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A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05791201
Recruitment Status : Recruiting
First Posted : March 30, 2023
Last Update Posted : May 2, 2024
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE March 16, 2023
First Posted Date  ICMJE March 30, 2023
Last Update Posted Date May 2, 2024
Actual Study Start Date  ICMJE May 16, 2023
Estimated Primary Completion Date May 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 16, 2023)
  • Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From Day 1 up to 24 months ]
  • Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT) [ Time Frame: From Baseline and at Day 90 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 16, 2023)
  • Part C: Change in peak C-peptide during MMTT [ Time Frame: From Baseline up to 24 months ]
  • Part C: Change in Average Total Daily Insulin Dose [ Time Frame: From Baseline up to 24 months ]
  • Part C: Proportion of Participants who are Insulin Independent at One Point in Time [ Time Frame: From Day 180 up to Day 365 ]
  • Part C: Change in HbA1c values [ Time Frame: From Baseline up to 24 months ]
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 A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
Official Title  ICMJE A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
Brief Summary The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Type 1 Diabetes
Intervention  ICMJE Drug: VX-264
Allogeneic human stem cell-derived islets.
Study Arms  ICMJE Experimental: VX-264
Intervention: Drug: VX-264
Publications * Not Provided

*   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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 16, 2023)		 17
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2026
Estimated Primary Completion Date May 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Clinical history of T1D with greater than or equal to (>=) 5 years duration
  • Participant is on a stable diabetic treatment
  • Consistent use of continuous glucose monitoring (CGM) for at least 4 weeks before Screening and willingness to use CGM for the duration of the study

Key Exclusion Criteria:

  • Prior islet cell transplant, organ transplant, or cell therapy

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: Medical Information 617-341-6777 medicalinfo@vrtx.com
Listed Location Countries  ICMJE Canada,   Germany,   Italy,   Netherlands,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05791201
Other Study ID Numbers  ICMJE VX22-264-101
2022-003318-35 ( 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 February 2024

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