A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes

Phase 3RecruitingNCT04786262

Vertex Pharmaceuticals Incorporated57 enrolled

Overview

This study will evaluate the safety, tolerability and efficacy of VX-880 infusion and VX-017 infusion in participants with type 1 diabetes (T1D) and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Diabetes Mellitus, Type 1 Impaired Hypoglycemic Awareness Severe Hypoglycemia

Interventions

VX-880BIOLOGICAL

Infused into the hepatic portal vein.

VX-017BIOLOGICAL

Infused into the hepatic portal vein.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Clinical history of T1D with \>=5 years of duration of insulin dependence * At least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment * Stable diabetic treatment * Consistent use of continuous glucose monitor (CGM) for at least 3 months 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

Locations (29)

Outcomes

Primary Outcomes

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Time frame: From VX-880 infusion to end of study (at least 5 years)

Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)

Time frame: 1 year after achieving insulin independence (following VX-880 infusion)

Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Time frame: From VX-017 infusion to end of study (at least 5 years)

Secondary Outcomes

Parts B and C: Proportion of Participants who are Insulin Independent

Time frame: At 1 year after VX-880 infusion

Parts B and C: Proportion of Participants Free from SHEs with a Glycated Hemoglobin (HbA1c) less than (<) 7.0%

Time frame: SHEs: from Day 90 through 1 year (inclusive) after VX-880 infusion; HbA1c < 7% at 1 year after VX-880 infusion

Parts B and C: Change From Baseline in Glycated Hemoglobin (HbA1c)

Time frame: At 1 year after VX-880 infusion

Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year

Time frame: From VX-880 infusion to end of study (at least 5 years)

Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Time frame: From VX-880 infusion up to end of study (at least 5 years)

Part D: Change from baseline in peak C-peptide during mixed-meal tolerance test (MMTT)

Central Contacts

Medical Information

CONTACT

617-341-6777 medicalinfo@vrtx.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

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...and 19 more locations