Clofarabine for Langerhans in Pedi
Primary Purpose
Langerhans Cell Histiocytosis
Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Clofarabine
Sponsored by
About this trial
This is an interventional treatment trial for Langerhans Cell Histiocytosis
Eligibility Criteria
Inclusion Criteria:
- Have failed first line treatment with prednisone and vinblastine
- Clinical evidence of involvement of hematopoietic system, liver or spleen
- Have failed salvage treatment with cladribine/cytarabine or are not considered to be eligible for such treatment
- Willing to use effective contraception during study and for six months after study treatment
- Adequate marrow and organ function
Exclusion Criteria:
- Pregnant or breastfeeding
- Have received chemotherapy or radiotherapy within 2 weeks of study entry
- Have not recovered from adverse events due to previously administered agents
- Receiving other study agents
- Taking drugs with known renal toxicity
- Use of alternative medicine during study treatment
- Uncontrolled intercurrent illness
- History of a different malignancy except if disease-free for at least five years or within five years for cervical cancer in situ and basal and squamous cell carcinoma of the skin
- Known to be HIV positive on antiretroviral therapy
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Low-Risk
High-Risk
Arm Description
Multifocal or Low-Risk Multi System Clofarabine, Low Dose, Two Cycles
High-risk Multi System Clofarabine, Standard Dose, Two Cycles
Outcomes
Primary Outcome Measures
Estimated Response Rate
To estimate the response rate of participants with recurrent LCH to clofarabine within each of two strata: a) low-risk participants with disease reactivation; and b) high-risk participants with risk-organ involvement that is refractory to standard treatment.
Secondary Outcome Measures
Estimated of Progression Free Survival
To estimate the progression-free survival after clofarabine treatment
Estimated survival with risk organ involvement
To estimate survival of participants with refractory multi-system LCH with risk organ involvement treated with clofarabine
Chemotherapy feasibility
To determine if clofarabine therapy can be delivered as planned ('chemotherapy feasibility'), and to describe toxicities of clofarabine in participants with LCH.
Full Information
NCT ID
NCT01796405
First Posted
February 19, 2013
Last Updated
August 24, 2015
Sponsor
Dana-Farber Cancer Institute
1. Study Identification
Unique Protocol Identification Number
NCT01796405
Brief Title
Clofarabine for Langerhans in Pedi
Official Title
Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis
Study Type
Interventional
2. Study Status
Record Verification Date
August 2015
Overall Recruitment Status
Withdrawn
Study Start Date
undefined (undefined)
Primary Completion Date
October 2017 (Anticipated)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Dana-Farber Cancer Institute
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if the drug is effective for treating different diseases. It also means that the FDA has not yet approved clofarabine for your disease.
Clofarabine is a chemotherapy drug that has been used in the treatment of leukemia in children and adults. Information from other research studies suggests that this drug may also be effective in patients with LCH.
The purpose of this study is to estimate the response rates of participants with recurrent LCH to clofarabine within each of two strata: a) low-risk participants with disease reactivation, and b) high-risk participants with risk-organ involvement.
Other purposes are to estimate the progression-free survival after clofarabine treatment, estimate survival of participants with refractory multi-system LCH with risk organ involvement treated with clofarabine and to describe toxicities of clofarabine in participants with LCH.
Detailed Description
If you are willing to participate in this research study you will be asked to undergo some screening test or procedure to find out if you are eligible. Many of these tests and procedures are likely to be part of regular care and may be done even if it turns out that you do not take part in the research study. If you have had some of these tests or procedures recently, they may or may not have to be repeated. These tests and procedures include the following: a medical history, performance status, blood tests, assessment of disease and pregnancy test. If these tests show that you are eligible to participate in the research study, you will begin the study treatment. If you do not meet the eligibility criteria, you will not be able to participate in this research study. After confirming eligibility you will also have a bone marrow test, electrocardiogram and urinalysis.
You will receive a maximum of 6 cycles of study treatment. Each treatment cycle will last approximately 4 weeks. In each cycle, you will receive the study drug clofarabine daily on the first 5 days (days 1 to 5) and rest for the remaining days. Clofarabine will be administered into the vein (IV) over two hours in the clinic or in the hospital. If you have high-risk disease, the dose of clofarabine will be higher during the first two cycles.
Before the start of each cycle, and at the end of your therapy, you will have a physical exam and you will be asked questions about your general health and specific questions about any problems that you are having and any medications you are taking. You will also have blood and urine tests done to monitor organ functions. You will also undergo an assessment of your disease by x-ray, CT scan, MRI or PET or bone scans, which wil be performed every two cycles and at the end of study treatment.
After the final dose of the study drug there will be a follow up period. Once every three months for the first year, twice yearly for the second and third years, and then annually, you will have a physical exam and be asked questions about your general health. You will also have blood and urine tests performed.
Twice yearly for the first three years, x-ray, CT scan, MRI, PET, and/or bone scans will be repeated if your doctor feels they are necessary.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Langerhans Cell Histiocytosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Low-Risk
Arm Type
Experimental
Arm Description
Multifocal or Low-Risk Multi System Clofarabine, Low Dose, Two Cycles
Arm Title
High-Risk
Arm Type
Experimental
Arm Description
High-risk Multi System Clofarabine, Standard Dose, Two Cycles
Intervention Type
Drug
Intervention Name(s)
Clofarabine
Primary Outcome Measure Information:
Title
Estimated Response Rate
Description
To estimate the response rate of participants with recurrent LCH to clofarabine within each of two strata: a) low-risk participants with disease reactivation; and b) high-risk participants with risk-organ involvement that is refractory to standard treatment.
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Estimated of Progression Free Survival
Description
To estimate the progression-free survival after clofarabine treatment
Time Frame
2 years
Title
Estimated survival with risk organ involvement
Description
To estimate survival of participants with refractory multi-system LCH with risk organ involvement treated with clofarabine
Time Frame
2 years
Title
Chemotherapy feasibility
Description
To determine if clofarabine therapy can be delivered as planned ('chemotherapy feasibility'), and to describe toxicities of clofarabine in participants with LCH.
Time Frame
2 years
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Have failed first line treatment with prednisone and vinblastine
Clinical evidence of involvement of hematopoietic system, liver or spleen
Have failed salvage treatment with cladribine/cytarabine or are not considered to be eligible for such treatment
Willing to use effective contraception during study and for six months after study treatment
Adequate marrow and organ function
Exclusion Criteria:
Pregnant or breastfeeding
Have received chemotherapy or radiotherapy within 2 weeks of study entry
Have not recovered from adverse events due to previously administered agents
Receiving other study agents
Taking drugs with known renal toxicity
Use of alternative medicine during study treatment
Uncontrolled intercurrent illness
History of a different malignancy except if disease-free for at least five years or within five years for cervical cancer in situ and basal and squamous cell carcinoma of the skin
Known to be HIV positive on antiretroviral therapy
12. IPD Sharing Statement
Learn more about this trial
Clofarabine for Langerhans in Pedi
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