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Flotetuzumab for the Treatment of Pediatric Recurrent or Refractory Acute Myeloid Leukemia
Phase 1 trial testing Cytarabine in Recurrent Acute Myeloid Leukemia in 16 participants. Participants enrolled and being followed up; not accepting new ones.
| Lead sponsor | Children's Oncology Group |
|---|---|
| Phase | Phase 1 |
| Status | Active, enrolled |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 16 |
| Start date | 22 January 2020 |
| Primary completion | 30 September 2022 |
| Estimated completion | 31 March 2026 |
| Sites | 19 locations across United States |
Children's Oncology Group — full company profile →
Under 20, any sex, with Recurrent Acute Myeloid Leukemia or Refractory Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number and percentage of patients experiencing dose limiting toxicities during cycle 1 that are possibly, probably, or definitely due to flotetuzumab by dose level and study part.
| Group | Value | 95% CI |
|---|---|---|
| Part A Dose Level 1: 500 Nanograms/kg/Day | 1 | |
| Part A Dose Level 2: 700 Nanograms/kg/Day | 1 |
Estimated maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of Flotetuzumab defined by the maximum dose at which fewer than one-third of toxicity-evaluable participants experience a DLT. RP2D was established by study committee based off the toxicity profile of DL1 (500 ng/kg/day).
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Flotetuzumab, Cytarabine) | 500 |
Median with Minimum and Maximum for the maximum (peak) serum concentration measured on days 3-9 post-administration by dose level and study part.
| Group | Value | 95% CI |
|---|---|---|
| Part A Dose Level 1: 500 Nanograms/kg/Day | 108.6 | 0.0 – 139.6 |
| Part A Dose Level 2: 700 Nanograms/kg/Day | 147.6 | 17.3 – 1835.4 |
Number of response evaluable participants with response (CR/PR) using revised AML International Working Group criteria for response including CR: M1 bone marrow (\<5% blasts), ANC at least 1000/mm3 and platelet count at least 100,000/mm3; CRp: platelet transfusion independence; CRi: without platelet transfusion independence, CRc: revision to normal karyotype; PR: decrease at least 50% in percentage of blasts to 5% to 25% in bone marrow aspirate.
| Group | Value | 95% CI |
|---|---|---|
| Part A Dose Level 1:500 Nanograms/kg/Day | 1 | |
| Part A Dose Level 2: 700 Nanograms/kg/Day | 1 |
Time frame: Up to 2 Years. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Stratum 1 Dose Level 1 | Stratum 1 Dose Level 2 |
|---|---|---|---|
| Platelet count decreased | Investigations | — | — |
| White blood cell decreased | Investigations | — | — |
| Anemia | Blood and lymphatic system disorders | — | — |
| Lymphocyte count decreased | Investigations | — | — |
| Hypokalemia | Metabolism and nutrition disorders | — | — |
| Neutrophil count decreased | Investigations | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Hypertension | Vascular disorders | — | — |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — | — |
| Anorexia | Metabolism and nutrition disorders | — | — |
| Cytokine release syndrome | Immune system disorders | — | — |
| Fever | General disorders | — | — |
| Hypotension | Vascular disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Sepsis | Infections and infestations | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Alanine aminotransferase increased | Investigations | — | — |
| Alkalosis | Metabolism and nutrition disorders | — | — |
| Aspartate aminotransferase increased | Investigations | — | — |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — |
| Blood bilirubin increased | Investigations | — | — |
| CD4 lymphocytes decreased | Investigations | — | — |
| Capillary leak syndrome | Vascular disorders | — | — |
| Reaction | System | Stratum 1 Dose Level 1 | Stratum 1 Dose Level 2 |
|---|---|---|---|
| Hypoalbuminemia | Metabolism and nutrition disorders | — | — |
| Cytokine release syndrome | Immune system disorders | — | — |
| Hyperglycemia | Metabolism and nutrition disorders | — | — |
| Hypophosphatemia | Metabolism and nutrition disorders | — | — |
| Hypocalcemia | Metabolism and nutrition disorders | — | — |
| Hyponatremia | Metabolism and nutrition disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Sinus tachycardia | Cardiac disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Alanine aminotransferase increased | Investigations | — | — |
| Hypomagnesemia | Metabolism and nutrition disorders | — | — |
| Weight gain | Investigations | — | — |
| Capillary leak syndrome | Vascular disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Fatigue | General disorders | — | — |
| Fever | General disorders | — | — |
| Headache | Nervous system disorders | — | — |
| Hypokalemia | Metabolism and nutrition disorders | — | — |
| Hypotension | Vascular disorders | — | — |
| Abdominal distension | Gastrointestinal disorders | — | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — | — |
| Aspartate aminotransferase increased | Investigations | — | — |
| Blood bilirubin increased | Investigations | — | — |
| Chills | General disorders | — | — |
| Creatinine increased | Investigations | — | — |
| Diarrhea | Gastrointestinal disorders | — | — |
| Edema limbs | General disorders | — | — |
| Hypermagnesemia | Metabolism and nutrition disorders | — | — |
| Hypertension | Vascular disorders | — | — |
| INR increased | Investigations | — | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — | — |
| Proteinuria | Renal and urinary disorders | — | — |
| Pruritus | Skin and subcutaneous tissue disorders | — | — |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | — | — |
| Sore throat | Respiratory, thoracic and mediastinal disorders | — | — |
| Activated partial thromboplastin time prolonged | Investigations | — | — |
| Anorexia | Metabolism and nutrition disorders | — | — |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — |
| Bruising | Injury, poisoning and procedural complications | — | — |
Most-reported serious reactions: Platelet count decreased, White blood cell decreased, Anemia, Lymphocyte count decreased, Hypokalemia, Neutrophil count decreased, Febrile neutropenia, Hypertension.
Data from ClinicalTrials.gov NCT04158739 adverse events section.
This phase I trial studies the side effects, best dose of flotetuzumab and how well it works in treating patients with acute myeloid leukemia (AML) that has come back (recurrent) or has not responded to treatment (refractory). This study also determines the safest dose of flotetuzumab to use in children with AML. As an immunotherapy, flotetuzumab may also cause changes in the body's normal immune system, which are also under study in this trial.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing