Glomerulosclerosis, Focal SegmentalPhase 2NCT04340362
Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
A Phase 2 study testing VX-147 in people with Glomerulosclerosis, Focal Segmental, run by Vertex Pharmaceuticals Incorporated at 44 sites in 4 countries.
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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Glomerulosclerosis, Focal Segmental
- What is being tested?
- VX-147
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 44 sitesin 4 countries
- Study dates
- 8 Jun 2020to 9 Dec 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- APOL1 genotype of G1/G1, G2/G2, or G1/G2
- FSGS diagnosed by kidney biopsy
Who cannot join
- 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.
Trial locations and contacts
44 sites in 4 countries.
44 sites in 4 countries
United States33 sites
Morehouse School of Medicine, Grady Memorial Hospital
Atlanta, Georgia, 30310
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Renal Associates of Baton Rouge
Baton Rouge, Louisiana, 70808
University of Alabama at Birmingham - The Kirklin Clinic
Birmingham, Alabama, 35294
Boston Medical Center
Boston, Massachusetts, 02118
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
SUNY Downstate
Brooklyn, New York, 11203
Urban Family Practice
Buffalo, New York, 14201
UNC Kidney Center Division of Nephrology & Hypertension
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Tryon Medical Partners
Charlotte, North Carolina, 28210
Durham Nephrology Associates, PA
Durham, North Carolina, 27704
Duke University School of Medicine - Duke Molecular Physiology Institute
Durham, North Carolina, 27710
Prolato Clinical Research Center
Houston, Texas, 77054
Privia Medical Group
Houston, Texas, 77095
Paragon Health, PC d/b/a Nephrology Center, PC
Kalamazoo, Michigan, 49007
Nevada Kidney Disease and Hypertension Centers
Las Vegas, Nevada, 89106
Georgia Nehphrology
Lawrenceville, Georgia, 30046
Central Georgia Kidney Specialists PC
Macon, Georgia, 31201
Kidney and Hypertension Specialists of Miami
Miami, Florida, 33150
Vanderbilt University VU
Nashville, Tennessee, 37232
Ochsner Medical Center - New Orleans
New Orleans, Louisiana, 70121
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
South Carolina Nephrology and Hypertension Center, Inc.
Orangeburg, South Carolina, 29118
Temple University Hospital
Philadelphia, Pennsylvania, 19140
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
California Institute of Renal Research
San Diego, California, 92123
Renal and Transplant Associates of New England, PC
Springfield, Massachusetts, 01107
St Louis Kidney Care
St Louis, Missouri, 63136
Nephrology and Hypertension Associates, LTD
Tupelo, Mississippi, 38801
The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
Washington D.C., District of Columbia, 20037
Howard University Hospital
Washington D.C., District of Columbia, 20060
Florida Premier Research Institute
Winter Park, Florida, 32789
France6 sites
Hopital Henri Mondor
Créteil
Hôpital Bicêtre AP-HP
Le Kremlin-Bicêtre
Bichat Hospital
Paris
Hopitaux Universitaires Est Parisien - Hopital Tenon
Paris
Service de Nephrologie - Hopital Universitaire Necker
Paris
Université Paris-Descartes / Hôpital Européen Georges Pompidou
Paris
United Kingdom4 sites
University Hospitals of Leicester NHS Trust - Leicester General Hospital
Leicester
Barts Health NHS Trust
London
The Medicines Evaluation Unit
Manchester
King's College Hospital NHS Foundation Trust - Guthrie Clinic
Southwark
About this study
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
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).
- Conditions
- Glomerulosclerosis, Focal Segmental
- Being tested
- VX-147
- Medical subject headings
- Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-147Drug
Also called IXP
Tablets for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| VX-147 | Experimental | 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. Receives: Drug: VX-147 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline in UPCR
Measured: From Baseline up to Week 13
Secondary outcomes (4)
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: From Baseline up to Week 17
Maximum Observed Concentration (Cmax) of VX-147
Measured: Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5
Observed Pre-dose Concentration (Ctrough) of VX-147
Measured: Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147
Measured: Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2020
- Study start8 Jun 2020
- Primary completion11 Nov 2021
- Study completion9 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Glomerulosclerosis, Focal SegmentalStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04340362
- Sponsor's protocol ID
- VX19-147-101
- Other IDs
- 2020-000185-42
- Status last verified
- Sep 2025
- First submitted
- 7 Apr 2020
- Last updated
- 24 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Publications
Egbuna O, Zimmerman B, Manos G, Fortier A, Chirieac MC, Dakin LA, Friedman DJ, Bramham K, Campbell K, Knebelmann B, Barisoni L, Falk RJ, Gipson DS, Lipkowitz MS, Ojo A, Bunnage ME, Pollak MR, Altshuler D, Chertow GM; VX19-147-101 Study Group. Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants. N Engl J Med. 2023 Mar 16;388(11):969-979. doi: 10.1056/NEJMoa2202396.
PubMed 36920755Bruggeman 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.
PubMed 33767059
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04340362
- Acronym / sponsor ID
- VX19-147-101
- Conditions
- Glomerulosclerosis, Focal Segmental
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 8 Jun 2020 (actual)
- Primary completion
- 11 Nov 2021 (actual)
- Study completion
- 9 Dec 2021 (actual)
- First posted
- 9 Apr 2020 (actual)
- Results
- Posted 10 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VX-147 | Experimental | Drug: VX-147All 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- APOL1 genotype of G1/G1, G2/G2, or G1/G2
- FSGS diagnosed by kidney biopsy
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VX-147Also: IXPTablets for oral administration. | Drug | VX-147 |
Primary outcome
Percent Change From Baseline in UPCR
Time frame: From Baseline up to Week 13
4 secondary outcomes
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
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
Observed Pre-dose Concentration (Ctrough) of VX-147
Time frame: Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36920755Egbuna O, Zimmerman B, Manos G, Fortier A, Chirieac MC, Dakin LA, Friedman DJ, Bramham K, Campbell K, Knebelmann B, Barisoni L, Falk RJ, Gipson DS, Lipkowitz MS, Ojo A, Bunnage ME, Pollak MR, Altshuler D, Chertow GM; VX19-147-101 Study Group. Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants. N Engl J Med. 2023 Mar 16;388(11):969-979. doi: 10.1056/NEJMoa2202396.
- Derived from this study: PubMed 33767059Bruggeman 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.
- Individual participant dataSharing planned: No. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
44 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VX-147 in people with Glomerulosclerosis, Focal Segmental, run by Vertex Pharmaceuticals Incorporated at 44 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 44 sites in United States, France, United Kingdom, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change From Baseline in UPCR (time frame: From Baseline up to Week 13). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04340362, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2025.