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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

100%

Hyperglycaemia

100%

Alanine aminotransferase increased

100%

Fatigue

67%

Blood alkaline phosphatase increased

67%

Oedema peripheral

67%

Pyrexia

67%

Headache

67%

Myalgia

67%

Aspartate aminotransferase increased

67%

Abdominal pain upper

67%

Diarrhoea

67%

Hypocalcaemia

67%

Weight increased

67%

Dyspnoea

67%

Pain in extremity

33%

Pain

33%

Rectal haemorrhage

33%

Pruritus

33%

Escherichia bacteraemia

33%

Capillary leak syndrome

33%

Febrile neutropenia

33%

Leukopenia

33%

Leukocytosis

33%

Oral pain

33%

Insomnia

33%

Proctalgia

33%

Glossodynia

33%

Malaise

33%

Abdominal pain

33%

Nausea

33%

Arthralgia

33%

Empyema

33%

Hyperkalaemia

33%

Hypokalaemia

33%

Back pain

33%

Stomatitis

33%

Hypotension

33%

Dyspnoea exertional

33%

Gingival pain

33%

Conjunctival haemorrhage

33%

Retching

33%

Decreased appetite

33%

Hypoalbuminaemia

33%

Deep vein thrombosis

33%

Epistaxis

33%

Contusion

33%

Oral mucosal blistering

33%

Hypomagnesaemia

33%

Rash erythematous

33%

Abdominal discomfort

33%

Soft tissue infection

33%

Vessel puncture site haematoma

100%

80%

60%

40%

20%

Study treatment Arm

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

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

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

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

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

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

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

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

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

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

Stage 1 - AML 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

~4020

Dexamethasone

2007

Completed Phase 4

~2650