Acute Myeloid LeukemiaPhase 1Pediatric AMLNCT05622591
ELU001 in Pediatric Subjects Who Have Relapsed and/or Refractory CBFA2T3-GLIS2-positive AML
A Phase 1 study testing ELU001 in people with Acute Myeloid Leukemia and AML, Childhood, run by Elucida Oncology.
Trial summary
This research study was planned to focus on a rare type of acute myeloid leukemia (with the subtype CBFA2T3::GLIS2 that overexpresses folate receptor alpha (FRα) (a protein on the surface of leukemia cells)) that has relapsed or is refractory. Relapse means the cancer has come back after treatment. Read more about this study
- Who is it for?
- Ages 1 month to 9 years
- Conditions the study is for
- Acute Myeloid Leukemia, AML, Childhood, Relapsed Pediatric AML
- What is being tested?
- ELU001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 7 Jun 2024to 7 Jun 2024
- 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 month to 9 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients must meet the following criteria to enroll in this study:
- Infants (>1 month) and children (≤9 years) at time of enrollment.
- Relapsed or refractory CBFA2T3::GLIS2 positive AML
- CNS1 or CNS2 during screening
- Performance Status: Lansky ≥ 50
- Adequate Organ Function including liver, kidney, and heart
Who cannot join
- Patients who meet any of the following are not eligible to enroll in this study:
- CNS3 Disease
- AML associated with congenital syndromes such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome or Diamond-Blackfan anemia, or bone marrow failure associated with inherited syndromes.
- Acute promyelocytic leukemia.
- Clinically significant active or chronic corneal disorder, particularly corneal epitheliopathy or any eye disorder that may predispose patient to this condition, or unable to comply with an age-appropriate ophthalmologic examination.
- Prior treatment with folate receptor-targeting anti-cancer agent(s) ≤ 21 days (or 2 half-lives must have elapsed before enrollment, whichever is longer), or received investigational anti-cancer treatment ≤ 4 weeks, or within a time interval less than at least 5 half-lives of the investigational agent, prior to starting study drug, whichever is shorter.
About this study
Official title: Dose Escalation Study to Evaluate the Safety and Tolerability of ELU001 in Pediatric Patients Who Have Relapsed and/or Refractory CBFA2T3::GLIS2 Positive Acute Myeloid Leukemia
This research study was planned to focus on a rare type of acute myeloid leukemia (with the subtype CBFA2T3::GLIS2 that overexpresses folate receptor alpha (FRα) (a protein on the surface of leukemia cells)) that has relapsed or is refractory. Relapse means the cancer has come back after treatment. Refractory means the cancer does not respond to treatment.
Read the detailed description
This study was planned as a Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D). This study will also evaluate the tolerability of ELU001.
ELU001 is not a drug approved by the FDA (Food and Drug Administration) yet.
- Being tested
- ELU001
- Keywords
- CBFA2T3-GLIS2, CBFA2T3::GLIS2, Refractory, Folate Receptor alpha, Acute Myeloid Leukemia, ELU001, Exatecan, C'Dot drug conjugate, C-Dot, C'Dot, Childhood, Nanoparticle, Folate Receptor, AML, Relapsed
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ELU001Drug
Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC)
| Study group | Role | What they receive |
|---|---|---|
| ELU001 | Experimental | Dose Escalation: Escalating doses of ELU001 Receives: Drug: ELU001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001
Establish the Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001 in pediatric patients with relapsed or refractory CBFA2T3::GLIS2 positive AML.
Measured: 28 days
Secondary outcomes (3)
Evaluate preliminary anti-leukemic activity of ELU001
Proportion of evaluable patients having achieved at least one of the following
- Complete Remission per IWG (CRIWG)
- Complete Remission With Partial Recovery of Platelet Count (CRp)
- Complete Remission with Incomplete Blood Count Recovery (CRi)
- Complete Remission for Minimal Residual Disease (CRm)
Duration of Complete Remission from CRIWG/CR/CRp/CRi to hematological relapse or death from any cause, whichever comes first
Measured: First dose of study drug until 42 days after last cycle.
Characterize the pharmacokinetics of ELU001
Measure the concentration of ELU001 in the blood.
This includes - Maximum Observed Concentration (Cmax), Time After Dosing at which Maximum Observed Concentration of Drug is Observed (tmax), Area Under the Curve to the End of the Dosing Period (AUC0-tau), and Area Under the Curve to the Last Measurable Concentration (AUC0-t), will be estimated. Other PK parameters, e.g., Terminal Elimination or Disposition Half-Life (T½), Volume of Distribution (Vd), Clearance Rate (CL), and C'Dot, payload on C'Dot
Measured: First dose of study drug until 42 days after last cycle.
Characterize the immunogenicity of ELU001
Percent incidence of Anti-Drug Antibodies (ADA) formation in the blood assessed from baseline until End-of-Treatment (EOT).
Measured: First dose of study drug until 42 days after last cycle.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2022
- Study start7 Jun 2024
- Primary completion7 Jun 2024
- Study completion7 Jun 2024
Why it stopped: Lack of Funding - no safety issue with Drug; Company Permanently Closed
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Dose Escalation
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Elucida Oncology
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05622591
- Sponsor's protocol ID
- ELU-FRα-AML-1
- Acronym
- Pediatric AML
- Status last verified
- Aug 2024
- First submitted
- 8 Nov 2022
- Last updated
- 7 Aug 2024
- 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 7 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose Escalation Study to Evaluate the Safety and Tolerability of ELU001 in Pediatric Patients Who Have Relapsed and/or Refractory CBFA2T3::GLIS2 Positive Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT05622591
- Acronym / sponsor ID
- Pediatric AML / ELU-FRα-AML-1
- Keywords
- CBFA2T3-GLIS2, CBFA2T3::GLIS2, Refractory, Folate Receptor alpha, Acute Myeloid Leukemia, ELU001, Exatecan, C'Dot drug conjugate
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of Funding - no safety issue with Drug; Company Permanently Closed
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Elucida OncologyIndustry (pharma, biotech, devices)
- Study start
- 7 Jun 2024 (actual)
- Primary completion
- 7 Jun 2024 (actual)
- Study completion
- 7 Jun 2024 (actual)
- First posted
- 18 Nov 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 research study was planned to focus on a rare type of acute myeloid leukemia (with the subtype CBFA2T3::GLIS2 that overexpresses folate receptor alpha (FRα) (a protein on the surface of leukemia cells)) that has relapsed or is refractory. Relapse means the cancer has come back after treatment. Refractory means the cancer does not respond to treatment.
Detailed description
This study was planned as a Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D). This study will also evaluate the tolerability of ELU001.
ELU001 is not a drug approved by the FDA (Food and Drug Administration) yet.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Dose Escalation
| Arm | Type | Receives |
|---|---|---|
| ELU001 | Experimental | Drug: ELU001Dose Escalation: Escalating doses of ELU001 |
Who may qualify
- Ages
- 1 month to 9 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Infants (>1 month) and children (≤9 years) at time of enrollment.
- Relapsed or refractory CBFA2T3::GLIS2 positive AML
- CNS1 or CNS2 during screening
- Performance Status: Lansky ≥ 50
- Adequate Organ Function including liver, kidney, and heart
Key exclusion criteria (6 listed)
- CNS3 Disease
- AML associated with congenital syndromes such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome or Diamond-Blackfan anemia, or bone marrow failure associated with inherited syndromes.
- Acute promyelocytic leukemia.
- Clinically significant active or chronic corneal disorder, particularly corneal epitheliopathy or any eye disorder that may predispose patient to this condition, or unable to comply with an age-appropriate ophthalmologic examination.
- Prior treatment with folate receptor-targeting anti-cancer agent(s) ≤ 21 days (or 2 half-lives must have elapsed before enrollment, whichever is longer), or received investigational anti-cancer treatment ≤ 4 weeks, or within a time interval less than at least 5 half-lives of the investigational agent, prior to starting study drug, whichever is shorter.
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 |
|---|---|---|
| ELU001Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC) | Drug | ELU001 |
Primary outcome
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001
Time frame: 28 days
Establish the Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001 in pediatric patients with relapsed or refractory CBFA2T3::GLIS2 positive AML.
3 secondary outcomes
Evaluate preliminary anti-leukemic activity of ELU001
Time frame: First dose of study drug until 42 days after last cycle.
Characterize the pharmacokinetics of ELU001
Time frame: First dose of study drug until 42 days after last cycle.
Characterize the immunogenicity of ELU001
Time frame: First dose of study drug until 42 days after last cycle.
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 7 Aug 2024.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ELU001 in people with Acute Myeloid Leukemia and AML, Childhood, run by Elucida Oncology.
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 6 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 7 Aug 2024. 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?
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001 (time frame: 28 days). 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 7 Aug 2024.