A Study of TRPC5 Channel Inhibitor in Patients With Diabetic Nephropathy, Focal Segmental Glomerulosclerosis, and Treatment-Resistant Minimal Change Disease

Phase 2

Terminated

• Conditions: Diabetes Complications; Glomerulonephritis; Kidney Diseases; Glomerulosclerosis, Focal Segmental; Nephrosis, Lipoid; Urologic Diseases; Nephrosis; Diabetic Nephropathies; Diabetes Mellitus; Endocrine System Diseases
• Interventions: Drug: GFB-887; Drug: Placebo

• Registration Number: NCT04387448
• Lead Sponsor: Goldfinch Bio, Inc.

Brief Summary

This is a phase 2a study evaluating the safety and tolerability of multiple ascending doses of GFB-887 in patients with diabetic nephropathy (DN), focal segmental glomerulosclerosis (FSGS), and treatment-resistant minimal change disease (TR-MCD).

Detailed Description

Approximately 125 patients will be enrolled in this study across the United States. Patients with DN and FSGS/TR-MCD will be randomized in 3 ascending dose cohorts to receive either GFB-887 or placebo.

Study Timeline

• Study First Submitted: 2020-05-11
• Study First Submitted QC: 2020-05-11
• Study First Posted: 2020-05-13
• Study Start: 2020-07-28
• Status Verified: 2022-11
• Primary Completion: 2022-11-01
• Study Completion: 2022-11-01
• Last Update Submitted: 2022-11-10
• Last Update Posted: 2022-11-15

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 96

Inclusion Criteria

• All patients:

  1. Male or female 18-75 years of age, of any race, at the time of signing informed consent.
  2. Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 at Screening.
  3. Currently receiving an angiotensin-converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB).

• For DN patients:

  1. Diagnosis of type 2 diabetes with glycated hemoglobin (HbA1c) level ≤11% at Screening.
  2. UACR ≥ 150 mg/g.

• For FSGS/TR-MCD patients:

  1. Diagnosis of FSGS based on either biopsy or genetic testing or TR-MCD based on biopsy.
  2. UPCR ≥ 1.0 g/g.

Exclusion Criteria

• All patients:

  1. Evidence of another (non-DN, non-FSGS/TR-MCD, respectively) kidney disease.
  2. History of malignancy, unless in remission for at least 5 years other than adequately treated basal cell or squamous cell skin cancer, cervical carcinoma in situ, or prostate cancer not expected to require treatment over the course of the study.
  3. History of any organ or bone marrow transplant, including kidney grafts.
  4. History of alcoholism or drug/chemical abuse within 12 months prior to Screening.

• For DN patients:

  1. Renal disease that requires immunosuppressive therapy (currently, or in the past).
  2. Body mass index (BMI) >45 kg/m2.

• For FSGS/TR-MCD patients:

  1. Currently on calcineurin inhibitors or history of resistance to calcineurin inhibitors.
  2. Body mass index (BMI) >40 kg/m2.
  3. Known history of severe or chronic hepatobiliary disease.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GFB-887 multiple ascending dose (MAD) active	GFB-887	GFB-887 active once-daily dosing
GFB-887 MAD placebo	Placebo	GFB-887 placebo once-daily dosing

Primary Outcome Measures

Name	Time	Method
Percentage change in Urine Albumin-to-Creatinine Ratio (UACR)	12 weeks
Percentage change in Urine Protein-to-Creatinine Ratio (UPCR)	12 weeks

Secondary Outcome Measures

Name	Time	Method
Proportion of FSGS/TR-MCD patients achieving a modified partial remission	12 weeks
Percentage change in 24-hour urine protein excretion	12 weeks
Percentage change in 24-hour urine albumin excretion	12 weeks
Proportion of patients (DN or FSGS/TR-MCD) with a UACR/UPCR decrease of at least 50% of baseline	12 weeks
Proportion of FSGS/TR-MCD patients achieving a complete remission	12 weeks
Incidence and severity of adverse events	12 weeks
Incidence of clinically significant changes in 12-lead electrocardiogram (ECG) parameters, vital signs measurements, and physical examinations	Approximately 12 weeks
Proportion of patients (DN or FSGS/TR-MCD) with a UACR/UPCR decrease of at least 30% of baseline	12 weeks
Incidence of clinically significant changes in laboratory parameters	12 weeks
Plasma PK parameters: area under the plasma concentration-time curve (AUC)	12 weeks
Proportion of patients (DN or FSGS/TR-MCD) with a UACR/UPCR decrease of at least 40% of baseline	12 weeks
Plasma pharmacokinetics (PK) parameters: maximum observed plasma concentration (Cmax)	12 weeks
Plasma PK parameters: time of the observed plasma concentration (Tmax)	12 weeks

Trial Locations

Locations (72)

University of Mississippi Medical Center; 🇺🇸 Jackson, Mississippi, United States

North American Research Institute; 🇺🇸 San Dimas, California, United States

DaVita Hartford North; 🇺🇸 Hartford, Connecticut, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Kidney & Hypertension Specialists of Miami; 🇺🇸 Miami, Florida, United States

University of Minnesota - Center for Pediatric Obesity Medicine; 🇺🇸 Minneapolis, Minnesota, United States

DaVita Pelican Point Dialysis; 🇺🇸 Las Vegas, Nevada, United States

Wayne State University School of Medicine; 🇺🇸 Detroit, Michigan, United States

Utah Kidney Research Institute; 🇺🇸 Salt Lake City, Utah, United States

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Aventiv Research - Phoenix; 🇺🇸 Mesa, Arizona, United States

Arizona Kidney Disease & Hypertension Centers (AKDHC); 🇺🇸 Scottsdale, Arizona, United States

Academic Medical Research Institute (AMRI); 🇺🇸 Glendale, California, United States

Amicis Research Center; 🇺🇸 Vacaville, California, United States

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

Valley Renal Medical Group; 🇺🇸 Northridge, California, United States

Respire Research - Palm Springs; 🇺🇸 Palm Springs, California, United States

Dr. Malvin Yan Inc.; 🇺🇸 S. Gate, California, United States

Valiance Clinical Research - Tarzana; 🇺🇸 Tarzana, California, United States

DaVita Mojave Sage Dialysis; 🇺🇸 Victorville, California, United States

Colorado Kidney Care (Denver Nephrology); 🇺🇸 Denver, Colorado, United States

Western Nephrology - Westminster; 🇺🇸 Wheat Ridge, Colorado, United States

Prohealth Research Center - Doral; 🇺🇸 Doral, Florida, United States

Premier Clinical Research Institute; 🇺🇸 Miami, Florida, United States

Florida Premier Research Institute - Clay Street; 🇺🇸 Winter Park, Florida, United States

Research by Design, LLC; 🇺🇸 Chicago, Illinois, United States

Adventist Health Partners; 🇺🇸 Hinsdale, Illinois, United States

University of Iowa College of Public Health; 🇺🇸 Iowa City, Iowa, United States

Southern Illinois University School of Medicine; 🇺🇸 Springfield, Illinois, United States

My Kidney Center, LLC; 🇺🇸 Manhattan, Kansas, United States

Louisiana Kidney Update, LLC; 🇺🇸 Lafayette, Louisiana, United States

Tufts Medical Center; 🇺🇸 Boston, Massachusetts, United States

Renal and Transplant Associates of New England, PC; 🇺🇸 Springfield, Massachusetts, United States

DaVita Clinical Research; 🇺🇸 Edina, Minnesota, United States

St. Clair Nephrology; 🇺🇸 Roseville, Michigan, United States

Nephrology & Hypertension Associates Ltd; 🇺🇸 Tupelo, Mississippi, United States

Clinical Research Consultants; 🇺🇸 Kansas City, Missouri, United States

Mount Sinai Medical Center; 🇺🇸 New York, New York, United States

Columbia University Medical Center; 🇺🇸 New York, New York, United States

Akron Nephrology Associates, Inc.; 🇺🇸 Akron, Ohio, United States

Eastern Nephrology Associates PLLC; 🇺🇸 New Bern, North Carolina, United States

Northeast Clinical Research Center; 🇺🇸 Bethlehem, Pennsylvania, United States

South Carolina Nephrology and Hypertension; 🇺🇸 Orangeburg, South Carolina, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Texas Tech University Health Sciences Center - Amarillo; 🇺🇸 Amarillo, Texas, United States

Arlington Nephrology, PC; 🇺🇸 Arlington, Texas, United States

Conroe Willis Medical Research; 🇺🇸 Conroe, Texas, United States

Renal Disease Research Institute; 🇺🇸 Dallas, Texas, United States

El Paso Kidney Specialists, PA; 🇺🇸 El Paso, Texas, United States

Xpress Trials LLC; 🇺🇸 Houston, Texas, United States

Prolato Clinical Research Center; 🇺🇸 Houston, Texas, United States

North Texas Kidney Disease Association; 🇺🇸 Lewisville, Texas, United States

Clinical Advancement Center, PLLC; 🇺🇸 San Antonio, Texas, United States

San Antonio Kidney Disease Center Physicians Group, PLLC; 🇺🇸 San Antonio, Texas, United States

Clear Lake Specialties; 🇺🇸 Webster, Texas, United States

Tidewater Kidney Specialists - Riverview; 🇺🇸 Norfolk, Virginia, United States

Providence Medical Research Center; 🇺🇸 Spokane, Washington, United States

Clinical Site Partners Leesburg, LLC; 🇺🇸 Leesburg, Florida, United States

Avanza Medical Research Center; 🇺🇸 Pensacola, Florida, United States

Coastal Nephrology Associates Research Center, LLC; 🇺🇸 Port Charlotte, Florida, United States

Masters of Clinical Research, Inc.; 🇺🇸 Augusta, Georgia, United States

Inova Clinical Trials and Research Center; 🇺🇸 Tyrone, Georgia, United States

The Ohio State University Wexner Medical Center; 🇺🇸 Columbus, Ohio, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Sierra Nevada Nephrology Consultants; 🇺🇸 Reno, Nevada, United States

Louisiana State University Health Sciences Center - School of Medicine - New Orleans; 🇺🇸 New Orleans, Louisiana, United States

Nephrology Associationes, P.A.; 🇺🇸 Newark, Delaware, United States

Boise Kidney and Hypertension Institute; 🇺🇸 Meridian, Idaho, United States

Southeast Renal Research Institute; 🇺🇸 Chattanooga, Tennessee, United States

University of Maryland Medical Center; 🇺🇸 Baltimore, Maryland, United States

University of Colorado Anschutz Medical Campus; 🇺🇸 Aurora, Colorado, United States

Rhode Island Hospital; 🇺🇸 East Providence, Rhode Island, United States