An Open-Label, Long-term Study of GFB-887 in Patients With Glomerular Kidney Diseases

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)

Study Overview

• Status: Terminated
• Conditions: Kidney Diseases; Glomerulonephritis; Nephritis; Nephrosis; Glomerulosclerosis; Focal Segmental Nephrosis; Lipoid Urologic Disease
• Intervention / Treatment: Drug: GFB-887

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).

• Study Type: Interventional
• Enrollment (Actual): 31
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90022
      • Academic Medical Research Institute (AMRI)
    • Northridge, California, United States, 91324
      • Amicis Research Center
    • Victorville, California, United States, 92395
      • Kidney and Hypertension Center - Apple Valley
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz Medical Center
    • Denver, Colorado, United States, 80230
      • Colorado Kidney Care (Denver Nephrology)
  • Idaho
    • Nampa, Idaho, United States, 83687
      • Boise Kidney and Hypertension Institute
  • Illinois
    • Hinsdale, Illinois, United States, 60521
      • NANI Research, LLC
  • Michigan
    • Roseville, Michigan, United States, 48066
      • St. Clair Nephrology
  • Missouri
    • Kansas City, Missouri, United States, 64111
      • Clinical Research Consultants
  • New York
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai
  • Ohio
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
  • Tennessee
    • Chattanooga, Tennessee, United States, 37404-2743
      • Southeast Renal Research Institute
  • Texas
    • Houston, Texas, United States, 77054
      • Prolato Clinical Research Center
    • San Antonio, Texas, United States, 78212
      • Clinical Advancement Center, PLLC
    • Webster, Texas, United States, 77598
      • Tranquility Research
  • Utah
    • Salt Lake City, Utah, United States, 84115
      • Utah Kidney Center
  • Washington
    • Spokane, Washington, United States, 99204
      • Providence Medical Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: GFB-887; GFB-887 is a potent, small molecule inhibitor of TRPC5.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of adverse events	Incidence and severity of adverse events	Approximately 3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent reduction in urine protein:creatinine ratio (UPCR) from baseline	Percent reduction in urine protein:creatinine ratio (UPCR) from baseline	Approximately 3 years
Proportion of participants achieving modified partial remission status	Proportion of participants achieving modified partial remission status	Approximately 3 years
Proportion of participants achieving complete remission status	Proportion of participants achieving complete remission status	Approximately 3 years
Proportion of participants with a UPCR decrease of at least 30% 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 40% from baseline	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 50% from baseline	Proportion of participants with a UPCR decrease of at least 50% from baseline	Approximately 3 years
Time to maximal percent reduction in UPCR from baseline	Time to maximal percent reduction in UPCR from baseline	Approximately 3 years
Summary of plasma pharmacokinetic (PK) concentrations: Dose proportionality	Dose proportionality of GFB-887	Approximately 3 years
Summary of Plasma PK concentrations (AUCinf)	Area under the plasma concentration-time curve from time zero to infinity	Approximately 3 years
Summary of Plasma PK concentrations (AUClast)	Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration	Approximately 3 years
Summary of Plasma PK concentrations (Cmax)	Maximum observed plasma concentration	Approximately 3 years
Changes in estimated glomerular filtration rate (eGFR) including slope	Glomerular filtration rate will be estimated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation based on serum creatinine	Approximately 3 years

Collaborators and Investigators

• Sponsor: Goldfinch Bio, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 27, 2021
• Primary Completion (Actual): November 2, 2022
• Study Completion (Actual): November 2, 2022

Study Registration Dates

• First Submitted: June 25, 2021
• First Submitted That Met QC Criteria: June 25, 2021
• First Posted (Actual): July 6, 2021

Study Record Updates

• Last Update Posted (Actual): November 15, 2022
• Last Update Submitted That Met QC Criteria: November 10, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Kidney Diseases; Glomerulonephritis; Nephritis; Nephrotic Syndrome; Nephrosis; Urologic Diseases
• Other Study ID Numbers: GFB-887-202

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No