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TerminatedNCT04317781Updated Jul 31, 2024Results posted

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

A Phase 2 interventional study of Tagraxofusp-erzs in Blastic Plasmacytoid Dendritic Cell Neoplasm, sponsored by M.D. Anderson Cancer Center. Terminated at 1 site in United States. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2024-07-31.

Sponsored by M.D. Anderson Cancer Center · Phase 2, Interventional, and Treatment

Why this study was terminated
The protocol was terminated early due to slow participant accrual and the sponsor not willing to provide the study drug.
Phase
Phase 2
Study type
Interventional
Enrollment
3
Allocation
Not applicable
Ages
2 Years and older
Sex
All
01

Study 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. Tagraxofusp may help find cancer cells that express IL-3 and kill them without harming normal cells.

Read the detailed description

PRIMARY OBJECTIVE:

I. 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:

I. 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.

02

Conditions studied

  • Blastic Plasmacytoid Dendritic Cell Neoplasm

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03

In context

Neoplasms

9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.

This study's enrollment of 3 is below the median of 50 across 7,253 interventional studies indexed under Neoplasms.

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Lead sponsor

M.D. Anderson Cancer Center is the lead sponsor of 2,999 studies on the registry; 581 are open to participants now.

Of its 599 completed or terminated interventional studies of FDA-regulated products, 402 (67%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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
  • 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

Exclusion criteria

Exclusion Criteria:

  • 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
  • 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
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Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
3 participants (actual)

Study arms

  • Experimental
    Treatment (tagraxofusp-erzs)

    Within 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.

    Biological: Tagraxofusp-erzs

Interventions

  • BiologicalTagraxofusp-erzs

    Given IV

    Also known as: Diphtheria Toxin(388)-Interleukin-3 Fusion Protein, DT(388)-IL3 Fusion Protein, DT388IL3 fusion protein, Elzonris, IL3R-targeting Fusion Protein SL-401, SL-401, Tagraxofusp, Tagraxofusp ERZS

06

What researchers measure

Primary outcomes

  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.

    Time frame: Up to 1 year

Secondary outcomes

  1. Progression Free Survival (PFS)

    Participant alive and disease free one year post transplant

    Time frame: 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

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

07

Results

Posted Jul 16, 2024
Limitations and caveats
The protocol was terminated early due to slow participant accrual and the sponsor not willing to provide the study drug. Stemline is the funder, not the sponsor.

Participant flow

All participants were registered at MD Anderson Cancer Center

Participant flow — Overall Study
MilestoneTreatment (Tagraxofusp-erzs)
Started3
Completed0
Not completed3
Withdrew: Adverse event1
Withdrew: Disease relapse1
Withdrew: Withdrawal by subject1

Outcome measures

PrimaryNumber of Participants That Received Planned Tagraxofusp Post Transplant

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

Time frame:
Up to 1 year
Reported as:
Count of participants · Participants
Number of Participants That Received Planned Tagraxofusp Post Transplant
ParticipantsTreatment (Tagraxofusp-erzs)
Number of Participants That Received Planned Tagraxofusp Post Transplant1
SecondaryProgression Free Survival (PFS)

Participant alive and disease free one year post transplant

Time frame:
From treatment start date to date of disease progression or death, assessed up to 1 year
Reported as:
Count of participants · Participants
Progression Free Survival (PFS)
ParticipantsTreatment (Tagraxofusp-erzs)
Progression Free Survival (PFS)2
SecondaryOverall Survival (OS)

Number of participants alive one year post transplant

Time frame:
From treatment start date to death, assessed up to 1 year
Reported as:
Count of participants · Participants
Overall Survival (OS)
ParticipantsTreatment (Tagraxofusp-erzs)
Overall Survival (OS)2

Adverse events

Collected over up to a year post transplant. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (Tagraxofusp-erzs)1/3 (33.3%)0/3 (0%)1/3 (33.3%)
Most frequent other events
Most frequent other events
EventTreatment (Tagraxofusp-erzs)
Elevated TransaminasesInvestigations1/3

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment (Tagraxofusp-erzs)
<=18 years0
Between 18 and 65 years2
>=65 years1
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (Tagraxofusp-erzs)
Female1
Male2
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment (Tagraxofusp-erzs)
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White3
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)Treatment (Tagraxofusp-erzs)
United States3
08

Study locations

1 site
  • M D Anderson Cancer Center
    Houston, Texas 77030, United States
09

References and documents

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 ↗

Study documents

  • Protocol and statistical analysis plan · Mar 29, 2023

Documents are hosted by the registry — open the source record to download them.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 31, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT04317781
Lead sponsor
M.D. Anderson Cancer Center
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Mar 23, 2020
Start date
Mar 27, 2020
Primary completion
Jul 11, 2023
Completion
Jul 11, 2023
Results posted
Jul 16, 2024
Last update
Jul 31, 2024

Study contacts

Qaiser Bashir, MS
principal investigator · M.D. Anderson Cancer Center

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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