Trial · NCT03987152
Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life
NCT03987152, Phase 3, Unknown, Radboud University Medical Center
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life. Trials, record NCT03987152. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03987152
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life
- NCT number
- NCT03987152
- Status
- Unknown
- Phases
- Phase 3
- Enrollment
- 75 participants, estimated
- Lead sponsor
- Radboud University Medical Center
- Conditions
- Vascular Malformations
- Interventions
- Sirolimus
- First posted
- 2019-06-14
- Last updated
- 2021-01-29
- Other registries' IDs
- eudract:2016-002157-38
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Quality of life using Sirolimus measured with survey (TAPQOL) | Change from baseline Quality of life at 6 months QoL in challenge phase, and 12 months QoL in Rechallenge phase. | Quality of life: Questionnaire for Preschool Children's Health-Related Quality of Life (TAPQOL). Preschool Children Quality of Life, parent-reported questionnaire clustered into 12 multi-item scales, with higher scores range 0-100) indicating better HRQOL) |
| Primary | Quality of life using Sirolimus measured with survey (PedsQl) | Change from baseline Quality of life at 6 months QoL in challenge phase, and 12 months QoL in Rechallenge phase. | Quality of life: Pediatric Quality of Life Inventory (PedsQl) (children) 23 items questionnaire, 0-100 scale, so that higher scores indicate better HRQOL (Health-Related Quality of Life). Psychosocial Health Summary Score, Physical Health Summary Score and Total score will be measured. |
| Primary | Quality of life using Sirolimus measured with survey (Research and development Rand-36). | hange from baseline Quality of life at 6 months QoL in challenge phase, and 12 months QoL in Rechallenge phase | Quality of life: Rand-36 (adults) eight health domains; physical functioning, social functioning, role limitations due to physical health problems, role limitations due to emotional problems, mental health, vitality, pain and general health perception. Outcomes at each domain will be defined on a scale from a minimum score of 0 to a maximum score of 100. A higher score is equivalent to a better health. |
| Primary | Difference in pain scores after using Sirolimus measured with VAS score | Daily pain scores will be compared after 6 months in Challenge phase, after Challenge phase: starts the phase, and pain during 12 months in the Rechallenge phase | Daily pain score (daily visual analogue scale (VAS-score 0-10) for children, and numeric rating scale (NRS-score 0-10) for adults) |
| Primary | Difference in pain scores after using Sirolimus measured with NRS score | Daily pain scores will be compared after 6 months in Challenge phase, after Challenge phase: starts the phase without Sirolimus treatment, and pain during 12 months in the Rechallenge phase | Daily pain score (daily numeric rating scale (NRS-score 0 no pain -10 extreme pain) for adults) |
| Secondary | Return of pain after treatment and duration of lowered pain or pain free period in days | After 6 months Sirolimus intake till one year follow up. | Amount of patients who have lowered pain or are pain free. Duration of lowered pain or pain free period in days. |
| Secondary | Growth/progression of vascular malformation | MRI baseline compared with MRI after 6 months in challenge phase and 12 months in rechallenge phase | MRI of the vascular malformation will be made at the beginning of the study and after six months of treatment with Sirolimus. An experienced radiologist will evaluate the evolution of the volume of the malformation. |
| Secondary | Rate and occurence of adverse events related to Sirolimus | 4 years | Adverse events: short and long term consequences of treatment with Sirolimus according to CTCAE version 5.0. |
| Secondary | Severity of adverse events related to Sirolimus | 4 years | Adverse events: short and long term consequences of treatment with Sirolimus according to CTCAE version 5.0. |
| Secondary | Genetic mutations in the vascular malformation that can predict outcome of treatment with Sirolimus using Single Molecule Molecular Inversion Probes (smMIPs) | 4 years | Secondary material that was obtained after surgery, stored in the HECOVAN biobank can be used for analyses. Comprehensive targeted Next Generation Sequencing screen using Unique Molecular Identifiers with a technical sensitivity of 1% mutant alleles was performed for frequently mutated positions using Single Molecule Molecular Inversion Probes. We will investigate if there is a correlation with a possible found mutation and painreduction or improvement of quality of life. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
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 |
|---|---|
| 37153086 | 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/NCT03987152
GET https://conductscience.org/api/trials/v1/NCT03987152/registered