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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vertex Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

6 Jul 2023 → ongoing

Decision date (initial)

2024-09-25

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-515583-32-00

EudraCT number

2022-003318-35

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

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)

Conditions and MedDRA coding

Type 1 Diabetes Mellitus

Study design 1 period

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

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 <50 pmol/L (<0.15 ng/mL)
4. 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.
5. Body habitus supportive of implantation of the planned number of VX- 264 units per study part and/or cohort.

Exclusion criteria 5

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 <10 U/day b) >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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. 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.
2. 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.
3. Part C: Change from baseline in peak C-peptide during MMTT at Day 90.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vertex Pharmaceuticals Inc.

Sponsor organisation

Vertex Pharmaceuticals Inc.

Address

50 Northern Avenue

City

Boston

Postcode

02210-1862

Country

United States

Scientific contact point

Organisation

Vertex Pharmaceuticals Inc.

Contact name

Clinical Trials and Medical Info

Public contact point

Organisation

Vertex Pharmaceuticals Inc.

Contact name

Clinical Trials and Medical Info

Locations

3 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 68 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications