Langerhans Cell HistiocytosisPhase 1NCT05477446
Safety and Efficacy of CD207 Targeted CAR-T Cell Therapy in Patients With R/R Langerhans Cell Histiocytosis
A Phase 1 study testing CD207 CAR-T cells in people with Langerhans Cell Histiocytosis, run by Beijing Friendship Hospital at 1 site in China.
Trial summary
This is a single-arm study to evaluate the efficacy and safety of CD207 targeted CAR-T cell therapy in relapsed and refractory langerhans cell histiocytosis. Read more about this study
- Who is it for?
- Ages 3 to 65 years
- Conditions the study is for
- Langerhans Cell Histiocytosis
- What is being tested?
- CD207 CAR-T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2022to 1 Aug 2025
- Phase
- Phase 1
- 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 3 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Relapsed or refractory langerhans cell histiocytosis, defined as: 1) any relapse during standard chemotherapy; 2) not responding to standard chemotherapy; 3) not achieving CR after first cycle of second-line chemotherapy for relapsed langerhans cell histiocytosis;
- 3-65 years old;
- Expected survival time ≥ 3 months;
- ECOG performance status of 0 or 1 (age ≥ 16 years) or Karnofsky performance status> 80 (age < 16 years) ;
- With single or venous blood collection standards, and no other cell collection contraindications;
- WBC ≥ 2.5×10^9/L ，LY ≥ 0.7×10^9/L，LY% ≥15%;
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- Serum creatinine ≤ 2.0 mg/dl;
- ALT/AST ≤ 2.5 x ULN;
- Total bilirubin ≤ 2.0 mg/dl;
- PT:INR<1.7, or PT is within 4s of the normal value;
- Ability and willingness to adhere to the study visit schedule and all protocol requirements.
Who cannot join
- Transduced CAR+ T lymphocytes<5%, or expansion <5 folds after stimulation with anti CD3/anti CD28 beads;
- Pregnant or breasting-feeding women;
- Active hepatitis B or hepatitis C infection;
- Patients with HIV infection;
- Uncontrolled active infection;
- Use of systemic corticosteroid therapy;
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- Have received gene therapy, or any other CAR-T treatment;
- Allergic to immunotherapy and related drugs;
- History of heart disease requiring treatment, or poorly controlled hypertension;
- Preceding and/or ongoing active ulcer or gastrointestinal bleeding;
- Have received allogeneic hematopoietic stem cell transplantation, or eligible for allogeneic hematopoietic stem cell transplantation;
- Severe central nervous system involvement;
- Severe lung involvement;
- Hyponatremia (serum sodium<125mmol/L);
- Hypokalemia (Serum kalium<3.5mmol/L);
- Those who need long-term anticoagulation treatment (warfarin or heparin);
- Those who need long-term antiplatelet treatment (aspirin, dose>300mg/d; clopidogrel, dose>75mg/d);
- Radiation therapy within 4 weeks prior to registration;
- Uncontrolled, symptomatic, intercurrent illness including but not limited to infection, congestive heart failure, unstable angina, cardiac arrhythmias, mental illness, and other diseases that in the opinion of the investigator would pose an unacceptable risk to the subject;
- Have a history of severe allergy;
- Current enrollment in another study;
- Patients with other contraindications considered unsuitable for participation in this study (according to investigator's judgement).
Trial locations and contacts
1 site in 1 country.
China1 site
Beijing Friendship Hospital, Capital Medical University
Beijing, 100050
Zhao Wang, PhD, 86-63139862, wangzhao@ccmu.edu.cn
About this study
Official title: Safety and Efficacy of CD207 Targeted CAR-T Cell Therapy in Patients With Relapsed and Refractory (R/R) Langerhans Cell Histiocytosis
This is a single-arm study to evaluate the efficacy and safety of CD207 targeted CAR-T cell therapy in relapsed and refractory langerhans cell histiocytosis.
Read the detailed description
There are limited options for treatment of r/r langerhans cell histiocytosis. CD207 is expressed on the membrane surface of langerhans cells，and it is an ideal target for CAR-T. In this study, investigators will evaluate the safety and efficacy of CD207 targeted CAR- T cell therapy in patients with r/r langerhans cell histiocytosis. The primary goal is safety and efficiency assessment, including incidence and severity of adverse events and overall response rate.
- Conditions
- Langerhans Cell Histiocytosis
- Being tested
- CD207 CAR-T cells
- Keywords
- CAR-T cell therapy, Langerhans Cell histiocytosis, CD207
- 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.
CD207 CAR-T cellsBiological
Single dose of CD207 CAR-T cells administered IV
| Study group | Role | What they receive |
|---|---|---|
| CD207 CAR-T cells | Experimental | Cohort 1 will receive 1 x 10^6 CAR+ T cells/kg. Cohort 2 will receive 3 x 10^6 CAR+ T cells/kg. Cohort 3 will receive 5 x 10^6 CAR+ T cells/kg. Cohort 4 will receive 1 x 10^7 CAR+ T cells/kg. Receives: Biological: CD207 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
The number of cases with complete response (CR) and partial response (PR) after treatment as a percentage of the total cases.
Measured: 2 years
Incidence and Severity of Adverse Events (AEs)
Treatment-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0)
Measured: 2 years
Secondary outcomes (4)
Progression Free Survival (PFS) of CD207 CAR-T cell therapy in patients with r/r langerhans cell histiocytosis
PFS will be assessed from the first CAR-T cell infusion to death from any cause or the first assessment of progression.
Measured: 2 years
Overall Survival (OS) of CD207 CAR-T cell therapy in patients with r/r langerhans cell histiocytosis
OS will be assessed from the first CAR-T cell infusion to death from any cause.
Measured: 2 years
Effects of CD207 CAR-T cells on human immune system
Dynamic changes of T cell subset and immune globulin.
Measured: 2 years
Metabolism of CAR T-cells in vivo
Absorption, distribution and metabolism of CD207-CAR T cells in vivo.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2022
- Study start1 Oct 2022 (estimated)
- Primary completion1 Jun 2025 (estimated)
- Study completion1 Aug 2025 (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 Beijing Friendship 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Friendship Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Beijing Boren Hospital
- Responsible party
- Principal investigator, Zhao Wang, Beijing Friendship Hospital
- Investigators
- ZHAO Wang, MD, Principal investigator, Beijing Friendship Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05477446
- Sponsor's protocol ID
- BRYY-IIT-LCYJ-2022-002
- Status last verified
- Jul 2022
- First submitted
- 18 Jul 2022
- Last updated
- 28 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of CD207 Targeted CAR-T Cell Therapy in Patients With Relapsed and Refractory (R/R) Langerhans Cell Histiocytosis
- ClinicalTrials.gov ID
- NCT05477446
- Acronym / sponsor ID
- BRYY-IIT-LCYJ-2022-002
- Conditions
- Langerhans Cell Histiocytosis
- Keywords
- CAR-T cell therapy, Langerhans Cell histiocytosis, CD207
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Beijing Friendship HospitalUniversity, hospital or other
- Collaborators
- Beijing Boren Hospital
- Study start
- 1 Oct 2022 (estimated)
- Primary completion
- 1 Jun 2025 (estimated)
- Study completion
- 1 Aug 2025 (estimated)
- First posted
- 28 Jul 2022 (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.
This is a single-arm study to evaluate the efficacy and safety of CD207 targeted CAR-T cell therapy in relapsed and refractory langerhans cell histiocytosis.
Detailed description
There are limited options for treatment of r/r langerhans cell histiocytosis. CD207 is expressed on the membrane surface of langerhans cells，and it is an ideal target for CAR-T. In this study, investigators will evaluate the safety and efficacy of CD207 targeted CAR- T cell therapy in patients with r/r langerhans cell histiocytosis. The primary goal is safety and efficiency assessment, including incidence and severity of adverse events and overall response rate.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD207 CAR-T cells | Experimental | Biological: CD207 CAR-T cellsCohort 1 will receive 1 x 10^6 CAR+ T cells/kg. Cohort 2 will receive 3 x 10^6 CAR+ T cells/kg. Cohort 3 will receive 5 x 10^6 CAR+ T cells/kg. Cohort 4 will receive 1 x 10^7 CAR+ T cells/kg. |
Who may qualify
- Ages
- 3 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Relapsed or refractory langerhans cell histiocytosis, defined as: 1) any relapse during standard chemotherapy; 2) not responding to standard chemotherapy; 3) not achieving CR after first cycle of second-line chemotherapy for relapsed langerhans cell histiocytosis;
- 3-65 years old;
- Expected survival time ≥ 3 months;
- ECOG performance status of 0 or 1 (age ≥ 16 years) or Karnofsky performance status> 80 (age < 16 years) ;
- With single or venous blood collection standards, and no other cell collection contraindications;
Key exclusion criteria (22 listed)
- Transduced CAR+ T lymphocytes<5%, or expansion <5 folds after stimulation with anti CD3/anti CD28 beads;
- Pregnant or breasting-feeding women;
- Active hepatitis B or hepatitis C infection;
- Patients with HIV infection;
- Uncontrolled active infection;
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 |
|---|---|---|
| CD207 CAR-T cellsSingle dose of CD207 CAR-T cells administered IV | Biological | CD207 CAR-T cells |
Primary outcome
Overall Response Rate (ORR)
Time frame: 2 years
The number of cases with complete response (CR) and partial response (PR) after treatment as a percentage of the total cases.
Incidence and Severity of Adverse Events (AEs)
Time frame: 2 years
Treatment-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0)
4 secondary outcomes
Progression Free Survival (PFS) of CD207 CAR-T cell therapy in patients with r/r langerhans cell histiocytosis
Time frame: 2 years
Overall Survival (OS) of CD207 CAR-T cell therapy in patients with r/r langerhans cell histiocytosis
Time frame: 2 years
Effects of CD207 CAR-T cells on human immune system
Time frame: 2 years
Metabolism of CAR T-cells in vivo
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2022.
- Individual participant dataSharing planned: No
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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.
ZHAO Wang, MD principal investigator
Beijing Friendship Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CD207 CAR-T cells in people with Langerhans Cell Histiocytosis, run by Beijing Friendship 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 11 inclusion and 22 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 28 Jul 2022. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 ZHAO Wang, MD (86-63139862, wangzhao@ccmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: 2 years). 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?
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 28 Jul 2022.