Phase 2
Completed N=9
Medical Treatment of "High-Risk" Neurofibromas
Neurofibromatosis 1
Source: ClinicalTrials.gov NCT00846430 ↗
Enrolled (actual)
9
Serious AEs
11.1%
Results posted
Aug 2023
Primary outcomePrimary: Improvement of Symptoms and Pain — 9 Participants
Summary
Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction and/or become life-threatening. Presently, there is no effective medical therapy to offer patients with "high-risk" plexiform neurofibromas, and surgery does not provide lasting help. This study will evaluate the effectiveness of two treatment combinations in patients with "high-risk" plexiform neurofibromas.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Improvement of Symptoms and Pain |
9 | — |
| PRIMARY At Least 50% Shrinkage in Tumor Measurements by Physical Examination |
7 | — |
| PRIMARY Response by MRI Measurements |
0; 1; 8; 2; 1; 6 | — |
| SECONDARY No Reported Psychological Toxicity Based Upon Psychological Evaluations |
8 | — |
Eligibility Criteria
Inclusion Criteria
- "High-Risk" Plexiform Neurofibromas associated with a diagnosis of NF1
- 2-30 years old (minimum bodyweight of 10 kilograms)
- Adequate renal function
Exclusion Criteria
- Previously untreated active optic glioma
- History of any previous allergy to study medications
- History of ischemic vascular disease
- Pregnancy / Breast feeding
Data sourced from ClinicalTrials.gov (NCT00846430). 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. Informational only — not medical advice.