Clinical Trial: ANF-Rho in the Treatment of Chronic Neutropenia

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Phase 2, Open-Label, Single Center, Efficacy, Safety, Tolerability, and Pharmacokinetic Study of ANF-Rho™ in Patients With Chronic Neutropenia

Brief Summary: A 6 month safety, tolerability, efficacy and pharmacokinetic study with ANF-Rho to treat patients with chronic neutropenia.

Detailed Summary: Chronic neutropenia patients who are eligible for participation will be screened and enrolled to received open-label ANF-Rho for a period of 6 months. Study participation will be separated into 2 periods, Induction (8 weeks) and Maintenance (16 weeks). Blood samples to measure neutrophil levels and biochemistry labs will be drawn throughout the study to monitor efficacy response and patient safety. Pharmacokinetic samples will be collected during both periods and questionnaires will be completed by the patient and investigator for bone pain, quality of life (QOL) and injection site reaction.
Sponsor: Prolong Pharmaceuticals

Current Primary Outcome: Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia. [ Time Frame: 6 months ]

Median Absolute Neutrophil Count (ANC) of ≥1.0 x 109/L over the 6 months total treatment period


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Serum concentration of ANF-Rho over time [ Time Frame: 6 months ]
    Pharmacokinetic parameter measuring ANF-Rho levels over time
  • The peak plasma concentration of ANF-Rho after administration (Cmax) over time [ Time Frame: 6 months ]
    Pharmacokinetic Parameter measuring ANF-Rho levels over time
  • Total ANF-Rho exposure (AUC) [ Time Frame: 6 months ]
    Pharmacokinetic parameter measuring ANF-Rho levels from time 0 through last dose.
  • Time to peak plasma concentration of ANF-Rho (Tmax) [ Time Frame: 6 months ]
    Pharmacokinetic parameter measuring the time to Cmax.
  • Half-Life of ANF-Rho (T1/2) [ Time Frame: 6 months ]
    Pharmacokinetic parameter to measure the time required for the concentration of the drug to reach half of its original value
  • Rate of subject reported infection related morbidities (infections, hospitalisations and antibiotic use) in Chronic Neutropenia patients treated with ANF Rho [ Time Frame: 6 months ]
  • Evaluate the effect of ANF Rho on the Quality of Life and Bone Pain in Chronic Neutropenia patients. [ Time Frame: 6 months ]
    Change in Quality of life over 6 months measured by the Short Form 36, change in bone pain over 6 months measured by the Bone Pain Questionnaire, Wong and Baker Scale and FLACC Behavioral Pain Assessment Scale
  • Evaluate safety and tolerability of subcutaneous injections of ANF Rho in chronic neutropenia patients. [ Time Frame: 6 months ]
    Composite endpoint with multiple vital signs, bone scans, biopsies, splenic ultrasounds, electrocardiographic assessments, clinical signs, and bio-analytical measures (e.g., biochemistry), and reported adverse events following repeated dosing.


Original Secondary Outcome: Same as current

Information By: Prolong Pharmaceuticals

Dates:
Date Received: August 10, 2015
Date Started: October 2015
Date Completion: October 2016
Last Updated: August 25, 2015
Last Verified: August 2015