Trial · NCT04042025

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025, Phase 3, Active not recruiting, Novartis Gene Therapies

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi. Trials, record NCT04042025. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04042025

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
NCT number: NCT04042025
Status: Active not recruiting
Phases: Phase 3
Enrollment: 85 participants, actual
Lead sponsor: Novartis Gene Therapies
Conditions: Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Interventions: Onasemnogene Abeparvovec-xioi
First posted: 2019-08-01
Last updated: 2026-09-24
Other registries' IDs: eudract:2019-002611-26

Source ClinicalTrials.gov

Registered outcomes20

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants Who Reach Developmental Milestones	Up to 5 years	Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.
Primary	Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score	Up to 5 years	The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability.
Primary	Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support	Up to 15 years	Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support.
Primary	Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support	Up to 5 years	Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.
Primary	Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings	Up to 5 years	The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures.
Primary	Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements	Up to 5 years	Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.
Primary	Change From Baseline in Height Measurements	Up to 5 years
Primary	Change From Baseline in Weight Measurements	Up to 5 years
Primary	Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments	Up to 5 years	Blood samples will be collected for hematology (including complete blood cell count) and chemistry.
Primary	Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments	Up to 5 years	Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.

1 to 10 of 20Source ClinicalTrials.gov

See all 20 registered outcomes

Outcomes0

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.

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
34383289	DERIVED

Source PubMed

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.

Tests1

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

Test	Named as
1-Back Task	Primary outcome

Source ConductCognition

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT04042025

GET https://conductscience.org/api/trials/v1/NCT04042025/registered

API reference For AI assistants