Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)Phase 1ABC123NCT03203369
Study to Evaluate the Safety and Clinical Activity of UCART123 in Patients With BPDCN
A Phase 1 study testing UCART123 in people with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), run by Cellectis S.A. at 1 site in United States.
Trial summary
A Phase 1 dose-finding study of Universal Chimeric Antigen Receptor T-cells targeting cluster of differentiation (CD) 123 (UCART123) administered intravenously to patients with relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), followed by a dose expansion phase in relapsed or refractory… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
- What is being tested?
- UCART123
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Jun 2017to 27 Jun 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Main Inclusion Criteria:
- Patients with a diagnosis BPDCN according to World Health Organization (WHO) classification confirmed by hematopathology;
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1;
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase 1, Open-label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of a Single Dose of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
A Phase 1 dose-finding study of Universal Chimeric Antigen Receptor T-cells targeting cluster of differentiation (CD) 123 (UCART123) administered intravenously to patients with relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), followed by a dose expansion phase in relapsed or refractory BPDCN patients or newly diagnosed BPDCN patients.
- Being tested
- UCART123
- Keywords
- Blastic plasmacytoid dendritic cell neoplasm, Natural Killer (NK) cell blastic lymphoma, Hematologic malignancies, Cluster of differentiation (CD) CD4+CD56+ hematodermic tumor, Acute leukemia and myeloid neoplasms, Chimeric Antigen Receptor T-cell (CAR-T) therapy, Allogeneic, Cluster of differentiation 4 (CD4), Cluster of differentiation 56 (CD56)
- Medical subject headings
- Blastic Plasmacytoid Dendritic Cell Neoplasm, Hematologic Neoplasms
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 given as a single dose following a lymphodepleting regimen.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Dose Escalation | Experimental | A single intravenous administration of UCART123. Dose escalation in Part 1 will include 3 doses ranging from 6.25 x 10^5 cells/kg to 6.25 x 10^6 cells/kg and continue until the Recommended Phase 2 Dose (RP2D) is identified. Receives: Biological: UCART123 |
| Part 2: Dose Expansion | Experimental | A single intravenous administration of UCART123 at the RP2D. 2 Cohorts: Patients with Relapsed/Refractory BPDCN and Newly Diagnosed BPDCN. Receives: Biological: UCART123 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, nature, and severity of adverse events and serious adverse events
Measured: Through day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2017
- Study start28 Jun 2017
- Primary completion27 Jun 2019
- Study completion27 Jun 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 Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)Studies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellectis S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ioana Kloos, MD, Study director, Cellectis S.A.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03203369
- Sponsor's protocol ID
- UCART123_02
- Acronym
- ABC123
- Status last verified
- Jul 2019
- First submitted
- 14 Jun 2017
- Last updated
- 31 Jul 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1, Open-label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of a Single Dose of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
- ClinicalTrials.gov ID
- NCT03203369
- Acronym / sponsor ID
- ABC123 / UCART123_02
- Keywords
- Blastic plasmacytoid dendritic cell neoplasm, Natural Killer (NK) cell blastic lymphoma, Hematologic malignancies, Cluster of differentiation (CD) CD4+CD56+ hematodermic tumor, Acute leukemia and myeloid neoplasms, Chimeric Antigen Receptor T-cell (CAR-T) therapy, Allogeneic, Cluster of differentiation 4 (CD4)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Cellectis S.A.Industry (pharma, biotech, devices)
- Study start
- 28 Jun 2017 (actual)
- Primary completion
- 27 Jun 2019 (actual)
- Study completion
- 27 Jun 2019 (actual)
- First posted
- 29 Jun 2017 (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.
A Phase 1 dose-finding study of Universal Chimeric Antigen Receptor T-cells targeting cluster of differentiation (CD) 123 (UCART123) administered intravenously to patients with relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), followed by a dose expansion phase in relapsed or refractory BPDCN patients or newly diagnosed BPDCN patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part 1: Dose Escalation | Experimental | Biological: UCART123A single intravenous administration of UCART123. Dose escalation in Part 1 will include 3 doses ranging from 6.25 x 10^5 cells/kg to 6.25 x 10^6 cells/kg and continue until the Recommended Phase 2 Dose (RP2D) is identified. |
| Part 2: Dose Expansion | Experimental | Biological: UCART123A single intravenous administration of UCART123 at the RP2D. 2 Cohorts: Patients with Relapsed/Refractory BPDCN and Newly Diagnosed BPDCN. |
Who may qualify
- Ages
- 18 to 80 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 given as a single dose following a lymphodepleting regimen. | Biological | Part 1: Dose Escalation, Part 2: Dose Expansion |
Primary outcome
Incidence, nature, and severity of adverse events and serious adverse events
Time frame: Through day 84
Results and safety
The study was stopped early (reason given: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2019.
- Individual participant dataSharing planned: Undecided
Related trials for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
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A Non-interventional Study of Participants With BPDCN Treated With Chemotherapy
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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.
Ioana Kloos, MD study director
Cellectis S.A.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing UCART123 in people with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), run by Cellectis S.A. at 1 site 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?
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 31 Jul 2019. 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 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: The trial was discontinued due to sponsor's decision and not a consequence of any safety concern.). 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?
Incidence, nature, and severity of adverse events and serious adverse events (time frame: Through day 84). 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 31 Jul 2019.