Focal Segmental Nephrosis, Glomerulonephritis, Glomerulosclerosis, Kidney Diseases, Lipoid Urologic Disease, Nephritis, Nephrosis
Conditions
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).
Interventions
DRUGGFB-887
GFB-887 is a potent, small molecule inhibitor of TRPC5.
Sponsors
Goldfinch Bio, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open label study extension
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events | Approximately 3 years | Incidence and severity of adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| Percent reduction in urine protein:creatinine ratio (UPCR) from baseline | Approximately 3 years | Percent reduction in urine protein:creatinine ratio (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 |
| Time to maximal percent reduction in UPCR from baseline | Approximately 3 years | Time to maximal percent reduction in UPCR from baseline |
Countries
United States
Outcome results
None listed