Clinical trial NCT04950114: An Open-Label, Long-term Study of GFB-887 in Patients With Glomerular Kidney Diseases
Official title: An Open-Label, Long-term Study of GFB-887 in Patients With Glomerular Kidney Diseases
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Kidney Diseases
Glomerulosclerosis
Focal Segmental Nephrosis
Lipoid Urologic Disease
Glomerulonephritis
Nephritis
Nephrosis
Lead sponsor: Goldfinch Bio, Inc.
Sponsor class: INDUSTRY
First posted: 2021-07-06
Enrollment: 31
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label study extension
Primary purpose: TREATMENT

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).

Eligibility criteria: 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

Primary outcomes:
measure: Incidence and severity of adverse events
timeFrame: Approximately 3 years
Secondary outcomes:
measure: Percent reduction in urine protein:creatinine ratio (UPCR) from baseline
timeFrame: Approximately 3 years
measure: Proportion of participants achieving modified partial remission status
timeFrame: Approximately 3 years
measure: Proportion of participants achieving complete remission status
timeFrame: Approximately 3 years
measure: Proportion of participants with a UPCR decrease of at least 30% from baseline
timeFrame: Approximately 3 years
measure: Proportion of participants with a UPCR decrease of at least 40% from baseline
timeFrame: Approximately 3 years
measure: Proportion of participants with a UPCR decrease of at least 50% from baseline
timeFrame: Approximately 3 years
measure: Time to maximal percent reduction in UPCR from baseline
timeFrame: Approximately 3 years
measure: Summary of plasma pharmacokinetic (PK) concentrations: Dose proportionality
timeFrame: Approximately 3 years
measure: Summary of Plasma PK concentrations (AUCinf)
timeFrame: Approximately 3 years
measure: Summary of Plasma PK concentrations (AUClast)
timeFrame: Approximately 3 years
measure: Summary of Plasma PK concentrations (Cmax)
timeFrame: Approximately 3 years
measure: Changes in estimated glomerular filtration rate (eGFR) including slope
timeFrame: Approximately 3 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Academic Medical Research Institute (AMRI)
state: California
city: Northridge
country: United States
facility: Amicis Research Center
state: California
city: Victorville
country: United States
facility: Kidney and Hypertension Center - Apple Valley
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Center
state: Colorado
city: Denver
country: United States
facility: Colorado Kidney Care (Denver Nephrology)
state: Colorado
city: Nampa
country: United States
facility: Boise Kidney and Hypertension Institute
state: Idaho
city: Hinsdale
country: United States
facility: NANI Research, LLC
state: Illinois
city: Roseville
country: United States
facility: St. Clair Nephrology
state: Michigan
city: Kansas City
country: United States
facility: Clinical Research Consultants
state: Missouri
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Chattanooga
country: United States
facility: Southeast Renal Research Institute
state: Tennessee
city: Houston
country: United States
facility: Prolato Clinical Research Center
state: Texas
city: San Antonio
country: United States
facility: Clinical Advancement Center, PLLC
state: Texas
city: Webster
country: United States
facility: Tranquility Research
state: Texas
city: Salt Lake City
country: United States
facility: Utah Kidney Center
state: Utah
city: Spokane
country: United States
facility: Providence Medical Research Center
state: Washington
Study officials:
References: