Trial · NCT01265030
A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis
NCT01265030, Phase 1, Phase 2, Completed, MaineHealth; results first posted 2023-06-26
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis. Trials, record NCT01265030. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01265030
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis
- NCT number
- NCT01265030
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- MaineHealth
- Conditions
- Desmoid Tumor
- Interventions
- Sirolimus
- First posted
- 2010-12-22
- Last updated
- 2023-06-26
- Results first posted
- 2023-06-26
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | 4 weeks | Changes to the mTOR pathway were determined using an immunohistochemical immunoreactive score (IRS). The components of the IRS for each phosphoprotein (p4EPB and pS706K) are as follows: the percentage of positive cells were scored as: 0 (0%); 1 (\<10%); 2 (11-50%); 3 (51-80%); 4(\>80%). The staining intensity were scored as: 0 (negative), 1 (weak), 2 (moderate), and 3 (strong). To derive the IRS, the percentage of positive cells and staining intensity were multiplied together, resulting in a value from 0 to 12. The score for patients after 4 weeks of sirolimus was compared to a group of control desmoid tumor samples from the UCLA tumor bank (n=68). Lower scores for patients following 4 weeks of sirolimus compared to control sample scores indicate a better outcome. |
| Secondary | Pain Levels After 4 Weeks of Sirolimus | 4 weeks | Pain assessments were performed using the validated numeric (age ≥ 10 years) and Wong-Baker FACES (≥3 and \< 10 years) pain rating scales at specified study time points including baseline, at week 1 and after 4 weeks of treatment (just prior to surgery). Both pain scales range from a value of 0 to 10 with 0 being equal to no pain and 10 being equal to the worst pain. The median pain score and pain score range for study participants at Week 1 and Prior to Surgery (Week 4) timepoints are provided. Lower pain scale values (median and upper limit of the range) at the Prior to Surrgery timepoint are considered a better outcome. |
| Secondary | Number of Participants Without Tumor Recurrence | 3 years from the end of therapy, a total duration of 3 years and 4 weeks | We evaluated the number of study participants whose tumor did not recur by 3 years from the date of surgery. |
| Secondary | Number of Participants With Grade 3 or Higher Toxicity Per CTCAE Definitions | 4 weeks | The safety and tolerability of patients receiving sirolimus was evaluated using Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 grading criteria. All reported grades of toxicity (1-5) were collected. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | 4 weeks | The 9 patients who were treated with sirolimus were compared to a group of control desmoid tumor samples from the UCLA tumor bank (n=68); no identifiable baseline or adverse event data were collected or available from participants who contributed control tumor samples due to anonymization. | score on a scale | MEAN |
| SECONDARY | Pain Levels After 4 Weeks of Sirolimus | 4 weeks | Pain scores were obtained weekly throughout the 4 week study period in all participants | score on a scale | MEDIAN |
| SECONDARY | Number of Participants Without Tumor Recurrence | 3 years from the end of therapy, a total duration of 3 years and 4 weeks | All trial participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Grade 3 or Higher Toxicity Per CTCAE Definitions | 4 weeks | All trial participants | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | Immunoreactive Score Results of p4EPB After 4 Weeks of Sirolimus for the Study Patients | 5.22 | Full Range 1 to 12 | score on a scale | |
| Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | Immunoreactive Score Results of pS706K After 4 Weeks of Sirolimus for the Study Patients | 7.44 | Full Range 0 to 12 | score on a scale | |
| Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | Immunoreactive Score Results of p4EPB for the Control Tumors | 4.47 | Full Range 0 to 12 | score on a scale | |
| Immunohistochemical Immunoreactive Score Results After 4 Weeks of Sirolimus Compared to Control Specimens | Immunoreactive Score Results of pS706K for the Control Tumors | 9.39 | Full Range 0 to 12 | score on a scale | |
| Pain Levels After 4 Weeks of Sirolimus | Week 1 | 1 | Full Range 0 to 3 | score on a scale | |
| Pain Levels After 4 Weeks of Sirolimus | Prior to Surgery | 0 | Full Range 0 to 3 | score on a scale | |
| Number of Participants Without Tumor Recurrence | Study Patients | 5 | Participants | ||
| Number of Participants With Grade 3 or Higher Toxicity Per CTCAE Definitions | Safety | 0 | 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 | 9 | |
| Overall Study | COMPLETED | Sirolimus | 9 | |
| Overall Study | NOT COMPLETED | Sirolimus | 0 |
Source ClinicalTrials.gov
Adverse events35
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 | Headache | Nervous system disorders | Sirolimus | 2 | 9 | 2 |
| other | Fatigue | General disorders | Sirolimus | 2 | 9 | 2 |
| other | Laryngeal inflammation | Respiratory, thoracic and mediastinal disorders | Sirolimus | 1 | 9 | 1 |
| other | Malaise | General disorders | Sirolimus | 1 | 9 | 1 |
| other | Weight loss | Investigations | Sirolimus | 1 | 9 | 1 |
| other | Dizziness | Nervous system disorders | Sirolimus | 1 | 9 | 1 |
| other | Oral pain | Gastrointestinal disorders | Sirolimus | 2 | 9 | 2 |
| other | Mucositis oral | Gastrointestinal disorders | Sirolimus | 5 | 9 | 5 |
| other | Rash maculo-papular | Skin and subcutaneous tissue disorders | Sirolimus | 1 | 9 | 1 |
| other | Hypertension | Vascular disorders | Sirolimus | 1 | 9 | 1 |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sirolimus | 0 | 9 |
| Total, other adverse events | Sirolimus | 7 | 9 |
| Total, serious adverse events | Sirolimus | 0 | 9 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT01265030
GET https://conductscience.org/api/trials/v1/NCT01265030/events?page=2