An Open-Label, Long-term Study of GFB-887 in Patients With Glomerular Kidney Diseases
- Conditions
- NephritisNephrosisFocal Segmental NephrosisGlomerulonephritisKidney DiseasesGlomerulosclerosisLipoid Urologic Disease
- Interventions
- Registration Number
- NCT04950114
- Lead Sponsor
- Goldfinch Bio, Inc.
- Brief Summary
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)
- Detailed Description
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).
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 31
- Participants with FSGS/TR-MCD who have completed the treatment phase from an interventional clinical study with GFB-887. 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.
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description 200 mg Dose Cohort GFB-887 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.
- Primary Outcome Measures
Name Time Method Incidence and severity of adverse events Approximately 3 years Incidence and severity of adverse events
- Secondary Outcome Measures
Name Time Method Summary of plasma pharmacokinetic (PK) concentrations: Dose proportionality Approximately 3 years Dose proportionality of GFB-887
Changes in estimated glomerular filtration rate (eGFR) including slope Approximately 3 years Glomerular filtration rate will be estimated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation based on serum creatinine
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 50% from baseline
Proportion of participants with a UPCR decrease of at least 30% from baseline Approximately 3 years Proportion of participants with a UPCR decrease of at least 30% from baseline
Summary of Plasma PK concentrations (AUClast) Approximately 3 years Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration
Percent reduction in urine protein:creatinine ratio (UPCR) from baseline Approximately 3 years Percent reduction in urine protein:creatinine ratio (UPCR) from baseline
Proportion of participants achieving modified partial remission status Approximately 3 years Proportion of participants achieving modified partial remission status
Proportion of participants achieving complete remission status Approximately 3 years Proportion of participants achieving complete remission status
Proportion of participants with a UPCR decrease of at least 40% from baseline Approximately 3 years Proportion of participants with a UPCR decrease of at least 40% from baseline
Time to maximal percent reduction in UPCR from baseline Approximately 3 years Time to maximal percent reduction in UPCR from baseline
Summary of Plasma PK concentrations (AUCinf) Approximately 3 years Area under the plasma concentration-time curve from time zero to infinity
Summary of Plasma PK concentrations (Cmax) Approximately 3 years Maximum observed plasma concentration
Trial Locations
- Locations (17)
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Southeast Renal Research Institute
🇺🇸Chattanooga, Tennessee, United States
Academic Medical Research Institute (AMRI)
🇺🇸Los Angeles, California, United States
Kidney and Hypertension Center - Apple Valley
🇺🇸Victorville, California, United States
Amicis Research Center
🇺🇸Northridge, California, United States
St. Clair Nephrology
🇺🇸Roseville, Michigan, United States
University of Colorado Anschutz Medical Center
🇺🇸Aurora, Colorado, United States
Colorado Kidney Care (Denver Nephrology)
🇺🇸Denver, Colorado, United States
NANI Research, LLC
🇺🇸Hinsdale, Illinois, United States
Boise Kidney and Hypertension Institute
🇺🇸Nampa, Idaho, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
Clinical Research Consultants
🇺🇸Kansas City, Missouri, United States
Prolato Clinical Research Center
🇺🇸Houston, Texas, United States
Tranquility Research
🇺🇸Webster, Texas, United States
Clinical Advancement Center, PLLC
🇺🇸San Antonio, Texas, United States
Utah Kidney Center
🇺🇸Salt Lake City, Utah, United States
Providence Medical Research Center
🇺🇸Spokane, Washington, United States