An Open-Label, Long-term Study of GFB-887 in Patients With Glomerular Kidney Diseases
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 31
- 试验地点
- 17
- 主要终点
- Incidence and severity of adverse events
研究概览
简要总结
This is an open-label Phase 2 study evaluating the long term safety and tolerability of GFB-887 in patients with focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD)
详细描述
Participants will be enrolled from the ongoing GFB-887 multiple ascending dose trial. Participants will be transitioned to a 200 mg QD dose level regardless of the dose received in the previous study although the data review team (DRT) and Medical Monitor may elect to decrease or increase the dose to minimize adverse events or improve clinical efficacy. The DRT may also elect to change dosing levels due to emerging data on GFB-887. Participants will take GFB-887 once daily at home. A phone visit will be conducted at Week 4 and at Week 8 to assess safety and tolerability. Participants will return to the clinic for follow up visits at Weeks 12, 24, 36, 48, and every 24 weeks thereafter through approximately 3 years from the time of the participant's first dose to evaluate long-term safety and durability of response (for up to approximately 13 scheduled in-clinic visits).
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with FSGS/TR-MCD who have completed the treatment phase from an interventional clinical study with GFB-
- •Participants who were discontinued for rising proteinuria from a GFB-887 interventional study may be considered for enrollment following consultation with the Medical Monitor.
- •Participants who enrolled in any other interventional study during the time between completion of the prior GFB-887 interventional study and this study may be considered for enrollment following consultation with the Medical Monitor.
排除标准
- •Participant is unable to take oral medications
- •Participant has an unstable medical condition based on medical history, physical examination, laboratory tests, ECGs, vital signs or is otherwise unstable in the judgement of the Investigator which would pose a risk to the participant or interfere with study evaluation, procedures, or completion
- •Evidence of significant hypersensitivity, intolerance, or allergy to any component of investigational product GFB-887
研究组 & 干预措施
200 mg Dose Cohort
Participants who received GFB-887 or placebo in GFB-887-201 will receive GFB-887 at a daily dose level of 200 mg regardless of original dose level.
干预措施: GFB-887 (Drug)
结局指标
主要结局
Incidence and severity of adverse events
时间窗: Approximately 3 years
Incidence and severity of adverse events
次要结局
- Summary of plasma pharmacokinetic (PK) concentrations: Dose proportionality(Approximately 3 years)
- Changes in estimated glomerular filtration rate (eGFR) including slope(Approximately 3 years)
- Proportion of participants with a UPCR decrease of at least 50% from baseline(Approximately 3 years)
- Proportion of participants with a UPCR decrease of at least 30% from baseline(Approximately 3 years)
- Summary of Plasma PK concentrations (AUClast)(Approximately 3 years)
- Percent reduction in urine protein:creatinine ratio (UPCR) from baseline(Approximately 3 years)
- Proportion of participants achieving modified partial remission status(Approximately 3 years)
- Proportion of participants achieving complete remission status(Approximately 3 years)
- Proportion of participants with a UPCR decrease of at least 40% from baseline(Approximately 3 years)
- Time to maximal percent reduction in UPCR from baseline(Approximately 3 years)
- Summary of Plasma PK concentrations (AUCinf)(Approximately 3 years)
- Summary of Plasma PK concentrations (Cmax)(Approximately 3 years)