Clinical trial · Interventional
Medical Treatment of "High-Risk" Neurofibromas
Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neurofibromatosis 1 | — | UNRESOLVED | — |
Interventions
Interventions (4)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Celecoxib (Celebrex) | Drug | Celecoxib | ALIAS |
| Peg-Interferon alpha-2b | Drug | Peginterferon Alfa-2b | ALIAS |
| Temozolomide (temodar) | Drug | Temozolomide | ALIAS |
| Vincristine Sulfate (Oncovin) | Drug | Vincristine | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Open-Label Intervention
- description
- This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study.
- interventionNames
- Drug: Peg-Interferon alpha-2b
- Drug: Celecoxib (Celebrex)
- Drug: Temozolomide (temodar)
- Drug: Vincristine Sulfate (Oncovin)
Primary outcomes (3)
- measure
- Improvement of Symptoms and Pain
- timeFrame
- Monthly physical exam first three months and then every three months after, for up to 36 months
- description
- Subjects will be evaluated for pain and Quality of Life scores
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 2 Years
- Maximum age
- 30 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available