Trial · NCT00021541

R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas

NCT00021541, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2012-09-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas. Trials, record NCT00021541. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00021541

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
NCT number: NCT00021541
Status: Completed
Phases: Phase 2
Enrollment: 62 participants, actual
Lead sponsor: National Cancer Institute (NCI)
Conditions: Neurofibroma, Plexiform, Neurofibromatosis Type I
Interventions: tipifarnib, placebo
First posted: 2001-07-23
Last updated: 2018-04-17
Results first posted: 2012-09-17

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Median Time to Progression	8 years	Median time to progression is defined as a greater than or equal to 20% increase increase in the sum of the volume of all index lesions based on volumetric analysis utilizing magnetic resonance imaging (MRI).Start of phase A or phase B to time of progression.
Primary	Number of Participants With Adverse Events	8 years	Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module.
Secondary	Quality of Life (QOL)	Baseline to pre cycle 4	Parents of participants aged 6-18 years completed the Impact of Pediatric Illness (IPI) Scale about their child prior to the start of cycles 1, 4, 7, and 10 and then after every 6 cycles. The IPI Scale assesses QOL in 4 domains: adaptive behavior, emotional functioning, medical/physical status, and cognitive functioning. Responses to the 43 items are made on a 5-point Likert scale (1-5) ranging from "not al all" to "a lot". Higher mean scores indicate better QOL. Parent total scores for participants on placebo were compared with scores from participants receiving tipifarnib on phase A.
Other	Median Time to Progression Using the Conventional 1-Dimensional Response Evaluation Criteria in Solid Tumors (RECIST) Method	8 years	Median time to progression is defined as ≥20% increase in diameter based on volumetric analysis using the 1-dimensional RECIST method. Start of phase A or phase B to time of progression.
Other	Median Time to Progression Using the 2-Dimensional World Health Organization (WHO) Solid Tumor Method	8 years	Median time to progression is defined as ≥25% increase in area based on volumetric analysis using the 2-dimensional WHO solid tumor method.

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	Median Time to Progression	8 years	phase A - 62 started and 2 were ineligible = 60 phase B - 43 started	Months	MEDIAN
PRIMARY	Number of Participants With Adverse Events	8 years	Adverse event data is in compliance with DSMB (Data Safety Monitoring Board).	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Quality of Life (QOL)	Baseline to pre cycle 4	Participants analyzed includes only those whose parents completed the IPI Scale at baseline and pre cycle 4 on phase A.	Total scores on a scale	MEAN
OTHER_PRE_SPECIFIED	Median Time to Progression Using the Conventional 1-Dimensional Response Evaluation Criteria in Solid Tumors (RECIST) Method	8 years	Phase B - Placebo group is not shown because this outcome measure only applies to the Phase A - Tipifarnib group.	Months	MEDIAN
OTHER_PRE_SPECIFIED	Median Time to Progression Using the 2-Dimensional World Health Organization (WHO) Solid Tumor Method	8 years	Phase B- Placebo group is not shown because this outcome measure only applies to the Phase A - Tipifarnib group.	Months	MEDIAN

Source ClinicalTrials.gov

Results by group11

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Median Time to Progression	Phase A - Tipifarnib	Placebo	10.6	95% Confidence Interval 8.1 to 18.3	Months
Median Time to Progression	Phase B - Placebo	Placebo	14.5	95% Confidence Interval 8.2 to 26.1	Months
Median Time to Progression	Phase A - Tipifarnib	Tipifarnib	19.2	95% Confidence Interval 8.1 to 30.3	Months
Median Time to Progression	Phase B - Placebo	Tipifarnib	13.3	95% Confidence Interval 8.0 to 19.6	Months
Number of Participants With Adverse Events	Tipifarnib & Placebo		59		Participants
Quality of Life (QOL)	Phase A - Tipifarnib	Baseline	3.69	Standard Deviation 0.43	Total scores on a scale
Quality of Life (QOL)	Phase B - Placebo	Baseline	3.70	Standard Deviation 0.53	Total scores on a scale
Quality of Life (QOL)	Phase A - Tipifarnib	Pre cycle 4	3.91	Standard Deviation 0.50	Total scores on a scale
Quality of Life (QOL)	Phase B - Placebo	Pre cycle 4	3.68	Standard Deviation 0.53	Total scores on a scale
Median Time to Progression Using the Conventional 1-Dimensional Response Evaluation Criteria in Solid Tumors (RECIST) Method	Phase A - Tipifarnib		NA		Months

1 to 10 of 11Source ClinicalTrials.gov

See all 11 results by group

Participant flow16

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Period 1-First Treatment	STARTED	Phase A -Tipifarnib	31
Period 1-First Treatment	STARTED	Phase B - Placebo	31
Period 1-First Treatment	Pts Who Progressed to Second Treatment	Phase A -Tipifarnib	20
Period 1-First Treatment	Pts Who Progressed to Second Treatment	Phase B - Placebo	23
Period 1-First Treatment	Ineligible Patients	Phase A -Tipifarnib	0
Period 1-First Treatment	Ineligible Patients	Phase B - Placebo	2
Period 1-First Treatment	COMPLETED	Phase A -Tipifarnib	20
Period 1-First Treatment	COMPLETED	Phase B - Placebo	23
Period 1-First Treatment	NOT COMPLETED	Phase A -Tipifarnib	11
Period 1-First Treatment	NOT COMPLETED	Phase B - Placebo	8

1 to 10 of 16Source ClinicalTrials.gov

See all 16 participant flow

Adverse events88

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	Abdominal pain	Gastrointestinal disorders	Tipifarnib & Placebo	20	60	26
other	Alkaline phosphatase	Metabolism and nutrition disorders	Tipifarnib & Placebo	2	60	2
other	Allergic rhinitis	Immune system disorders	Tipifarnib & Placebo	1	60	1
other	Alopecia	Skin and subcutaneous tissue disorders	Tipifarnib & Placebo	2	60	2
other	Anorexia	Gastrointestinal disorders	Tipifarnib & Placebo	10	60	11
other	Anxiety	Psychiatric disorders	Tipifarnib & Placebo	1	60	1
other	Bicarbonate	Metabolism and nutrition disorders	Tipifarnib & Placebo	17	60	21
other	Bilirubin	Hepatobiliary disorders	Tipifarnib & Placebo	8	60	9
other	Blurred vision	Eye disorders	Tipifarnib & Placebo	1	60	1
other	Bruising	Injury, poisoning and procedural complications	Tipifarnib & Placebo	1	60	1

1 to 10 of 88Source ClinicalTrials.gov

See all 88 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Tipifarnib & Placebo	0	60
Total, other adverse events	Tipifarnib & Placebo	59	60
Total, serious adverse events	Tipifarnib & Placebo	9	60

Source ClinicalTrials.gov

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
7626654	BACKGROUND	PubMed
8953130	BACKGROUND	PubMed
10656436	BACKGROUND	PubMed
24500418	RESULT	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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT00021541

GET https://conductscience.org/api/trials/v1/NCT00021541/events?page=2

API reference For AI assistants