Trial · NCT05335876
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
NCT05335876, Phase 3, Active not recruiting, Novartis Pharmaceuticals
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials. Trials, record NCT05335876. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05335876
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
- NCT number
- NCT05335876
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 20 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- onasemnogene abeparvovec
- First posted
- 2022-04-20
- Last updated
- 2026-07-09
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of participants with treatment-emergent serious adverse events (SAEs) | 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 |
| Primary | Number of participants with treatment emergent Adverse Events of Special Interest (AESI) | 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. |
| Secondary | The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist | 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. |
| Secondary | The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist | 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. |
| Secondary | Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score | 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. |
| Secondary | Change from Baseline in the Revised Upper Limb Module (RULM) total score | 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. |
| Secondary | Systolic and diastolic blood pressure (mmHg) | Up to Year 5 | |
| Secondary | Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min) | Up to Year 5 | |
| Secondary | Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min) | Up to Year 5 | |
| Secondary | Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius) | Up to Year 5 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05335876
GET https://conductscience.org/api/trials/v1/NCT05335876/registered