Phase 2
N=23
Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas
Neurofibromatosis Type 1 · Precancerous Condition
Bottom Line
View on ClinicalTrials.gov: NCT00030264 ↗Enrolled (actual)
23
Serious AEs
26.1%
Results posted
Aug 2018
Primary outcome: Primary: Time to Disease Progression — 25.8; 29.2; 25.8 Months
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Methotrexate (Drug); Vinblastine (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Children's Hospital of Philadelphia
- Primary completion
- Dec 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Disease Progression |
25.8; 29.2; 25.8 | — |
Summary
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining methotrexate with vinblastine may be effective treatment for neurofibromatosis type 1 associated with progressive plexiform neurofibromas.
PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have neurofibromatosis type 1 associated with progressive plexiform neurofibromas.
Eligibility Criteria
Inclusion Criteria
- Progressive, debilitating, severely disfiguring or life-threatening plexiform neurofibroma (PN) which is not surgically resectable and for which there is no other standard medical management. Histologic confirmation of tumor is not required in the presence of consistent clinical and radiographic findings. However, if any clinical observation or scan suggests possible malignant transformation, the tumor must be biopsied prior to therapy. In addition to PN, all study subjects must have at least one other diagnostic criteria for Neurofibromatosis type 1 (NF1) listed below:
- 6 or more café-au-lait spots > 0.5 cm in prepubertal subjects or > 1.5 cm in postpubertal subjects
- freckling in the axilla or groin
- optic glioma
- 2 or more lisch nodules
- a distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex)
- a first degree relative with NF1
- Adequate bone marrow, renal, hepatic function:
- Absolute Neutrophil Count (ANC)> 1000 and platelet count >100,000 prior to initiation of therapy
- must have normal renal function: Blood Urea Nitrogen (BUN)/Creatinine 60. Patients who are wheelchair bound because of paralysis should be considered "ambulatory" when they are mobile and active in their wheelchairs rather than actually ambulatory.
- A Pregnancy test must be negative for females of childbearing age.
- Informed consent: All patients or their legal guardians (if the patient is less than 18 years old) must sign an approved document of informed consent indicating their understanding of the investigational nature and the risks of this study before beginning therapy. When appropriate pediatric patients will be included in all discussion in order to obtain verbal assent.
Exclusion Criteria
- Pregnant females are excluded
- Patient has had treatment with an investigational agent within the past 30 days.
- Ongoing radiation therapy, chemotherapy, hormonal therapy directed at the tumor or immunotherapy.
- Inability to return for follow-up visits or obtain follow-up studies required to assess toxicity and response to therapy.
Data sourced from ClinicalTrials.gov (NCT00030264). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.