Clofarabine for Langerhans Cell Histiocytosis

Recruiting in Palo Alto (17 mi)

+13 other locations

Overseen byBarbara Degar, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Dana-Farber Cancer Institute

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.

Eligibility Criteria

This trial is for people of all ages with active Langerhans Cell Histiocytosis (LCH) or related disorders, who have tried at least one treatment before. They must not be pregnant, breastfeeding, or have had certain other cancers within the last 5 years. Participants need to have a performance score above 70%, normal kidney and liver function tests, and can't be on drugs that could harm their kidneys during the study.

Inclusion Criteria

Provide signed written informed consent

For stratum 1, you have tried one type of chemotherapy in the past and it didn't work. For stratum 2, if you have RDD, you have tried corticosteroid treatment and it didn't work. If you have ECD with a specific gene mutation, you have tried a certain medication and it didn't work, or you are not eligible for that treatment.

Your liver enzyme levels are not too high, unless it's due to a specific condition called LCH.

+16 more

Exclusion Criteria

I am not taking kidney-harming drugs during my clofarabine treatment.

I do not have any severe illnesses or social situations that would stop me from following the study's requirements.

I am not using any herbal or alternative medicines that could affect the study drug.

+6 more

Participant Groups

The drug being tested is clofarabine for treating LCH and similar conditions. The study will look at how well it works in patients who've already tried other treatments without success. It's given under controlled conditions to monitor its effectiveness and safety.

2Treatment groups

Experimental Treatment

Group I: Recurrent or Refractory Langerhans Cell Histiocytosis (LCH) + ClofarabineExperimental Treatment1 Intervention

Participants with recurrent or refractory LCH defined as with multi-focal or multi-system disease who have recurred (or have refractory disease) after at least one prior systemic chemotherapy regimen. Patient will receive Clofarabine administered via IV on days 1-5, 25 mg/m2/day per cycle for 2 cycles. At the end of cycle 2 if no disease progression, will continue with same dose for maintenance treatment for 4 additional cycles.

Group II: LCH-related disorders + ClofarabineExperimental Treatment1 Intervention

Participants with LCH-related disorders defined as who require systemic chemotherapy including participants with Rosai Dorfman Disease (RDD) who have not responded to or recurred after treatment with corticosteroids. Erdheim Chester Disease (ECD) subjects who have confirmed presence of BRAF V600E mutation must have not responded to, have recurred after, or be unable to receive treatment with a BRAF inhibitor. Patient will receive Clofarabine administered via IV on days 1-5, 25 mg/m2/day per cycle for 2 cycles. At the end of cycle 2 if no disease progression, will continue with same dose for maintenance treatment for 4 additional cycles.

Clofarabine is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Clolar for: