Spinal Muscular Atrophy (SMA)Phase 3SPECTRUMNCT05335876
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
A Phase 3 study testing onasemnogene abeparvovec in people with Spinal Muscular Atrophy (SMA), run by Novartis Pharmaceuticals at 32 sites in 19 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 100 years
- Conditions the study is for
- Spinal Muscular Atrophy (SMA)
- What is being tested?
- onasemnogene abeparvovec
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 32 sitesin 19 countries
- Study dates
- 19 Dec 2022to 30 Apr 2031
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participated in an OAV101 clinical trial.
- Written informed consent must be obtained before any assessment is performed.
- Patient/Parent/legal guardian willing and able to comply with study procedures.
Who cannot join
- There are no exclusion criteria for this study.
Trial locations and contacts
32 sites in 19 countries.
32 sites in 19 countries
China6 sites
Novartis Investigative Site
Beijing, 100034
Novartis Investigative Site
Beijing, 100069
Novartis Investigative Site
Beijing, 100730
Novartis Investigative Site
Chengdu, Sichuan, 610041
Novartis Investigative Site
Chongqing, Chongqing Municipality, 400010
Novartis Investigative Site
Guangzhou, Guangdong, 510623
France4 sites
Novartis Investigative Site
Bron, 69677
Novartis Investigative Site
Garches, 92380
Novartis Investigative Site
Strasbourg, 67000
Novartis Investigative Site
Toulouse, 31059
Brazil2 sites
Novartis Investigative Site
Curitiba, Paraná, 81520-060
Novartis Investigative Site
São Paulo, São Paulo, 05403 000
Japan2 sites
Novartis Investigative Site
Kurume, Fukuoka, 830-0011
Novartis Investigative Site
Shinjuku Ku, Tokyo, 1628666
Malaysia2 sites
Novartis Investigative Site
Kuala Lumpur, 50300
Novartis Investigative Site
Kuala Lumpur, 59100
Taiwan2 sites
Novartis Investigative Site
Kaohsiung City, 80756
Novartis Investigative Site
Taipei, 10002
United Kingdom2 sites
Novartis Investigative Site
London, WC1N 3JH
Novartis Investigative Site
Newcastle upon Tyne, NE1 4LP
United States1 site
Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507
About this study
Official title: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
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.
Read the 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.
- Conditions
- Spinal Muscular Atrophy (SMA)
- Being tested
- onasemnogene abeparvovec
- 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
- Medical subject headings
- Muscular Atrophy, Spinal, Muscular Atrophy, Bulbo-Spinal Atrophy, X-Linked, Muscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
onasemnogene abeparvovecBiological
Also called Zolgensma
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.
| Study group | Role | What they receive |
|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec | Experimental | Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302) Receives: Biological: onasemnogene abeparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent serious adverse events (SAEs)
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
Measured: Up to Year 5
Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
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.
Measured: Up to Year 5
Secondary outcomes (9)
The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist
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.
Measured: Up to Year 5
The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist
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.
Measured: Up to Year 5
Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
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.
Measured: Up to Year 5
Change from Baseline in the Revised Upper Limb Module (RULM) total score
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.
Measured: Up to Year 5
Systolic and diastolic blood pressure (mmHg)
Measured: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)
Measured: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)
Measured: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)
Measured: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).
Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.
Measured: Up to Year 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2022
- Study start19 Dec 2022
- Primary completion29 Apr 2031 (estimated)
- Study completion30 Apr 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy (SMA)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05335876
- Sponsor's protocol ID
- COAV101A12308
- Acronym
- SPECTRUM
- Status last verified
- Jul 2026
- First submitted
- 12 Apr 2022
- Last updated
- 9 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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/.
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
- ClinicalTrials.gov ID
- NCT05335876
- Acronym / sponsor ID
- SPECTRUM / COAV101A12308
- Conditions
- Spinal Muscular Atrophy (SMA)
- Keywords
- Zolgensma, OAV101, AVXS 101, gene therapy, Muscle atrophy, SBMA, spinal and bulbar muscular atrophy, spinal muscular atrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 19 Dec 2022 (actual)
- Primary completion
- 29 Apr 2031 (estimated)
- Study completion
- 30 Apr 2031 (estimated)
- First posted
- 20 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec | Experimental | Biological: onasemnogene abeparvovecPatients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302) |
Who may qualify
- Ages
- 0 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Participated in an OAV101 clinical trial.
- Written informed consent must be obtained before any assessment is performed.
- Patient/Parent/legal guardian willing and able to comply with study procedures.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| onasemnogene abeparvovecAlso: ZolgensmaOnasemnogene 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. | Biological | Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec |
Primary outcome
Number of participants with treatment-emergent serious adverse events (SAEs)
Time frame: Up to Year 5
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
Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
Time frame: Up to Year 5
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.
9 secondary outcomes
The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist
Time frame: Up to Year 5
The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist
Time frame: Up to Year 5
Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
Time frame: Up to Year 5
Change from Baseline in the Revised Upper Limb Module (RULM) total score
Time frame: Up to Year 5
Systolic and diastolic blood pressure (mmHg)
Time frame: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)
Time frame: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)
Time frame: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)
Time frame: Up to Year 5
Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).
Time frame: Up to Year 5
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2026.
- Individual participant dataSharing planned: Yes. 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/.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
32 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing onasemnogene abeparvovec in people with Spinal Muscular Atrophy (SMA), run by Novartis Pharmaceuticals at 32 sites in 19 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in China, France, Brazil, Japan, Malaysia and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-emergent serious adverse events (SAEs) (time frame: Up to Year 5). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2026.