Clinical trial NCT04340362: Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Glomerulosclerosis, Focal Segmental
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2020-04-09
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Percent Change From Baseline in UPCR
timeFrame: From Baseline up to Week 13
Secondary outcomes:
measure: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: From Baseline up to Week 17
measure: Maximum Observed Concentration (Cmax) of VX-147
timeFrame: Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5
measure: Observed Pre-dose Concentration (Ctrough) of VX-147
timeFrame: Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
measure: Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147
timeFrame: Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham - The Kirklin Clinic
state: Alabama
city: San Diego
country: United States
facility: California Institute of Renal Research
state: California
city: Washington
country: United States
facility: The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
state: District of Columbia
city: Washington
country: United States
facility: Howard University Hospital
state: District of Columbia
city: Miami
country: United States
facility: Kidney and Hypertension Specialists of Miami
state: Florida
city: Winter Park
country: United States
facility: Florida Premier Research Institute
state: Florida
city: Atlanta
country: United States
facility: Morehouse School of Medicine, Grady Memorial Hospital
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Lawrenceville
country: United States
facility: Georgia Nehphrology
state: Georgia
city: Macon
country: United States
facility: Central Georgia Kidney Specialists PC
state: Georgia
city: Baton Rouge
country: United States
facility: Renal Associates of Baton Rouge
state: Louisiana
city: New Orleans
country: United States
facility: Ochsner Medical Center - New Orleans
state: Louisiana
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Springfield
country: United States
facility: Renal and Transplant Associates of New England, PC
state: Massachusetts
city: Kalamazoo
country: United States
facility: Paragon Health, PC d/b/a Nephrology Center, PC
state: Michigan
city: Tupelo
country: United States
facility: Nephrology and Hypertension Associates, LTD
state: Mississippi
city: Saint Louis
country: United States
facility: St Louis Kidney Care
state: Missouri
city: Las Vegas
country: United States
facility: Nevada Kidney Disease and Hypertension Centers
state: Nevada
city: Brooklyn
country: United States
facility: SUNY Downstate
state: New York
city: Buffalo
country: United States
facility: Urban Family Practice
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Chapel Hill
country: United States
facility: UNC Kidney Center Division of Nephrology & Hypertension
state: North Carolina
city: Charlotte
country: United States
facility: Tryon Medical Partners
state: North Carolina
city: Durham
country: United States
facility: Durham Nephrology Associates, PA
state: North Carolina
city: Durham
country: United States
facility: Duke University School of Medicine - Duke Molecular Physiology Institute
state: North Carolina
city: Philadelphia
country: United States
facility: Temple University Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Orangeburg
country: United States
facility: South Carolina Nephrology and Hypertension Center, Inc.
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University VU
state: Tennessee
city: Houston
country: United States
facility: Prolato Clinical Research Center
state: Texas
city: Houston
country: United States
facility: Privia Medical Group
state: Texas
city: Créteil
country: France
facility: Hopital Henri Mondor
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre AP-HP
city: Paris
country: France
facility: Bichat Hospital
city: Paris
country: France
facility: Hopitaux Universitaires Est Parisien - Hopital Tenon
city: Paris
country: France
facility: Service de Nephrologie - Hopital Universitaire Necker
city: Paris
country: France
facility: Université Paris-Descartes / Hôpital Européen Georges Pompidou
city: San Juan
country: Puerto Rico
facility: GCM Medical Group, PSC
city: Leicester
country: United Kingdom
facility: University Hospitals of Leicester NHS Trust - Leicester General Hospital
city: London
country: United Kingdom
facility: Barts Health NHS Trust
city: Manchester
country: United Kingdom
facility: The Medicines Evaluation Unit
city: Southwark
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust - Guthrie Clinic
Study officials:
References:
citation: Bruggeman LA, Sedor JR, O'Toole JF. Apolipoprotein L1 and mechanisms of kidney disease susceptibility. Curr Opin Nephrol Hypertens. 2021 May 1;30(3):317-323. doi: 10.1097/MNH.0000000000000704.