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Protein-Drug Conjugate

Tagraxofusp for Blood Cancers

Phase 1

Recruiting

Research Sponsored by Therapeutic Advances in Childhood Leukemia Consortium

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at the end of cycle 1 (21 days for part 1, and 28 days for part 2)

Awards & highlights

No Placebo-Only Group

Summary

This trial is examining the safety of tagraxofusp, a novel agent that targets CD123, in pediatric patients with relapsed/refractory hematologic malignancies. The trial includes two parts: a monotherapy phase and a combination chemotherapy phase. The goal is to 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.

Who is the study for?

This trial is for children and young adults aged 1 to 21 with relapsed or refractory hematologic malignancies expressing CD123. Eligible participants include those with various types of leukemia, lymphoma, and myelodysplastic syndrome who have experienced multiple relapses or did not respond to at least two chemotherapy cycles. Patients must have adequate organ function and agree to use contraception if applicable.

What is being tested?

The study tests Tagraxofusp alone and in combination with other chemotherapies (like Hydrocortisone, Dexamethasone) in pediatric patients. It aims to determine the safe dosage levels, describe side effects, understand how the drug works in the body, and improve survival rates for these cancers.

What are the potential side effects?

Possible side effects may include reactions related to diphtheria toxin such as fever or nausea; however specific side effects are being studied as part of this trial's purpose.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at the end of cycle 1 (21 days for part 1, and 28 days for part 2)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at the end of cycle 1 (21 days for part 1, and 28 days for part 2)

This trial's timeline: 3 weeks for screening, Varies for treatment, and at the end of cycle 1 (21 days for part 1, and 28 days for part 2) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Side effects data

From 2020 Phase 1 & 2 trial • 138 Patients • NCT02113982

80%

Hypoalbuminaemia

60%

Pyrexia

60%

Oedema peripheral

50%

Nausea

50%

Fatigue

50%

Aspartate aminotransferase increased

50%

Alanine aminotransferase increased

50%

Thrombocytopenia

50%

Chills

40%

Neutropenia

40%

Hypotension

30%

Blood creatinine increased

30%

Back pain

30%

Capillary leak syndrome

30%

Hyperglycaemia

30%

Dyspnoea

30%

Hypertension

20%

Hyperuricaemia

20%

Pneumonia

20%

Mental status changes

20%

Hypomagnesaemia

20%

Hypocalcaemia

20%

Anaemia

20%

Blood alkaline phosphatase increased

20%

Decreased appetite

20%

Abdominal pain

20%

Urinary tract infection

20%

Hypokalaemia

20%

Haematuria

20%

Cough

20%

Lymphocytosis

20%

Vomiting

20%

Febrile neutropenia

20%

Pruritus

20%

Lymphopenia

20%

Tumour lysis syndrome

20%

Activated partial thromboplastin time prolonged

20%

Hypophosphataemia

10%

Non Cardiac Chest Pain

10%

Insomnia

10%

Escherichia Sepsis

10%

Diarrhoea

10%

Epistaxis

10%

Musculoskeletal pain

10%

Headache

10%

Anxiety

10%

Infusion related reaction

10%

Eye pain

10%

Abdominal pain upper

10%

Hyperbilirubinaemia

10%

Blood fibrinogen decreased

10%

Stomatitis

10%

Hypoxia

10%

Constipation

10%

Blood lactate dehydrogenase increased

10%

Bone pain

10%

Acute kidney injury

10%

Rash

10%

Deep vein thrombosis

10%

Blood creatine phosphokinase increased

10%

Metabolic Encephalopathy

10%

Asthenia

10%

Leukopenia

10%

Dry skin

10%

Hypermagnesaemia

10%

Proteinuria

10%

Dyspnoea exertional

10%

Nasal congestion

10%

Pain in extremity

10%

Musculoskeletal chest pain

10%

Leukocytosis

10%

International normalised ratio increased

10%

Weight increased

10%

Hypernatraemia

10%

Hyponatraemia

10%

Neuropathy peripheral

10%

Transaminases increased

10%

Aspartate Aminotransferase Increased

10%

Dizziness

100%

80%

60%

40%

20%

Study treatment Arm

R/R - Stage 2 - BPDCN Indication: 12 µg/kg/Day Dosage

1L - Stage 4 - BPDCN Indication: 12 µg/kg/Day Dosage

1L - Stage 1 - BPDCN Indication: 7 µg/kg/Day Dosage

R/R - Stage 4 - BPDCN Indication: 12 µg/kg/Day Dosage

1L - Stage 3 - BPDCN Indication: 12 µg/kg/Day Dosage

Stage 1 - AML Indication: 9 µg/kg/Day Dosage

1L - Stage 2 - BPDCN Indication: 12 µg/kg/Day Dosage

Stage 1 - AML Indication: 12 µg/kg/Day Dosage

Stage 1 - AML Indication: 7 µg/kg/Day Dosage

Stage 1 - AML Indication: 16 µg/kg/Day Dosage

1L - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage

Stage 2 - AML Indication: 12 µg/kg/Day Dosage

Stage 2 - AML Indication: 16 µg/kg/Day Dosage

R/R - Stage 1 - BPDCN Indication: 12 µg/kg/Day Dosage

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Part 2 - Cohort CExperimental Treatment5 Interventions

Tagraxofsup -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

Group II: Part 2 - Cohort BExperimental Treatment6 Interventions

Tagraxofsup -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

Group III: Part 2 - Cohort AExperimental Treatment6 Interventions

Tagraxofsup -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

Group IV: Part 1Experimental Treatment4 Interventions

Tagraxofusp -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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cytarabine

2016

Completed Phase 3

~3330

Dexamethasone

2007

Completed Phase 4

~2650