Study of the efficacy, safety, tolerability and drug blood levlrs of ANF-RHO to treat patients with low white blood cells count

Phase 1

• Conditions: MedDRA version: 20.0Level: LLTClassification code 10066702Term: Chronic neutropeniaSystem Organ Class: 100000013471; Chronic neutropenia; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-004539-18-FR
• Lead Sponsor: Prolong Pharmaceuticals, LLC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-07-03
• Last Update Posted: 15 October 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

1) Patients 12 years of age or older
2) Patients with established Chronic, cyclic or idiopathic severe neutropenia defined as Median ANC < 0.5 x 109/L at diagnosis (both with and without demonstrated genetic lesion) having an indication for treatment by GCSF. Patients on GCSF & PEG-GCSF treatment are still eligible to participate after a washout period of 7 days (after GCSF) or 14 days after PEG-GSCF, after the stopping of the drug
3) Signed and dated informed written consent/assent by the patient/parent
4) 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

Are the trial subjects under 18? yes
Number of subjects for this age range: 3
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

A subject will not be enrolled if they meet any of the following criteria:
1) 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
2) Progressive malignant disease or malignancy history
3) Presence of macrophage activation syndrome before the diagnosis of neutropenia
4) Presence of a permanent proteinuria before the diagnosis of neutropenia
5) Clinical Significant Abnormal Renal, Cardiac, Hepatic Function or Blood Coagulation
6) Patient has a chronic infection such as hepatitis B virus(HBV), hepatitis C Virus (HCV) or Human immunodeficiency virus( HIV) or history of tuberculosis
7) Association with anemia <8 g/dL or thrombocytopenia (except iron deficiency anemia inflammatory except Shwachman syndrome) before the diagnosis of neutropenia
8) Drug abuse, substance abuse, or alcohol abuse
9) Use of any other investigational drug at the time of enrollment, or within 5 half-lives prior to enrollment, whichever is longer
10) Patients unwilling and/or who are not capable of ensuring compliance with the provisions of the study protocol
11) 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
12) Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are 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 (e.g., calendar, ovulation, symptothermal, post ovulation methods) is not acceptable
13) For patients with glycogen storage disease and G6PC3 neutropenia, Pulmonary hypertension should be determined by ECHO and if positive patient is not eligible for the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified