Trial · NCT00634270
A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
NCT00634270, Phase 2, Completed, University of Alabama at Birmingham; results first posted 2017-02-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas. Trials, record NCT00634270. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00634270
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
- NCT number
- NCT00634270
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 58 participants, actual
- Lead sponsor
- University of Alabama at Birmingham
- Conditions
- Neurofibromatosis Type 1
- Interventions
- Sirolimus
- First posted
- 2008-03-13
- Last updated
- 2017-11-17
- Results first posted
- 2017-02-14
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time to Disease Progression Based on Volumetric MRI | 24 Months Stratum 1 | Median time to progression in Stratum 1 as defined as an increase of at least 20% of the volume of the primary lesion. Note: Since Stratum 2 looked at response rate only, median time to progression was not reviewed for this outcome. |
| Primary | Results in Objective Radiographic Responses Based on Volumetric MRI Measurements in Children and Adults With NF1 and Inoperable PN in the Absence of Documented Radiographic Progression at Trial Entry | 48 weeks Stratum 2 | Stratum 2 outcome - Response |
| Primary | Toxicity | 24 weeks Stratum 1 / 48 weeks Stratum 2 | Number of participants experiencing adverse events |
| Primary | To Characterize the Pharmacokinetic Profile of Sirolimus Administered to This Patient Population (Clearance Liters/Hour (L/h)) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | An iterative 2-stage Bayesian method was used for the PK parameter analyses |
| Primary | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population Using Liters/Hour Per Population Median Weight of 70kg (L/h70kg) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | An iterative 2-stage Bayesian method was used for the PK parameter analyses |
| Primary | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population - (Clearance (L/h Per 1.85 m^2) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | An iterative 2-stage Bayesian Method was used for the PK parameter analyses |
| Primary | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population- Therapeutic Dose (mg/m^2 Per Dose) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | An iterative 2-stage Bayesian Method was used for the PK parameter analyses |
| Primary | To Characterize the Pharmacokinetic Profile of Sirolimus in When Administered to This Patient Population - Therapeutic Dose (mg/kg Per Dose) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | An iterative 2-stage Bayesian Method was used for the PK parameter analyses |
| Primary | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population - Therapeutic Dose (mg/kg)^0.75 | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | An iterative 2-stage Bayesian method was used for the PK parameter analyses |
| Secondary | To Evaluate the Quality of Life During Treatment With Sirolimus by Assessing Preliminary Correlations of Response With Quality-of-life Outcomes | 24 weeks Stratum 1 / 48 weeks Stratum 2 | Self-reported, age-appropriate PedsQL Scale. Assessments included: Inventory for physical function, emotional function, social function and school function - number system 0-4 was used with 4 being the worse maximum threshold); inventory for chronic illness used a 5-point likert scale - 5 being the worst maximum threshold; Skindex-Teen used a scale of 0 to 100 - the higher the number the more frequent the experience; Pain intensity was measured using a line with a happy and sad face - marks toward the sad face indicated more intense pain; and, the McGill Pain Questionnaire - higher values indicating worse pain of a scale from 0-3. All assessments were combined for an overall PedsQL score by rating each item 0-4, then reverse transforming each to a 0 - 100 scale. The total scores were calculated by averaging the item scores, with higher scores being better. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Time to Disease Progression Based on Volumetric MRI | 24 Months Stratum 1 | All evaluable participants. Note: In Stratum 2 the value was not assessed as time to progression therefore the appropriate response would be N/A. Stratum 2 was reported as response rate only. | Months | MEDIAN |
| PRIMARY | Results in Objective Radiographic Responses Based on Volumetric MRI Measurements in Children and Adults With NF1 and Inoperable PN in the Absence of Documented Radiographic Progression at Trial Entry | 48 weeks Stratum 2 | Identify the index plexiform neurofibroma(s) for 3-D MRI evaluation based on prior imaging studies. The criteria for response was \<20% increase in volume using RECIST v1.0. Response for 48 weeks was only assessed for Stratum 2. | participants | NUMBER |
| PRIMARY | Toxicity | 24 weeks Stratum 1 / 48 weeks Stratum 2 | All evaluable participants for Stratum 1 and 2 combined. | Participants | NUMBER |
| PRIMARY | To Characterize the Pharmacokinetic Profile of Sirolimus Administered to This Patient Population (Clearance Liters/Hour (L/h)) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | Pediatric Patients (3 through 18) with Neurofibromatosis Type 1 - Clearance liters/hour (L/h). Stratum 2 did not meet the 6 month response criteria; therefore, was not analyzed for pharmacokinetic profiles. | Clearance liters/hour (L/h) | MEAN |
| PRIMARY | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population Using Liters/Hour Per Population Median Weight of 70kg (L/h70kg) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | Stratum 1 used allometrically scaled clearance (clearance scaled to a 70kg individual). Stratum 2 did not meet the 6 month response criteria; therefore, was not analyzed for pharmacokinetic profiles. | L/h/70kg | MEAN |
| PRIMARY | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population - (Clearance (L/h Per 1.85 m^2) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | Stratum 1, Neurofibromatosis Type 1 Patients Clearance (L/h per 1.85 m\^2). Stratum 2 did not meet the 6 month response criteria; therefore, was not analyzed for pharmacokinetic profiles. | L/h per 1.85 m^2 | MEAN |
| PRIMARY | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population- Therapeutic Dose (mg/m^2 Per Dose) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | Stratum 1, Neurofibromatosis Type 1 patients - Therapeutic Dose (mg/m\^2 per dose). Stratum 2 did not meet the 6 month response criteria; therefore, was not analyzed for pharmacokinetic profiles. | Therapeutic Dose mg/m^2 per dose | MEAN |
| PRIMARY | To Characterize the Pharmacokinetic Profile of Sirolimus in When Administered to This Patient Population - Therapeutic Dose (mg/kg Per Dose) | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | Stratum 1, Neurofibromatosis Type 1 patients (Ages 3 to 18) Therapeutic Dose (mg/kg per dose). Stratum 2 did not meet the 6 month response criteria; therefore, was not analyzed for pharmacokinetic profiles. | Therapeutic Dose (mg/kg per dose) | MEAN |
| PRIMARY | To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population - Therapeutic Dose (mg/kg)^0.75 | Pre-dose; Day 1 at 0.5 hours, 1.0 hours, 2.0 hours, 3.0 hours, 4.0 hours, 6.0 hours, 8.0 hours, and 10.0 to 12.0 hours | Stratum 1, Neurofibromatosis Type 1 patients (ages 3 to 18) - Therapeutic Dose (mg/kg)\^0.75. Stratum 2 did not meet the 6 month response criteria; therefore, was not analyzed for pharmacokinetic profiles. | Therapeutic Dose (mg/kg)^0.75 | MEAN |
| SECONDARY | To Evaluate the Quality of Life During Treatment With Sirolimus by Assessing Preliminary Correlations of Response With Quality-of-life Outcomes | 24 weeks Stratum 1 / 48 weeks Stratum 2 | Stratum 1 patients with Neurofibromatosis Type 1; all ages - Change from Baseline to Course 3. Stratum 2 did not meet the requirements for response at the 6 month time point. Therefore, Stratum 2 was not analyzed for this aim. | Units on a scale | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Time to Disease Progression Based on Volumetric MRI | Stratum 1 | 15.4 | 95% Confidence Interval 14.3 to 23.7 | Months | |
| Results in Objective Radiographic Responses Based on Volumetric MRI Measurements in Children and Adults With NF1 and Inoperable PN in the Absence of Documented Radiographic Progression at Trial Entry | Stratum 2 | 12 | participants | ||
| Toxicity | Stratum 1 | 49 | Participants | ||
| Toxicity | Stratum 2 | 13 | Participants | ||
| To Characterize the Pharmacokinetic Profile of Sirolimus Administered to This Patient Population (Clearance Liters/Hour (L/h)) | Stratum 1 | 11.8 | Standard Deviation 4.6 | Clearance liters/hour (L/h) | |
| To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population Using Liters/Hour Per Population Median Weight of 70kg (L/h70kg) | Stratum 1 | 23.7 | Standard Deviation 6.7 | L/h/70kg | |
| To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population - (Clearance (L/h Per 1.85 m^2) | Stratum 1 | 6.4 | Standard Deviation 2.5 | L/h per 1.85 m^2 | |
| To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population- Therapeutic Dose (mg/m^2 Per Dose) | Stratum 1 | 2.0 | Standard Deviation 0.7 | Therapeutic Dose mg/m^2 per dose | |
| To Characterize the Pharmacokinetic Profile of Sirolimus in When Administered to This Patient Population - Therapeutic Dose (mg/kg Per Dose) | Stratum 1 | 0.08 | Standard Deviation 0.04 | Therapeutic Dose (mg/kg per dose) | |
| To Characterize the Pharmacokinetic Profile of Sirolimus When Administered to This Patient Population - Therapeutic Dose (mg/kg)^0.75 | Stratum 1 | 0.14 | Standard Deviation 0.05 | Therapeutic Dose (mg/kg)^0.75 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Stratum 1 | 49 | |
| Overall Study | STARTED | Stratum 2 | 13 | |
| Overall Study | COMPLETED | Stratum 1 | 46 | |
| Overall Study | COMPLETED | Stratum 2 | 12 | |
| Overall Study | NOT COMPLETED | Stratum 1 | 3 | |
| Overall Study | NOT COMPLETED | Stratum 2 | 1 |
Source ClinicalTrials.gov
Adverse events192
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 | Stratum 1 | 2 | 46 | 2 |
| other | Headache | Nervous system disorders | Stratum 2 | 1 | 12 | 1 |
| other | Vomiting | Gastrointestinal disorders | Stratum 1 | 4 | 46 | 4 |
| other | Vomiting | Gastrointestinal disorders | Stratum 2 | 0 | 12 | 0 |
| other | Low ANC | Investigations | Stratum 1 | 1 | 46 | 1 |
| other | Low ANC | Investigations | Stratum 2 | 0 | 12 | 0 |
| other | Low WBC | Investigations | Stratum 1 | 1 | 46 | 1 |
| other | Low WBC | Investigations | Stratum 2 | 0 | 12 | 0 |
| other | Pain - Stomach | Gastrointestinal disorders | Stratum 1 | 1 | 46 | 1 |
| other | Pain - Stomach | Gastrointestinal disorders | Stratum 2 | 0 | 12 | 0 |
1 to 10 of 192Source ClinicalTrials.gov
See all 192 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Stratum 1 | ||
| Total, all-cause mortality | Stratum 2 | ||
| Total, other adverse events | Stratum 1 | 8 | 46 |
| Total, other adverse events | Stratum 2 | 3 | 12 |
| Total, serious adverse events | Stratum 1 | 20 | 46 |
| Total, serious adverse events | Stratum 2 | 4 | 12 |
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Stratum 1 vs Stratum 2: Toxicity | Trial comparisons |
| Stratum 1 vs Stratum 2: To Evaluate the Role of Apolipoprotein E Genotypes as Predictors for Development of Hyperlipidemia During Therapy With Sirolimus. | Trial comparisons |
| Stratum 1 vs Stratum 2: serious adverse events | Trial comparisons |
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/NCT00634270
GET https://conductscience.org/api/trials/v1/NCT00634270/events?page=2