Langerhans Cell HistiocytosisPhase 2NCT01796405
Clofarabine for Langerhans in Pedi
A Phase 2 study testing Clofarabine in people with Langerhans Cell Histiocytosis, run by Dana-Farber Cancer Institute.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Langerhans Cell Histiocytosis
- What is being tested?
- Clofarabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Start not givento Oct 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
About this study
Official title: Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis
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.
Read more
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.
Read the 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.
- Conditions
- Langerhans Cell Histiocytosis
- Being tested
- Clofarabine
- Medical subject headings
- Histiocytosis, Langerhans-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClofarabineDrug
| Study group | Role | What they receive |
|---|---|---|
| Low-Risk | Experimental | Multifocal or Low-Risk Multi System Clofarabine, Low Dose, Two Cycles Receives: Drug: Clofarabine |
| High-Risk | Experimental | High-risk Multi System Clofarabine, Standard Dose, Two Cycles Receives: Drug: Clofarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Secondary outcomes (3)
Estimated of Progression Free Survival
To estimate the progression-free survival after clofarabine treatment
Measured: 2 years
Estimated survival with risk organ involvement
To estimate survival of participants with refractory multi-system LCH with risk organ involvement treated with clofarabine
Measured: 2 years
Chemotherapy feasibility
To determine if clofarabine therapy can be delivered as planned ('chemotherapy feasibility'), and to describe toxicities of clofarabine in participants with LCH.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2013 (estimated)
- Primary completionOct 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Langerhans Cell HistiocytosisStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Carlos Rodriguez-Galindo, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01796405
- Sponsor's protocol ID
- 12-050
- Status last verified
- Aug 2015
- First submitted
- 19 Feb 2013
- Last updated
- 25 Aug 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis
- ClinicalTrials.gov ID
- NCT01796405
- Acronym / sponsor ID
- 12-050
- Conditions
- Langerhans Cell Histiocytosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Primary completion
- Oct 2017 (estimated)
- First posted
- 21 Feb 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Low-Risk | Experimental | Drug: ClofarabineMultifocal or Low-Risk Multi System Clofarabine, Low Dose, Two Cycles |
| High-Risk | Experimental | Drug: ClofarabineHigh-risk Multi System Clofarabine, Standard Dose, Two Cycles |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Clofarabine | Drug | High-Risk, Low-Risk |
Primary outcome
Estimated Response Rate
Time frame: 2 years
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.
3 secondary outcomes
Estimated of Progression Free Survival
Time frame: 2 years
Estimated survival with risk organ involvement
Time frame: 2 years
Chemotherapy feasibility
Time frame: 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Clofarabine in people with Langerhans Cell Histiocytosis, run by Dana-Farber Cancer Institute.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 25 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Estimated Response Rate (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2015.