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Trial record 1 of 2 for:    vx-147
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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 : November 14, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE April 7, 2020
First Posted Date  ICMJE April 9, 2020
Last Update Posted Date November 14, 2022
Actual Study Start Date  ICMJE June 8, 2020
Actual Primary Completion Date November 11, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 7, 2020)
Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 13 [ Time Frame: Baseline, Week 13 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 7, 2020)
  • 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 ]
  • Maximum observed concentration (Cmax) of VX-147 [ Time Frame: Pre-dose up to Week 13 ]
  • Observed pre-dose concentration (Ctrough) of VX-147 [ Time Frame: Pre-dose ]
  • Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-147 [ Time Frame: Pre-dose up to Week 13 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Glomerulosclerosis, Focal Segmental
Intervention  ICMJE Drug: VX-147
Tablets for oral administration
Study Arms  ICMJE Experimental: VX-147
Subjects will receive VX-147 orally at Dose 1 for 2 weeks and at Dose 2 for 11 weeks.
Intervention: Drug: VX-147
Publications * 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. Review.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 14, 2021)
16
Original Estimated Enrollment  ICMJE
 (submitted: April 7, 2020)
10
Actual Study Completion Date  ICMJE December 9, 2021
Actual Primary Completion Date November 11, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Puerto Rico,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04340362
Other Study ID Numbers  ICMJE VX19-147-101
2020-000185-42 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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.
Current Responsible Party Vertex Pharmaceuticals Incorporated
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date November 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP