Acute Lymphoblastic LeukemiaPhase 1NCT05168748
CD19- and CD22-directed CAR-T Cell Therapy in Patients With Acute Lymphoblastic Leukemia
A Phase 1 study testing IMJ995 single agent in people with Acute Lymphoblastic Leukemia, run by Novartis Pharmaceuticals.
Trial summary
This is a first-in-human study to evaluate the feasibility, safety and preliminary antitumor efficacy of autologous chimeric antigen receptor (CAR) T cells targeting both CD19 and CD22, manufactured with T-Charge(TM) process. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Acute Lymphoblastic Leukemia
- What is being tested?
- IMJ995 single agent
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 24 Jan 2023to 13 Aug 2026
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All patients:
- Evidence of CD19 and/or CD22 cell surface expression on B-ALL blasts in bone marrow or peripheral blood by flow cytometry at time of relapse or prior to study entry.
- Pediatric, adolescent and young adult ALL patients:
- 1 - 29 years of age at the time of informed consent form (ICF) signature.
- Relapsed or refractory CD19+ and/or CD22+ ALL after 3 or more lines of treatment OR after allogeneic HCT.
- Must have received a CD19-directed CAR-T treatment (with or without blinatumomab), unless prior loss of CD19 cell surface expression occurred or have not been eligible for CD19 directed CAR-T treatment.
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- Lansky (age < 16 years), Karnofsky (age 16-25 years) performance status ≥ 60%. ECOG (age >25 years) performance status that is either 0 or 1 at screening.
- Adult ALL patients aged ≥30 years:
- ≥30 years of age at the time of informed consent form (ICF) signature.
- Refractory or relapsed CD19+ and/or CD22+ ALL including at least one of the following:
- After allogeneic HCT
- After 2 or more lines of treatment, including blinatumomab and/or inotuzumab
- Primary refractory disease (defined as failure to achieve a CR at the end of at least 1 induction chemotherapy)
- First relapse occurring within 12 months from first remission
- ECOG performance status that is either 0 or 1 at screening.
Who cannot join
- Allogeneic HCT within 12 weeks prior to screening.
- Presence of isolated extra-medullary disease, testicular involvement or bulky disease
- Patients with concomitant genetic syndromes associated with bone marrow failure states: such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome.
- Patients with Burkitt's lymphoma/leukemia
- History of active neurological auto immune or inflammatory disorders
- Other protocol-defined inclusion/exclusion criteria may apply.
About this study
Official title: Phase I, Open Label, Multicenter, Dose Escalation and Expansion Study of IMJ995 in Acute Lymphoblastic Leukemia
This is a first-in-human study to evaluate the feasibility, safety and preliminary antitumor efficacy of autologous chimeric antigen receptor (CAR) T cells targeting both CD19 and CD22, manufactured with T-Charge(TM) process. CAR-T cells will be investigated as single agent in pediatric and adult acute lymphoblastic leukemia (ALL).
Read the detailed description
This is a phase I, open label, multicenter, dose escalation and expansion study of IMJ995. The study will investigate single agent IMJ995 in two independent groups of acute lymphoblastic leukemia (ALL) patients:
- Pediatric, adolescent and young adult (AYA) ALL patients up to 29 years old
- Adult ALL patients (≥30 years old) safety cohort The pediatric and AYA ALL group consists of two parts: a dose escalation part to evaluate feasibility, characterize safety and identify the recommended dose (RD) of IMJ995, and a dose expansion part to further characterize safety, cellular kinetics and assess preliminary antitumor activity. Once the RD of IMJ995 is determined for this group, the corresponding expansion part may commence.
Once the RD of IMJ995 is determined for the pediatric and AYA group, a safety cohort for adult ALL patients ≥30 years old may commence in parallel to the above mentioned expansion part.
- Conditions
- Acute Lymphoblastic Leukemia
- Being tested
- IMJ995 single agent
- Keywords
- CAR-T, IMJ995, CD19, CD22, acute lymphoblastic leukemia (ALL), pediatric, adolescent
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IMJ995 single agentDrug
Single intravenous administration of IMJ995
| Study group | Role | What they receive |
|---|---|---|
| IMJ995 in ALL | Experimental | Dose escalation and expansion of IMJ995 single agent in ALL Receives: Drug: IMJ995 single agent |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and nature of Dose Limiting Toxicities (Dose Escalation part only, in pediatric, adolescent and young adult ALL patients)
Dose recommendation for IMJ995 in pediatric, adolescent and young adult ALL patients
Measured: 28 days
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) (pediatric, adolescent and young adult ALL patients)
Safety and tolerability
Measured: 24 months
Number of patients infused with planned target dose
Manufacture success rate
Measured: 24 months
Secondary outcomes (8)
Incidence and nature of DLTs during the first 28 days after IMJ995 infusion (safety cohort for adult ALL).
Dose recommendation in adult ALL
Measured: 28 days
Cellular kinetics of IMJ995 (maximum drug concentration - Cmax)
CAR transgene levels will be measured by flow cytometry and quantitative polymerase chain reaction (qPCR) in peripheral blood. PK parameters will be determined using non-compartmental methods for IMJ995.
Measured: 24 months
Cellular kinetics of IMJ995 (area under the drug concentration-time curve - AUC)
CAR transgene levels will be measured by flow cytometry and quantitative polymerase chain reaction (qPCR) in peripheral blood. PK parameters will be determined using non-compartmental methods for IMJ995.
Measured: 24 months
Number of participants with anti-CAR19 and/or anti-CAR22 antibodies
Humoral immunogenicity
Measured: 24 months
Change from baseline in interferon (IFN)-gamma levels in peripheral blood mononuclear cells (PBMCs)
Cellular immunogenicity
Measured: 24 months
Antitumor activity assessed by Complete Remission / Complete Remission with Incomplete Hematologic Recovery (CR/ CRi).
Antitumor activity
Measured: 24 months
Antitumor activity assessed by duration of response.
Duration of response
Measured: 24 months
Incidence and severity of AEs and SAEs after IMJ995 infusion (safety cohort for adult ALL).
Safety and tolerability
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2021
- Study start24 Jan 2023 (estimated)
- Primary completion13 Aug 2026 (estimated)
- Study completion13 Aug 2026 (estimated)
Why it stopped: Sponsor's decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05168748
- Sponsor's protocol ID
- CIMJ995A12101
- Other IDs
- 2021-000677-89
- Status last verified
- Sep 2022
- First submitted
- 3 Nov 2021
- Last updated
- 21 Dec 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, Open Label, Multicenter, Dose Escalation and Expansion Study of IMJ995 in Acute Lymphoblastic Leukemia
- ClinicalTrials.gov ID
- NCT05168748
- Acronym / sponsor ID
- CIMJ995A12101
- Conditions
- Acute Lymphoblastic Leukemia
- Keywords
- CAR-T, IMJ995, CD19, CD22, acute lymphoblastic leukemia (ALL), pediatric, adolescent
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Sponsor's decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Jan 2023 (estimated)
- Primary completion
- 13 Aug 2026 (estimated)
- Study completion
- 13 Aug 2026 (estimated)
- First posted
- 23 Dec 2021 (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 first-in-human study to evaluate the feasibility, safety and preliminary antitumor efficacy of autologous chimeric antigen receptor (CAR) T cells targeting both CD19 and CD22, manufactured with T-Charge(TM) process. CAR-T cells will be investigated as single agent in pediatric and adult acute lymphoblastic leukemia (ALL).
Detailed description
This is a phase I, open label, multicenter, dose escalation and expansion study of IMJ995. The study will investigate single agent IMJ995 in two independent groups of acute lymphoblastic leukemia (ALL) patients:
- Pediatric, adolescent and young adult (AYA) ALL patients up to 29 years old
- Adult ALL patients (≥30 years old) safety cohort The pediatric and AYA ALL group consists of two parts: a dose escalation part to evaluate feasibility, characterize safety and identify the recommended dose (RD) of IMJ995, and a dose expansion part to further characterize safety, cellular kinetics and assess preliminary antitumor activity. Once the RD of IMJ995 is determined for this group, the corresponding expansion part may commence.
Once the RD of IMJ995 is determined for the pediatric and AYA group, a safety cohort for adult ALL patients ≥30 years old may commence in parallel to the above mentioned expansion part.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IMJ995 in ALL | Experimental | Drug: IMJ995 single agentDose escalation and expansion of IMJ995 single agent in ALL |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Evidence of CD19 and/or CD22 cell surface expression on B-ALL blasts in bone marrow or peripheral blood by flow cytometry at time of relapse or prior to study entry.
- 1 - 29 years of age at the time of informed consent form (ICF) signature.
- Relapsed or refractory CD19+ and/or CD22+ ALL after 3 or more lines of treatment OR after allogeneic HCT.
- Must have received a CD19-directed CAR-T treatment (with or without blinatumomab), unless prior loss of CD19 cell surface expression occurred or have not been eligible for CD19 directed CAR-T treatment.
- Lansky (age < 16 years), Karnofsky (age 16-25 years) performance status ≥ 60%. ECOG (age >25 years) performance status that is either 0 or 1 at screening.
Key exclusion criteria (6 listed)
- Allogeneic HCT within 12 weeks prior to screening.
- Presence of isolated extra-medullary disease, testicular involvement or bulky disease
- Patients with concomitant genetic syndromes associated with bone marrow failure states: such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome.
- Patients with Burkitt's lymphoma/leukemia
- History of active neurological auto immune or inflammatory disorders
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 |
|---|---|---|
| IMJ995 single agentSingle intravenous administration of IMJ995 | Drug | IMJ995 in ALL |
Primary outcome
Incidence and nature of Dose Limiting Toxicities (Dose Escalation part only, in pediatric, adolescent and young adult ALL patients)
Time frame: 28 days
Dose recommendation for IMJ995 in pediatric, adolescent and young adult ALL patients
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) (pediatric, adolescent and young adult ALL patients)
Time frame: 24 months
Safety and tolerability
Number of patients infused with planned target dose
Time frame: 24 months
Manufacture success rate
8 secondary outcomes
Incidence and nature of DLTs during the first 28 days after IMJ995 infusion (safety cohort for adult ALL).
Time frame: 28 days
Cellular kinetics of IMJ995 (maximum drug concentration - Cmax)
Time frame: 24 months
Cellular kinetics of IMJ995 (area under the drug concentration-time curve - AUC)
Time frame: 24 months
Number of participants with anti-CAR19 and/or anti-CAR22 antibodies
Time frame: 24 months
Change from baseline in interferon (IFN)-gamma levels in peripheral blood mononuclear cells (PBMCs)
Time frame: 24 months
Antitumor activity assessed by Complete Remission / Complete Remission with Incomplete Hematologic Recovery (CR/ CRi).
Time frame: 24 months
Antitumor activity assessed by duration of response.
Time frame: 24 months
Incidence and severity of AEs and SAEs after IMJ995 infusion (safety cohort for adult ALL).
Time frame: 24 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing IMJ995 single agent in people with Acute Lymphoblastic Leukemia, run by Novartis Pharmaceuticals.
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 15 inclusion and 6 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: withdrawn, as of the sponsor's last update on 21 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Incidence and nature of Dose Limiting Toxicities (Dose Escalation part only, in pediatric, adolescent and young adult ALL patients) (time frame: 28 days). The study lists 3 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 21 Dec 2022.