ANF-Rho in the Treatment of Chronic Neutropenia

NCT02533362 | Withdrawn Phase 1 DMC

• 1 other identifier: PGCN-002
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Neutropenia, Severe Chronic

Outcome Measures

Primary Outcomes (1)

• Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia.

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

  6 months

Secondary Outcomes (8)

• Serum concentration of ANF-Rho over time

  6 months

• The peak plasma concentration of ANF-Rho after administration (Cmax) over time

  6 months

• Total ANF-Rho exposure (AUC)

  6 months

• Time to peak plasma concentration of ANF-Rho (Tmax)

  6 months

• Half-Life of ANF-Rho (T1/2)

  6 months

Study Arms (1)

ANF-Rho

EXPERIMENTAL

pegfilgrastim Anti-Neutropenic Factor (ANF)

Drug: ANF-Rho

Interventions

ANF-Rho DRUG

Pegylated version of recombinant human granulocyte-colony stimulating factor (G-CSF)

Also known as: pegfilgrastim ANF

ANF-Rho

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients 1 years of age or older
• Patients with established chronic neutropenia defined as Median Absolute Neutrophil Count (ANC) \< 0.5 x 109/L (both with and without demonstrated genetic lesion) having an indication for treatment, including:
• Independent of hematological parameters, all patients with: Shwachman-Diamond syndrome (SDS), Barth's syndrome or other inherited diseases associated with neutropenia (exclude Glycogen Storage Disease 1b)
• Patients on granulocyte-colony stimulating factor (GCSF) \& PEG-GCSF treatment are still eligible to participate after a washout period of 7 days, after the stopping of the drug
• Signed and dated informed written consent/assent by the patient/parent
• Women of childbearing potential with a negative serum pregnancy test and using a reliable method of contraception during the study period. Male study participants also agreeing to use contraception for the study period.

You may not qualify if:

• Evidence of chromosomal abnormalities, myelodysplasia, hematologic malignancy, aplastic anemia, systemic lupus erythematosus, or rheumatoid arthritis (Felty's syndrome) or if the neutropenia was drug-induced
• Progressive malignant disease or malignancy history
• Presence of macrophage activation syndrome before the diagnosis of neutropenia
• Clinical Significant Abnormal Renal, Cardiac, Hepatic or Blood Coagulation disease.
• Chronic infection such as hepatitis B virus (HBV), hepatitis C Virus (HCV) or Human immunodeficiency virus( HIV) or history of tuberculosis
• Association with anemia, thrombocytopenia (low blood platelets) before the diagnosis of neutropenia.
• Drug abuse, substance abuse, or alcohol abuse
• Use of any other investigational drug at the time of enrollment, or within 5 half-lives prior to enrollment, whichever is longer
• Patients unwilling and/or who are not capable of ensuring compliance with the provisions of the study protocol
• Women who are pregnant or breastfeeding
• Women of childbearing potential who do not use an approved method of birth control. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post ovulation methods) is not acceptable
• Patients with known DNA loss-of-function mutations in GCSFR and RUNX1 genes

Sponsors & Collaborators

• Prolong Pharmaceuticals: lead

MeSH Terms

Conditions

Neutropenia, severe chronic

Study Officials

• Hemant Misra, PhD

  Prolong Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 10, 2015
• First Posted: August 26, 2015
• Study Start: October 1, 2015
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: March 6, 2019

Record last verified: 2015-08