Trial · NCT04174157
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
NCT04174157, Recruiting, Novartis Pharmaceuticals
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA). Trials, record NCT04174157. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04174157
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
- NCT number
- NCT04174157
- Status
- Recruiting
- Enrollment
- 700 participants, estimated
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- Prospective observational registry, Zolgensma
- First posted
- 2019-11-22
- Last updated
- 2025-01-31
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in probability of survival of all patients with SMA using Kaplan Meier method to estimate | Based on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up. | |
| Primary | Change from baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in infants with pre-symptomatic or type I SMA | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | CHOP INTEND score ranges from 0 to 64 with higher scores indicating higher motor function |
| Primary | Change from baseline Hammersmith Infant Neurological Examination (HINE) in infants with pre-symptomatic, type I or type II SMA | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | HINE score range from 0 to 26 with higher scores indicating more development. |
| Primary | Change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) for patients with type II and III SMA | Baseline and every 6months through 2 years of follow up, then annually through 15 years of follow up | HFMSE score range from 0 to 66 with the higher scores indicating more development. |
| Primary | Incidence of treatment emergent adverse events | Through 15 years of follow up | |
| Primary | Incidence of treatment emergent serious adverse events | Through 15 years of follow up | |
| Primary | Incidence of treatment emergent adverse events related to therapy | Through 15 years of follow up | |
| Primary | Incidence of treatment emergent thrombocytopenia, hepatotoxicity and cardiac adverse events | Through 15 years of follow up | |
| Secondary | Change from baseline in rates of hospitalization | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | |
| Secondary | Change from baseline in Zarit Burden Interview | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | Zarit Burden Total Score ranges from 0 to 88. A higher score correlates with higher level of burden. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 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.
Papers3
The papers the trial lists on ClinicalTrials.gov.
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/NCT04174157
GET https://conductscience.org/api/trials/v1/NCT04174157/registered