AML, ChildhoodPhase 1NCT05077423
A Phase 1 Trial of CD33xCD3 BsAb in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
A Phase 1 study testing CD33*CD3 BsAb in people with AML, Childhood, run by Y-mAbs Therapeutics at 13 sites in United States.
Trial summary
Pediatric patients (<21 years at study entry) with relapsed or refractory acute myeloid leukemia (AML) will be treated with CD33*CD3 a bispecific antibody to investigate the safety and tolerability of the drug. Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- AML, Childhood
- What is being tested?
- CD33*CD3 BsAb
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 13 sitesUnited States
- Study dates
- 25 May 2022to 1 Dec 2022
- 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 2 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed informed consent from legal guardian(s), patient and/or child obtained in accordance with local regulations. Pediatric patients must provide assent as required by local regulations
- Age ≥2 years, and ≤21 years, and a minimum body weight of ≥11 kg
- Histologically confirmed relapsed or refractory AML (except acute promyelocytic leukemia) with no therapeutic options that may provide clinical benefit. Disease burden ≥5.0% in the bone marrow meets definition for enrollment.
- Karnofsky performance status ≥50 for ≥16 years / Lansky performance status ≥50 for <16 years
- White blood cells (WBC) ≤25 x 109/L (may receive hydroxyurea to bring WBC count down prior to first dose of CD33xCD3 BsAb and during Cycle 1 or low dose cytarabine up to 48 h prior to first dose of CD33xCD3 BsAb)
- Central Nervous System (CNS) disease as per Children's Oncology Group
Show all 11Show fewer
- Patients must have the status of CNS1 and no clinical signs or neurologic symptoms suggestive of CNS leukemia, such as cranial palsy
- Patients with CNS3 or CNS2 status may receive antecedent intrathecal chemotherapy to achieve CNS1 status prior to trial entry
- Patients with a history of CNS chloromatous disease are required to have no radiographic evidence of disease prior to enrollment
- Has acceptable liver and kidney laboratory values
- Patient must have recovered from acute toxic effects of prior anti-cancer therapies prior to first dose of CD33xCD3 BsAb
Who cannot join
- History of uncontrolled seizure. If on anti-convulsant and/or seizures are well controlled as per treating physician enrollment is acceptable
- Acute promyelocytic leukemia with PML-RARA genetic abnormality according to WHO classification or t(15;17)
- Isolated extramedullary AML
- Clinically significant graft-versus-host disease (GvHD) secondary to prior allogeneic transplantation. No immunosuppressive therapy for ≥14 days prior to first dose, except for topical corticosteroids for minor rash (<5% of BSA) or adrenal replacement therapy
- Patient known to have one of the following genetic syndromes: Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Nijmegen breakage syndrome, Kostmann syndrome, Shwachman Diamond syndrome or any known bone marrow failure syndrome where increased risk for toxicity may be expected as judged by the Investigator
- Treatment with another investigational agent under the following conditions:
- Within two weeks (four weeks for biologics) before first administration of CD33xCD3 BsAb; or
- Patient has persistent toxicities from prior anti-leukemic therapies which are determined to be relevant by the Investigator
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Children's of Alabama/University of Alabama at Birmingham
Birmingham, Alabama, 35233
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Riley Hospital for Children - Indiana University
Indianapolis, Indiana, 46202-5225
St Jude Children's Research Hospital
Memphis, Tennessee, 38105
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Children's Hospital of Orange County
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
UCSF Benioff Children's Hospital
San Francisco, California, 94158
Washington University School of Medicine
St Louis, Missouri, 63110
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase 1, Open-label, Dose-escalation Trial of CD33xCD3 Bispecific Antibody in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
Pediatric patients (<21 years at study entry) with relapsed or refractory acute myeloid leukemia (AML) will be treated with CD33*CD3 a bispecific antibody to investigate the safety and tolerability of the drug.
Read the detailed description
This is an open label, first in human dose escalation trial in pediatric patients with relapsed or refractory acute myeloid leukemia to assess the safety and tolerability of increasing doses of CD33xCD3 BsAb administered subcutaneously.
A modified Bayesian Optimal Interval Design (mBOIN) design will be applied. The trial will start with accelerated titration using single patient cohorts until one grade ≥2 AE not clearly associated to underlying disease, thereafter the trial will continue with mBOIN titration.
- Conditions
- AML, Childhood
- Being tested
- CD33*CD3 BsAb
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD33*CD3 BsAbDrug
Also called CD33xCD3
CD33xCD3 is a bispecific monoclonal antibody, which specifically targets CD33 on the AML blasts and CD3 on the T cells
| Study group | Role | What they receive |
|---|---|---|
| Subcutaneous administration of CD33*CD3 BsAb up to 12 cycles | Experimental | Subcutaneous administration of CD33*CD3 BsAb up to 12 cycles Receives: Drug: CD33*CD3 BsAb |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of dose limiting toxicities (DLTs)
Occurrence of DLTs during a DLT period .
Measured: 28 days
Occurrence of Adverse Events
Occurrence of Adverse Events during the trial
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2021
- Study start25 May 2022
- Primary completion1 Dec 2022
- Study completion1 Dec 2022
Why it stopped: Study terminated due to business priorities
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, ChildhoodStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- open-label, single-arm, dose-escalation consisting of up to 12 cycles
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Y-mAbs Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Children's Oncology Group
- Responsible party
- Sponsor
- Investigators
- Jessica Pollard, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05077423
- Sponsor's protocol ID
- 801
- Status last verified
- Nov 2022
- First submitted
- 30 Sep 2021
- Last updated
- 30 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- 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 30 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-label, Dose-escalation Trial of CD33xCD3 Bispecific Antibody in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT05077423
- Acronym / sponsor ID
- 801
- Conditions
- AML, Childhood
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated due to business priorities
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Y-mAbs TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Children's Oncology Group
- Study start
- 25 May 2022 (actual)
- Primary completion
- 1 Dec 2022 (actual)
- Study completion
- 1 Dec 2022 (actual)
- First posted
- 14 Oct 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.
Pediatric patients (<21 years at study entry) with relapsed or refractory acute myeloid leukemia (AML) will be treated with CD33*CD3 a bispecific antibody to investigate the safety and tolerability of the drug.
Detailed description
This is an open label, first in human dose escalation trial in pediatric patients with relapsed or refractory acute myeloid leukemia to assess the safety and tolerability of increasing doses of CD33xCD3 BsAb administered subcutaneously.
A modified Bayesian Optimal Interval Design (mBOIN) design will be applied. The trial will start with accelerated titration using single patient cohorts until one grade ≥2 AE not clearly associated to underlying disease, thereafter the trial will continue with mBOIN titration.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- open-label, single-arm, dose-escalation consisting of up to 12 cycles
| Arm | Type | Receives |
|---|---|---|
| Subcutaneous administration of CD33*CD3 BsAb up to 12 cycles | Experimental | Drug: CD33*CD3 BsAbSubcutaneous administration of CD33*CD3 BsAb up to 12 cycles |
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Signed informed consent from legal guardian(s), patient and/or child obtained in accordance with local regulations. Pediatric patients must provide assent as required by local regulations
- Age ≥2 years, and ≤21 years, and a minimum body weight of ≥11 kg
- Histologically confirmed relapsed or refractory AML (except acute promyelocytic leukemia) with no therapeutic options that may provide clinical benefit. Disease burden ≥5.0% in the bone marrow meets definition for enrollment.
- Karnofsky performance status ≥50 for ≥16 years / Lansky performance status ≥50 for <16 years
- White blood cells (WBC) ≤25 x 109/L (may receive hydroxyurea to bring WBC count down prior to first dose of CD33xCD3 BsAb and during Cycle 1 or low dose cytarabine up to 48 h prior to first dose of CD33xCD3 BsAb)
Key exclusion criteria (8 listed)
- History of uncontrolled seizure. If on anti-convulsant and/or seizures are well controlled as per treating physician enrollment is acceptable
- Acute promyelocytic leukemia with PML-RARA genetic abnormality according to WHO classification or t(15;17)
- Isolated extramedullary AML
- Clinically significant graft-versus-host disease (GvHD) secondary to prior allogeneic transplantation. No immunosuppressive therapy for ≥14 days prior to first dose, except for topical corticosteroids for minor rash (<5% of BSA) or adrenal replacement therapy
- Patient known to have one of the following genetic syndromes: Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Nijmegen breakage syndrome, Kostmann syndrome, Shwachman Diamond syndrome or any known bone marrow failure syndrome where increased risk for toxicity may be expected as judged by the Investigator
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 |
|---|---|---|
| CD33*CD3 BsAbAlso: CD33xCD3CD33xCD3 is a bispecific monoclonal antibody, which specifically targets CD33 on the AML blasts and CD3 on the T cells | Drug | Subcutaneous administration of CD33*CD3 BsAb up to 12 cycles |
Primary outcome
Occurrence of dose limiting toxicities (DLTs)
Time frame: 28 days
Occurrence of DLTs during a DLT period .
Occurrence of Adverse Events
Time frame: 52 weeks
Occurrence of Adverse Events during the trial
Results and safety
The study was stopped early (reason given: Study terminated due to business priorities). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2023.
- 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.
Jessica Pollard, MD principal investigator
Dana-Farber Cancer Institute
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CD33*CD3 BsAb in people with AML, Childhood, run by Y-mAbs Therapeutics at 13 sites in United States.
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 8 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: stopped early, as of the sponsor's last update on 30 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. 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?
The study was stopped early (reason given: Study terminated due to business priorities). Results are not available in the source record.
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?
Occurrence of dose limiting toxicities (DLTs) (time frame: 28 days). 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 30 May 2023.