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

Primary Purpose

Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
VX-880
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diabetes Mellitus, Type 1

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Clinical history of T1D with > 5 years of duration
  • 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

Sites / Locations

  • City of HopeRecruiting
  • UHealth Diabetes Research InstituteRecruiting
  • Northwestern Organ Transplant CenterRecruiting
  • University of ChicagoRecruiting
  • Massachusetts General HospitalRecruiting
  • Hospital of the University of PennsylvaniaRecruiting
  • University of Pittsburgh Medical Center MontefioreRecruiting
  • VCU Medical Center, RichmondRecruiting
  • University of WisconsinRecruiting
  • University of Alberta, EdmontonRecruiting
  • McGill University Health CentreRecruiting
  • Toronto General Hospital (TGH)Recruiting
  • Vancouver General HospitalRecruiting
  • CHU LilleRecruiting
  • Hôpitaux Universitaires de StrasbourgRecruiting
  • Dresden Center for Islet TransplantationRecruiting
  • IRCCS Ospedale San RaffaeleRecruiting
  • Leiden UniversityRecruiting
  • Oslo University HospitalRecruiting
  • Hopiteaux Universitaires de GeneveRecruiting
  • Churchill HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-880

Arm Description

Outcomes

Primary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Proportion of Participants Free of Severe Hypoglycemic Events With Either a Glycosylated Hemoglobin (HbA1c) <7.0% or a ≥ 1% Reduction in HbA1c From Baseline

Secondary Outcome Measures

Proportion of Participants who are Insulin Independent
Changes in Stimulated C-peptide

Full Information

First Posted
March 3, 2021
Last Updated
October 17, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04786262
Brief Title
A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes
Official Title
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-880 in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 29, 2021 (Actual)
Primary Completion Date
January 2024 (Anticipated)
Study Completion Date
January 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
17 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VX-880
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
VX-880
Other Intervention Name(s)
Formerly known as STx-02
Intervention Description
Infused into the hepatic portal vein.
Primary Outcome Measure Information:
Title
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
From VX-880 infusion to end of study (up to 5 years)
Title
Proportion of Participants Free of Severe Hypoglycemic Events With Either a Glycosylated Hemoglobin (HbA1c) <7.0% or a ≥ 1% Reduction in HbA1c From Baseline
Time Frame
At 1 year after VX-880 infusion
Secondary Outcome Measure Information:
Title
Proportion of Participants who are Insulin Independent
Time Frame
At 1 year after VX-880 infusion
Title
Changes in Stimulated C-peptide
Time Frame
At 1 year after VX-880 infusion

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Clinical history of T1D with > 5 years of duration 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-341-6777
Email
medicalinfo@vrtx.com
Facility Information:
Facility Name
City of Hope
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Individual Site Status
Recruiting
Facility Name
UHealth Diabetes Research Institute
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Name
Northwestern Organ Transplant Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Individual Site Status
Recruiting
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Name
Hospital of the University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Pittsburgh Medical Center Montefiore
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Individual Site Status
Recruiting
Facility Name
VCU Medical Center, Richmond
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Wisconsin
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Alberta, Edmonton
City
Edmonton
Country
Canada
Individual Site Status
Recruiting
Facility Name
McGill University Health Centre
City
Montreal
Country
Canada
Individual Site Status
Recruiting
Facility Name
Toronto General Hospital (TGH)
City
Toronto
Country
Canada
Individual Site Status
Recruiting
Facility Name
Vancouver General Hospital
City
Vancouver
Country
Canada
Individual Site Status
Recruiting
Facility Name
CHU Lille
City
Lille
Country
France
Individual Site Status
Recruiting
Facility Name
Hôpitaux Universitaires de Strasbourg
City
Strasbourg
Country
France
Individual Site Status
Recruiting
Facility Name
Dresden Center for Islet Transplantation
City
Dresden
Country
Germany
Individual Site Status
Recruiting
Facility Name
IRCCS Ospedale San Raffaele
City
Milan
Country
Italy
Individual Site Status
Recruiting
Facility Name
Leiden University
City
Leiden
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Oslo University Hospital
City
Oslo
Country
Norway
Individual Site Status
Recruiting
Facility Name
Hopiteaux Universitaires de Geneve
City
Geneve
Country
Switzerland
Individual Site Status
Recruiting
Facility Name
Churchill Hospital
City
Headington
State/Province
Oxford
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Learn more about this trial

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

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