AzTrials
AzTrials

Acute Myeloid LeukemiaPhase 2NCT04342962

Tagraxofusp in Patients With CD123+ or With BPDCN-IPh-like Acute Myeloid Leukemia

A Phase 2 study testing tagraxofusp in people with Acute Myeloid Leukemia, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 10 sites in Italy.

Terminated

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

Official record
Search all trials

Trial summary

Non-randomized, open-label, multicenter phase II Study for the treatment of 25 R/R BPDCN-IF (CD123/CD4/CD56 positive) AML patients and 25 patients presenting R/R AML CD123+, but negative for either, or both, CD4 and CD56. Patients will be treated with 12 mcg/kg/day of tagraxofusp for 5 days, for at least 4 cicles. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
tagraxofusp
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
10 sitesItaly
Study dates
16 Feb 2021to 18 Jan 2024
Phase
Phase 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

  • The patient has evidence of AML in the peripheral blood and/or bone marrow with either BPDCN-IF [CD123/CD4/CD56 (+)] or with AML that is CD123+ but negative for either, or both, CD4 and CD56.
  • The patient is ≥18 years old.
  • The patient must be refractory to at least one previous line of conventional therapy (either high dose therapy or hypomethylating agents) or relapsed after receiving conventional therapy (a maximum of two previous line of therapy is admitted).
  • The patient has an Eastern Cooperative Oncology Group (ECOG) performance score (PS) of 0 to 2.
  • The patient has adequate baseline organ function, including cardiac, renal, and hepatic function:
  • Left ventricular ejection fraction (LVEF) ≥institutional lower limit of normal as measured by multigated acquisition (MUGA) scan or 2-dimensional (2-D) echocardiography(ECHO) within 21 days before start of therapy and no clinically significant abnormalities on a 12-lead electrocardiogram (ECG).
Show all 14Show fewer
  • Serum creatinine ≤1.5 mg/dL (133 μmol/L).
  • Serum albumin ≥3.2 g/dL (32 g/L) (albumin infusions are not permitted to enable eligibility).
  • Bilirubin ≤1.5 mg/dL (26 μmol/L).
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 times the upper limit of normal (ULN).
  • If the patient is a woman of childbearing potential (WOCBP), she must have a negative serum or urine pregnancy test at screeningwithin 1 week before treatment.
  • The patient has signed informed consent before initiation of any study-specific procedures or treatment.
  • The patient is able to adhere to the study visit schedule and other protocol requirements, including follow-up for survival assessment.
  • The patient (male and female) agrees to use acceptable contraceptive methods for the duration of time on the study and continue to use acceptable contraceptive methods for 1 week after the last infusion of tagraxofusp.

Who cannot join

  • The patient has a diagnosis of acute promyelocytic leukemia (APL; FAB subtype M3).
  • The patient has persistent clinically significant toxicities of Grade≥2 from previous chemotherapy (excluding alopecia, nausea, fatigue, and liver function tests [as mandated in the inclusion criteria]).
  • The patient has received treatment with chemotherapy, wide-field radiation, or biologic therapy within 14 days of study entry.
  • The patient has received treatment with an investigational agent within 14 days of study entry.
  • The patient has previously received treatment with tagraxofusp.
  • The patient has an active malignancy and/or cancer history (excluding antecedent MDS) that may confound the assessment of the study endpoints. Patients with a past cancer history (within 2 years of entry) with substantial potential for recurrence and/or ongoing active malignancy will be evaluated on a case by case basis. Patients with the following neoplastic diagnoses are eligible: non-melanoma skin cancer, carcinoma in situ, cervical intraepithelial neoplasia, organ-confined prostate cancer with no evidence of progressive disease.
Show all 16Show fewer
  • The patient has clinically significant cardiovascular disease (eg, uncontrolled or any New York Heart Association Class 3 or 4 congestive heart failure, uncontrolled angina, history of myocardial infarction, unstable angina or stroke within 6 months before study entry, uncontrolled hypertension or clinically significant arrhythmias not controlled by medication).
  • The patient has uncontrolled, clinically significant pulmonary disease (eg, chronic obstructive pulmonary disease, pulmonary hypertension) that, in the opinion of the Investigator, would put the patient at significant risk for pulmonary complications during the study.
  • The patient has known active or suspected central nervous system (CNS) leukemia. If suspected, CNS leukemia should be ruled out with relevant imaging and/or examination of cerebrospinal fluid.
  • The patient is receiving immunosuppressive therapy - with the exception of low-dose prednisone (≤10 mg/day) - for treatment or prophylaxis of graft-versus-host disease (GVHD). If the patient has been on immunosuppressive treatment or prophylaxis for GVHD, the treatment(s) must have been discontinued at least 14 days before study treatment and there must be no evidence of Grade ≥2 GVHD.
  • The patient has uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, disseminated intravascular coagulation, or psychiatric illness/social situations that would limit compliance with study requirements.
  • The patient is pregnant or breastfeeding.
  • The patient has known positive status for human immunodeficiency virus or active or chronic hepatitis B or hepatitis C (Patients with positive serology for HBV can be enrolled and must receive antiviral prophylaxis - i.e lamivudine or entcavir).
  • The patient is oxygen-dependent.
  • The patient has any medical condition that, in the opinion of the Investigator, places the patient at an unacceptably high risk for toxicities.
  • The patient has AML and requires more than 1 g/day of hydroxyurea (Hydroxyureaispermittedatdoses of ≤1 g/day.)

Trial locations and contacts

10 sites in 1 country.

10 sites in 1 country

Italy10 sites

  • Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia

    Bergamo

  • Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia

    Bologna

  • Asst Degli Spedali Civili Di Brescia - Uo Ematologia

    Brescia

  • Aou Policlinico Vittorio Emanuele, Po Ospedaliero "G. Rodolico" - Uo Ematologia Con Trapianto Di Midollo Osseo - Catania

    Catania

  • Irccs Aou San Martino - Genova - Uo Clinica Ematologica

    Genova

  • Asst Grande Ospedale Metropolitano Niguarda - Milano - Sc Ematologia

    Milan

  • Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora

    Milan

  • Ao Ospedali Riuniti Villa Sofia Cervello - Palermo - Uo Ematologia Ad Indirizzo Oncologico

    Palermo

  • Ao Di Perugia, Ospedale S. Maria Della Misericordia - Ematologia E Trapianto Midollo Osseo

    Perugia

  • Aou Senese - Uoc Ematologia E Trapianti

    Siena

Other trials in Italy

About this study

Official title: Tagraxofusp in Patients With CD123+ or With Blastic Plasmacytoid Dendritic Cell Neoplasm Immunophenotype-like Acute Myeloid Leukemia

Non-randomized, open-label, multicenter phase II Study for the treatment of

  • 25 R/R BPDCN-IF (CD123/CD4/CD56 positive) AML patients and
  • 25 patients presenting R/R AML CD123+, but negative for either, or both, CD4 and CD56.

Patients will be treated with 12 mcg/kg/day of tagraxofusp for 5 days, for at least 4 cicles.

Being tested
tagraxofusp
Keywords
CD123+ Acute Myeloid Leukemia, Blastic Plasmacytoid Dendritic Cell Leukemia, Relapsed, Refractory
Medical subject headings
Leukemia, Myeloid, Acute, Recurrence

What participants receive

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

  • tagraxofuspDrug

    Tagraxofusp is provided as an intravenous (IV) injectable and administered as a 15-minute IV infusion. Cycle 1 will include a 2-day dosing period for the first 3 enrolled patients (Days 1-2), a 2-day plus an optional 3rd day for patients 4-6 (Days 1-2 + Day 3 if patient meets criteria for continued dosing) and a 3-day dosing period for patients 7 and beyond (Days 1-3). Cycle 2 and beyond will include a 5-day dosing period for all patients (Days 1-5). In all cycles and schedules, dose delays up to Day 10 of each cycle will be allowed for resolution of toxicities.

Study groups in NCT04342962
Study groupRoleWhat they receive
Experimental armExperimental12 mcg/kg/day of tagraxofusp for 5 days, for at least 4 cycles of therapy; each cycle is 21 days. Patients will receive the study drug until disease progression or in case of toxicity.
Receives: Drug: tagraxofusp

What is being measured

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

  1. The objective response rate (ORR)

    Evaluate the activity of tagraxofusp, in terms of ORR (PR + CR + CRi), in patients with CD123+ or BlasticPlasmacytoid Dendritic Cell Neoplasm-like phenotype (BPDCN-IF) Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML).

    Measured: 4 months

Secondary outcomes (6)

  1. Rate of AEs and SAEs

    Safety analysis according to CTCAE criteria

    Measured: 28 months

  2. Overall survival (OS)

    Overall survival

    Measured: 24 months

  3. Event Free Survival (EFS)

    Event Free Survival

    Measured: 24 months

  4. Disease Free Survival (DFS)

    Disease Free Survival

    Measured: 24 months from response assessment

  5. Cumulative incidence of relapse (CIR)

    Cumulative incidence of relapse

    Measured: 24 months from response assessment

  6. Percentage of patients undergoing allogeneic stem cell transplantation

    in MRD-negative CR

    Measured: 12 months

Timeline

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

  1. First posted13 Apr 2020
  2. Study start16 Feb 2021
  3. Primary completion18 Jan 2024
  4. Study completion18 Jan 2024

Why it stopped: poor enrollment

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
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04342962
Sponsor's protocol ID
AML2020
Status last verified
Jan 2025
First submitted
9 Apr 2020
Last updated
30 Jan 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing tagraxofusp in people with Acute Myeloid Leukemia, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 10 sites in Italy.

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 14 inclusion and 16 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 30 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Italy. 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?

The study was stopped early (reason given: poor enrollment). 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?

The objective response rate (ORR) (time frame: 4 months). 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 NCT04342962.

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 30 Jan 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in