Acute Myeloid LeukaemiaPhase 1NCT04106076
Phase I Study of UCART123 in Patient With Adverse Genetic Risk Acute Myeloid Leukemia
A Phase 1 study testing UCART123 in people with Acute Myeloid Leukaemia, run by Cellectis S.A..
Trial summary
This is a Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute Myeloid Leukaemia
- What is being tested?
- UCART123
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 11 Jul 2019to 5 Dec 2019
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Main Inclusion Criteria:
- Patients newly diagnosed with CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined as per ELN guidelines (Döhner et al., 2017)
- Eastern Cooperative Oncology Group performance status of 0 or 1
- No prior gene or experimental cellular therapy
- No organ dysfunction that in the opinion of the investigator precludes intensive induction chemotherapy or cellular therapy
About this study
Official title: Phase I, Open Label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of Multiple Infusions of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor) in Patients With Adverse Genetic Risk Acute Myeloid Leukaemia
This is a Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). The purpose of this study is to evaluate the safety and clinical activity of multiple infusions of UCART123 and to determine the Maximum Tolerated Dose (MTD).
- Conditions
- Acute Myeloid Leukaemia
- Being tested
- UCART123
- Keywords
- Acute Myeloid Leukaemia, Newly diagnosed AML, ELN adverse genetic risk group, Chimeric Antigen Receptor T-Cell (CAR-T) therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN)
- 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.
UCART123Biological
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor
| Study group | Role | What they receive |
|---|---|---|
| Dose escalation | Experimental | Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified. Receives: Biological: UCART123 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AE/SAE/DLT [Safety and Tolerability]
Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study
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 posted26 Sep 2019
- Study start11 Jul 2019
- Primary completion5 Dec 2019
- Study completion5 Dec 2019
Why it stopped: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukaemiaStudies 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
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellectis S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ghulam Mufti, Pr, Principal investigator, Kings college London NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04106076
- Sponsor's protocol ID
- UCART123_03
- Status last verified
- Jul 2020
- First submitted
- 25 Sep 2019
- Last updated
- 14 Jul 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, Open Label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of Multiple Infusions of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor) in Patients With Adverse Genetic Risk Acute Myeloid Leukaemia
- ClinicalTrials.gov ID
- NCT04106076
- Acronym / sponsor ID
- UCART123_03
- Conditions
- Acute Myeloid Leukaemia
- Keywords
- Acute Myeloid Leukaemia, Newly diagnosed AML, ELN adverse genetic risk group, Chimeric Antigen Receptor T-Cell (CAR-T) therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Cellectis S.A.Industry (pharma, biotech, devices)
- Study start
- 11 Jul 2019 (actual)
- Primary completion
- 5 Dec 2019 (actual)
- Study completion
- 5 Dec 2019 (actual)
- First posted
- 26 Sep 2019 (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 Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). The purpose of this study is to evaluate the safety and clinical activity of multiple infusions of UCART123 and to determine the Maximum Tolerated Dose (MTD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose escalation | Experimental | Biological: UCART123Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| UCART123Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor | Biological | Dose escalation |
Primary outcome
Incidence of AE/SAE/DLT [Safety and Tolerability]
Time frame: 24 months
Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study
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 14 Jul 2020.
- Individual participant dataSharing planned: Undecided
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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.
Ghulam Mufti, Pr principal investigator
Kings college London NHS Foundation Trust
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing UCART123 in people with Acute Myeloid Leukaemia, run by Cellectis S.A..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 14 Jul 2020. 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 of AE/SAE/DLT [Safety and Tolerability] (time frame: 24 months). 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 14 Jul 2020.