Trial · NCT00272844
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
NCT00272844, Phase 1, Phase 2, Completed, Boston Children's Hospital; results first posted 2017-10-20
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome. Trials, record NCT00272844. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00272844
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
- NCT number
- NCT00272844
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 23 participants, actual
- Lead sponsor
- Boston Children's Hospital
- Conditions
- Smith-Lemli-Opitz Syndrome
- Interventions
- crystalline cholesterol oil-based suspension
- First posted
- 2006-01-09
- Last updated
- 2017-10-20
- Results first posted
- 2017-10-20
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Responders | Every 3-6 months for an approximate median of 5 years | Responders was defined as an increase in total serum cholesterol and a decrease in 7-DHC (7-Dehydrocholesterol), and 8-DHC (8-Dehydrocholesterol) were measured on all participants. |
| Secondary | Number of Growth Responders | Every 3-6 months for an approximate median of 5 years | Growth response was defined as an increase in general health, growth, and behavior. |
| Secondary | Number of Participants With Improved Neuropsychological Development | Every 3-6 months for an approximate median of 5 years | Improved neuropsychological development is defined as progressively achieving developmental milestones |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Responders | Every 3-6 months for an approximate median of 5 years | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Growth Responders | Every 3-6 months for an approximate median of 5 years | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Improved Neuropsychological Development | Every 3-6 months for an approximate median of 5 years | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group3
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Responders | Cholesterol Supplementation | 18 | Participants | ||
| Number of Growth Responders | Cholesterol Supplementation | 18 | Participants | ||
| Number of Participants With Improved Neuropsychological Development | Cholesterol Supplementation | 18 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cholesterol Supplementation | 23 | |
| Overall Study | COMPLETED | Cholesterol Supplementation | 18 | |
| Overall Study | NOT COMPLETED | Cholesterol Supplementation | 5 |
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 totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cholesterol Supplementation | ||
| Total, other adverse events | Cholesterol Supplementation | 0 | 23 |
| Total, serious adverse events | Cholesterol Supplementation | 0 | 23 |
Source ClinicalTrials.gov
Papers5
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/NCT00272844
GET https://conductscience.org/api/trials/v1/NCT00272844/registered