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Anti-metabolites

Clofarabine for Langerhans Cell Histiocytosis

Phase 2

Waitlist Available

Led By Barbara Degar, MD

Research Sponsored by Dana-Farber Cancer Institute

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Performance Score > 70% (use Lansky score for age < 16 and Karnofsky score for age = >16)

Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent

Must not have

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

Use of alternative medications (e.g., herbal or botanical that could interfere with clofarabine) is not permitted during the entire study period

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if a drug called clofarabine can treat Langerhans Cell Histiocytosis (LCH) and other histiocytic disorders.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Clofarabine may cause side effects like nausea, vomiting, diarrhea, fever, rash, fatigue, liver problems (increased bilirubin), blood disorders such as low counts of white cells or platelets which might increase infection risk.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am mostly active and can care for myself.

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I understand the study's risks and benefits and can consent.

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My kidney function is normal or meets the required levels for my age.

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My kidney function is normal or only slightly reduced.

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I have been diagnosed with Langerhans Cell Histiocytosis or a related disorder.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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

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I am not pregnant or breastfeeding.

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I have not had a stem cell transplant, serious infections, hepatitis B or C, or cirrhosis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Response Rate (OR) of LCH Cohort

Histiocytosis, Langerhans-Cell

Secondary study objectives

1-year Overall Survival (OS)

1-year Progression Free Survival (PFS)

Number of Participants With at Least One Grade 3 or Higher Treatment-Related Toxicity

Side effects data

From 2020 Phase 3 trial • 256 Patients • NCT01471444

95%

Nausea

95%

Mucositis

82%

Elevated transminitis

79%

Infections

55%

Skin GvHD

50%

ATG induced fevers

45%

Fluid overload

32%

Diarrhea

32%

Neutropenic fevers

29%

Hypertension

27%

Upper GI GvHD

22%

Renal insufficiency

19%

Liver GvHD

18%

Chronic ocular GvHD

15%

ATG induced skin rash

15%

BK virus associated hemorrhagic cystitis

15%

Chronic oral GvHD

14%

GI GvHD

12%

Pneumonitis

11%

Rash

Headaches

Palmar-plantar erythrodysesthesia

Chronic lung GvGHD

ABO incompatibility

Dysrhythmia

Viral Infections

Cardiomyopathy

Poor graft function

Allergic reaction to ATG

Fungal Infections

Ascites

Hemorrhagic cystits

Diffused alveolar hemorrhage

Thrombocytopenia

Skin GVHD

Bacterial Infections

PRES

Hemochromatosis

Transcient secondary graft failure

100%

80%

60%

40%

20%

Study treatment Arm

Arm A (Flu+Bu)

Arm B (Flu+Clo+Bu)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Clofarabine

2007

Completed Phase 3

~1130

0 / 9Eligibility criteria met

Find a Location

Who is running the clinical trial?

SanofiIndustry Sponsor

2,203 Previous Clinical Trials

4,036,670 Total Patients Enrolled

Dana-Farber Cancer InstituteLead Sponsor

1,108 Previous Clinical Trials

357,212 Total Patients Enrolled

St. Baldrick's FoundationOTHER

18 Previous Clinical Trials

9,113 Total Patients Enrolled

Media Library

Clofarabine (Anti-metabolites) Clinical Trial Eligibility Overview. Trial Name: NCT02425904 — Phase 2

$Langerhans Cell Histiocytosis Research Study Groups: LCH-related disorders + Clofarabine, Recurrent or Refractory Langerhans Cell Histiocytosis (LCH) + Clofarabine$