Trial · NCT03987152
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 12 of 12
Download this page as JSON| 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. |
| Secondary | Pharmacogenetic profile in the vascular malformation that can predict outcome of treatment with Sirolimus | 4 years | Genotyping will be performed using Taqman assays or Kompetitive Allele Specific PCR (KASPTM) assays in saliva swabs. Pharmacogenetic profiles (slow, intermediate, and extensive metabolizer) will be determined. Subjects compared on the outcome of sirolimus effectiveness for pain, and QoL stratified by CYP3A4. |
| Secondary | Cost-effectiveness of administration of Sirolimus: Quality Adjusted Life Year (QALY) estimate for each patient | 4 years | Via standardized questionnaires developed by the iMTA (institute for Medical Technology Assessment). The second part of the cost analysis consists of determining the cost prices for each volume of consumption. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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.
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