Langerhans Cell HistiocytosisPhase 2NCT04121819
AraC for Newly Diagnosed Adult Langerhans Cell Histiocytosis
A Phase 2 study testing Cytarabine in people with Langerhans Cell Histiocytosis, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
Langerhans cell histiocytosis (LCH) is a rare, heterogeneous histiocytic disorder occurring in patients of all ages from neonates to the elderly. The current standard treatment protocol for children with de novo multisystem LCH is vinblastine plus prednisone. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Langerhans Cell Histiocytosis
- What is being tested?
- Cytarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2019to 31 Dec 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- • Histologically confirmed diagnosis of LCH.
- Patients were newly diagnosed or did not receive prior systemic treatment of LCH (patients who had received radiotherapy alone were allowed).
- Age ≥18 years and ≤75 years.
- LCH involved multisystem or multifocal single system.
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Patients must have adequate renal, liver, and bone marrow function as defined by the following criteria:
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- Absolute neutrophil count ≥1500 cells per mm3 or ≥500 cells per mm3 in the case of known hematopoietic system involvement by LCH.
- Platelet count ≥100000 cells per mm3 or ≥20000 cells per mm3 in the case of known hematopoietic system involvement by LCH.
- Creatinine clearance [according to Cockcroft formula] ≥60 mL/min.
- Aspartate aminotransferase and alanine aminotransferase ≤2·5×upper limit of normal [ULN], and total bilirubin ≤2·5×ULN; or ≤10×ULN in the case of known liver involvement by LCH.
- No active or untreated infection.
- No cardiac abnormalities.
- Subject provide written informed consent.
- A female is eligible to enter and participate in this study if she is of:
- Non-childbearing potential including ω Any female who has had a surgical procedure rendering her incapable of becoming pregnant.
- ω Subjects have experienced total cessation of menses for more than 1 year and be greater than 45 years in age.
- ⎫ Childbearing potential, including any female who has had a negative serum pregnancy test within 2 weeks prior to the first dose of study treatment, and agrees to use adequate contraception.
- Male subjects must use an effective barrier method of contraception during the study and for 90 days following the last course of MA if sexually active with a childbearing potential
Who cannot join
- • Non-langerhans cell histiocytosis.
- Patients had concurrent malignancies.
- Patients who had received any treatment except radiotherapy for LCH.
- History of myocardial infarction, or unstable angina, or New York Heart Association (NYHA) Grade III-IV within 6 months prior to Day 1.
- Women who were pregnant or of childbearing potential.
- Known HIV seropositive, active hepatitis C infection, and/or hepatitis B (defined as HCV RNA
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- ≥103 copies or HBV DNA ≥103 copies at screening).
- Major surgical procedure within 28 days prior to the first dose of study treatment.
- Presence of uncontrolled infection.
- Evidence of active bleeding or bleeding diathesis.
- Any serious and/or unstable pre-existing medical, psychiatric, or other condition that could interfere with subject's safety, provision of informed consent, or compliance to study procedures.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing
Xinxin Cao
About this study
Official title: Cytarabine Monotherapy for Adult Patients With Newly Diagnosed Langerhans Cell Histiocytosis: A Single Arm, Single Center, Prospective Phase 2 Study
Langerhans cell histiocytosis (LCH) is a rare, heterogeneous histiocytic disorder occurring in patients of all ages from neonates to the elderly. The current standard treatment protocol for children with de novo multisystem LCH is vinblastine plus prednisone. This regimen has never been proven effective for adults in a prospective study, since the only prospective trial evaluating the efficacy of a vinblastine/prednisone regimen in adults was prematurely closed due to unacceptable toxicities. A retrospective study showed an advantage for cytarabine monotherapy compared with vinblastine/prednisone in bone LCH patients. This phase 2, prospective, single-center study is designed to evaluate the efficacy and safety of cytarabine monotherapy in adults with newly diagnosed MS-LCH or LCH with multifocal single system (SS-m) involvement.
- Conditions
- Langerhans Cell Histiocytosis
- Being tested
- Cytarabine
- Keywords
- Langerhans cell histiocytosis, adult, newly diagnosed
- 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.
CytarabineDrug
cytarabine 100mg/m2 d1-5 subcutaneous
| Study group | Role | What they receive |
|---|---|---|
| AraC | Experimental | cytarabine 100mg/m2 d1-5 subcutaneous Receives: Drug: Cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival (EFS)
Events were defined as a poor response to AraC, reactivation after AraC therapy and death from any cause.
Measured: the duration from initiation of AraC treatment to the date of a first event or date of death from any cause, whichever come first, assessed up to 5 years
Secondary outcomes (2)
Overall response rate
the cumulative number of patients with either non-active disease or regressive disease (signs and symptoms were improved with no new lesions) after AraC therapy
Measured: on 12 months
Overall survival
Overall survival
Measured: the duration from initiation of AraC treatment to the date of death or last follow-up, assessed up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2019
- Study start1 Oct 2019
- Primary completion31 Dec 2021 (estimated)
- Study completion31 Dec 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Langerhans Cell HistiocytosisStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Model description
- AraC
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Cao Xinxin, Peking Union Medical College Hospital
- Investigators
- Jian Li, MD, Study director, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04121819
- Sponsor's protocol ID
- PUMCHNDLCH2
- Status last verified
- Jun 2021
- First submitted
- 7 Oct 2019
- Last updated
- 8 Jun 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cytarabine Monotherapy for Adult Patients With Newly Diagnosed Langerhans Cell Histiocytosis: A Single Arm, Single Center, Prospective Phase 2 Study
- ClinicalTrials.gov ID
- NCT04121819
- Acronym / sponsor ID
- PUMCHNDLCH2
- Conditions
- Langerhans Cell Histiocytosis
- Keywords
- Langerhans cell histiocytosis, adult, newly diagnosed
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Oct 2019 (actual)
- Primary completion
- 31 Dec 2021 (estimated)
- Study completion
- 31 Dec 2021 (estimated)
- First posted
- 10 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Langerhans cell histiocytosis (LCH) is a rare, heterogeneous histiocytic disorder occurring in patients of all ages from neonates to the elderly. The current standard treatment protocol for children with de novo multisystem LCH is vinblastine plus prednisone. This regimen has never been proven effective for adults in a prospective study, since the only prospective trial evaluating the efficacy of a vinblastine/prednisone regimen in adults was prematurely closed due to unacceptable toxicities. A retrospective study showed an advantage for cytarabine monotherapy compared with vinblastine/prednisone in bone LCH patients. This phase 2, prospective, single-center study is designed to evaluate the efficacy and safety of cytarabine monotherapy in adults with newly diagnosed MS-LCH or LCH with multifocal single system (SS-m) involvement.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- AraC
| Arm | Type | Receives |
|---|---|---|
| AraC | Experimental | Drug: Cytarabinecytarabine 100mg/m2 d1-5 subcutaneous |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- • Histologically confirmed diagnosis of LCH.
- ω Subjects have experienced total cessation of menses for more than 1 year and be greater than 45 years in age.
- ⎫ Childbearing potential, including any female who has had a negative serum pregnancy test within 2 weeks prior to the first dose of study treatment, and agrees to use adequate contraception.
- Male subjects must use an effective barrier method of contraception during the study and for 90 days following the last course of MA if sexually active with a childbearing potential
Key exclusion criteria (11 listed)
- • Non-langerhans cell histiocytosis.
- ≥103 copies or HBV DNA ≥103 copies at screening).
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 |
|---|---|---|
| Cytarabinecytarabine 100mg/m2 d1-5 subcutaneous | Drug | AraC |
Primary outcome
Event-free survival (EFS)
Time frame: the duration from initiation of AraC treatment to the date of a first event or date of death from any cause, whichever come first, assessed up to 5 years
Events were defined as a poor response to AraC, reactivation after AraC therapy and death from any cause.
2 secondary outcomes
Overall response rate
Time frame: on 12 months
Overall survival
Time frame: the duration from initiation of AraC treatment to the date of death or last follow-up, assessed up to 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2021.
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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.
Jian Li, MD study director
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cytarabine in people with Langerhans Cell Histiocytosis, run by Peking Union Medical College Hospital at 1 site in China.
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 18 inclusion and 11 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: status not confirmed, as of the sponsor's last update on 8 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xinxin Cao, MD (69155027, caoxinxin@pumch.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free survival (EFS) (time frame: the duration from initiation of AraC treatment to the date of a first event or date of death from any cause, whichever come first, assessed up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Jun 2021.