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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 belowSelect...
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:Select...
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
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 2022 Phase 3 trial • 727 Patients • NCT0208540898%
White blood cell decreased
96%
Platelet count decreased
94%
Neutrophil count decreased
91%
Anemia
73%
Aspartate aminotransferase increased
71%
Alanine aminotransferase increased
59%
Blood bilirubin increased
55%
Febrile neutropenia
46%
Alkaline phosphatase increased
34%
Hypomagnesemia
26%
Creatinine increased
23%
Lymphocyte count decreased
12%
Lung infection
12%
Hypermagnesemia
8%
Sepsis
8%
Hypokalemia
7%
Hypophosphatemia
6%
Dyspnea
6%
Rash maculo-papular
5%
Fatigue
5%
Hypoxia
5%
Infections and infestations - Other
5%
Hypotension
5%
Hypocalcemia
4%
Acute kidney injury
4%
Catheter related infection
4%
Hyponatremia
4%
Anorexia
4%
Diarrhea
3%
Hypertension
3%
Hyperglycemia
3%
Generalized muscle weakness
3%
Hypoalbuminemia
3%
Respiratory failure
3%
Urinary tract infection
2%
Atrial fibrillation
2%
Enterocolitis infectious
2%
Epistaxis
2%
Nausea
1%
Acidosis
1%
Skin infection
1%
Soft tissue infection
1%
Multi-organ failure
1%
Confusion
1%
Pruritus
1%
Enterocolitis
1%
Non-cardiac chest pain
1%
Cardiac disorders - Other, specify
1%
Rectal hemorrhage
1%
Edema limbs
1%
Intracranial hemorrhage
1%
Investigations - Other, specify
1%
Heart failure
1%
Sinus tachycardia
1%
Supraventricular tachycardia
1%
Colitis
1%
Vomiting
1%
Upper respiratory infection
1%
Weight loss
1%
Bone pain
1%
Pain in extremity
1%
Headache
1%
Hiccups
1%
Pulmonary edema
1%
Adult respiratory distress syndrome
1%
Left ventricular systolic dysfunction
1%
Sinusitis
1%
Blood and lymphatic disorders - Other
1%
Dehydration
1%
Tumor lysis syndrome
1%
Stroke
1%
Syncope
1%
Vasovagal reaction
1%
Abdominal pain
1%
Thromboembolic event
1%
Mucositis oral
1%
Lower gastrointestinal hemorrhage
1%
Typhlitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm B (Induction: Clofarabine)
Arm F (Maintenance: Decitabine)
Arm A (Induction: Daunorubicin + Cytarabine)
Arm C (Consolidation: Cytarabine)
Arm D (Consolidation: Clofarabine)
Arm E (Maintenance: Observation)
Arm G (Transplant)
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
2011
Completed Phase 3
~2530
Find a Location
Who is running the clinical trial?
SanofiIndustry Sponsor
2,216 Previous Clinical Trials
4,047,607 Total Patients Enrolled
Dana-Farber Cancer InstituteLead Sponsor
1,113 Previous Clinical Trials
358,856 Total Patients Enrolled
St. Baldrick's FoundationOTHER
18 Previous Clinical Trials
9,113 Total Patients Enrolled
Cookies for Kids' CancerOTHER
6 Previous Clinical Trials
333 Total Patients Enrolled
North American Consortium for HistiocytosisOTHER
4 Previous Clinical Trials
1,752 Total Patients Enrolled
Barbara Degar, MDPrincipal InvestigatorDana-Farber Cancer Institute
Media Library
Eligibility Criteria:
This trial includes the following eligibility 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.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.I am not using any herbal or alternative medicines that could affect the study drug.Your liver enzyme levels are not too high, unless it's due to a specific condition called LCH.I had cancer before, but it was either skin cancer, cervical cancer in situ, or another type that's been in remission for over 5 years.Your alkaline phosphatase level is not more than 2.5 times the normal limit for the testing lab.I haven't had chemotherapy or radiotherapy in the last 2 weeks, or 6 weeks for specific drugs, and have recovered from any side effects.I am not pregnant or breastfeeding.I have not had a stem cell transplant, serious infections, hepatitis B or C, or cirrhosis.I am not HIV-positive or on antiretroviral therapy.I am mostly active and can care for myself.My age does not limit my participation.My disease is currently active.My organs are functioning well.My kidney function is normal or meets the required levels for my age.I have no restrictions on my past treatments.I am not pregnant and will use birth control during and 6 months after the study.I understand the study's risks and benefits and can consent.My blood cell counts are within a healthy range, unless my condition affects my blood.My kidneys are working well.My kidney function is normal or only slightly reduced.You have enough infection-fighting white blood cells in your body.Your platelet count is at least 75,000 per microliter.Your total bilirubin level should be within a certain range.I have been diagnosed with Langerhans Cell Histiocytosis or a related disorder.
Research Study Groups:
This trial has the following groups:- Group 1: LCH-related disorders + Clofarabine
- Group 2: Recurrent or Refractory Langerhans Cell Histiocytosis (LCH) + Clofarabine
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.