1N 3JH
Novartis Investigative Site Recruiting
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05335876    
Other Study ID Numbers: COAV101A12308
First Posted: April 20, 2022    Key Record Dates
Last Update Posted: April 4, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

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/.
URL: https://www.clinicalstudydatarequest.com/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
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
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
 Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases