Trial Outcomes & Findings for Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies (NCT NCT00975819)
NCT ID: NCT00975819
Last Updated: 2026-01-06
Results Overview
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.
COMPLETED
PHASE2
61 participants
6 months
2026-01-06
Participant Flow
Enrollment opened October 2009, closed to enrollment March 2014.
Participant milestones
| Measure |
Sirolimus
sirolimus: liquid dosing based on trough levels
|
|---|---|
|
Overall Study
STARTED
|
61
|
|
Overall Study
COMPLETED
|
46
|
|
Overall Study
NOT COMPLETED
|
15
|
Reasons for withdrawal
| Measure |
Sirolimus
sirolimus: liquid dosing based on trough levels
|
|---|---|
|
Overall Study
Lack of Efficacy
|
8
|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Physician Decision
|
2
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Protocol Violation
|
1
|
Baseline Characteristics
Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
Baseline characteristics by cohort
| Measure |
Sirolimus
n=61 Participants
sirolimus: liquid dosing based on trough levels
|
|---|---|
|
Age, Categorical
<=18 years
|
41 Participants
n=9 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=9 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=9 Participants
|
|
Age, Continuous
|
8.1 years
n=9 Participants
|
|
Sex: Female, Male
Female
|
35 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=9 Participants
|
|
Region of Enrollment
United States
|
61 participants
n=9 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: Sixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6.
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.
Outcome measures
| Measure |
Sirolimus
n=57 Participants
Responsiveness to sirolimus by the end of course 6
|
|---|---|
|
Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score
Partial Response
|
47 Participants
|
|
Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score
Progressive Disease
|
7 Participants
|
|
Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score
Stable Disease
|
3 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: 53 were evaluable at the end of course 12.
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.
Outcome measures
| Measure |
Sirolimus
n=53 Participants
Responsiveness to sirolimus by the end of course 6
|
|---|---|
|
Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score
Partial Response
|
45 Participants
|
|
Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score
Progressive Disease
|
8 Participants
|
Adverse Events
All Grade 2 and Higher Adverse Events Attributable to Sirolimus
Serious adverse events
| Measure |
All Grade 2 and Higher Adverse Events Attributable to Sirolimus
n=61 participants at risk
All Grade 2 and Higher Adverse Events Attributable to Sirolimus Per CTCAE Version 3.0, by category
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
1.6%
1/61 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
1.6%
1/61 • Number of events 1
|
|
Cardiac disorders
Hypertension
|
1.6%
1/61 • Number of events 1
|
|
Infections and infestations
Cellulitis
|
13.1%
8/61 • Number of events 14
|
|
Infections and infestations
Bacteremia
|
1.6%
1/61 • Number of events 1
|
|
Infections and infestations
Sepsis
|
1.6%
1/61 • Number of events 1
|
|
Infections and infestations
Urinary Tract Infection
|
3.3%
2/61 • Number of events 2
|
|
Infections and infestations
Infection- Colon
|
1.6%
1/61 • Number of events 1
|
|
Infections and infestations
CVC port infection
|
3.3%
2/61 • Number of events 2
|
|
Infections and infestations
Airway/ lung
|
4.9%
3/61 • Number of events 4
|
|
Infections and infestations
Immunodeficency
|
1.6%
1/61 • Number of events 1
|
|
Infections and infestations
Phlebitis
|
1.6%
1/61 • Number of events 1
|
|
Injury, poisoning and procedural complications
Fracture
|
1.6%
1/61 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
3.3%
2/61 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Dyspenea
|
1.6%
1/61 • Number of events 1
|
|
General disorders
Fever
|
8.2%
5/61 • Number of events 8
|
|
General disorders
Weight Loss
|
1.6%
1/61 • Number of events 2
|
|
General disorders
Dehydration
|
1.6%
1/61 • Number of events 1
|
|
General disorders
Pain- Abdomen- NOS
|
4.9%
3/61 • Number of events 4
|
|
Blood and lymphatic system disorders
Anemia
|
1.6%
1/61 • Number of events 1
|
|
Blood and lymphatic system disorders
Platelets
|
1.6%
1/61 • Number of events 1
|
|
Blood and lymphatic system disorders
Lymphedema
|
1.6%
1/61 • Number of events 1
|
|
Blood and lymphatic system disorders
Neutropenia
|
1.6%
1/61 • Number of events 1
|
|
Gastrointestinal disorders
Appendix
|
1.6%
1/61 • Number of events 1
|
|
Gastrointestinal disorders
Colitis
|
1.6%
1/61 • Number of events 1
|
Other adverse events
| Measure |
All Grade 2 and Higher Adverse Events Attributable to Sirolimus
n=61 participants at risk
All Grade 2 and Higher Adverse Events Attributable to Sirolimus Per CTCAE Version 3.0, by category
|
|---|---|
|
Blood and lymphatic system disorders
Blood Bone Marrow
|
8.2%
5/61 • Number of events 5
|
Additional Information
Denise M. Adams, M.D.
Children's Hospital of Philadelphia
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place