Trial · NCT03837184

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

NCT03837184, Phase 3, Completed, Novartis Gene Therapies; results first posted 2022-01-11

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies. Trials, record NCT03837184. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03837184

On this page

Trial

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

Title: Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
NCT number: NCT03837184
Status: Completed
Phases: Phase 3
Enrollment: 2 participants, actual
Lead sponsor: Novartis Gene Therapies
Conditions: Spinal Muscular Atrophy Type I
Interventions: Onasemnogene Abeparvovec-xioi
First posted: 2019-02-12
Last updated: 2026-01-26
Results first posted: 2022-01-11

Source ClinicalTrials.gov

Registered outcomes2

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

List	Outcome	Time frame	Description
Primary	Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds	From Baseline up to 18 Months of Age Visit	Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight unsupported for at least 10 seconds.
Secondary	Event-free Survival at 14 Months of Age	From Baseline up to 14 Months of Age	Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.

Source ClinicalTrials.gov

Outcomes2

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds	From Baseline up to 18 Months of Age Visit	Intent-to-Treat (ITT) population - Symptomatic participants with biallelic deletion mutations of survival of motor neuron 1 (SMN1) (exon 7/8 common homozygous deletions) and 2 copies of survival of motor neuron 2 (SMN2) without the known gene modifier mutation (c.859G\>C) who received an IV infusion of AVXS-101 at less than 180 days of age.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Event-free Survival at 14 Months of Age	From Baseline up to 14 Months of Age	ITT population - Symptomatic participants with biallelic deletion mutations of SMN1 (exon 7/8 common homozygous deletions) and 2 copies of SMN2 without the known gene modifier mutation (c.859G\>C) who received an IV infusion of AVXS-101 at less than 180 days of age.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group2

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds	AVXS-101		1		Participants
Event-free Survival at 14 Months of Age	AVXS-101		2		Participants

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	AVXS-101	2
Overall Study	COMPLETED	AVXS-101	2
Overall Study	NOT COMPLETED	AVXS-101	0

Source ClinicalTrials.gov

Adverse events21

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Respiratory distress	Respiratory, thoracic and mediastinal disorders	AVXS-101	1	2	1
other	Pharyngitis	Infections and infestations	AVXS-101	1	2	1
other	Rhonchi	Respiratory, thoracic and mediastinal disorders	AVXS-101	1	2	1
other	Tachypnoea	Respiratory, thoracic and mediastinal disorders	AVXS-101	1	2	1
other	Pyrexia	General disorders	AVXS-101	1	2	3
other	Dysphagia	Gastrointestinal disorders	AVXS-101	2	2	3
other	Gastrointestinal hypomotility	Gastrointestinal disorders	AVXS-101	1	2	1
other	Gastrooesophageal reflux disease	Gastrointestinal disorders	AVXS-101	1	2	1
other	Upper gastrointestinal haemorrhage	Gastrointestinal disorders	AVXS-101	1	2	1
other	Failure to thrive	Metabolism and nutrition disorders	AVXS-101	2	2	2

1 to 10 of 21Source ClinicalTrials.gov

See all 21 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	AVXS-101	0	2
Total, other adverse events	AVXS-101	2	2
Total, serious adverse events	AVXS-101	1	2

Source ClinicalTrials.gov

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.

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.

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/NCT03837184

GET https://conductscience.org/api/trials/v1/NCT03837184/events?page=2

API reference For AI assistants