Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
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| ClinicalTrials.gov Identifier: NCT00053937 |
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Recruitment Status
:
Completed
First Posted
: February 6, 2003
Last Update Posted
: April 30, 2015
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RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Pirfenidone may slow the growth or prevent further development of plexiform neurofibromas.
PURPOSE: Phase I trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and plexiform neurofibroma.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Neurofibromatosis Type 1 Precancerous Condition | Drug: pirfenidone | Phase 1 |
OBJECTIVES:
- Determine the maximum tolerated dose or "comparable dose" of pirfenidone in pediatric patients with neurofibromatosis type 1 and inoperable, symptomatic plexiform neurofibromas.
- Determine the toxic effects of this drug in these patients.
- Determine the plasma pharmacokinetics of this drug in these patients.
- Determine, preliminarily, if this drug could be beneficial for pediatric patients with refractory solid tumors.
- Assess the quality of life of patients treated with this drug.
OUTLINE: This is an open-label, multicenter, dose-escalation study.
Patients receive oral pirfenidone three times daily on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of pirfenidone until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
Quality of life is assessed at baseline, before course 4, and then after every 6 courses.
PROJECTED ACCRUAL: A total of 3-18 patients will be accrued for this study within 18 months.
| Study Type : | Interventional (Clinical Trial) |
| Masking: | None (Open Label) |
| Primary Purpose: | Prevention |
| Official Title: | Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas |
| Study Start Date : | December 2002 |
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| Ages Eligible for Study: | 3 Years to 21 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
- Diagnosis of neurofibromatosis type 1 (NF1) AND
-
Plexiform neurofibromas
- Neurofibromas that have grown along the length of a nerve and may involve multiple fascicles and branches (spinal neurofibromas involve 2 or more levels with connection between the levels or extending laterally along the nerve)
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Potential to cause significant morbidity such as:
- Head and neck lesions that could compromise airway or great vessels
- Brachial or lumbar plexus lesions that could cause nerve compression and loss of function
- Lesions that could result in major deformity (e.g., orbital lesions) or significant cosmetic problems
- Lesions of the extremity that cause limb hypertrophy or loss of function
- Painful lesions
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Meets at least 1 other diagnostic criteria for NF1
- 6 or more cafe-au-lait spots (at least 0.5 cm in prepubertal patients or at least 1.5 cm in postpubertal patients)
- Freckling in the axilla or groin
- Optic glioma
- 2 or more Lisch nodules
- Distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
- First-degree relative with NF1
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Measurable plexiform neurofibromas
- At least 3 cm in 1 dimension
- Tumor resection not feasible
- No history of malignant peripheral nerve sheath tumor or other cancer
- No evidence of an active optic glioma requiring chemotherapy or radiotherapy
- No malignant glioma
PATIENT CHARACTERISTICS:
Age
- 3 to 21
Performance status
- Karnofsky 50-100% (over 10 years of age)
- Lansky 50-100% (10 years and under)
Life expectancy
- Not specified
Hematopoietic
- Absolute granulocyte count at least 1,500/mm^3
- Hemoglobin at least 9.0 g/dL
- Platelet count at least 150,000/mm^3
Hepatic
- Bilirubin normal
- SGPT no greater than 2 times upper limit of normal
- No clinically significant hepatic dysfunction that would preclude study participation
Renal
- Creatinine normal for age OR
- Creatinine clearance at least 70 mL/min
Cardiovascular
- No clinically significant cardiac dysfunction that would preclude study participation
Pulmonary
- No clinically significant pulmonary dysfunction that would preclude study participation
Other
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception during and for 2 months after study
- Must be able to take pirfenidone orally
- No serious infections
- No clinically significant unrelated systemic illness or organ dysfunction that would preclude study participation
PRIOR CONCURRENT THERAPY:
Biologic therapy
- At least 30 days since prior immunotherapy
- No concurrent immunotherapy
- No concurrent hematopoietic growth factors
Chemotherapy
- At least 30 days since prior chemotherapy
- No concurrent chemotherapy directed at the tumor
Endocrine therapy
- At least 30 days since prior hormonal therapy directed at the tumor
- No concurrent hormonal therapy directed at the tumor
Radiotherapy
- At least 90 days since prior radiotherapy to the site of the plexiform neurofibroma
- No concurrent radiotherapy directed at the tumor
Surgery
- Not specified
Other
- Recovered from prior therapy
- More than 30 days since prior investigational agents
- No prior pirfenidone
- No other concurrent investigational agents
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00053937
| United States, Alabama | |
| University of Alabama at Birmingham Comprehensive Cancer Center | |
| Birmingham, Alabama, United States, 35294-3300 | |
| United States, District of Columbia | |
| Children's National Medical Center | |
| Washington, District of Columbia, United States, 20010-2970 | |
| United States, Illinois | |
| Children's Memorial Hospital - Chicago | |
| Chicago, Illinois, United States, 60614 | |
| United States, Maryland | |
| Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | |
| Baltimore, Maryland, United States, 21231 | |
| Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support | |
| Bethesda, Maryland, United States, 20892 | |
| United States, Massachusetts | |
| Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute | |
| Boston, Massachusetts, United States, 02115 | |
| United States, Minnesota | |
| Mayo Clinic Cancer Center | |
| Rochester, Minnesota, United States, 55905 | |
| United States, Missouri | |
| Washington University School of Medicine | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, New York | |
| Beth Israel Medical Center - Singer Division | |
| New York, New York, United States, 10128 | |
| University Hospital at State University of New York - Upstate Medical University | |
| Syracuse, New York, United States, 13210 | |
| United States, Ohio | |
| Cleveland Clinic Taussig Cancer Center | |
| Cleveland, Ohio, United States, 44195 | |
| United States, Oregon | |
| Cancer Institute at Oregon Health and Science University | |
| Portland, Oregon, United States, 97239-3098 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Children's Hospital of Pittsburgh | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| United States, Texas | |
| Texas Children's Cancer Center | |
| Houston, Texas, United States, 77030-2399 | |
| Study Chair: | Brigitte C. Widemann, MD | National Cancer Institute (NCI) |
| ClinicalTrials.gov Identifier: | NCT00053937 History of Changes |
| Obsolete Identifiers: | NCT00050453 |
| Other Study ID Numbers: |
CDR0000269598 NCI-03-C-0058A |
| First Posted: | February 6, 2003 Key Record Dates |
| Last Update Posted: | April 30, 2015 |
| Last Verified: | April 2004 |
Keywords provided by National Cancer Institute (NCI):
|
plexiform neurofibroma neurofibromatosis type 1 |
Additional relevant MeSH terms:
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Neurofibromatoses Neurofibromatosis 1 Neurofibroma Neurofibroma, Plexiform Precancerous Conditions Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Neoplasms by Histologic Type Neoplasms Neoplastic Syndromes, Hereditary Neurocutaneous Syndromes Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn |
Peripheral Nervous System Diseases Neuromuscular Diseases Peripheral Nervous System Neoplasms Nervous System Neoplasms Pirfenidone Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents, Non-Steroidal Analgesics, Non-Narcotic Anti-Inflammatory Agents Antirheumatic Agents Antineoplastic Agents |