Trial · NCT00076102
Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
NCT00076102, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2012-07-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas. Trials, record NCT00076102. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00076102
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
- NCT number
- NCT00076102
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Neurofibromatosis 1, Neurofibroma, Plexiform
- Interventions
- Pirfenidone
- First posted
- 2004-01-14
- Last updated
- 2018-04-23
- Results first posted
- 2012-07-12
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Median Time to Disease Progression | 5 years | Time to progression is defined as greater than or equal to 20% increase in plexiform neurofibromas (PN) volume on magnetic resonance imaging (MRI). |
| Primary | Number of Participants With Adverse Events | 5 years | Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module. |
| Primary | Percentage of Participants Who Had an Objective Response Rate | ≥4 weeks | Objective response rate is defined as a complete response (CR) or partial response (PR). Complete response is a complete resolution of all measurable or palpable soft tissue tumors for ≥4 weeks and no appearance of new lesions. Partial response is a ≥50% reduction in the sum of the volume of all index lesions for ≥4 weeks. |
| Secondary | Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Baseline | Quality of life was assessed by the Impact of Pediatric Illness scale for children 6-18 years of age. The child's primary caregiver completed the proxy Parent Form and children answered either the self-report Child or Adolescent (11-18 years) Form. The parallel IPI Scale forms assess four domains: adaptive behavior, emotional functioning, medical/physical status, and cognitive functioning. Responses to the 43 items are made on a 3-or5-point Likert scale (1 to 5 for Parent and Adolescent Form and 1, 3, 5 for the Child Form) ranging from "not at all" to "a lot". Item scores are transformed to a scale of 0-100, and then mean scores are calculated for the four domains and total scale with higher scores indicating better QOL. Baseline comparisons between child and parent total and domain scores were performed. |
| Secondary | Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale | prior to cycles 1, 4, 7 and 10. | Quality of life was assessed by the Impact of Pediatric Illness scale for children 6-18 years of age. The child's primary caregiver completed the proxy Parent Form and children answered either the self-report Child or Adolescent (11-18 years) Form prior to cycles 1, 4, 7 and 10. The parallel IPI Scale forms assess four domains: adaptive behavior, emotional functioning, medical/physical status, and cognitive functioning. Responses to the 43 items are made on a 3-or5-point Likert scale (1 to 5 for Parent and Adolescent Form and 1, 3, 5 for the Child Form) ranging from "not at all" to "a lot". Item scores are transformed to a scale of 0-100, and then mean scores are calculated for the four domains and total scale with higher scores indicating better QOL. |
| Secondary | Number of Participants With A Response Evaluation Determined by the Comparison of One-Dimensional (1D) Magnetic Resonance Imaging | Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression | Index lesions will be followed for progression by 1D magnetic resonance imaging. Progression is defined as a ≥20% increase in the volume of at least one of the index plexiform neurofibromas compared to the pretreatment volume measured prior to the start of treatment. |
| Secondary | Number of Participants With A Response Evaluation Determined by the Comparison of Two-Dimensional (2D) Magnetic Resonance Imaging | Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression | Index lesions will be followed for progression by 2D magnetic resonance imaging. Progression is defined as a ≥20% increase in the volume of at least one of the index plexiform neurofibromas compared to the pretreatment volume measured prior to the start of treatment. |
| Secondary | Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging | Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter, approximately 5 years | Index lesions will be followed for progression by 3D magnetic resonance imaging. Compete response is a complete resolution of all measurable or palpable soft tissue tumors for ≥4 weeks and no appearance of new lesions. Partial response is a ≥50% reduction in the sum of the volume of all index lesions for ≥4 weeks. Progression is defined as a ≥20% increase in the volume of at least one of the index plexiform neurofibromas compared to the pretreatment volume measured prior to the start of treatment. Stable disease is a \<20% increase, and \<25% decrease in the sum of the volume of all index lesions for ≥4 weeks. Minor response is a ≥25% but \<50% reduction in the sum of the volume of all index lesions for ≥4 weeks. |
| Secondary | Number of Participants Who Contributed to the Tissue Bank | 5 years | Tumor specimens from patients who undergo tumor surgery or biopsies for clinical reasons. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Median Time to Disease Progression | 5 years | Months | MEDIAN | |
| PRIMARY | Number of Participants With Adverse Events | 5 years | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Percentage of Participants Who Had an Objective Response Rate | ≥4 weeks | percentage of participants | NUMBER | |
| SECONDARY | Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Baseline | Of the 36 patients enrolled, 28 were within the age range of the IPI Scale at baseline. Two patients did not have any QOL forms completed; one did not have a baseline evaluation, and six patients had missing follow-up evaluations. Thus, QOL data is presented for 19 subjects. | Scores on a scale | MEAN |
| SECONDARY | Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale | prior to cycles 1, 4, 7 and 10. | Of the 36 pts enrolled, 28 were within the age range of the IPI Scale at baseline. 2 pts did not have any QOL forms completed; 1 did not have a baseline \& 6 pts had missing f/u evals. QOL data is presented for 19 pts. Due to pts not completing forms at random f/u evals, some time points have fewer than 19 pts included in the longitudinal analysis. | scores on a scale | MEAN |
| SECONDARY | Number of Participants With A Response Evaluation Determined by the Comparison of One-Dimensional (1D) Magnetic Resonance Imaging | Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression | This outcome measure was not done because only 3D imaging was performed. Due to a detailed comparison of 1D-2D and 3D imaging for another study (Tipifarnib R115777) the investigator determined that a detailed comparison of 1D-2D and 3D analysis would really add no new knowledge. Therefore only 3D analysis was performed for this trial. | ||
| SECONDARY | Number of Participants With A Response Evaluation Determined by the Comparison of Two-Dimensional (2D) Magnetic Resonance Imaging | Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression | This outcome measure was not done because only 3D imaging was performed. Due to a detailed comparison of 1D-2D and 3D imaging for another study (Tipifarnib R115777) the investigator determined that a detailed comparison of 1D-2D and 3D analysis would really add no new knowledge. Therefore only 3D analysis was performed for this trial. | ||
| SECONDARY | Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging | Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter, approximately 5 years | Five patients were not analyzed due to clinical progression (n=1), plexiform neurofibroma surgery (n=1), progression in a pre-existing brain tumor (n=1), and refusal of further therapy (n=2). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Contributed to the Tissue Bank | 5 years | Participants declined to contribute specimens for the tissue bank. |
Source ClinicalTrials.gov
Results by group31
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Median Time to Disease Progression | Pirfenidone | 13.2 | 95% Confidence Interval 8.2 to 18.6 | Months | |
| Number of Participants With Adverse Events | Pirfenidine | 36 | Participants | ||
| Percentage of Participants Who Had an Objective Response Rate | Pirfenidine | Complete Response | 0 | percentage of participants | |
| Percentage of Participants Who Had an Objective Response Rate | Pirfenidine | Partial Response | 0 | percentage of participants | |
| Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Pirfenidine | Total score parent | 72.7 | Standard Deviation 10.7 | Scores on a scale |
| Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Pirfenidine | Total score child | 69.1 | Standard Deviation 12.0 | Scores on a scale |
| Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Pirfenidine | Parent proxy-adaptive behavior | 72.1 | Standard Deviation 10.2 | Scores on a scale |
| Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Pirfenidine | Child Self-Report-adaptive behavior | 76.7 | Standard Deviation 11.8 | Scores on a scale |
| Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Pirfenidine | Parent proxy - emotional functioning | 72.4 | Standard Deviation 13.4 | Scores on a scale |
| Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline | Pirfenidine | Child Self-Report-emotional functioning | 63.7 | Standard Deviation 14.7 | Scores on a scale |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pirfenidone | 36 | |
| Overall Study | COMPLETED | Pirfenidone | 31 | |
| Overall Study | NOT COMPLETED | Pirfenidone | 5 |
Source ClinicalTrials.gov
Adverse events128
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 | ALT, SGPT (serum glutamic pyruvic transaminase) | Investigations | Pirfenidone | 2 | 36 | 2 |
| other | Albumin, serum low (hypoalbuminemia) | Metabolism and nutrition disorders | Pirfenidone | 5 | 36 | 7 |
| other | Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip) | Immune system disorders | Pirfenidone | 7 | 36 | 8 |
| other | Anorexia | Gastrointestinal disorders | Pirfenidone | 9 | 36 | 22 |
| other | Atrophy, skin | Skin and subcutaneous tissue disorders | Pirfenidone | 1 | 36 | 1 |
| other | Bicarbonate, serum low | Investigations | Pirfenidone | 4 | 36 | 7 |
| other | Bronchospasm, wheezing | Respiratory, thoracic and mediastinal disorders | Pirfenidone | 1 | 36 | 2 |
| other | Calcium, serum-high (hypercalcemia) | Metabolism and nutrition disorders | Pirfenidone | 4 | 36 | 5 |
| other | Calcium, hypocalcemia | Metabolism and nutrition disorders | Pirfenidone | 9 | 36 | 13 |
| other | Cardiac arrhythmia-Other, specify- sinus tachycardia | Cardiac disorders | Pirfenidone | 1 | 36 | 1 |
1 to 10 of 128Source ClinicalTrials.gov
See all 128 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pirfenidone | 0 | 36 |
| Total, other adverse events | Pirfenidone | 36 | 36 |
| Total, serious adverse events | Pirfenidone | 3 | 36 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
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/NCT00076102
GET https://conductscience.org/api/trials/v1/NCT00076102/results?page=2