NCT02533362撤回1 期
A Phase 2, Open-Label, Single Center, Efficacy, Safety, Tolerability, and Pharmacokinetic Study of ANF-Rho™ in Patients With Chronic Neutropenia
Prolong Pharmaceuticals0 个研究点开始时间: 2015年10月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 发起方
- 主要终点
- Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia.
研究概览
简要总结
A 6 month safety, tolerability, efficacy and pharmacokinetic study with ANF-Rho to treat patients with chronic neutropenia.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
ANF-Rho
Experimental
pegfilgrastim Anti-Neutropenic Factor (ANF)
干预措施: ANF-Rho (Drug)
结局指标
主要结局
Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia.
时间窗: 6 months
Median Absolute Neutrophil Count (ANC) of ≥1.0 x 109/L over the 6 months total treatment period
次要结局
- Rate of subject reported infection related morbidities (infections, hospitalisations and antibiotic use) in Chronic Neutropenia patients treated with ANF Rho(6 months)
- Evaluate the effect of ANF Rho on the Quality of Life and Bone Pain in Chronic Neutropenia patients.(6 months)
- Serum concentration of ANF-Rho over time(6 months)
- Time to peak plasma concentration of ANF-Rho (Tmax)(6 months)
- The peak plasma concentration of ANF-Rho after administration (Cmax) over time(6 months)
- Total ANF-Rho exposure (AUC)(6 months)
- Evaluate safety and tolerability of subcutaneous injections of ANF Rho in chronic neutropenia patients.(6 months)
- Half-Life of ANF-Rho (T1/2)(6 months)
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