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

Type 1 Diabetes Clinical Trial

Official title:

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05791201
• Other study ID #: VX22-264-101
• Secondary ID: 2022-003318-35
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: May 16, 2023
• Est. completion date: May 2026

Study information

• Verified date: February 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: Medical Information
• Phone: 617-341-6777
• Email: medicalinfo[@]vrtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 17
• Est. completion date: May 2026
• Est. primary completion date: May 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus, Type 1
• Type 1 Diabetes

Intervention

Drug:
• VX-264
Allogeneic human stem cell-derived islets.

Locations

Country	Name	City	State
Canada	University of Alberta, Edmonton	Edmonton
Canada	Toronto General Hospital (TGH)	Toronto
Canada	Vancouver General Hospital	Vancouver
Germany	Dresden Center for Islet Transplantation	Dresden
Italy	IRCCS Ospedale San Raffaele	Milan
Netherlands	Leiden University	Leiden
Switzerland	Hopiteaux Universitaires de Geneve	Geneve
United Kingdom	Churchill Hospital	Headington
United Kingdom	Royal Victoria Infirmary	Newcastle Upon Tyne
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Chicago	Chicago	Illinois
United States	University of Wisconsin	Madison	Wisconsin
United States	UHealth Diabetes Research Institute	Miami	Florida
United States	Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania
United States	University of Pittsburgh Medical Center Montefiore	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Italy,  Netherlands,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Part B and Part C: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)		From Baseline and at Day 90
Secondary	Part C: Change in peak C-peptide during MMTT		From Baseline up to 24 months
Secondary	Part C: Change in Average Total Daily Insulin Dose		From Baseline up to 24 months
Secondary	Part C: Proportion of Participants who are Insulin Independent at One Point in Time		From Day 180 up to Day 365
Secondary	Part C: Change in HbA1c values		From Baseline up to 24 months