Tipifarnib for Rhabdomyosarcoma

Phase-Based Progress Estimates
Rhabdomyosarcoma+69 More
Tipifarnib - Drug
< 65
All Sexes
What conditions do you have?

Study Summary

This trial studies how well tipifarnib works in treating patients with advanced solid tumors, lymphoma, or histiocytic disorders that have a HRAS gene alteration.

Eligible Conditions
  • Rhabdomyosarcoma
  • Adrenal Gland Diseases
  • Hepatoblastoma
  • Glioma
  • Recurrent Melanoma
  • Recurrent WHO Grade 2 Glioma
  • Malignant Solid Neoplasms
  • Medulloblastoma
  • Refractory Ependymoma
  • Histiocytosis
  • Recurrent Ectomesenchymoma
  • Ependymoma
  • recurrent Ewing's Sarcoma
  • Kidney
  • Recurrent Langerhans Cell Histiocytosis
  • Recurrent Malignant Germ Cell Tumor
  • Recurrent Malignant Gliomas
  • Neuroblastoma
  • Lymphoma, Non-Hodgkin
  • Osteosarcoma
  • Recurrent Peripheral Primitive Neuroectodermal Tumor
  • Recurrent Rhabdoid Tumor
  • Recurrent Rhabdoid Tumor of the Kidney
  • Recurrent Soft Tissue Sarcoma
  • Carcinoma
  • Refractory Ewing Sarcoma
  • Refractory Soft Tissue Sarcomas
  • Refractory Thyroid Gland Carcinoma
  • Refractory WHO Grade 2 Glioma
  • Refractory Adrenal Gland Pheochromocytoma
  • Refractory Hepatoblastoma
  • Refractory Malignant Germ Cell Tumor
  • Refractory Malignant Glioma
  • Refractory Osteosarcoma
  • Refractory Peripheral Primitive Neuroectodermal Tumor
  • Refractory Rhabdoid Tumor
  • Refractory Medulloblastoma
  • Refractory Melanoma
  • Refractory Neuroblastoma

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: Up to 7 years

Year 7
Year 2
Objective response rate (complete response + partial response) in pediatric patients treated with tipifarnib
Percentage of patients experiencing grade 3 or higher adverse events
Year 7
Progression free survival (PFS)
Up to 7 years
Biomarker analysis

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

Arm B (Clinical Observation)
17%White blood cell decreased
16%Platelet count decreased
This histogram enumerates side effects from a completed undefined Phase 3 trial (NCT00093470) in the Arm B (Clinical Observation) ARM group. Side effects include: Anemia with 19%, White blood cell decreased with 17%, Platelet count decreased with 16%, Fatigue with 1%.

Trial Design

1 Treatment Group

Treatment (tipifarnib)
1 of 1
Experimental Treatment

49 Total Participants · 1 Treatment Group

Primary Treatment: Tipifarnib · No Placebo Group · Phase 2

Treatment (tipifarnib)
Experimental Group · 1 Intervention: Tipifarnib · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 7 years

Who is running the clinical trial?

National Cancer Institute (NCI)Lead Sponsor
12,990 Previous Clinical Trials
41,297,589 Total Patients Enrolled
86 Trials studying Rhabdomyosarcoma
16,611 Patients Enrolled for Rhabdomyosarcoma
Christine A PratilasPrincipal InvestigatorChildren's Oncology Group

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Lesions only detected by nuclear medicine studies (e.g.
Bone marrow infiltration except that detected by MIBG scan for neuroblastoma.
You must have a body surface area > 0.29 m^2 at enrollment.
You have malignant fluid collections (e.g.
Elevated tumor markers in plasma or CSF.
You have previously had radiotherapy for cancer.
The size of the tumor does not meet the measurement requirements for RECIST 1.1.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 30th, 2021

Last Reviewed: October 31st, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

How old are they?
18 - 65100.0%
What site did they apply to?
Eastern Maine Medical Center100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%