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

Primary Purpose

Glomerulosclerosis, Focal Segmental

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-147
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glomerulosclerosis, Focal Segmental

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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
  • Subjects with known sickle cell disease
  • Solid organ or Bone marrow transplant

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • University of Alabama at Birmingham - The Kirklin Clinic
  • California Institute of Renal Research
  • The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
  • Howard University Hospital
  • Kidney and Hypertension Specialists of Miami
  • Florida Premier Research Institute
  • Morehouse School of Medicine, Grady Memorial Hospital
  • Children's Healthcare of Atlanta
  • Georgia Nehphrology
  • Central Georgia Kidney Specialists PC
  • Renal Associates of Baton Rouge
  • Ochsner Medical Center - New Orleans
  • Boston Medical Center
  • Beth Israel Deaconess Medical Center
  • Renal and Transplant Associates of New England, PC
  • Paragon Health, PC d/b/a Nephrology Center, PC
  • Nephrology and Hypertension Associates, LTD
  • St Louis Kidney Care
  • Nevada Kidney Disease and Hypertension Centers
  • SUNY Downstate
  • Urban Family Practice
  • Icahn School of Medicine at Mount Sinai
  • UNC Kidney Center Division of Nephrology & Hypertension
  • Tryon Medical Partners
  • Durham Nephrology Associates, PA
  • Duke University School of Medicine - Duke Molecular Physiology Institute
  • Temple University Hospital
  • Children's Hospital of Pittsburgh of UPMC
  • Medical University of South Carolina
  • South Carolina Nephrology and Hypertension Center, Inc.
  • Vanderbilt University VU
  • Prolato Clinical Research Center
  • Privia Medical Group
  • Hopital Henri Mondor
  • Hôpital Bicêtre AP-HP
  • Bichat Hospital
  • Hopitaux Universitaires Est Parisien - Hopital Tenon
  • Service de Nephrologie - Hopital Universitaire Necker
  • Université Paris-Descartes / Hôpital Européen Georges Pompidou
  • GCM Medical Group, PSC
  • University Hospitals of Leicester NHS Trust - Leicester General Hospital
  • Barts Health NHS Trust
  • The Medicines Evaluation Unit
  • King's College Hospital NHS Foundation Trust - Guthrie Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-147

Arm Description

All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.

Outcomes

Primary Outcome Measures

Percent Change From Baseline in UPCR

Secondary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Maximum Observed Concentration (Cmax) of VX-147
Observed Pre-dose Concentration (Ctrough) of VX-147
Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147

Full Information

First Posted
April 7, 2020
Last Updated
June 17, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04340362
Brief Title
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

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
June 8, 2020 (Actual)
Primary Completion Date
November 11, 2021 (Actual)
Study Completion Date
December 9, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glomerulosclerosis, Focal Segmental

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VX-147
Arm Type
Experimental
Arm Description
All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.
Intervention Type
Drug
Intervention Name(s)
VX-147
Other Intervention Name(s)
IXP
Intervention Description
Tablets for oral administration.
Primary Outcome Measure Information:
Title
Percent Change From Baseline in UPCR
Time Frame
From Baseline up to Week 13
Secondary Outcome Measure Information:
Title
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
From Baseline up to Week 17
Title
Maximum Observed Concentration (Cmax) of VX-147
Time Frame
Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5
Title
Observed Pre-dose Concentration (Ctrough) of VX-147
Time Frame
Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
Title
Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147
Time Frame
Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
University of Alabama at Birmingham - The Kirklin Clinic
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
California Institute of Renal Research
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20037
Country
United States
Facility Name
Howard University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20060
Country
United States
Facility Name
Kidney and Hypertension Specialists of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33150
Country
United States
Facility Name
Florida Premier Research Institute
City
Winter Park
State/Province
Florida
ZIP/Postal Code
32789
Country
United States
Facility Name
Morehouse School of Medicine, Grady Memorial Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30310
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Georgia Nehphrology
City
Lawrenceville
State/Province
Georgia
ZIP/Postal Code
30046
Country
United States
Facility Name
Central Georgia Kidney Specialists PC
City
Macon
State/Province
Georgia
ZIP/Postal Code
31201
Country
United States
Facility Name
Renal Associates of Baton Rouge
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Facility Name
Ochsner Medical Center - New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Facility Name
Boston Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Beth Israel Deaconess Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Renal and Transplant Associates of New England, PC
City
Springfield
State/Province
Massachusetts
ZIP/Postal Code
01107
Country
United States
Facility Name
Paragon Health, PC d/b/a Nephrology Center, PC
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49007
Country
United States
Facility Name
Nephrology and Hypertension Associates, LTD
City
Tupelo
State/Province
Mississippi
ZIP/Postal Code
38801
Country
United States
Facility Name
St Louis Kidney Care
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63136
Country
United States
Facility Name
Nevada Kidney Disease and Hypertension Centers
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89106
Country
United States
Facility Name
SUNY Downstate
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11203
Country
United States
Facility Name
Urban Family Practice
City
Buffalo
State/Province
New York
ZIP/Postal Code
14201
Country
United States
Facility Name
Icahn School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
UNC Kidney Center Division of Nephrology & Hypertension
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Tryon Medical Partners
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28210
Country
United States
Facility Name
Durham Nephrology Associates, PA
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27704
Country
United States
Facility Name
Duke University School of Medicine - Duke Molecular Physiology Institute
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Temple University Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19140
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
South Carolina Nephrology and Hypertension Center, Inc.
City
Orangeburg
State/Province
South Carolina
ZIP/Postal Code
29118
Country
United States
Facility Name
Vanderbilt University VU
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Prolato Clinical Research Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77054
Country
United States
Facility Name
Privia Medical Group
City
Houston
State/Province
Texas
ZIP/Postal Code
77095
Country
United States
Facility Name
Hopital Henri Mondor
City
Créteil
Country
France
Facility Name
Hôpital Bicêtre AP-HP
City
Le Kremlin-Bicêtre
Country
France
Facility Name
Bichat Hospital
City
Paris
Country
France
Facility Name
Hopitaux Universitaires Est Parisien - Hopital Tenon
City
Paris
Country
France
Facility Name
Service de Nephrologie - Hopital Universitaire Necker
City
Paris
Country
France
Facility Name
Université Paris-Descartes / Hôpital Européen Georges Pompidou
City
Paris
Country
France
Facility Name
GCM Medical Group, PSC
City
San Juan
ZIP/Postal Code
00917
Country
Puerto Rico
Facility Name
University Hospitals of Leicester NHS Trust - Leicester General Hospital
City
Leicester
Country
United Kingdom
Facility Name
Barts Health NHS Trust
City
London
Country
United Kingdom
Facility Name
The Medicines Evaluation Unit
City
Manchester
Country
United Kingdom
Facility Name
King's College Hospital NHS Foundation Trust - Guthrie Clinic
City
Southwark
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.
Citations:
PubMed Identifier
33767059
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.
Results Reference
derived

Learn more about this trial

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

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