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Hematologic MalignancyPhase 1NCT05476770

Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies

A Phase 1 study testing Tagraxofusp, Fludarabine and Cytarabine in people with Hematologic Malignancy and AML, run by Therapeutic Advances in Childhood Leukemia Consortium at 31 sites in 2 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Read more about this study

Who is it for?
Ages 1 year to 21 years
Conditions the study is for
Hematologic Malignancy, AML, ALL
What is being tested?
Tagraxofusp, Fludarabine and Cytarabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
31 sitesin 2 countries
Study dates
11 Nov 2022to 11 Nov 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

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 1 year to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age
  • Patients must be ≥ 1 and ≤21 years of age at the time of study enrollment.
  • Diagnosis
  • Relapsed and/or refractory hematologic malignancy (including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma).
  • Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local institution.
  • Disease Status:
Show all 70Show fewer
  • Monotherapy, Part 1
  • Second or greater relapse; or
  • Refractory after 2 or more chemotherapy cycles; or
  • First relapse after primary chemotherapy-refractory disease; or
  • BPDCN in first relapse or refractory after 1 or more chemotherapy cycles
  • Combination therapy, Part 2
  • First or greater relapse; or
  • Refractory after 2 or more chemotherapy cycles; or
  • BPDCN in first relapse or refractory after 1 or more chemotherapy cycles
  • For relapsed/refractory leukemia, patients must have:
  • >5% blasts in the bone marrow aspirate or biopsy by morphology or flow cytometry
  • Patients with 1% - 5% blasts are eligible for Part 2, Cohort C (only), if A single bone marrow sample with flow cytometry and at least one other test (e.g. karyotype, FISH, PCR, or NGS) shows ≥ 1% leukemic blasts and/or flow cytometry demonstrates a stable or rising level of disease on two serial bone marrows.
  • For relapsed/refractory non-Hodgkin or Hodgkin lymphoma, patients must have:
  • Histologic verification of relapse
  • Measurable disease documented by radiographic criteria or bone marrow
  • Patients in Part 1 may have sites of non-CNS extramedullary disease, but no CNS disease. Patients in Part 2 may have CNS disease and/or other non-CNS extramedullary disease. No cranial irradiation is allowed during the protocol therapy.
  • Patients with Down syndrome are eligible to participate in Part 1 only.
  • Performance Level
  • Karnofsky > 50% for patients > 16 years of age and Lansky > 50% for patients ≤ 16 years of age (See Appendix I for Performance Scales). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Prior Therapy
  • Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy, defined as resolution of all such toxicities to ≤ Grade 2 or lower per the inclusion/exclusion criteria.
  • Myelosuppressive chemotherapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study. At least 14 day must have elapsed since the completion of myelosuppressive therapy. However, individuals may receive any of the following medications within 14 days without a "wash-out period":
  • Hydroxyurea: Hydroxyurea can be initiated and/or continued for up to 24 hours prior to the start of protocol therapy.
  • "Maintenance-style" therapy: therapy including vincristine (dosed a maximum of one-time weekly), oral 6-mercaptopurine, oral methotrexate (dosed a maximum of one-time weekly), intrathecal therapy (dosed a maximum of one-time weekly) and/or dexamethasone (dosed at ≤3 mg/m2/dose twice daily) or prednisone (dosed at ≤20 mg/m2/dose twice daily) can be continued for up to 24 hours prior to entering the study.
  • Hematopoietic stem cell transplant: Patients who have experienced their relapse after a HSCT are eligible, provided they have no evidence of acute or chronic Graft-versus-Host Disease (GVHD) and are at least 100 days post-transplant at the time of enrollment.
  • Hematopoietic growth factors: It must have been at least 7 days since the completion of therapy with granulocyte colony stimulating factor (GCSF) or other growth factors at the time of enrollment. It must have been at least 14 days since the completion of therapy with pegfilgrastim (Neulasta®).
  • Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.
  • Monoclonal antibodies: Maximum of 3 half-lives of the antibody or 21 days (whichever is shorter) must have elapsed after the last dose of monoclonal antibody.
  • Immunotherapy: At least 30 days from last infusion of chimeric antigen receptor T cell (CART) therapy or tumor vaccine.
  • Radiation Therapy (XRT):
  • ≥ 84 days must have passed, from the end of therapy, if patient received prior total body irradiation (TBI).
  • ≥ 42 days must have passed, from the end of therapy, if patient received craniospinal irradiation (CSI).
  • ≥ 14 days must have passed after whole brain radiotherapy or stereotactic radiation therapy.
  • No washout period is required for:
  • Extramedullary site other than CNS that is a maximum 10 x 10 cm total radiation non-CNS field. If the field is > 10 x 10 cm, a 14-day washout period is required. ii. Local ocular radiotherapy as long as subject has measurable/evaluable disease outside the radiation port.
  • Patients that have received other non-tagraxofusp CD123 targeting agents are eligible. Patients that have previously received tagraxofusp are not eligible.
  • Organ Function Requirements
  • Adequate Bone Marrow Function Defined as:
  • Patients should not be known to be refractory to red blood cell or platelet transfusions.
  • Blood counts are not required to be normal prior to enrollment on trial. However, platelet count must be ≥20,000/mm3 to initiate therapy (may receive platelet transfusions).
  • Adequate Renal Function Defined as:
  • Patient must have a calculated creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73m2 OR a normal serum creatinine based on age/gender in the chart below:
  • Maximum Serum Creatinine (mg/dL):
  • 1 to < 2 years old - Male: 0.6, Female: 0.6
  • 2 to < 6 years old - Male:0.8, Female: 0.8
  • 6 to < 10 years old - Male: 1, Female: 1
  • 10 to < 13 years old - Male: 1.2, Female: 1.2
  • 13 to < 16 years old - Male: 1.5, Female: 1.4
  • ≥ 16 years old - Male: 1.7, Female: 1.4
  • The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.
  • Adequate Liver Function Defined as:
  • Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x institutional upper limit of normal for age
  • SGPT (ALT) and SGOT (AST) must be less than 3x institutional upper limit of normal.
  • Serum albumin ≥3.2 g/dL (albumin infusion independent).
  • Adequate Cardiac Function Defined as:
  • Shortening fraction of ≥27% by echocardiogram, or
  • Ejection fraction of ≥ 50% by gated radionuclide study/echocardiogram.
  • Adequate Pulmonary Function Defined as:
  • Pulse oximetry > 94% on room air (> 90% if at high altitude)
  • No evidence of dyspnea at rest and no exercise intolerance.
  • Reproductive Function
  • Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 2 weeks prior to enrollment.
  • Female patients with infants must agree not to breastfeed their infants while on this study.
  • Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study and for 12 weeks after the last dose of tagraxofusp.

Who cannot join

  • Disease Status:
  • Patients with CNS disease are not eligible for Part 1.
  • Patients with isolated CNS disease are not eligible for Part 1 or Part 2.
  • Patients with isolated non-CNS disease are eligible for Part 1 and Part 2.
  • Concomitant Medications
  • Corticosteroids - Patients receiving corticosteroids for disease control who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.
Show all 17Show fewer
  • Investigational Drugs - Patients who are currently receiving another investigational drug are not eligible. The definition of "investigational" for use in this protocol means any drug that is not licensed by the FDA, Health Canada or the Therapeutic Goods Administration to be sold in the countries they govern. (United States, Canada and Australia)
  • Anti-cancer Agents - Patients who are currently receiving or may receive while on therapy, other anti-cancer agents, radiation therapy or immunotherapy are not eligible [with the exceptions being laid out in the inclusion criteria under 'Prior Therapy']. Intrathecal chemotherapy (at the discretion of the primary oncologist) may be given up to one week prior to the initiation of study treatment (day 1 therapy).
  • Anti-GVHD or agents to prevent organ rejection post-transplant - Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial. At least 4 weeks must have elapsed after the last dose of GVHD meds.
  • Infection Criteria - Patients are excluded if they have:
  • Positive blood culture within 48 hours of study enrollment;
  • Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection. Fever that is determined to be due to tumor burden is allowed if patients have documented negative blood cultures for at least 48 hours prior to enrollment and no concurrent signs or symptoms of active infection or hemodynamic instability.
  • A positive fungal culture within 30 days of study enrollment.
  • Active fungal, viral, bacterial, or protozoal infection requiring IV treatment. Chronic prophylaxis therapy to prevent infections is allowed.
  • Patients will be excluded if they have a known allergy to any of the drugs used in the study.
  • Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures, interfere with consent, study participation, follow up, or interpretation of study results.
  • Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.

Trial locations and contacts

31 sites in 2 countries. Contact the study team or a site near you to ask about taking part.

Benjamin N Brookhart contact

323-361-5429

bbrookhart@chla.usc.edu

Ellynore Florendo contact

323-361-3022

eflorendo@chla.usc.edu

31 sites in 2 countries

United States29 sites, 29 recruiting

  • C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109-0914

    Rajen Mody, MD

    Recruiting

  • Children's Healthcare of Atlanta, Emory University

    Atlanta, Georgia, 30322

    Melinda Pauly, MD

    Not yet recruiting

  • Johns Hopkins University

    Baltimore, Maryland, 21231

    Pat Brown, MD

    Not yet recruiting

  • National Cancer Institute, Pediatric Oncology Branch

    Bethesda, Maryland, 20892

    Nirali Shah, MD

    Not yet recruiting

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

    Andrew Place, MD

    Not yet recruiting

  • Carolina-Levine Children's Hospital

    Charlotte, North Carolina, 28204

    Joel Kaplan, MD

    Not yet recruiting

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

    Jenna Rossoff, MD

    Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

    Robin Norris, MD

    Recruiting

  • Rainbow Babies

    Cleveland, Ohio, 44106

    Rachel Egler, MD

    Not yet recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

    Susan Vear-Colace, MD

    Not yet recruiting

  • University of Texas, Southwestern

    Dallas, Texas, 75235

    Tamra Slone, MD

    Recruiting

  • Children's Hospital Colorado

    Denver, Colorado, 80045

    Margaret Macy, MD

    Recruiting

  • Cook Children's Hospital

    Fort Worth, Texas, 76104

    Kenneth Heym, MD

    Recruiting

  • Texas Children's Hospital/Baylor College of Medicine

    Houston, Texas, 77030

    Eric Schafer, MD

    Recruiting

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202

    Sandeep Batra, MD

    Recruiting

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

    Alan Wayne, MD

    Recruiting

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

    Jeffrey Rubnitz, MD

    Not yet recruiting

  • University of Miami

    Miami, Florida, 33136

    Julio Barredo, MD

    Not yet recruiting

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226

    Zachary Graff, MD

    Recruiting

  • Children's Hospital and Clinics of Minnesota

    Minneapolis, Minnesota, 55404

    Nathan Gossai, MD

    Nathan Gossai, MD

    Not yet recruiting

  • Children's Hospital New York-Presbyterian

    New York, New York, 10032

    Nobuko Hijiya, MD

    Not yet recruiting

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

    Maria Sulis, MD, MS

    Recruiting

  • Children's Hospital Orange County

    Orange, California, 92868

    Van Thu Huynh, MD

    Not yet recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Sue Rheingold, MD

    Not yet recruiting

  • Oregon Health & Science University

    Portland, Oregon, 97239

    Bill Chang, MD

    Not yet recruiting

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

    Anupam Verma, MD

    Recruiting

  • UCSF School of Medicine

    San Francisco, California, 94143-0106

    Michelle Hermiston, MD

    Recruiting

  • Seattle Children's Hospital

    Seattle, Washington, 98105

    Adam Lamble, MD

    Not yet recruiting

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

    Reuven Schore, MD

    Recruiting

Other trials in United States

Australia2 sites, 2 recruiting

  • Sydney Children's Hospital

    Sydney

    Tracey O'Brien, MD

    Not yet recruiting

  • Children's Hospital at Westmead

    Westmead, New South Wales

    Luciano Dalla-Pozza, MD

    Recruiting

Other trials in Australia

About this study

Official title: A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies

Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). This trial aims to examine the safety of this novel agent in pediatric patients with relapsed/refractory hematologic malignancies.

The mechanism by which tagraxofusp kills cells is distinct from that of conventional chemotherapy. Tagraxofusp directly targets CD123 that is present on tumor cells, but is expressed at lower or levels or absent on normal hematopoietic stem cells. Tagraxofusp also utilizes a payload that is not cell cycle dependent, making it effective against both highly proliferative tumor cells and also quiescent tumor cells.

Read more

The rationale for clinical development of tagraxofusp for pediatric patients with hematologic malignancies is based on the ubiquitous and high expression of CD123 on many of these diseases, as well as the highly potent preclinical activity and robust clinical responsiveness in adults observed to date.

This trial includes two parts: a monotherapy phase and a combination chemotherapy phase. This design will provide further monotherapy safety data and confirm the FDA approved pediatric dose, as well as provide safety data when combined with chemotherapy.

The goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to describe the toxicities, pharmacokinetics, and pharmacodynamic properties of tagraxofusp in pediatric patients.

About 54 children and young adults will participate in this study. Patients with Down syndrome will be included in part 1 of the study.

Being tested
Tagraxofusp, Fludarabine, Cytarabine, Dexamethasone, Vincristine
Medical subject headings
Hematologic Neoplasms, Blastic Plasmacytoid Dendritic Cell Neoplasm, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, B-Cell, Lymphoma, T-Cell, Hodgkin Disease, Leukemia, Biphenotypic, Acute

What participants receive

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

  • TagraxofuspDrug

    Also called Elzonris

    Dose will be assigned at study entry. Give IV over 15 minutes.

  • FludarabineDrug

    30 mg/m^2 will be given IV over 30 minutes on days 1-5.

    Infusion will start 30 minutes after start of tagraxofusp on days 4 and 5.

  • CytarabineDrug

    Also called Cytosar

    2000 mg/m2 intravenously will be given daily over 1-3 hours for 5 days on days 1 through 5. Infusion will begin 4 hours after start of fludarabine. Because of an increased risk of neurotoxicity, it is recommended that IT cytarabine be separated from high dose IV cytarabine administration by at least 24 hours on C1D1.

  • DexamethasoneDrug

    Also called Decadron

    • 20 mg/m2/day divided BID (max 40 mg/day) given orally on days 1 through 5 and 15 through 19. The two doses should be separated by at least 8 hours.
    • Any oral formulation of dexamethasone is acceptable.
    • IV may be given if oral formulation is not tolerated

  • VincristineDrug

    Also called Oncovin

    • 1.5 mg/m2 (maximum dose 2 mg) given intravenously as an IV push over 1-5 minutes or infusion via minibag as per institutional policy on days 1, 8, 15, and 22.
    • Infusion will start 30 minutes after start of tagraxofusp on day 8.

  • AzacitidineDrug

    Also called AZA, Vidaza

    • 75 mg/m2 subcutaneously or intravenously will be given daily over 15 minutes for 5 days on days 1 through 5.
    • Azacitidine will be given 30-60 minutes before beginning the tagraxofusp infusion.

  • MethotrexateDrug

    Also called MTX, Amethopterin

    Give intrathecally:

    • 8 mg for patients age 1-1.99
    • 10 mg for patients age 2-2.99
    • 12 mg for patients 3-8.99 years of age
    • 15 mg for patients ≥9 years of age

  • Cytarabine ITDrug

    Also called Cytosar

    Give intrathecally:

    • 30 mg for patients age 1-1.99
    • 50 mg for patients age 2-2.99
    • 70 mg for patients ≥3 years of age

    If given as part of Triple IT Therapy:

    AML Patients:

    Age 1-1.99 - 24 mg Age 2-2.99 - 30 mg Age ≥3 years of age - 36 mg

    AML Patients:

    Age 1-1.99 - 16 mg Age 2-2.99 - 20 mg Age 3-8.99 - 24 mg Age ≥9 years of age - 30 mg

  • HydrocortisoneDrug

    Given intrathecally.

    AML Patients:

    Age 1-1.99 - 16 mg Age 2-2.99 - 20 mg Age ≥3 years of age - 24 mg

    AML Patients:

    Age 1-1.99 - 8 mg Age 2-2.99 - 10 mg Age 3-8.99 - 12 mg Age ≥9 years of age - 15 mg

Study groups in NCT05476770
Study groupRoleWhat they receive
Part 1ExperimentalTagraxofusp -Days 1-5 IT Therapy (may include methotrexate, cytarabine, or triple IT) * Day 1 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator
Receives: Drug: Tagraxofusp; Drug: Methotrexate; Drug: Cytarabine IT; Drug: Hydrocortisone
Part 2 - Cohort AExperimentalTagraxofsup -Days 4-8 Fludarabine -Days 1-5 Cytarabine -Days 1-5 IT Therapy (may include methotrexate, cytarabine, or triple IT) CNS1 IT Therapy * Day 1 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator CNS2/3 IT Therapy * Days 1, 8, 15, and 22 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator
Receives: Drug: Tagraxofusp; Drug: Fludarabine; Drug: Cytarabine; Drug: Methotrexate; Drug: Cytarabine IT; Drug: Hydrocortisone
Part 2 - Cohort BExperimentalTagraxofsup -Days 8-12 Dexamethasone -Days 1-5 Vincristine -Days 1, 8, 15, and 22 IT Therapy (may include methotrexate, cytarabine, or triple IT) CNS1 IT Therapy * Day 1 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator CNS2/3 IT Therapy * Days 1, 8, 15, and 22 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator
Receives: Drug: Tagraxofusp; Drug: Dexamethasone; Drug: Vincristine; Drug: Methotrexate; Drug: Cytarabine IT; Drug: Hydrocortisone
Part 2 - Cohort CExperimentalTagraxofsup -Days 1-5 Azacitidine -Days 1-5 IT Therapy (may include methotrexate, cytarabine, or triple IT) CNS1 IT Therapy * Day 1 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator CNS2/3 IT Therapy * Days 1, 8, 15, and 22 * Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator
Receives: Drug: Tagraxofusp; Drug: Azacitidine; Drug: Methotrexate; Drug: Cytarabine IT; Drug: Hydrocortisone

What is being measured

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

  1. Occurrence of dose limiting toxicity (DLT) during cycle 1 of therapy

    The incidence of dose limiting toxicity (DLT) will be measured at different dose levels.

    Measured: At the end of Cycle 1 (21 days for Part 1, and 28 days for Part 2)

Timeline

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

  1. First posted27 Jul 2022
  2. Study start11 Nov 2022
  3. Primary completion11 Nov 2025 (estimated)
  4. Study completion11 Nov 2027 (estimated)

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
54 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05476770
Sponsor's protocol ID
T2020-006
Status last verified
Dec 2024
First submitted
25 Jul 2022
Last updated
6 Dec 2024
Results posted
No
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 study testing Tagraxofusp, Fludarabine and Cytarabine in people with Hematologic Malignancy and AML, run by Therapeutic Advances in Childhood Leukemia Consortium at 31 sites in 2 countries.

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 70 inclusion and 17 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: looking for participants now, as of the sponsor's last update on 6 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 31 sites in United States, Australia. 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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Benjamin N Brookhart (323-361-5429, bbrookhart@chla.usc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Occurrence of dose limiting toxicity (DLT) during cycle 1 of therapy (time frame: At the end of Cycle 1 (21 days for Part 1, and 28 days for Part 2)). 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 NCT05476770.

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 6 Dec 2024.

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Clinical Trial NCT05476770 | AzTrials