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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 21 November 2022
Main ID:  NCT04340362
Date of registration: 07/04/2020
Prospective Registration: Yes
Primary sponsor: Vertex Pharmaceuticals Incorporated
Public title: Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
Scientific 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
Date of first enrolment: June 8, 2020
Target sample size: 16
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT04340362
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
France Puerto Rico United Kingdom United States
Contacts
Key inclusion & exclusion 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

- Subjects with known sickle cell disease

- Solid organ or Bone marrow transplant

Other protocol defined Inclusion/Exclusion criteria may apply.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All
Health Condition(s) or Problem(s) studied
Glomerulosclerosis, Focal Segmental
Intervention(s)
Drug: VX-147
Primary Outcome(s)
Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 13 [Time Frame: Baseline, Week 13]
Secondary Outcome(s)
Maximum observed concentration (Cmax) of VX-147 [Time Frame: Pre-dose up to Week 13]
Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-147 [Time Frame: Pre-dose up to Week 13]
Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [Time Frame: From Baseline up to Week 17]
Observed pre-dose concentration (Ctrough) of VX-147 [Time Frame: Pre-dose]
Secondary ID(s)
2020-000185-42
VX19-147-101
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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