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

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ClinicalTrials.gov Identifier: NCT04340362
Recruitment Status : Completed
First Posted : April 9, 2020
Last Update Posted : January 11, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in subjects with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).

Condition or disease Intervention/treatment Phase
Glomerulosclerosis, Focal Segmental Drug: VX-147 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : June 8, 2020
Actual Primary Completion Date : November 11, 2021
Actual Study Completion Date : December 9, 2021

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Arms and Interventions
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Arm Intervention/treatment
Experimental: VX-147
Subjects will receive VX-147 orally at Dose 1 for 2 weeks and at Dose 2 for 11 weeks.
 Drug: VX-147
Tablets for oral administration


Outcome Measures
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Primary Outcome Measures :
  1. Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 13 [ Time Frame: Baseline, Week 13 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Maximum observed concentration (Cmax) of VX-147 [ Time Frame: Pre-dose up to Week 13 ]
  3. Observed pre-dose concentration (Ctrough) of VX-147 [ Time Frame: Pre-dose ]
  4. Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-147 [ Time Frame: Pre-dose up to Week 13 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04340362


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT04340362    
Other Study ID Numbers: VX19-147-101
2020-000185-42 ( EudraCT Number )
First Posted: April 9, 2020    Key Record Dates
Last Update Posted: January 11, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Glomerulosclerosis, Focal Segmental
Glomerulonephritis
Nephritis
Kidney Diseases
Urologic Diseases