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Trial · NCT03421977

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

NCT03421977, Active not recruiting, Novartis Gene Therapies

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Long-Term Follow-up Study for Patients From AVXS-101-CL-101. Trials, record NCT03421977. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03421977

On this page

Trial

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

Title
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
NCT number
NCT03421977
Status
Active not recruiting
Enrollment
13 participants, actual
Lead sponsor
Novartis Gene Therapies
Conditions
Spinal Muscular Atrophy 1
Interventions
Onasemnogene Abeparvovec-xioi
First posted
2018-02-05
Last updated
2026-09-22

Source ClinicalTrials.gov

Registered outcomes1

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

ListOutcomeTime frameDescription
PrimaryLong-Term Safety15 years
The primary objective is to collect long term safety data of patients with SMA Type 1 who were treated with onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial by assessing incidence of SAEs and Adverse Events of Special Interest

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.

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMIDListed asSource
33999158DERIVEDPubMed
34383289DERIVEDPubMed
42016922DERIVEDPubMed

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/NCT03421977

GET https://conductscience.org/api/trials/v1/NCT03421977/registered