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

Sponsor

Goldfinch Bio, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04950114

Collaborator

(none)

Enrollment

Locations

Arms

49.2

Anticipated Duration (Months)

2.1

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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)

Condition or Disease	Intervention/Treatment	Phase
Kidney Diseases Glomerulosclerosis Focal Segmental Nephrosis Lipoid Urologic Disease Glomerulonephritis Nephritis Nephrosis	Drug: GFB-887	Phase 2

Detailed Description

Participants will be enrolled from the ongoing GFB-887 multiple ascending dose trial. Participants will be maintained on the same 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 Design

Study Type:

Interventional

Anticipated Enrollment :

35 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Open label study extensionOpen label study extension

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Jul 27, 2021

Anticipated Primary Completion Date :

Sep 1, 2025

Anticipated Study Completion Date :

Sep 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 120 mg Dose Cohort Participants who received GFB-887 or placebo at dose level 2 (120 mg) in GFB-887-201 will receive the same daily dose level upon enrollment. Participants who received GFB-887 or placebo at dose level 1 in GFB-887-201 will receive GFB-887 at the dose level 2 (120mg).	Drug: GFB-887 GFB-887 is a potent, small molecule inhibitor of TRPC5.
Experimental: 360 mg Dose Cohort Participants who received GFB-887 or placebo at dose level 3 (360 mg) in GFB-887-201 will receive the same daily dose level upon enrollment	Drug: GFB-887 GFB-887 is a potent, small molecule inhibitor of TRPC5.

Arm

Intervention/Treatment

Experimental: 120 mg Dose Cohort

Participants who received GFB-887 or placebo at dose level 2 (120 mg) in GFB-887-201 will receive the same daily dose level upon enrollment. Participants who received GFB-887 or placebo at dose level 1 in GFB-887-201 will receive GFB-887 at the dose level 2 (120mg).

Drug: GFB-887

GFB-887 is a potent, small molecule inhibitor of TRPC5.

Experimental: 360 mg Dose Cohort

Participants who received GFB-887 or placebo at dose level 3 (360 mg) in GFB-887-201 will receive the same daily dose level upon enrollment

Drug: GFB-887

GFB-887 is a potent, small molecule inhibitor of TRPC5.

Outcome Measures

Primary Outcome Measures

Incidence and severity of adverse events [Approximately 3 years]
Incidence and severity of adverse events

Secondary Outcome Measures

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 30% from baseline [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 40% 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 50% from baseline [Approximately 3 years]
Proportion of participants with a UPCR decrease of at least 50% 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 pharmacokinetic (PK) concentrations: Dose proportionality [Approximately 3 years]
Dose proportionality of GFB-887
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 (AUClast) [Approximately 3 years]
Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration
Summary of Plasma PK concentrations (Cmax) [Approximately 3 years]
Maximum observed plasma concentration
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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

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