Langerhans Cell HistiocytosisPhase 2NCT02425904
Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders
A Phase 2 study testing Clofarabine in people with Langerhans Cell Histiocytosis, run by Dana-Farber Cancer Institute at 15 sites in 2 countries.
Trial summary
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Langerhans Cell Histiocytosis
- What is being tested?
- Clofarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 15 sitesin 2 countries
- Study dates
- May 2015to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Prior diagnosis of Langerhans Cell Histiocytosis (stratum 1) or LCH-related disorder (stratum 2) established by standard diagnostic criteria and confirmed histologically.
- Evidence of active disease (histological confirmation of reactivation or progression 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.
- In stratum 1, patients must have failed one prior systemic chemotherapy regimen. In stratum 2, RDD patients must have failed treatment with corticosteroid. ECD patients who have confirmed BRAF V600E mutation must have failed treatment with a BRAF inhibitor or are not considered to be eligible for such treatment.
Show all 19Show fewer
- There is no limitation of amount or the type of prior therapy or drugs.
- 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 cells/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:
- Pediatric Population (patients < 18 years): Creatinine within normal limits or calculated creatinine clearance greater than or equal to 90 ml/min/1.73 m2 as calculated by the Schwartz formula for estimated glomerular filtration rate (GFR) where GFR (ml/min/1.73 m2) = k x Height (cm)/serum creatinine (mg/dl). k is a proportionality constant which varies with age and is a function of urinary creatinine excretion per unit of body size; 0.45 up to 12 months of age; 0.55 children and adolescent girls; and 0.70 adolescent boys.
- Adult Population (patients >= 18 years): Serum creatinine less than or equal to 1.0 mg/dL; if serum creatinine >1.0 mg/dL, then the estimated glomerular filtration rate (GFR) must be >60 mL/min/1.73 m2 as calculated by the Modification of Diet in Renal Disease equation where Predicted GFR (ml/min/1.73 m2) = 186 x (Serum Creatinine)-1.154 x (age in years)-0.203 x (0.742 if patient is female) x (1.212 if patient is black), where serum creatinine is measured in mg/dL.
- Alkaline phosphatase ≤ 2.5 x institutional upper limit of normal
Who cannot join
- 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. Corticosteroid treatment is allowed.
- Participants may not be receiving any other investigational agents targeting Histiocytosis.
- Clofarabine is excreted primarily by the kidneys. Therefore, drugs with known renal toxicity (e.g.vancomycin, amphotericin B, acyclovir, cyclosporin, methotrexate, tacrolimus) should be avoided to the extent possible during the 5 days of clofarabine treatment in each cycle or, if required, administered cautiously and with close monitoring.
- 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 is a nucleoside analog with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk of adverse events in nursing infants secondary to treatment of the mother with clofarabine, breastfeeding should be discontinued if the mother is treated with clofarabine. These potential risks may also apply to other agents used in this study.
Show all 9Show fewer
- 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 are ineligible because of the potential for pharmacokinetic interactions with clofarabine. In addition, these individuals are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in participants receiving combination antiretroviral therapy when indicated.
Trial locations and contacts
15 sites in 2 countries.
15 sites in 2 countries
United States14 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Children's Hospital
Houston, Texas, 77030
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital of Los Angeles
Los Angeles, California, 90027
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Mount Sinai Medical Center
New York, New York, 10029
The Children's Hospital of Pennsylvania
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
University of California San Francisco Medical Center
San Francisco, California, 94143
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
Read the detailed description
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.
- Conditions
- Langerhans Cell Histiocytosis
- Being tested
- Clofarabine
- Keywords
- Langerhans Cell Histiocytosis
- Medical subject headings
- Histiocytosis, Langerhans-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClofarabineDrug
Also called Clofarex, Clolar
second-generation purine nucleoside analog
| Study group | Role | What they receive |
|---|---|---|
| Recurrent or Refractory Langerhans Cell Histiocytosis (LCH) + Clofarabine | Experimental | 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. Receives: Drug: Clofarabine |
| LCH-related disorders + Clofarabine | Experimental | 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. Receives: Drug: Clofarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: 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).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2015 (estimated)
- Study startMay 2015
- Primary completionJan 2020
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Langerhans Cell HistiocytosisStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi, St. Baldrick's Foundation, Cookies for Kids' Cancer, North American Consortium for Histiocytosis
- Responsible party
- Principal investigator, Barbara Degar, MD, Dana-Farber Cancer Institute
- Investigators
- Barbara Degar, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02425904
- Sponsor's protocol ID
- 15-005
- Status last verified
- Jun 2026
- First submitted
- 18 Apr 2015
- Last updated
- 23 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Degar BA, Abla O, Campbell PK, Friedmann AM, Henry MM, Hermiston ML, Hogarty M, Jacobsen ED, Jubran R, Kao PC, Koch VB, Kumar AR, Ladisch S, London WB, McClain KL, Stine KC, Wistinghausen B, Rodriguez-Galindo C. Clofarabine for relapsed/refractory LCH and non-LC histiocytosis. Blood Adv. 2026 Jul 28;10(14):5087-5096. doi: 10.1182/bloodadvances.2026019922.
PubMed 42126265
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders
- ClinicalTrials.gov ID
- NCT02425904
- Acronym / sponsor ID
- 15-005
- Conditions
- Langerhans Cell Histiocytosis
- Keywords
- Langerhans Cell Histiocytosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Sanofi, St. Baldrick's Foundation, Cookies for Kids' Cancer, North American Consortium for Histiocytosis
- Study start
- May 2015 (actual)
- Primary completion
- Jan 2020 (actual)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 24 Apr 2015 (estimated)
- Results
- Posted 16 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Recurrent or Refractory Langerhans Cell Histiocytosis (LCH) + Clofarabine | Experimental | Drug: ClofarabineParticipants 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. |
| LCH-related disorders + Clofarabine | Experimental | Drug: ClofarabineParticipants 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (19 listed)
- Prior diagnosis of Langerhans Cell Histiocytosis (stratum 1) or LCH-related disorder (stratum 2) established by standard diagnostic criteria and confirmed histologically.
- Evidence of active disease (histological confirmation of reactivation or progression 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.
Key exclusion criteria (9 listed)
- 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. Corticosteroid treatment is allowed.
- Participants may not be receiving any other investigational agents targeting Histiocytosis.
- Clofarabine is excreted primarily by the kidneys. Therefore, drugs with known renal toxicity (e.g.vancomycin, amphotericin B, acyclovir, cyclosporin, methotrexate, tacrolimus) should be avoided to the extent possible during the 5 days of clofarabine treatment in each cycle or, if required, administered cautiously and with close monitoring.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ClofarabineAlso: Clofarex, Clolarsecond-generation purine nucleoside analog | Drug | LCH-related disorders + Clofarabine, Recurrent or Refractory Langerhans Cell Histiocytosis (LCH) + Clofarabine |
Primary outcome
Response Rate (OR) of LCH Cohort
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.
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.
Response Rate of LCH-related Disorders Cohort
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.
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.
3 secondary outcomes
1-year Progression Free Survival (PFS)
Time frame: At 1 year
1-year Overall Survival (OS)
Time frame: At 1 year
Number of Participants With at Least One Grade 3 or Higher Treatment-Related Toxicity
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).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42126265Degar BA, Abla O, Campbell PK, Friedmann AM, Henry MM, Hermiston ML, Hogarty M, Jacobsen ED, Jubran R, Kao PC, Koch VB, Kumar AR, Ladisch S, London WB, McClain KL, Stine KC, Wistinghausen B, Rodriguez-Galindo C. Clofarabine for relapsed/refractory LCH and non-LC histiocytosis. Blood Adv. 2026 Jul 28;10(14):5087-5096. doi: 10.1182/bloodadvances.2026019922.
Related trials for Langerhans Cell Histiocytosis
RecruitingObservational study
Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study
Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis
RecruitingObservational study
Registry for Patients With Erdheim-Chester Disease and Other Histiocytoses
Erdheim-Chester Disease
Not yet recruitingObservational study
Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
RecruitingPhase 2
A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis
RecruitingObservational study
Hemophagocytic Lymphohistiocytosis After Allogeneic Stem Cell Transplantation
Hemophagocytic Lymphohistiocytosis (HLH)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Barbara Degar, MD principal investigator
Dana-Farber Cancer Institute
15 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Clofarabine in people with Langerhans Cell Histiocytosis, run by Dana-Farber Cancer Institute at 15 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response Rate (OR) of LCH Cohort (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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02425904, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2026.