Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

Glomerulosclerosis, Focal Segmental Clinical Trial

Official title:

A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04340362
• Other study ID #: VX19-147-101
• Secondary ID: 2020-000185-42
• Status: Completed
• Phase: Phase 2
• Start date: June 8, 2020
• Est. completion date: December 9, 2021

Study information

• Verified date: June 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: December 9, 2021
• Est. primary completion date: November 11, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Key Inclusion Criteria:

• APOL1 genotype of G1/G1, G2/G2, or G1/G2

• FSGS diagnosed by kidney biopsy

Key Exclusion Criteria:

• Evidence of non-APOL1-mediated FSGS

• Participants with known sickle cell disease

• Solid organ or Bone marrow transplant

Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Glomerulosclerosis, Focal Segmental

Intervention

Drug:
• VX-147
Tablets for oral administration.

Locations

Country	Name	City	State
France	Hopital Henri Mondor	Créteil
France	Hôpital Bicêtre AP-HP	Le Kremlin-Bicêtre
France	Bichat Hospital	Paris
France	Hopitaux Universitaires Est Parisien - Hopital Tenon	Paris
France	Service de Nephrologie - Hopital Universitaire Necker	Paris
France	Université Paris-Descartes / Hôpital Européen Georges Pompidou	Paris
Puerto Rico	GCM Medical Group, PSC	San Juan
United Kingdom	University Hospitals of Leicester NHS Trust - Leicester General Hospital	Leicester
United Kingdom	Barts Health NHS Trust	London
United Kingdom	The Medicines Evaluation Unit	Manchester
United Kingdom	King's College Hospital NHS Foundation Trust - Guthrie Clinic	Southwark
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Morehouse School of Medicine, Grady Memorial Hospital	Atlanta	Georgia
United States	Renal Associates of Baton Rouge	Baton Rouge	Louisiana
United States	University of Alabama at Birmingham - The Kirklin Clinic	Birmingham	Alabama
United States	Beth Israel Deaconess Medical Center	Boston	Massachusetts
United States	Boston Medical Center	Boston	Massachusetts
United States	SUNY Downstate	Brooklyn	New York
United States	Urban Family Practice	Buffalo	New York
United States	UNC Kidney Center Division of Nephrology & Hypertension	Chapel Hill	North Carolina
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Tryon Medical Partners	Charlotte	North Carolina
United States	Duke University School of Medicine - Duke Molecular Physiology Institute	Durham	North Carolina
United States	Durham Nephrology Associates, PA	Durham	North Carolina
United States	Privia Medical Group	Houston	Texas
United States	Prolato Clinical Research Center	Houston	Texas
United States	Paragon Health, PC d/b/a Nephrology Center, PC	Kalamazoo	Michigan
United States	Nevada Kidney Disease and Hypertension Centers	Las Vegas	Nevada
United States	Georgia Nehphrology	Lawrenceville	Georgia
United States	Central Georgia Kidney Specialists PC	Macon	Georgia
United States	Kidney and Hypertension Specialists of Miami	Miami	Florida
United States	Vanderbilt University VU	Nashville	Tennessee
United States	Ochsner Medical Center - New Orleans	New Orleans	Louisiana
United States	Icahn School of Medicine at Mount Sinai	New York	New York
United States	South Carolina Nephrology and Hypertension Center, Inc.	Orangeburg	South Carolina
United States	Temple University Hospital	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	St Louis Kidney Care	Saint Louis	Missouri
United States	California Institute of Renal Research	San Diego	California
United States	Renal and Transplant Associates of New England, PC	Springfield	Massachusetts
United States	Nephrology and Hypertension Associates, LTD	Tupelo	Mississippi
United States	Howard University Hospital	Washington	District of Columbia
United States	The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension	Washington	District of Columbia
United States	Florida Premier Research Institute	Winter Park	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  France,  Puerto Rico,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent Change From Baseline in UPCR		From Baseline up to Week 13
Secondary	Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)		From Baseline up to Week 17
Secondary	Maximum Observed Concentration (Cmax) of VX-147		Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5
Secondary	Observed Pre-dose Concentration (Ctrough) of VX-147		Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
Secondary	Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147		Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5