Trial · NCT00568698
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
NCT00568698, Phase 1, Phase 2, Completed, Stanford University; results first posted 2019-10-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients. Trials, record NCT00568698. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00568698
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
- NCT number
- NCT00568698
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Stanford University
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Hydroxyurea, Placebo to match hydroxyurea
- First posted
- 2007-12-06
- Last updated
- 2019-10-02
- Results first posted
- 2019-10-02
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety: Frequency of Adverse Events/Lab Abnormalities | Up to 8 years, 1 month | |
| Primary | Efficacy: Length of Survival (LOS) and Age of Ventilator Dependence (AVD) | Up to 8 years, 1 month | |
| Secondary | Motor Unit Number Estimation (MUNE) | Up to 8 years, 1 month | |
| Secondary | Biomarker Assays: SMN Protein and SMN mRNA | Up to 8 years, 1 month |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety: Frequency of Adverse Events/Lab Abnormalities | Up to 8 years, 1 month | Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted. | ||
| PRIMARY | Efficacy: Length of Survival (LOS) and Age of Ventilator Dependence (AVD) | Up to 8 years, 1 month | Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted. | ||
| SECONDARY | Motor Unit Number Estimation (MUNE) | Up to 8 years, 1 month | Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted. | ||
| SECONDARY | Biomarker Assays: SMN Protein and SMN mRNA | Up to 8 years, 1 month | Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted. |
Source ClinicalTrials.gov
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | All Participants | 29 | |
| Overall Study | COMPLETED | All Participants | 18 | |
| Overall Study | NOT COMPLETED | All Participants | 11 |
Source ClinicalTrials.gov
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 totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Hydroxyurea | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Hydroxyurea | 0 | 0 |
| Total, other adverse events | Placebo | 0 | 0 |
| Total, serious adverse events | Hydroxyurea | 0 | 0 |
| Total, serious adverse events | Placebo | 0 | 0 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT00568698
GET https://conductscience.org/api/trials/v1/NCT00568698/registered