Study of drug combinations including irinotecan, ifosfamide, vincristine, dactinomycin, regorafenib and other agents for children and adults with rhabdomyosarcoma
This clinical trial is studying Rhabdomyosarcoma, a type of cancer that develops in muscle tissue. The study will test several cancer medications including regorafenib, irinotecan, cyclophosphamide, doxorubicin, temozolomide, ifosfamide, vincristine, dactinomycin, and vinorelbine in different combinations.
The main purpose is to find better treatment options for both newly diagnosed and relapsed rhabdomyosarcoma in children and adults. The study will evaluate different timing and doses of chemotherapy and radiation therapy. Some patients will receive standard treatment while others will receive new combinations of medications.
The trial will last several years, with an estimated completion date in 2030. Throughout the study, researchers will monitor how well the treatments work by measuring survival rates and checking if the cancer returns. They will also track side effects and complications from the treatments.
1Initial diagnosis and qualification
You must have a confirmed diagnosis of rhabdomyosarcoma (except pleomorphic type)
A medical assessment will determine if you qualify as High Risk (HR) or Very High Risk (VHR) patient
2Frontline treatment – initial phase
Treatment will begin within 60 days after diagnostic biopsy/surgery
You will receive one of two medication combinations:
Option 1: Standard therapy with ifosfamide, vincristine, and actinomycin D (IVA)
Option 2: New combination adding irinotecan to the standard therapy (IrIVA)
3Radiotherapy options
If you are 2 years or older, you may receive radiation therapy
The timing of radiation treatment depends on your specific situation:
It may be given before surgery (pre-operative)
It may be given after surgery (post-operative)
The radiation dose will be determined based on your individual risk factors
4Maintenance treatment
After initial treatment, you will receive maintenance therapy with vinorelbine and cyclophosphamide
For High Risk patients: 6 cycles of treatment, with possibility of 6 additional cycles
For Very High Risk patients: 12 cycles of treatment, with possibility of 12 additional cycles
5Treatment for disease relapse
If the disease returns, you may receive one of these combinations:
Option 1: vincristine, irinotecan, and temozolomide (VIrT)
Option 2: vincristine, irinotecan, and regorafenib (VIrR)
Who Can Join the Study?
Confirmed diagnosis of rhabdomyosarcoma (a type of muscle cancer), except for the pleomorphic type
Age requirements vary depending on study group:
At least 6 months old for most study groups
Over 12 months and up to 25 years for some treatments
At least 2 years old for radiation therapy
No previous cancer treatment except surgery (for newly diagnosed patients)
Good liver function:
Normal or slightly elevated bilirubin levels
Liver enzymes less than 2.5 times the normal limit
Adequate blood counts:
White blood cells (neutrophils) at least 1.0 x 10^9/L
Platelets at least 80 x 10^9/L
Good kidney function with adequate creatinine clearance
Negative pregnancy test for females who can become pregnant
Agreement to use birth control during treatment and:
12 months after treatment for females
6 months after treatment for males
Must be healthy enough to receive treatment
Written informed consent from patient and/or parent/legal guardian
Who Cannot Join the Study?
Patients who do not have confirmed diagnosis of Rhabdomyosarcoma (a type of soft tissue cancer)
Patients younger than 6 months or older than 21 years of age
Patients with severe heart problems that would make chemotherapy unsafe
Patients with severe kidney or liver dysfunction that would interfere with treatment
Patients who have received any other cancer treatment within the last 30 days
Pregnant or breastfeeding women
Patients who are unable to follow the study protocol due to serious medical or psychological conditions
Patients who have participated in another clinical trial within the past 30 days
Patients who are allergic to any of the study medications (irinotecan, ifosfamide, vincristine, actinomycin D, vinorelbine, cyclophosphamide, regorafenib, or temozolomide)
Patients with active, uncontrolled infections
Patients who cannot undergo required imaging procedures
Patients who do not meet the required blood test values for safe treatment
Irinotecan
A chemotherapy medication used to treat various types of cancer. In this trial, it is being tested in combination with other medications for treating rhabdomyosarcoma.
Ifosfamide
A chemotherapy medication that works by stopping cancer cells from dividing and growing. It is used as part of combination therapy for treating rhabdomyosarcoma.
Vincristine
A chemotherapy medication that prevents cancer cells from dividing. It is commonly used in combination with other medications to treat various types of cancer, including rhabdomyosarcoma.
Actinomycin D
A chemotherapy medication that works by interfering with cancer cell DNA. It is used as part of combination therapy for treating rhabdomyosarcoma.
Vinorelbine
A chemotherapy medication that works by stopping cancer cells from separating into new cells. In this trial, it is being tested as part of maintenance therapy.
Cyclophosphamide
A chemotherapy medication that works by slowing or stopping cell growth. In this trial, it is being used as part of maintenance therapy and can be given orally.
Regorafenib
A targeted therapy medication that works by blocking several proteins that signal cancer cells to grow. It is being tested in combination with other medications for treating relapsed rhabdomyosarcoma.
Temozolomide
A chemotherapy medication that works by stopping cancer cells from making new DNA. In this trial, it is being used as part of combination therapy for treating relapsed rhabdomyosarcoma.
Rhabdomyosarcoma – A rare type of cancer that develops in soft tissue, specifically in muscle cells that are in the process of developing. It most commonly occurs in children and teens, forming in muscles that are attached to bones. The disease can start in various parts of the body, including the head and neck area, arms, legs, trunk, or in internal organs. As the tumor grows, it can cause swelling, pain, or changes in the affected area. The cancer cells can spread to other parts of the body through the blood or lymph system if not addressed.
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