Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis
a study on Langerhans Cell Histiocytosis
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis( LCH )
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational intervention, to learn whether the drug works in treating a specific disease, in this case, clofarabine to treat LCH.
"Investigational" means that the intervention is still being studied. It also means that the FDA (the U.S. Food and Drug Administration) has not yet approved clofarabine for your disease.
Clofarabine is a chemotherapy drug that has been used and is approved by the FDA for the treatment of leukemia in children and adults. Information from other research studies suggests that this drug may also be effective in participants with LCH.
Langerhans Cell Histiocytosis Histiocytosis Histiocytosis, Langerhans-Cell Clofarabine A-Recurrent Low-Risk Multi-System LCH B-Refractory High-Risk Multi-System LCH
You can join if…
- Prior diagnosis of Langerhans Cell Histiocytosis established by standard diagnostic criteria and confirmed histologically
- Evidence of disease reactivation or progression after standard treatment against LCH(histological confirmation is not required)
- Performance Score > 70% (use Lansky score for age < 16 and Karnofsky score for age =>16).
- Patients of all ages will be eligible.
- Provide signed written informed consent.
- Patients have failed first line treatment with prednisone and vinblastine. There is no limitation of amount or the type of prior therapy or drugs.
- Patients with 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.
- Female patients of childbearing potential must have a negative serum pregnancy test within 14 days prior to enrollment. Male and female patients must use an effective contraceptive method during the study and for a minimum of 6 months after study treatment.
- Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.
- Participants must have adequate marrow functions as defined below, except those with involvement of hematopoietic system for whom these criteria can be waived:
- Absolute neutrophil count ≥ 750/µL
- Platelets ≥75,000/µL
- Participants must have adequate organ functions as defined below:
- Total bilirubin ≤ 2.5x institutional upper limit of normal
- AST (SGOT)/ALT (SGPT) < 2.5 X institutional upper limit of normal unless it is related to involvement by LCH
- Adequate renal function defined as:
- A serum creatinine ≤ institutional upper limit of normal or
- Creatinine clearance or radioisotope GFR ≥60 mL/min/1.73 m2
- Alkaline phosphatase ≤ 2.5 x institutional upper limit of normal
You CAN'T join if...
- Participants who have had chemotherapy or radiotherapy within 2 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 2 weeks earlier.
- Participants may not be receiving any other investigational agents targeting LCH.
- Use of alternative medications (e.g., herbal or botanical that could interfere with clofarabine) is not permitted during the entire study period.
- Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study because clofarabine has potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk of adverse events in nursing infants, breastfeeding should be discontinued if the mother is treated with clofarabine.
- Individuals with a history of a different malignancy are ineligible except for the following circumstances. Individuals with a history of other malignancies are eligible if they have been disease-free for at least 5 years and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with the following cancers are eligible if diagnosed and treated within the past 5 years: cervical cancer in situ, and basal cell or squamous cell carcinoma of the skin.
- Patients with a history of prior hematopoietic stem cell transplantation(HSCT),elevated conjugated serum bilirubin at study entry, uncontrolled systemic fungal,bacterial, or other infection, a history of hepatitis B or C infection or a history of cirrhosis.
- Individuals who are known to be HIV-positive on combination antiretroviral therapy.
- University of California San Francisco Medical Center accepting new patients
San Francisco California 94143 United States
- Texas Children's Hospital accepting new patients
Houston Texas 77030 United States
Please contact me about this study
We will not share your information with anyone other than the team in charge of this study. Submitting your contact information does not obligate you to participate in research.
The study team should get back to you in a few business days.
You will also receive an email with next steps. Check your junk/spam folder if needed.
If you do not hear from the study team, please call 888-689-8273 and tell them you’re interested in study number NCT02425904.