VX-264 for Type 1 diabetes mellitus

Inclusion criteria

• {"criterion_text":"- Subjects (male and female) between the ages of 18 and 65 years (inclusive) on the date of first informed consent."}
• {"criterion_text":"- HbA1c ≥6.0% and ≤ 9.5%."}
• {"criterion_text":"- 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 <50 pmol/L (<0.15 ng/mL)"}
• {"criterion_text":"- Consistent use of CGM for at least 4 weeks before Screening and willingness to use only the study-provided CGM for the duration of the study."}
• {"criterion_text":"- Body habitus supportive of implantation of the planned number of VX- 264 units per study part and/or cohort."}

Exclusion criteria

• {"criterion_text":"- Prior islet cell transplant, organ transplant, or cell therapy."}
• {"criterion_text":"- Advanced complications associated with diabetes including untreated proliferative retinopathy, diabetic nephropathy, skin ulcers, or amputations attributable to diabetes."}
• {"criterion_text":"- Insulin requirement: a) Parts A and B >40 U/day, or <10 U/day b) >0.8 U/kg/day."}
• {"criterion_text":"- Subjects with ≥2 or more episodes of severe hypoglycemia in the 12 months prior to signing of informed consent at Screening."}
• {"criterion_text":"- 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."}

Primary endpoints

• {"endpoint_text":"- Part A: 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, number of subjects with VX 264 units explanted due to AEs or ADEs and/or unit integrity issues.","definition_or_measurement_approach":"Safety and tolerability assessed by TEAEs/SAEs, ADEs/SADEs, device deficiencies, clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging (ultrasound, MRI), retinopathy evaluation, and counts of subjects receiving fewer than target VX-264 units or having units explanted."}
• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Safety and tolerability assessed by TEAEs/SAEs, ADEs/SADEs, device deficiencies, clinical laboratory assessments, vital signs, standard 12-lead ECGs, imaging (ultrasound, MRI), retinopathy evaluation, and counts of subjects receiving fewer than target VX-264 units or having units explanted."}
• {"endpoint_text":"- Part C: Change from baseline in peak C-peptide during MMTT at Day 90.","definition_or_measurement_approach":"Measured change from baseline in peak C-peptide during a Mixed-Meal Tolerance Test (MMTT) at Day 90."}

Methods

• K1_Recruitment Arrangements (country-specific) documents listed for Netherlands, Italy, Germany (study-specific recruitment arrangements).
• MyTomorrows platform: Landing pages and patient navigator scripts (MyTomorrows Landing Page Copy, Landing Page Screenshots, Patient Navigator Scripts) — country-specific copies for Netherlands, Italy, Germany.
• Digital advertising: Facebook advertisements (country-specific: Netherlands, Italy, Germany) and Google advertisements (country-specific).
• Digital materials: MyTomorrows Landing Page, MyTomorrows Google and Facebook ads, landing page screenshots (country-specific).
• Printed materials and direct outreach: Posters (Study Specific and T1D Specific), Recruitment Brochure, Patient Invitation to Trial Letter (country-specific).
• HCP / site engagement: PI to Physician Letter, HCP Slide Deck (country-specific), Research Institute Posting, Press Release Text, Study Fact Sheets.

Netherlands

Earliest CTIS Part Ii Submission Date

20-09-2024

Latest Decision Or Authorization Date

08-09-2025

Processing Time Days

353

Number Of Sites

1

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

27-09-2024

Latest Decision Or Authorization Date

10-09-2025

Processing Time Days

348

Number Of Sites

2

Number Of Participants

1

Germany

Earliest CTIS Part Ii Submission Date

24-09-2024

Latest Decision Or Authorization Date

18-09-2025

Processing Time Days

359

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Vertex Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States