Trial · NCT04404764
Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study
NCT04404764, Completed, Hospital Israelita Albert Einstein
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study. Trials, record NCT04404764. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04404764
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study
- NCT number
- NCT04404764
- Status
- Completed
- Enrollment
- 155 participants, actual
- Lead sponsor
- Hospital Israelita Albert Einstein
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Nusinersen Injectable Product
- First posted
- 2020-05-27
- Last updated
- 2021-04-27
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Expanded Hammersmith Functional Motor Scale | Baseline | Hammersmith Functional Motor Scale-Expanded (HFMSE) scores range from 0 to 66, with higher scores indicating better motor function. |
| Secondary | Revised Upper Limb Module | In the inclusion of the study | Revised Upper Limb Module (RULM) scores range from 0 to 37, with higher scores indicating better function. |
| Secondary | WHO motor milestones | Unique evaluation at the time of inclusion | The six World Health Organization (WHO) motor milestones are sitting without support, standing with assistance, hands and knees crawling, walking with assistance, standing alone, and walking alone. |
| Secondary | Disease duration | At the time of inclusion in the study | Time between diagnosis and age at inclusion in the study |
| Secondary | Clinical features | Unique evaluation | SMN2 (gene copy number); |
| Secondary | History of hospitalizations | Documented in the period prior to the inclusion of the study | Records of need for hospitalizations |
| Secondary | History and characterization of previous surgical procedures | In the period prior to the inclusion of the study | History of comorbidities |
| Secondary | Treatment with nusinersen | Registration of the dose used at the time of inclusion in the study | To undergo intrathecal administration of nusinersen at a dose of 12 mg |
Source ClinicalTrials.gov
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 |
|---|---|
| 42104555 | 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.
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/NCT04404764
GET https://conductscience.org/api/trials/v1/NCT04404764/registered