Trial · NCT00975819
Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
NCT00975819, Phase 2, Completed, Denise Martin Adams; results first posted 2026-01-06
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies. Trials, record NCT00975819. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00975819
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
- NCT number
- NCT00975819
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 61 participants, actual
- Lead sponsor
- Denise Martin Adams
- Conditions
Kaposiform Hemangioendotheliomas, Tufted Angioma, Capillary Venous Lymphatic Malformation, Venous Lymphatic Malformation, Microcystic Lymphatic Malformation, Mucocutaneous Lymphangiomatosis and Thrombocytopenia, Capillary Lymphatic Arterial Venous Malformations, PTEN Overgrowth Syndrome With Vascular Anomaly, Lymphangiectasia Syndromes
- Interventions
- sirolimus
- First posted
- 2009-09-11
- Last updated
- 2026-01-06
- Results first posted
- 2026-01-06
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | 6 months | Measures were determined by 3 distinct methods: radiologic evaluation, functional impairment score (clinical measurement of disease), and health-related quality of life. (HRQOL). The most common radiologic evaluation was MRI but other studies including CT and X-rays were included. HRQOL was assessed using the Pediatric Quality of Life Inventory 4.0 (3-18 years) ad infant Scales (\< or = to 2 years) and the Functional Assessment of Chronic Illness System (\> 18 years). Third method was the functional impairment score which was adopted from the measure of organ function that have been validated in the quantification of adverse even results from medical therapies and procedures. Patients needed to have complete response, normalization in quality of life and functional impairment score to have a complete response. In order to have a partial response they needed to have improvement in all three areas of assessment and not worsening of any others. |
| Primary | Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | 12 months | Measures were determined by 3 distinct methods: radiologic evaluation, functional impairment score (clinical measurement of disease), and health-related quality of life. (HRQOL). The most common radiologic evaluation was MRI but other studies including CT and X-rays were included. HRQOL was assessed using the Pediatric Quality of Life Inventory 4.0 (3-18 years) ad infant Scales (\< or = to 2 years) and the Functional Assessment of Chronic Illness System (\> 18 years). Third method was the functional impairment score which was adopted from the measure of organ function that have been validated in the quantification of adverse even results from medical therapies and procedures. Patients needed to have complete response, normalization in quality of life and functional impairment score to have a complete response. In order to have a partial response they needed to have improvement in all three areas of assessment and not worsening of any others. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | 6 months | Sixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | 12 months | 53 were evaluable at the end of course 12. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group5
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | Sirolimus | Partial Response | 47 | Participants | |
| Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | Sirolimus | Progressive Disease | 7 | Participants | |
| Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | Sirolimus | Stable Disease | 3 | Participants | |
| Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | Sirolimus | Partial Response | 45 | Participants | |
| Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score | Sirolimus | Progressive Disease | 8 | 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 | Sirolimus | 61 | |
| Overall Study | COMPLETED | Sirolimus | 46 | |
| Overall Study | NOT COMPLETED | Sirolimus | 15 |
Source ClinicalTrials.gov
Adverse events26
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Blood Bone Marrow | Blood and lymphatic system disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 5 | 61 | 5 |
| serious | Pain- Abdomen- NOS | General disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 3 | 61 | 4 |
| serious | Anemia | Blood and lymphatic system disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Platelets | Blood and lymphatic system disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Lymphedema | Blood and lymphatic system disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Neutropenia | Blood and lymphatic system disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Appendix | Gastrointestinal disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Colitis | Gastrointestinal disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Diarrhea | Gastrointestinal disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
| serious | Vomiting | Gastrointestinal disorders | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 1 | 61 | 1 |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | ||
| Total, other adverse events | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 5 | 61 |
| Total, serious adverse events | All Grade 2 and Higher Adverse Events Attributable to Sirolimus | 24 | 61 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 30672136 | DERIVED |
Source PubMed
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD003712 | Phase II Study of Rapamycin for Complicated Vascular Anomalies IND Exempt | ADAMS, DENISE MARTIN | CINCINNATI CHILDRENS HOSP MED CTR | FOOD AND DRUG ADMINISTRATION | 2009 to 2012 |
Source NIH RePORTER
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/NCT00975819
GET https://conductscience.org/api/trials/v1/NCT00975819/events?page=2