A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05791201
Collaborator
(none)
17
1
37
Study Details
Study Description
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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 1/Phase 2 |
Study Design
Study Type:
Interventional
Anticipated Enrollment
:
17 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
Anticipated Study Start Date
:
Apr 1, 2023
Anticipated Primary Completion Date
:
May 1, 2026
Anticipated Study Completion Date
:
May 1, 2026
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: VX-264
|
Drug: VX-264
Allogeneic human stem cell-derived islets.
|
Outcome Measures
Primary Outcome Measures
- Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [From Day 1 up to 24 months]
- Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT) [From Baseline and at Day 90]
Secondary Outcome Measures
- Part C: Change in peak C-peptide during MMTT [From Baseline up to 24 months]
- Part C: Change in Average Total Daily Insulin Dose [From Baseline up to 24 months]
- Part C: Proportion of Participants who are Insulin Independent at One Point in Time [From Day 180 up to Day 365]
- Part C: Change in HbA1c values [From Baseline up to 24 months]
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Key Inclusion Criteria:
-
Clinical history of T1D with greater than (>) 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.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT05791201
Other Study ID Numbers:
- VX22-264-101
First Posted:
Mar 30, 2023
Last Update Posted:
Mar 30, 2023
Last Verified:
Mar 1, 2023
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms: