Relapse/Refractory B-cell Acute Lymphoblastic LeukemiaPhase 1/2NCT06635330
Safety and Efficacy of CAR T Cell Therapy in Patients with R/r B-ALL
A Phase 1/2 study testing anti-CD19 CAR T cell therapy in people with Relapse/Refractory B-cell Acute Lymphoblastic Leukemia, run by Kara Yakhteh Tajhiz Azma Company at 1 site in Iran.
Trial summary
The goal of this clinical trial is to evaluate the safety and efficacy of CD19 CAR-T cells in pediatric patients of all genders, aged 2 to 18 years, with relapsing or refractory B cell acute lymphoblastic leukemia (r/r B-ALL). The main questions it aims to answer are as following: 1. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Relapse/Refractory B-cell Acute Lymphoblastic Leukemia
- What is being tested?
- anti-CD19 CAR T cell therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteIran
- Study dates
- 20 May 2024to 22 Sep 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Ages 2 to 18 years with relapsed or refractory CD19+ B-ALL
- Presence of disease in the bone marrow
- Able to tolerate the apheresis process
- Life expectancy \> 12 weeks
- Lansky or Karnofsky score \> 50%
- At least 7 days passed since the last chemotherapy and the last treatment with corticosteroids
- Informed consent
- Having potential donor for stem cell transplantation
Who cannot join
- Presence of active malignancy other than the disease under study
- Chloroma and leukemic infiltration on MRI or significant neurological symptoms
- Any CNS disorder
- Presence of active GVHD
- Radiation therapy within last 14 days
- History of Anti-CD19 or Anti-CD20 therapy
Show all 9Show fewer
- Donor lymphocyte injection or other cell therapy methods within the last 30 days
- Presence of severe active infection
- Organ dysfunction
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Iran1 site, 1 recruiting
- Recruiting
Pediatric cell and gene therapy research center, Children medical center
Tehran, Tehran Province, 1419733151
Amir Ali Hamidieh, +989121593270, aahamidieh@tums.ac.ir
About this study
Official title: A Phase I/II Single Arm Study, Safety and Efficacy Assessment of the CD19 CAR T Cell on Pediatric Patients with Relapsing or Refractory B Cell Acute Lymphoblastic Leukemia (r/r B-ALL)
The goal of this clinical trial is to evaluate the safety and efficacy of CD19 CAR-T cells in pediatric patients of all genders, aged 2 to 18 years, with relapsing or refractory B cell acute lymphoblastic leukemia (r/r B-ALL). The main questions it aims to answer are as following:
- What is the percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)?
- What is the rate of Event-free survival at first month and 2-3 months after intervention?
- What is the rate of Overall survival at first month and at 3 months after the intervention?
Read the detailed description
B-cell acute lymphoblastic leukemia (B-ALL), as the most common type of pediatric tumor, is identified by unregulated cell proliferation of immature lymphoid cells that can infiltrate the bone marrow and blood. Also, relapse and refractory B-ALL (R/R B-ALL) is the main reason of global mortality due to the constraints of combination chemotherapy.
Over the past few years, substantial advancements have been made in treatment of ALL, specifically in the R/R context. Chimeric antigen receptor T (CAR-T) cells are a type of cancer immunotherapy treatment that function through modification of patient T cells to express CAR antigen on their surface. CAR-T cells aimed at CD19 have demonstrated promising activity in treatment of r/r B-ALL. In this study we aim to evaluate safety and efficacy of Anti-CD19 CAR T cell therapy in children with R/R B-ALL.
- Being tested
- anti-CD19 CAR T cell therapy
- Medical subject headings
- Recurrence, Burkitt Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-CD19 CAR T cell therapyBiological
Anti-CD19 CAR-T cell therapy for R/R B-ALL pediatric patients. For patients 50 kg and less: 0.2 to 5 in ten to the power of six live CAR+ T cells per kilogram of body weight/ For patients over 50 kg: 0.1 to 2.5 in ten to the power of eight live CAR+ T cells (without considering weight).
| Study group | Role | What they receive |
|---|---|---|
| Anti-CD19 CAR-T treatment group | Experimental | Not described Receives: Biological: anti-CD19 CAR T cell therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
Measured: First month and 2-3 months after intervention
Overall survival
Measured: First month and 3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4
Measured: First month and 3 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
Measured: First month and 3 months after intervention
Event-free survival
Measured: First month and 2-3 months after intervention
Secondary outcomes (9)
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
Measured: 6 months and 12 months after intervention
Investigation of Minimal residual disease in patient
Measured: First month and 2-3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4
Measured: 6 months and 12 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
Measured: 6 months and 12 months after intervention
Incidence of tumor lysis syndrome (TLS)
Measured: Months 1, 3, 6, and 12 after the intervention
Incidence of leukopenia
Measured: Months 1, 3, 6, and 12 after the intervention
Incidence of infection
Measured: Months 1, 3, 6, and 12 after the intervention
Event-free survival
Measured: 6 months and 12 months after intervention
Overall survival
Measured: 6 months and 12 months after intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2024
- Study start20 May 2024
- Primary completion22 Sep 2026 (estimated)
- Study completion22 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapse/Refractory B-cell Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kara Yakhteh Tajhiz Azma Company
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06635330
- Sponsor's protocol ID
- IR.NREC.1403.003
- Status last verified
- Oct 2024
- First submitted
- 1 Oct 2024
- Last updated
- 10 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Single Arm Study, Safety and Efficacy Assessment of the CD19 CAR T Cell on Pediatric Patients with Relapsing or Refractory B Cell Acute Lymphoblastic Leukemia (r/r B-ALL)
- ClinicalTrials.gov ID
- NCT06635330
- Acronym / sponsor ID
- IR.NREC.1403.003
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Kara Yakhteh Tajhiz Azma CompanyResearch network
- Study start
- 20 May 2024 (actual)
- Primary completion
- 22 Sep 2026 (estimated)
- Study completion
- 22 Sep 2027 (estimated)
- First posted
- 10 Oct 2024 (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.
The goal of this clinical trial is to evaluate the safety and efficacy of CD19 CAR-T cells in pediatric patients of all genders, aged 2 to 18 years, with relapsing or refractory B cell acute lymphoblastic leukemia (r/r B-ALL). The main questions it aims to answer are as following:
- What is the percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)?
- What is the rate of Event-free survival at first month and 2-3 months after intervention?
- What is the rate of Overall survival at first month and at 3 months after the intervention?
Detailed description
B-cell acute lymphoblastic leukemia (B-ALL), as the most common type of pediatric tumor, is identified by unregulated cell proliferation of immature lymphoid cells that can infiltrate the bone marrow and blood. Also, relapse and refractory B-ALL (R/R B-ALL) is the main reason of global mortality due to the constraints of combination chemotherapy.
Over the past few years, substantial advancements have been made in treatment of ALL, specifically in the R/R context. Chimeric antigen receptor T (CAR-T) cells are a type of cancer immunotherapy treatment that function through modification of patient T cells to express CAR antigen on their surface. CAR-T cells aimed at CD19 have demonstrated promising activity in treatment of r/r B-ALL. In this study we aim to evaluate safety and efficacy of Anti-CD19 CAR T cell therapy in children with R/R B-ALL.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Anti-CD19 CAR-T treatment group | Experimental | Biological: anti-CD19 CAR T cell therapy |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Ages 2 to 18 years with relapsed or refractory CD19+ B-ALL
- Presence of disease in the bone marrow
- Able to tolerate the apheresis process
- Life expectancy \> 12 weeks
- Lansky or Karnofsky score \> 50%
Key exclusion criteria (9 listed)
- Presence of active malignancy other than the disease under study
- Chloroma and leukemic infiltration on MRI or significant neurological symptoms
- Any CNS disorder
- Presence of active GVHD
- Radiation therapy within last 14 days
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 |
|---|---|---|
| anti-CD19 CAR T cell therapyAnti-CD19 CAR-T cell therapy for R/R B-ALL pediatric patients. For patients 50 kg and less: 0.2 to 5 in ten to the power of six live CAR+ T cells per kilogram of body weight/ For patients over 50 kg: 0.1 to 2.5 in ten to the power of eight live CAR+ T cells (without considering weight). | Biological | Anti-CD19 CAR-T treatment group |
Primary outcome
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
Time frame: First month and 2-3 months after intervention
Overall survival
Time frame: First month and 3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4
Time frame: First month and 3 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
Time frame: First month and 3 months after intervention
Event-free survival
Time frame: First month and 2-3 months after intervention
9 secondary outcomes
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
Time frame: 6 months and 12 months after intervention
Investigation of Minimal residual disease in patient
Time frame: First month and 2-3 months after intervention
Incidence of cytokine release syndrome: grade 3 and 4
Time frame: 6 months and 12 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
Time frame: 6 months and 12 months after intervention
Incidence of tumor lysis syndrome (TLS)
Time frame: Months 1, 3, 6, and 12 after the intervention
Incidence of leukopenia
Time frame: Months 1, 3, 6, and 12 after the intervention
Incidence of infection
Time frame: Months 1, 3, 6, and 12 after the intervention
Event-free survival
Time frame: 6 months and 12 months after intervention
Overall survival
Time frame: 6 months and 12 months after intervention
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2024.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing anti-CD19 CAR T cell therapy in people with Relapse/Refractory B-cell Acute Lymphoblastic Leukemia, run by Kara Yakhteh Tajhiz Azma Company at 1 site in Iran.
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 8 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: looking for participants now, as of the sponsor's last update on 10 Oct 2024. 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 Iran. 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 combined 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.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Setayesh Sadeghi (+989124779968, sadeghi.setayesh@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi) (time frame: First month and 2-3 months after intervention). The study lists 5 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 10 Oct 2024.