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Liposome-encapsulated Daunorubicin-Cytarabine, Fludarabine Phosphate, Cytarabine, and Filgrastim in Treating Younger Patients With Relapsed or Refractory Acute Myeloid Leukemia
Phase 1, PHASE2 trial testing Cytarabine in Acute Myeloid Leukemia Post Cytotoxic Therapy in 38 participants. Completed in 30 June 2023.
| Lead sponsor | Children's Oncology Group |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 38 |
| Start date | 2 May 2016 |
| Primary completion | 31 December 2018 |
| Estimated completion | 30 June 2023 |
| Sites | 73 locations across Canada, United States |
Children's Oncology Group — full company profile →
Adults 1 to 21, any sex, with Acute Myeloid Leukemia Post Cytotoxic Therapy or Recurrent Childhood 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 of patients in the dose-finding phase with a dose-limiting toxicity, graded using the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 1 |
Best response (complete response or complete remission with partial platelet recovery) after up to 2 cycles of therapy, where response is assessed using the revised Acute Myeloid Leukemia International Working Group Criteria. Percentage of responders is calculated using the methods of Jung and Kim. 90% confidence interval is determined using the methods of Koyama and Chen.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 68.30 | 52.89 – 78.02 |
Response (complete response or complete remission with partial or incomplete platelet recovery) after first cycle of therapy, where response is assessed using the revised Acute Myeloid Leukemia International Working Group Criteria. Percentage of responders is calculated by the total of number of patients with complete response or complete remission with partial or incomplete platelet recovery divided by the number of patients evaluable for response after the first cycle. 95% confidence interval is determined using a binomial exact method.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 75.68 | 58.80 – 88.23 |
Geometric mean liposome-encapsulated daunorubicin clearance following IV infusion will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 94.7 | 47.5 – 193.0 |
Geometric mean liposome-encapsulated daunorubicin volume of distribution following IV infusion will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 3827.7 | 2165.0 – 6596.0 |
Median liposome-encapsulated daunorubicin time of maximum observed plasma concentration will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 2 | 1.42 – 2.07 |
Geometric mean liposome-encapsulated daunorubicin area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 1288010.3 | 761251.0 – 1715266.0 |
Geometric mean liposome-encapsulated cytarabine clearance following IV infusion will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 71.76 | 37.5 – 151.0 |
Geometric mean liposome-encapsulated cytarabine volume of distribution following IV infusion will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 4158.0 | 2642.0 – 7081.0 |
Median liposome-encapsulated cytarabine time of maximum observed plasma concentration will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 5 | 1.92 – 5.07 |
Geometric mean liposome-encapsulated cytarabine area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration will be determined for patients in the dose-finding phase.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (CPX-351 and FLAG) | 4418582.5 | 2765108.0 – 6382600.0 |
Time frame: While patients were on Protocol Therapy (up to 8 weeks) or during follow-up (up to 3 years).. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Treatment (CPX-351 and FLAG) |
|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | — |
| Infections and infestations - Other, specify | Infections and infestations | — |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | — |
| Death NOS | General disorders | — |
| Typhlitis | Gastrointestinal disorders | — |
| Fever | General disorders | — |
| Lung infection | Infections and infestations | — |
| Hyperglycemia | Metabolism and nutrition disorders | — |
| Dysphagia | Gastrointestinal disorders | — |
| Gastrointestinal disorders - Other, specify | Gastrointestinal disorders | — |
| Mucositis oral | Gastrointestinal disorders | — |
| Anorectal infection | Infections and infestations | — |
| Catheter related infection | Infections and infestations | — |
| Kidney infection | Infections and infestations | — |
| Skin infection | Infections and infestations | — |
| Small intestine infection | Infections and infestations | — |
| Soft tissue infection | Infections and infestations | — |
| Blood bilirubin increased | Investigations | — |
| Ejection fraction decreased | Investigations | — |
| Lymphocyte count decreased | Investigations | — |
| White blood cell decreased | Investigations | — |
| Anorexia | Metabolism and nutrition disorders | — |
| Headache | Nervous system disorders | — |
| Nervous system disorders - Other, specify | Nervous system disorders | — |
| Syncope | Nervous system disorders | — |
| Reaction | System | Treatment (CPX-351 and FLAG) |
|---|---|---|
| Electrocardiogram QT corrected interval prolonged | Investigations | — |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — |
| Hypokalemia | Metabolism and nutrition disorders | — |
| General disorders and administration site conditions - Other, specify | General disorders | — |
| Alanine aminotransferase increased | Investigations | — |
| Infections and infestations - Other, specify | Infections and infestations | — |
| Skin infection | Infections and infestations | — |
| Aspartate aminotransferase increased | Investigations | — |
| Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | — |
| Enterocolitis infectious | Infections and infestations | — |
| Mucosal infection | Infections and infestations | — |
| Upper respiratory infection | Infections and infestations | — |
| Ejection fraction decreased | Investigations | — |
| GGT increased | Investigations | — |
| Anorexia | Metabolism and nutrition disorders | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — |
| Anemia | Blood and lymphatic system disorders | — |
| Ventricular arrhythmia | Cardiac disorders | — |
| Rectal pain | Gastrointestinal disorders | — |
| Multi-organ failure | General disorders | — |
| Device related infection | Infections and infestations | — |
| Periorbital infection | Infections and infestations | — |
| Investigations - Other, specify | Investigations | — |
| Platelet count decreased | Investigations | — |
| Hyperglycemia | Metabolism and nutrition disorders | — |
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | — |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | — |
| Respiratory, thoracic and mediastinal disorders - Other, specify | Respiratory, thoracic and mediastinal disorders | — |
| Skin and subcutaneous tissue disorders - Other, specify | Skin and subcutaneous tissue disorders | — |
| Hypotension | Vascular disorders | — |
Most-reported serious reactions: Febrile neutropenia, Infections and infestations - Other, specify, Rash maculo-papular, Death NOS, Typhlitis, Fever, Lung infection, Hyperglycemia.
Data from ClinicalTrials.gov NCT02642965 adverse events section.
This phase I/II trial studies the side effects and best dose of liposome-encapsulated daunorubicin-cytarabine when given with fludarabine phosphate, cytarabine, and filgrastim and to see how well they work in treating younger patients with acute myeloid leukemia that has come back after treatment (relapsed) or is not responding to treatment (is refractory). Liposome-encapsulated daunorubicin-cytarabine is made up of two chemotherapy drugs, cytarabine and daunorubicin hydrochloride, and works to stop cancer cell growth by blocking the cells from dividing. Drugs used in chemotherapy, such as fludarabine phosphate and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Filgrastim may increase the production of blood cells and may help the immune system recover from the side effects of chemotherapy. Giving liposome-encapsulated daunorubicin-cytarabine followed by fludarabine phosphate, cytarabine, and filgrastim may be a better treatment for patients with relapsed acute myeloid leukemia and may cause fewer side effects to the heart, a common effect of other chemotherapy treatments for acute myeloid leukemia.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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