This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
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 and other histiocytic disorders.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
25
second-generation purine nucleoside analog
Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Response Rate (OR) of LCH Cohort
The OR was defined as the proportion of participants achieving certain response on treatment among LCH patients, criteria were defined per protocol. Better response were defined as achieving complete disease resolution (NAD) or disease regression (AD better); intermediate response defined as stable or unchanged disease status; worse response defined as disease progression.
Time frame: Disease was evaluated at baseline and the end of cycle 2. Treatment continued after cycle 2 until disease progression or unacceptable toxicity. LCH treatment duration has a median of 5.8 months with range 2.1-7 months.
Response Rate of LCH-related Disorders Cohort
The OR was defined as the proportion of participants achieving certain response on treatment among LCH-related disorder patients, criteria were defined per protocol. A clinical response of progressive metabolic disease (PMD) defined as CT-based target lesion abnormal and bone marrow new or recurrent involvement; partial metabolic response (PMR) defined as CT-based target lesion decreasing or disease regressed.
Time frame: Disease was evaluated at baseline and the end of cycle 2. Treatment continued after cycle 2 until disease progression or unacceptable toxicity. Treatment duration has a median of 5.5 months with range 1.7-5.6 months.
1-year Progression Free Survival (PFS)
1-year PFS defined as the proportion of patients that survival progression free at 1 year. PFS based on the duration of time from study entry to documented disease progression (PD) or death. Per protocol criteria: where time to event for PFS is the time from study enrollment until the time of first occurrence of new lesions, progressive disease, or death from any cause, or until last contact if no event occurs.
Time frame: At 1 year
1-year Overall Survival (OS)
1-year OS defined as the proportion of patients that survival at 1 year. OS calculated as the time from enrollment until death or censored at date last known alive.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California San Francisco Medical Center
San Francisco, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Mount Sinai Medical Center
New York, New York, United States
...and 5 more locations
Time frame: At 1 year
Number of Participants With at Least One Grade 3 or Higher Treatment-Related Toxicity
All grade 3 or higher adverse events (AE) with treatment attribution of possibly, probably or definite based on CTCAEv4.0 as reported on case report forms were counted. Number of Participants with at least one Grade 3 or Higher Treatment-Related Toxicity defined as number of patients experiencing at least one treatment-related grade 3 or higher AE of any type during the time of observation.
Time frame: Disease was evaluated at baseline and the end of cycle 2. Treatment continued after cycle 2 until disease progression or unacceptable toxicity. LCH treatment duration is median 5.8 with range 2.1-7 months; LCH-related treatment duration is 5.5 (1.7-5.6).