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Blastic Plasmacytoid Dendritic Cell NeoplasmPhase 2NCT04317781

Tagraxofusp in Treating Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm After Stem Cell Transplant

A Phase 2 study testing Tagraxofusp-erzs in people with Blastic Plasmacytoid Dendritic Cell Neoplasm, run by M.D. Anderson Cancer Center at 1 site 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

This phase II trial studies the side effects of tagraxofusp in treating patients with blastic plasmacytoid dendritic cell neoplasm after stem cell transplant. Tagraxofusp is a type of immunotoxin that is made by linking a protein called IL-3 to a toxic substance. Read more about this study

Who is it for?
Ages 2 years and older
What is being tested?
Tagraxofusp-erzs
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteUnited States
Study dates
27 Mar 2020to 11 Jul 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Eligible patients will be aged >= 18 years. Pediatric patients age 2 years and older will be considered on a case by case basis.
  • Diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN) according to World Health Organization (WHO) classification or confirmed by hematopathology
  • The patients must be in partial response or better
  • > 30 days post-transplant without active or chronic infections
  • Karnofsky performance status >= 60%; Lansky >= 60
  • Left ventricular ejection fraction (LVEF) >= institutional lower limit of normal by multigated acquisition (MUGA) scan or echocardiogram within 30 days of first protocol treatment
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  • Diffusion capacity of the lung for carbon monoxide (DLCO) > 40% of predicted value (corrected for hemoglobin) within 3 months of registration
  • Forced expiratory volume in 1 second (FEV1) > 40% of predicted value within 3 months of registration
  • Forced vital capacity (FVC) > 40% of predicted value within 3 months of registration
  • Serum creatinine =< 1.5 mg/dL (133 mmol/L)
  • Serum albumin >= 3.2 g/dL (or >= 32 g/L) without IV albumin within the previous 72 hours
  • Bilirubin =< 1.5 x the upper limit of normal ([ULN] except patients with Gilbert syndrome in whom bilirubin level of > 1.5 x ULN will be allowed)
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2.5 times ULN
  • Hemoglobin >= 8 g/dL with or without transfusion in the last 7 days
  • Absolute neutrophil count (ANC) >= 1000 without granulocyte colony stimulating factor (GCSF) or granulocyte-macrophage colony-stimulating factor (GMCSF) in the last 2 weeks prior to screening
  • Platelets >= 50,000micro/mL
  • For allo-HCT, no >= grade 2 visceral (gut or liver) acute graft versus host disease (GVHD) and no >= grade 3 or any other acute GVHD (patients with chronic GVHD will be allowed at the discretion of the investigator)
  • Patient agrees to use acceptable contraceptive methods for the duration of time in the study, and to continue to use acceptable contraceptive methods for 2 months after the last tagraxofusp infusion
  • Woman of child bearing potential (WOCBP) with a negative serum or urine pregnancy test within 14 days of tagraxofusp treatment
  • Patient or patient's legal representative, parent(s) or guardian able to sign informed consent
  • The patient can adhere to the study visit schedule and other protocol requirements, including follow-up for survival assessment

Who cannot join

  • The patient has persistent clinically significant non-hematologic toxicities >= grade 2 (excluding alopecia, nausea, and fatigue)
  • Evidence of central nervous system (CNS) involvement
  • Uncontrolled and active pulmonary disease
  • Requirement for oxygen treatment
  • Receiving chemotherapy, radiotherapy or other anti-cancer therapy within 14 days of first dose of study drug. There must be at least a 6-week interval from the last immunotherapy therapy
  • Uncontrolled infection
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  • Human immunodeficiency virus (HIV)/hepatitis B and/or C
  • Any history of invasive malignancy in the last 2 years excluding any malignancy such as cervical cancer or skin cancer (excluding melanoma) that is considered cured at the time of screening
  • Pregnant or breast-feeding woman
  • Patient has uncontrolled intercurrent illness or medical/psychiatric condition that would limit compliance with study requirements or that would in the investigator's opinion place the patient at an unacceptably high risk for toxicities
  • Clinical significant cardiopulmonary disease including uncontrolled or New York Heart Association (NYHA) class 3 or 4 congestive heart failure, uncontrolled angina, uncontrolled hypertension, uncontrolled arrhythmia, myocardial infarction or stroke within 6 months of first protocol treatment or corrected QT (QTc) > 480 ms

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M D Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: Tagraxofusp (SL-401) Therapy for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Patients Post-Autologous or Post-Allogeneic Hematopoietic Cell Transplantation

This phase II trial studies the side effects of tagraxofusp in treating patients with blastic plasmacytoid dendritic cell neoplasm after stem cell transplant. Tagraxofusp is a type of immunotoxin that is made by linking a protein called IL-3 to a toxic substance. Tagraxofusp may help find cancer cells that express IL-3 and kill them without harming normal cells.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To evaluate the safety of tagraxofusp-erzs (tagraxofusp) in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) after autologous (auto) or allogeneic (allo) hematopoietic cell transplantation (HCT).

SECONDARY OBJECTIVES:

  1. To estimate progression-free survival (PFS) in patients with BPDCN receiving maintenance therapy with tagraxofusp after auto-HCT or allo-HCT.

II. To estimate the overall survival (OS) in patients with BPDCN receiving maintenance therapy with tagraxofusp after auto-HCT or allo-HCT.

OUTLINE:

Within day 45 and 180 after stem cell transplant, patients receive tagraxofusp-erzs intravenously (IV) over 15 minutes on days 1-3 of cycles 1-4 and days 1-2 of subsequent cycles. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for up to 1 year.

Being tested
Tagraxofusp-erzs
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.

  • Tagraxofusp-erzsBiological

    Also called Diphtheria Toxin(388)-Interleukin-3 Fusion Protein, DT(388)-IL3 Fusion Protein, DT388IL3 fusion protein, Elzonris

    Given IV

Study groups in NCT04317781
Study groupRoleWhat they receive
Treatment (tagraxofusp-erzs)ExperimentalWithin day 45 and 180 after stem cell transplant, patients receive tagraxofusp-erzs IV over 15 minutes on days 1-3 of cycles 1-4 and days 1-2 of subsequent cycles. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
Receives: Biological: Tagraxofusp-erzs

What is being measured

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

  1. Number of Participants That Received Planned Tagraxofusp Post Transplant

    Participants completed at least 75% of planned tagraxofusp doses in at least 4 cycles of therapy.

    Measured: Up to 1 year

Secondary outcomes (2)

  1. Progression Free Survival (PFS)

    Participant alive and disease free one year post transplant

    Measured: From treatment start date to date of disease progression or death, assessed up to 1 year

  2. Overall Survival (OS)

    Number of participants alive one year post transplant

    Measured: From treatment start date to death, assessed up to 1 year

Timeline

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

  1. First posted23 Mar 2020
  2. Study start27 Mar 2020
  3. Primary completion11 Jul 2023
  4. Study completion11 Jul 2023

Why it stopped: The protocol was terminated early due to slow participant accrual and the sponsor not willing to provide the study drug.

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 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
NCT04317781
Sponsor's protocol ID
2018-0646
Other IDs
NCI-2019-03832, 2018-0646
Status last verified
Jul 2024
First submitted
19 Mar 2020
Last updated
31 Jul 2024
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Wang SY, Thomassen K, Kurch L, Opitz S, Franke GN, Bach E, Platzbecker U, Kayser S. Combination of Tagraxofusp and Azacitidine Is an Effective Option for Relapsed Blastic Plasmacytoid Dendritic Cell Neoplasm After Allogeneic Hematopoietic Stem-Cell Transplantation. Clin Lymphoma Myeloma Leuk. 2021 Jul;21(7):e579-e582. doi: 10.1016/j.clml.2021.02.008. Epub 2021 Mar 2. No abstract available.

    PubMed 33795208

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tagraxofusp-erzs in people with Blastic Plasmacytoid Dendritic Cell Neoplasm, run by M.D. Anderson Cancer Center 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?

The sponsor lists 21 inclusion and 11 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 31 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 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 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?

Yes. Results are posted in the registry (first posted 16 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants That Received Planned Tagraxofusp Post Transplant (time frame: Up to 1 year). 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 NCT04317781, which also lists 1 linked publication.

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 2024.

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