Phase 2
N=16
Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
Glomerulosclerosis, Focal Segmental
Bottom Line
View on ClinicalTrials.gov: NCT04340362 ↗Enrolled (actual)
16
Serious AEs
6.3%
Results posted
Jul 2023
Primary outcome: Primary: Percent Change From Baseline in UPCR — -47.7; -47.5; -47.6 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- VX-147 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- Nov 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in UPCR |
-47.7; -47.5; -47.6 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
3; 0; 12; 1 | — |
| SECONDARY Maximum Observed Concentration (Cmax) of VX-147 |
0.168; 0.846 | — |
| SECONDARY Observed Pre-dose Concentration (Ctrough) of VX-147 |
0.153; 0.123; 0.379; 0.492; 0.446; 0.529 | — |
| SECONDARY Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147 |
15.3 | — |
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.
Data sourced from ClinicalTrials.gov (NCT04340362). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.