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Randomized Phase 2, Dose-finding Efficacy, Safety Study of ANF-RHO™ Versus Neulasta® in Chemotherapy-Induced Neutropenia

Phase 2
Terminated
Conditions
Chemotherapy-induced Neutropenia
Interventions
Drug: ANF-RHO™
Registration Number
NCT03559387
Lead Sponsor
Prolong Pharmaceuticals
Brief Summary

Randomized, Open-Label study to determine the dose, efficacy, safety and pharmacokinetic profile of ANF-RHO™ with once-per-cycle injection in comparison with Neulasta in Breast Cancer patients at high risk of developing Chemotherapy-Induced Neutropenia

Detailed Description

Forty Eight (48) adult female, chemotherapy treatment-naïve, stage I to III, breast cancer patients scheduled to receive FEC100 (3 cycles)/docetaxel (3 cycles) myelosuppressive chemotherapy will be enrolled into the study after they meet all the inclusion/exclusion criteria. Four (4) Cohorts of 12 patients each will be studied; they will be randomized either in the ANF-RHO™ treatment arm at different doses (10 μg/kg, 20 μg/kg, or 30 μg/kg, for the cohorts 1-3, respectively) or Neulasta® (6 mg / 0.6 ml SC injection, for cohort 4). For the cohorts 1-3, 12 patients from each of the respective ANF-RHO™ cohorts will be randomized with four patients from cohort 4 (Neulasta®). Patients in the ANF-RHO™ cohorts will receive study drug on Day 1 of each Chemotherapy cycle. Patients in the Neulasta® cohort will receive study drug on Day 2 of each Chemotherapy cycle. Doses of ANF-RHO™/Neulasta® will be provided for a total of 6 cycles (21 days each). The total duration of the study 129 ± 2 days (126 Days of treatment period followed by end of study visit)

Recruitment & Eligibility

Status
TERMINATED
Sex
Female
Target Recruitment
9
Inclusion Criteria
  1. Adult female patients, 18 years of age or older

  2. Signed and dated written consent/assent by the patient or legally authorized representative

  3. Histologically confirmed non-metastatic breast cancer

  4. ECOG performance status ≤ 2

  5. Myelosuppressive chemotherapy naive

  6. Scheduled to receive and anticipated to complete the following chemotherapy regimen

    1. FEC (fluorouracil/epirubicin (100) / cyclophosphamide) (3 cycles);
    2. Docetaxel (3 cycles) chemotherapy
  7. White blood cells (WBC) ≥ 3 × 10^9/L; Absolute neutrophil count (ANC) ≥ 2.0 × 10^9/L; platelet count ≥ 100 × 10^9/L; and hemoglobin ≥ 10 g/dL (6.2 mmol/L)

  8. Adequate cardiac function (e.g. LVEF > 50% as determined by standard care) and adequate hepatic function (e.g. liver transaminases < 2.5 x ULN)

  9. Women of childbearing potential with a negative serum pregnancy test and using a highly effective method of birth control (i.e. one that results in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives and intrauterine devices (IUDs)). Periodic abstinence is not an acceptable contraceptive method during the study period.

Exclusion Criteria

A subject will not be enrolled if any they meet any of the following criteria:

  1. Known hypersensitivity to E.coli derived products or polyethylene glycol
  2. No other malignancy except carcinoma in situ and basal-cell and squamous cell carcinoma of the skin, unless the other malignancy was treated ≥ 5 years ago with curative intent
  3. Evidence of myelodysplasia, aplastic anemia, myelofibrosis, rheumatoid arthritis, systemic lupus erythematosus, or sickle cell disease
  4. Clinical diagnosis or history of chronic infection such as hepatitis B virus (HBV), hepatitis C virus (HCV) or Human immunodeficiency virus (HIV) or history of tuberculosis
  5. Previous exposure to filgrastim, perfilgrastim or lipegfilgrastim within 30 days before randomization
  6. Treatment with systemically active antibiotics within 72 hours before chemotherapy
  7. Chronic use of oral corticosteroids
  8. Participation in a pharmacological clinical trial within 30 days before randomization
  9. Clinical diagnosis of drug abuse or substance abuse within 30 days prior to screening
  10. Documented alcohol abuse within 30 days prior to screening
  11. Unwilling and/or not capable of ensuring compliance with the provisions of the study protocol
  12. Pregnant or breastfeeding women where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive serum HCG laboratory test
  13. Other serious medical condition that would prevent individual from receiving protocol treatment

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
ANF-RHO™ANF-RHO™Subjects will receive the ANF-RHO™ dose with a volume equivalent to 10 µg/kg, 20 µg/kg and 30 µg/kg as a subcutaneous injection.
Neulasta®Neulasta®Neulasta® will be administered to the subjects at a dose of 6.0 mg in 0.6 ml as a subcutaneous injection.
Primary Outcome Measures
NameTimeMethod
Duration of neutropenia grade 1 or worse (absolute neutrophil count [ANC] ≤ 2.0 x 10^9/L) in the first cycle of chemotherapy (FE100C).21 days
Secondary Outcome Measures
NameTimeMethod
Incidence and duration of infection and infection-related events based on use of antibiotics during all chemotherapy cycles~ 128 ± 2 days
Clinically meaningful changes in vital signs during all chemotherapy cycles - Assessment of Blood pressure~ 128 ± 2 days

Assessment of Blood pressure (systolic and diastolic) helps determine the safety of the medications under study - ANF-RHO and Neulasta

Clinically meaningful changes in vital signs during all chemotherapy cycles - Assessment of Heart rate~ 128 ± 2 days

Assessment of Heart rate helps determine the safety of the medications under study - ANF-RHO and Neulasta

Incidence of bone pain, determined by a numerical rating scale, as well as other reported adverse events~ 128 ± 2 days
Pharmacokinetic profile of ANF-RHO and Neulasta - Measurement of Time taken to reach the maximum concentration (Tmax)~ 128 ± 2 days

Tmax is the time taken to reach the maximum concentration (Cmax) after administration of a dose.

Duration of severe neutropenia (ANC < 0.5 x 10^9/L) during the first chemotherapy cycle (21-day cycle FE100C)21 days
Duration of severe neutropenia (ANC < 0.5 x 10^9/L) during the fourth chemotherapy cycle (21-day cycle docetaxel)21 days
Incidence of severe neutropenia (ANC < 0.5 x 10^9/L) during all chemotherapy cycles~ 128 ± 2 days
Incidence and duration of moderate (ANC ≥ 50 x 10^9/L) leukocytosis during all chemotherapy cycles~ 128 ± 2 days
Clinically meaningful changes in vital signs during all chemotherapy cycles - Assessment of Respiratory rate~ 128 ± 2 days

Assessment of Respiratory rate helps determine the safety of the medications under study - ANF-RHO and Neulasta

Pharmacokinetic profile of ANF-RHO and Neulasta - Measurement of Maximum Plasma Concentration (Cmax)~ 128 ± 2 days

Cmax is the maximum concentration of the drug (either ANF-RHO or Neulasta) that is achieved after administration of a dose

Duration of neutropenia grade 1 or worse (absolute neutrophil count [ANC] ≤ 2.0 x 10^9/L) in the fourth cycle of chemotherapy (docetaxel).21 days
Incidence and duration of febrile neutropenia defined as peak temperature ≥38.0°C for two readings over two hours and ANC < 0.5 x 10^9/L, during all chemotherapy cycles~ 128 ± 2 days
Incidence and duration of infection and infection-related events based on the need for hospitalization during all chemotherapy cycles~ 128 ± 2 days
Incidence and duration of severe (ANC ≥ 100 x 10^9/L) leukocytosis during all chemotherapy cycles~ 128 ± 2 days
Clinically meaningful changes in vital signs during all chemotherapy cycles - Assessment of Body Temperature~ 128 ± 2 days

Assessment of Respiratory rate helps determine the safety of the medications under study - ANF-RHO and Neulasta

Duration of bone pain, determined by a numerical rating scale, as well as other reported adverse events~ 128 ± 2 days
Incidence of anti-drug antibodies to ANF-RHO and Neulasta~ 128 ± 2 days
Incidence and duration of febrile neutropenia defined as peak temperature ≥38.5°C and ANC < 0.5 x 10^9/L, during all chemotherapy cycles~ 128 ± 2 days
Severity of bone pain, determined by a numerical rating scale, as well as other reported adverse events~ 128 ± 2 days
Site of bone pain, determined by a numerical rating scale, as well as other reported adverse events~ 128 ± 2 days
Pharmacokinetic profile of ANF-RHO and Neulasta - Measurement of half-life (T1/2)~ 128 ± 2 days

T1/2 is the time taken for ANF-RHO and Neulasta to reach half the value of their initial concentrations. T1/2 determination helps in understanding the duration for which ANF-RHO or Neulasta would be active upon administration.

Pharmacokinetic profile of ANF-RHO and Neulasta - Area Under the Curve (AUC[0-∞])~ 128 ± 2 days

Similar to AUC\[0-t\] which is estimated till time 't', in AUC\[0-∞\], the estimation is done on the concentration of the drug to an infinite time. Calculation of the AUC\[0-∞\], helps understand how much of the drug is available in the body at extremely low concentrations, beyond the limits of measurable concentrations.

Pharmacokinetic profile of ANF-RHO and Neulasta - Area Under the Curve (AUC[0-t])~ 128 ± 2 days

AUC\[0-t\] is an important parameter for determining as to how much of the drug is available in the body after administration of a drug formulation, in this case ANF-RHO vs Neulasta. Understanding the AUC for both these drug formulations will help us to determine the efficacy and safety profiles of these medicines.

Pharmacokinetic profile of ANF-RHO and Neulasta - Area Under the Curve (AUC[0-t]/AUC[0-∞])~ 128 ± 2 days

The parameter AUC\[0-t\]/AUC\[0-∞\] is useful to calculate the fraction of the Total AUC that was added due to the extrapolated AUC (AUC\[0-∞\]). It helps understand if the methods employed for determining the total AUC and drug's availability in the body are correct.

Trial Locations

Locations (8)

Institut de cancérologie Jean Godinot

🇫🇷

Reims, France

Hôpital Saint Louis - Center des Maladies du Sein

🇫🇷

Paris, France

Strasbourg Oncologie Libérale

🇫🇷

Strasbourg, France

Erasmus Medical Center

🇳🇱

Rotterdam, Netherlands

CHU de Tours

🇫🇷

Tours, France

Ikazia Ziekenhuis

🇳🇱

Rotterdam, Netherlands

Maasstad Ziekenhuis

🇳🇱

Rotterdam, Netherlands

Franciscus Gasthuis & Vlietland

🇳🇱

Schiedam, Netherlands

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