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Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)Phase 1/2NCT04109482

Trial to Evaluate the Safety and Efficacy of MB-102 in Patients With BPDCN.

A Phase 1/2 study testing MB-102, Fludarabine and Cyclophosphamide in people with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), run by Mustang Bio at 4 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

A phase 1/2 study to assess the safety and efficacy of MB-102 in patients with relapsed or refractory BPDCN Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
MB-102, Fludarabine and Cyclophosphamide
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
4 sitesUnited States
Study dates
17 Feb 2020to 17 May 2023
Phase
Phase 1/2
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Blastic Plasmacytoid Dendritic Cell Neoplasm
  • Patients with a diagnosis of BPDCN according to WHO classification (Arber et al., 2016) confirmed by hematopathology and histological/cytological evidence of BPDCN in the peripheral blood, bone marrow, spleen, lymph nodes, skin and/or other sites who have failed one prior therapy.
  • General Inclusion Criteria
  • Male and female patients ≥ 18 years of age at the time of consent.
  • Written informed consent in accordance with federal, local, and institutional guidelines.
  • Must be able to adhere to the study visit schedule and other protocol requirements.
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  • Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
  • Meet the following laboratory criteria:
  • Absolute lymphocyte count (ALC) > 100/mm3
  • ALT/SGPT and AST/SGOT < 2.5x the upper limit of normal (ULN) unless due to underlying disease state
  • Calculated creatinine clearance ≥ 45.0 mL/min as estimated by Cockcroft Gault and dialysis independent
  • Total bilirubin ≤ 3.0 mg/dL
  • Patients with Gilbert's Syndrome must have a total bilirubin < 5.0 mg/dL.
  • Serum albumin ≥ 3.2 g/dL
  • Cardiac ejection fraction ≥ 45%, with no evidence of pericardial effusion as determined by an echocardiogram (ECHO) or if not available, a multigated acquisition scan (MUGA).
  • Females participants of childbearing potential must have a negative serum test.
  • Patients must agree to use a highly effective method of contraception if procreative potential exists from the start of the study until one year after the completion of lymphodepletion for females and 4 months after completion of lymphodepletion for males.
  • Patients with a previously treated malignancy if treatment of that malignancy was completed greater than 2 years before screening and the patient has no evidence of disease at the time of screening.
  • Patients who have previously undergone allogenic or autologous bone marrow transplants are allowed.
  • Centrally confirmed CD-123 positivity on the bone marrow, or for patients without bone marrow involvement local pathology assessments within 28 days from Screening, showing evidence of CD-123 positivity of skin/lymph node biopsy.

Who cannot join

  • Patients with a corticosteroid dependence on doses greater than physiological replacement i.e., prednisone no more than 7.5 mg/day or hydrocortisone less than 12mg/m2/day.
  • Contraindication or hypersensitivity to fludarabine or cyclophosphamide.
  • Hypersensitivity or known history of allergic reactions attributed to tocilizumab, Cetuximab, or other anti-EGFR -monoclonal antibodies.
  • Immunotherapy treatments within 28 days prior to leukapheresis.
  • Previous treatment with anti-CD123 CAR-T treatment.
  • Previous treatment with non-CAR-T anti-CD123 agents is allowed e.g. tagraxofusp-erzs.
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  • Previous treatment with any other antileukemic or investigational agent within 7 days of leukapheresis.
  • Hydroxyurea is allowed up to 3 days prior to leukapheresis.
  • Patients with history or active seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement.
  • Patients with known CNS leukemic involvement that are refractory to intrathecal chemotherapy and/or cranio-spinal radiation that have NOT been effectively treated to complete remission (defined as < 5 WBC/mm3 and no blasts in CSF).
  • Patients with active Graft versus Host Disease (GVHD).
  • Acute active infection
  • Patients being administered prophylactic antibiotics, antivirals, or antifungals are permitted.
  • Patients who have any form of primary immunodeficiency, such as severe combined immunodeficiency disease, human immunodeficiency virus (HIV), or acquired immune deficiency syndrome (AIDS).
  • Active infection with hepatitis B or C.
  • Patients requiring supplemental oxygen or mechanical ventilation or oxygen saturation < 92% on room air.
  • Patients with an oxygen saturation < 92%, a pulmonary function test with a result of Diffusing capacity of the lungs for carbon monoxide (DLCO) of ≥ 40% of predicted and a forced expiratory volume in one second (FEV1) > 45% predicted will be accepted.
  • Patients with decompensated hepatic cirrhosis/liver failure.
  • Pregnant or lactating females.
  • Any other clinically significant medical disease or condition that, in the investigator's opinion, may interfere with protocol adherence or a patient's ability to give informed consent.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • City of Hope Medical Center

    Duarte, California, 91010

  • Duke University

    Durham, North Carolina, 27710

  • MD Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: A Phase 1/2, Open Label, Multicenter Trial to Assess the Safety and Efficacy of MB-102 in Patients With Relapsed or Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm

A phase 1/2 study to assess the safety and efficacy of MB-102 in patients with relapsed or refractory BPDCN

Read the detailed description

The Phase 1 portion of the study will determine the maximum tolerated dose of MB-102.

The Phase 2 portion of the trial will evaluate the efficacy of MB-102 in relapsed or refractory BPDCN.

Being tested
MB-102, Fludarabine, Cyclophosphamide
Keywords
CD123 CAR-T, BPDCN, Blastic plasmacytoid dendritic cell neoplasm
Medical subject headings
Blastic Plasmacytoid Dendritic Cell Neoplasm

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MB-102Biological

    Also called CD123 CAR-T

    The study drug, MB-102 consists of adoptively transferred T cells that are genetically modified using a self-inactivating (SIN) lentiviral vector to express a CD123-specific, CD28-costimulatory chimeric antigen receptor (CAR) as well as a truncated human epidermal growth factor receptor (EGFRt) (CD123.CD28.CD3ζ.EGFRt+T cells) derived from autologous leukapheresis which is administered after a lymphodepletion regimen.

    Single dose of MB-102 up to 600 x 10 6 CART-T+ cells (Day 0) as defined by Phase 1 will be administered.

  • FludarabineDrug

    Also called Fludara

    Fludarabine 30 mg/m2/day IV (3 days) on days -5, -4, and -3

    • A 20% dose reduction (24 mg/m2/day IV (3 days) on days -5, -4, and -3) is required for patients with moderately impaired renal function (creatine clearance ≤ 70 mL/min).

  • CyclophosphamideDrug

    Also called Cytoxan

    Cyclophosphamide 300 - 500 mg/m2/day IV (3 days) on days -5, -4, and -3

Study groups in NCT04109482
Study groupRoleWhat they receive
Relapsed or Refractory BPDCNExperimentalTreatment with MB-102.
Receives: Biological: MB-102; Drug: Fludarabine; Drug: Cyclophosphamide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1: Safety and Tolerability as measured by the number of patients with treatment related adverse events

    Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 in Phase 1

    Measured: 28 Days

  2. Phase 1: Maximum Tolerated Dose (MTD) and recommended Phase 2 dose

    To determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose of MB-102

    Measured: 28 Days

  3. Phase 2: Response Rate of patients with BPDCN

    Relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm is measured by a response rate which consists of Complete Response and clinical Complete Response and Complete Response with incomplete hematologic recovery (CR + CRc + CRi) at day 28 post infusion

    Measured: up to 3 years

Secondary outcomes (8)

  1. Phase 2: BPDCN - DOR

    Duration of Response

    Measured: up to 3 years

  2. Phase 2: BPDCN - PFS

    Progression-Free Survival

    Measured: up to 3 years

  3. Phase 2: BPDCN - OS

    overall survival

    Measured: up to 3 years

  4. Phase 2: BPDCN - MRD

    CR MRD- Response Rate for patients with CR and CRi

    Measured: up to 3 years

  5. Phase 2 - Adverse events

    Incidence of treatment-emergent AEs (TEAEs), including SAEs, therapy-related AEs or death.

    Measured: up to 3 years

  6. Phase 2 -Change from Baseline in the European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0.

    The European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0 is an integrated, modular approach for evaluating the quality of life of patients participating in international clinical trials. The questionnaire is designed to measure cancer patients' physical, psychological and social functions. The questionnaire is composed of 5 multi-item scales (physical, role, social, emotional and cognitive functioning) and 9 single items (pain, fatigue, financial impact, appetite loss, nausea/vomiting, diarrhea, constipation, sleep disturbance and quality of life).

    It utilizes a four-point scales for the first 28 questions which are coded with response categories as "Not at all", "A little", "Quite a bit" and "Very much.". the final two question consist of an overall physical condition questions which have employed a 7-point response scale where the higher number indicates a better overall health.

    Measured: up to 3 years

  7. Phase 2 - Change from Baseline in the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Version 4.0.

    The Functional Assessment of Cancer treatment for cancer, and a transplant-specific module, bone marrow transplant (BMT) concerns, that addresses disease and treatment-related questions specific to BMT.

    It utilizes a 5 point scale assessing physical, social, emotional, functional and other well-being concerns. Response categories are coded as "Not at all", "A little bit", "Somewhat", "Quite a bit" and "Very much.".

    Measured: up to 3 years

  8. Phase 2 - Number of patients showing evidence of replication competent lentivirus

    To confirm the absence of replication competent lentivirus

    Measured: up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Sep 2019
  2. Study start17 Feb 2020
  3. Primary completion17 May 2023
  4. Study completion17 May 2023

Why it stopped: Business reasons.

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04109482
Sponsor's protocol ID
MB102-CD123-001
Status last verified
Jun 2023
First submitted
25 Sep 2019
Last updated
22 Jul 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing MB-102, Fludarabine and Cyclophosphamide in people with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), run by Mustang Bio at 4 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 20 inclusion and 20 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 22 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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: Business reasons.). 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?

Phase 1: Safety and Tolerability as measured by the number of patients with treatment related adverse events (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?

On the ClinicalTrials.gov record for NCT04109482.

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 22 Jul 2024.

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