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

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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2022-003318-35-DE

Enrollment

Registered

2022-11-28

Start date

2023-09-25

Completion date

Unknown

Last updated

2024-09-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Type 1 Diabetes Mellitus MedDRA version: 20.0 Level: PT Classification code 10012601 Term: Diabetes mellitus System Organ Class: 10027433 - Metabolism and nutrition disorders

Interventions

Product Code: VX-264 Pharmaceutical Form: Implant INN or Proposed INN: VX-264 Other descriptive name: VX-264 Concentration unit: Other Concentration type: not less then Concentration number: 75000000-

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects (male and female) between the ages of 18 and 65 years (inclusive) on the date of first informed consent. 2. HbA1c =6.0% and =9.5%. 3. Clinical history and laboratory evidence of T1D based on the American Diabetes Association/European Association for the Study of Diabetes algorithm 13 for investigation of suspected T1D including: o insulin dependence for =5 years at time of Screening, and o peak stimulated C-peptide level during MMTT =65 years) yes F.1.3.1 Number of subjects for this age range 2

Exclusion criteria

Exclusion criteria: 1. Prior islet cell transplant, organ transplant, or cell therapy. 2. Advanced complications associated with diabetes including untreated proliferative retinopathy, diabetic nephropathy, skin ulcers, or amputations attributable to diabetes. 3. Insulin requirement: a) Parts A and B >40 U/day, or 55 U/day, or 0.8 U/kg/day. 4. Subjects with =2 or more episodes of severe hypoglycemia in the 12 months prior to signing of informed consent at Screening. 5. Previous abdominal or abdominal wall surgery, or history of peritonitis, that can impact VX-264 placement or put the patient at higher risk of surgical complications Please refer to Section 8.2 of the Protocol for additional exclusion criteria.

Design outcomes

Primary

Measure	Time frame
Main Objective: 1. Evaluate the safety and tolerability of VX-264 in subjects with T1D (Part A and Part B). 2. Evaluate VX-264 function in subjects with T1D (Part B and Part C).;Secondary Objective: 1. Evaluate the effect of VX-264 on exogenous insulin dose (Part C). 2. Evaluate the effect of VX-264 on insulin independence (Part C). 3. Evaluate the effect of VX-264 on metabolic control (Part C). 4. Evaluate the safety and tolerability of VX-264 (Part C).;Primary end point(s): Part A: Safety and tolerability based on treatment-emergent adverse events (TEAEs; including incidence and severity of adverse events [AEs] and serious adverse events [SAEs]), adverse device effects (ADEs; including serious adverse device effects [SADEs] and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs, number of subjects with VX 264 units explanted due to AEs or ADEs and/or unit integrity issues. Part B: - Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues. Part C: Change from baseline in peak C-peptide during MMTT at Day 90.;Timepoint(s) of evaluation of this end point: Please refer to the protocol	—

Measure

Time frame

Main Objective: 1. Evaluate the safety and tolerability of VX-264 in subjects with T1D (Part A and Part B). 2. Evaluate VX-264 function in subjects with T1D (Part B and Part C).;Secondary Objective: 1. Evaluate the effect of VX-264 on exogenous insulin dose (Part C). 2. Evaluate the effect of VX-264 on insulin independence (Part C). 3. Evaluate the effect of VX-264 on metabolic control (Part C). 4. Evaluate the safety and tolerability of VX-264 (Part C).;Primary end point(s): Part A: Safety and tolerability based on treatment-emergent adverse events (TEAEs; including incidence and severity of adverse events [AEs] and serious adverse events [SAEs]), adverse device effects (ADEs; including serious adverse device effects [SADEs] and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs, number of subjects with VX 264 units explanted due to AEs or ADEs and/or unit integrity issues. Part B: - Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues. Part C: Change from baseline in peak C-peptide during MMTT at Day 90.;Timepoint(s) of evaluation of this end point: Please refer to the protocol

—

Secondary

Measure	Time frame
Secondary end point(s): Part C: • Change from baseline in peak C-peptide during MMTT over time • Change from baseline in C-peptide AUC during MMTT over time • Proportion of subjects with peak C-peptide =100, =200, =400, and =1000 pmol/L (=0.30, =0.60, =1.21, and =3.02 ng/mL) during MMTT over time • Change from baseline in average total daily insulin dose over time • Proportion of subjects with a reduction of at least 50% insulin dose from baseline and HbA1c <7.0% at Day 365 • Proportion of subjects who are insulin independent at one point in time between Day 180 and Day 365 • Change from baseline on metabolic control: HbA1c values; continuous glucose monitoring (CGM)-derived time-in-range (TIR) over time; glucose variability (CGM, CV%); and glucose management indicator (GMI) • Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues;Timepoint(s) of evaluation of this end point: Please refer to the protocol	—

Measure

Time frame

Secondary end point(s): Part C: • Change from baseline in peak C-peptide during MMTT over time • Change from baseline in C-peptide AUC during MMTT over time • Proportion of subjects with peak C-peptide =100, =200, =400, and =1000 pmol/L (=0.30, =0.60, =1.21, and =3.02 ng/mL) during MMTT over time • Change from baseline in average total daily insulin dose over time • Proportion of subjects with a reduction of at least 50% insulin dose from baseline and HbA1c <7.0% at Day 365 • Proportion of subjects who are insulin independent at one point in time between Day 180 and Day 365 • Change from baseline on metabolic control: HbA1c values; continuous glucose monitoring (CGM)-derived time-in-range (TIR) over time; glucose variability (CGM, CV%); and glucose management indicator (GMI) • Safety and tolerability based on TEAEs (including incidence and severity of AEs and SAEs), ADEs (including SADEs and device deficiencies), clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging assessments (ultrasound and magnetic resonance imaging), retinopathy evaluation, number of subjects receiving less than the target number of VX 264 units due to AEs or ADEs, number of subjects with VX 264 units explanted due to AEs and/or unit integrity issues;Timepoint(s) of evaluation of this end point: Please refer to the protocol

—

Countries

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

Contacts

Public ContactClinical Trials and Medical Info

Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com+1877 634 8789

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026