Trials / Active Not Recruiting
Active Not RecruitingNCT04284774
Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Tipifarnib in Patients With Tumors Harboring HRAS Genomic Alterations
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 5 (actual)
- Sponsor
- National Cancer Institute (NCI) · NIH
- Sex
- All
- Age
- 12 Months – 21 Years
- Healthy volunteers
- Not accepted
Summary
This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a genetic alteration in the gene HRAS. Tipifarnib may block the growth of cancer cells that have specific genetic changes in a gene called HRAS and may reduce tumor size.
Detailed description
PRIMARY OBJECTIVE: I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with tipifarnib with advanced solid tumors (including central nervous system \[CNS\] tumors), lymphomas or histiocytic disorders that harbor activating genetic alterations in HRAS. SECONDARY OBJECTIVES: I. To estimate the progression free survival in pediatric patients treated with tipifarnib with advanced solid tumors (including CNS tumors), lymphomas or histiocytic disorders that harbor activating genetic alterations in HRAS. II. To obtain information about the tolerability of tipifarnib in children and adolescents with relapsed or refractory cancer. EXPLORATORY OBJECTIVES: I. To evaluate other biomarkers as predictors of response to tipifarnib and specifically, whether tumors that harbor different missense mutations or variant allele frequency will demonstrate differential response to tipifarnib treatment. II. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA). OUTLINE: Patients receive tipifarnib tablets 350 mg/m\^2/dose orally (PO) or via nasogastric or gastric tube twice daily (BID) (maximum 600 mg/dose BID) with food on days 1-7 and 15-21. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days, then periodically thereafter.
Conditions
- Malignant Solid Neoplasm
- Recurrent Adrenal Gland Pheochromocytoma
- Recurrent Ectomesenchymoma
- Recurrent Ependymoma
- Recurrent Ewing Sarcoma
- Recurrent Hepatoblastoma
- Recurrent Kidney Wilms Tumor
- Recurrent Langerhans Cell Histiocytosis
- Recurrent Malignant Germ Cell Tumor
- Recurrent Malignant Glioma
- Recurrent Medulloblastoma
- Recurrent Melanoma
- Recurrent Neuroblastoma
- Recurrent Non-Hodgkin Lymphoma
- Recurrent Osteosarcoma
- Recurrent Peripheral Primitive Neuroectodermal Tumor
- Recurrent Rhabdoid Tumor
- Recurrent Rhabdoid Tumor of the Kidney
- Recurrent Rhabdomyosarcoma
- Recurrent Soft Tissue Sarcoma
- Recurrent Thyroid Gland Carcinoma
- Recurrent WHO Grade 2 Glioma
- Refractory Adrenal Gland Pheochromocytoma
- Refractory Ependymoma
- Refractory Ewing Sarcoma
- Refractory Hepatoblastoma
- Refractory Langerhans Cell Histiocytosis
- Refractory Malignant Germ Cell Tumor
- Refractory Malignant Glioma
- Refractory Medulloblastoma
- Refractory Melanoma
- Refractory Neuroblastoma
- Refractory Non-Hodgkin Lymphoma
- Refractory Osteosarcoma
- Refractory Peripheral Primitive Neuroectodermal Tumor
- Refractory Rhabdoid Tumor
- Refractory Rhabdoid Tumor of the Kidney
- Refractory Rhabdomyosarcoma
- Refractory Soft Tissue Sarcoma
- Refractory Thyroid Gland Carcinoma
- Refractory WHO Grade 2 Glioma
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Tipifarnib | Tipifarnib tablets |
Timeline
- Start date
- 2020-10-13
- Primary completion
- 2024-03-31
- Completion
- 2027-09-30
- First posted
- 2020-02-26
- Last updated
- 2025-12-04
- Results posted
- 2025-06-03
Locations
172 sites across 2 countries: United States, Puerto Rico
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04284774. Inclusion in this directory is not an endorsement.