Trial · NCT01365468
Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)
NCT01365468, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2016-05-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1). Trials, record NCT01365468. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01365468
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of RAD001 in Treating Plexiform Neurofibromas (PN) Associated With Neurofibromatosis (NF1)
- NCT number
- NCT01365468
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 9 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Plexiform Neurofibroma Associated With Neurofibromatosis Type 1
- Interventions
- Everolimus (RAD001)
- First posted
- 2011-06-03
- Last updated
- 2016-05-12
- Results first posted
- 2016-05-12
- Limitations and caveats
- Study got terminated because of poor patient's accrual.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Time to Disease Progression (TTP) Based on Change in Volumetric MRI Measurements in Children and Adults (In Stratum I Only) | Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days) | This endpoint was planned to be analyzed for only Stratum 1 patients. Progression of disease defined as a ≥ 20% increase in the volume (by volumetric MRI) of at least one of the index plexiform neurofibromas (PN) compared to the pretreatment volume measured prior to the start of the current treatment phase. |
| Primary | Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only) | Screening, after course #6, then every 6 months and end of treatment(1 course=28days) | Response was assessed at the time that a follow up volumetric MRI scan is performed (after course 6 and then every 6 months and at the end of treatment). * Complete response (CR): complete resolution of all measurable or palpable PN for ≥ 28days and no appearance of new lesions. * Partial response (PR): A ≥ 20% reduction in the sum of the volume of all index PN lesions for ≥ 28days. * Stable disease (SD): A \< 20% increase and \< 20% decrease in the sum of the volume of all index PN lesions for ≥ 28days. |
| Primary | Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months) | Adverse events were assessed according to the NCI Common Toxicity Criteria for Adverse Events (CTCAE) version 4.0. If CTCAE grading does not exist for an adverse event, the severity of mild, moderate, severe, and life-threatening, corresponding to grades 1 - 4 respectively, were used. CTCAE grade 5 (death) was not used in this study. |
| Other | Number of Patients With Clinical Response | Screening, Day 1, after course #3, #6, #12, #18, #24, End of Treatment (1 course = 28 days) | Clinical response is defined as improvement of function, performance status, or decrease in PN related pain persisting for at least 28 days on treatment. |
| Other | Physician's Global Assessment of Clinical Condition (PGA) of Skin Lesions | Screening, after course #3, #6, #12, #18, #24, End of Treatment (1 course = 28 days) | The Physician"s Global Assessment of Clinical Condition (PGA) is a 7-point grading scale for the investigator's assessment of the overall extent of improvement or worsening of the patient"s skin disease as compared to baseline. Responses must be confirmed by at least two assessments separated in time by at least 4 weeks. The grading ranges from 0 to 6; 0 is Completely clear where as 6 is for worse condition. A complete clinical response (CCR) requires a grading of 0 indicating the absence of disease (histological confirmation is not required). Grades 1, 2, and 3 constitute partial response, indicating improvement of at least 50 percent, but less than 100 percent improvement. |
Source ClinicalTrials.gov
Outcomes5
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 (TTP) Based on Change in Volumetric MRI Measurements in Children and Adults (In Stratum I Only) | Screening, after course #6, #12, #18, #24, End of Treatment(1 course=28days) | The Full Analysis Set (FAS) consisted of all enrolled patients. | Days | MEDIAN |
| PRIMARY | Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only) | Screening, after course #6, then every 6 months and end of treatment(1 course=28days) | The Full Analysis Set (FAS) consisted of all enrolled patients. | Patients | NUMBER |
| PRIMARY | Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | From the time ICF was signed until 28 days after End of Treatment (up to a maximum of 25 months) | The Safety Population consisted of all patients who received at least one dose of study treatment and had at least one post-baseline safety assessment. | Patients | NUMBER |
| OTHER_PRE_SPECIFIED | Number of Patients With Clinical Response | Screening, Day 1, after course #3, #6, #12, #18, #24, End of Treatment (1 course = 28 days) | Study got terminated because of poor patient's accrual. Enrolled patients were less than planned number of patients required for analysis. Hence, planned analysis was not done. | ||
| OTHER_PRE_SPECIFIED | Physician's Global Assessment of Clinical Condition (PGA) of Skin Lesions | Screening, after course #3, #6, #12, #18, #24, End of Treatment (1 course = 28 days) | Study got terminated because of poor patient's accrual. Enrolled patients were less than planned number of patients required for analysis. Hence, planned analysis was not done. |
Source ClinicalTrials.gov
Results by group12
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 (TTP) Based on Change in Volumetric MRI Measurements in Children and Adults (In Stratum I Only) | Stratum 1 | NA | 95% Confidence Interval NA to NA | Days | |
| Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only) | Stratum 2 | Complete Response | 0 | Patients | |
| Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only) | Stratum 2 | Partial Response | 0 | Patients | |
| Number of Patients With Objective Radiographic Responses Based on Volumetric MRI Measurements (In Stratum 2 Only) | Stratum 2 | Stable Disease | 5 | Patients | |
| Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | Stratum 1 | At least one Grade 1 AE | 4 | Patients | |
| Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | Stratum 2 | At least one Grade 1 AE | 5 | Patients | |
| Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | Stratum 1 | At least one Grade 2 AE | 4 | Patients | |
| Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | Stratum 2 | At least one Grade 2 AE | 5 | Patients | |
| Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | Stratum 1 | At least one Grade 3 AE | 1 | Patients | |
| Number of Patients With Adverse Events Assessed by Common Toxicity Criteria for Adverse Events (CTCAE) V.04 | Stratum 2 | At least one Grade 3 AE | 0 | Patients |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 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 | 4 | |
| Overall Study | STARTED | Stratum 2 | 5 | |
| Overall Study | COMPLETED | Stratum 1 | 0 | |
| Overall Study | COMPLETED | Stratum 2 | 5 | |
| Overall Study | NOT COMPLETED | Stratum 1 | 4 | |
| Overall Study | NOT COMPLETED | Stratum 2 | 0 |
Source ClinicalTrials.gov
Adverse events116
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 | Ear pain | Ear and labyrinth disorders | Stratum 1 | 2 | 4 | |
| other | Ear pain | Ear and labyrinth disorders | Stratum 2 | 0 | 5 | |
| other | External ear inflammation | Ear and labyrinth disorders | Stratum 1 | 0 | 4 | |
| other | External ear inflammation | Ear and labyrinth disorders | Stratum 2 | 1 | 5 | |
| other | Ocular hyperaemia | Eye disorders | Stratum 1 | 1 | 4 | |
| other | Ocular hyperaemia | Eye disorders | Stratum 2 | 0 | 5 | |
| other | Abdominal pain | Gastrointestinal disorders | Stratum 1 | 1 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Stratum 2 | 2 | 5 | |
| other | Diarrhoea | Gastrointestinal disorders | Stratum 1 | 2 | 4 | |
| other | Diarrhoea | Gastrointestinal disorders | Stratum 2 | 0 | 5 |
1 to 10 of 116Source ClinicalTrials.gov
See all 116 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 | 4 | 4 |
| Total, other adverse events | Stratum 2 | 5 | 5 |
| Total, serious adverse events | Stratum 1 | 1 | 4 |
| Total, serious adverse events | Stratum 2 | 0 | 5 |
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Stratum 1 vs Stratum 2: serious adverse events |
Source 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/NCT01365468
GET https://conductscience.org/api/trials/v1/NCT01365468/events?page=2