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

Diabetes Mellitus, Type 1 Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04786262
• Other study ID #: VX20-880-101
• Secondary ID: 2022-002292-11
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: March 29, 2021
• Est. completion date: February 2030

Study information

• Verified date: June 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: Medical Information
• Phone: 617-341-6777
• Email: medicalinfo[@]vrtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 37
• Est. completion date: February 2030
• Est. primary completion date: February 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Biological:
• VX-880
Infused into the hepatic portal vein.

Locations

Country	Name	City	State
Canada	University of Alberta, Edmonton	Edmonton
Canada	McGill University Health Centre	Montreal
Canada	Toronto General Hospital (TGH)	Toronto
Canada	Vancouver General Hospital	Vancouver
France	CHU Lille	Lille
France	Centre de recherche en Biomédecine de Strasbourg	Strasbourg
Germany	Dresden Center for Islet Transplantation	Dresden
Italy	IRCCS Ospedale San Raffaele	Milan
Netherlands	Leiden University	Leiden
Norway	Oslo University Hospital	Oslo
Switzerland	Hopiteaux Universitaires de Geneve	Geneve
United Kingdom	Churchill Hospital	Headington	Oxford
United Kingdom	Cardiovascular, Metabolic Medicine and Sciences, King's College London	London
United Kingdom	The Newcastle upon Tyne Hospitals NHS Foundation Trust	Newcastle Upon Tyne
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Northwestern Organ Transplant Center	Chicago	Illinois
United States	University of Chicago	Chicago	Illinois
United States	City of Hope	Duarte	California
United States	University of Wisconsin	Madison	Wisconsin
United States	UHealth Diabetes Research Institute	Miami	Florida
United States	Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania
United States	University of Pittsburgh Medical Center Montefiore	Pittsburgh	Pennsylvania
United States	VCU Medical Center, Richmond	Richmond	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)		From VX-880 infusion to end of study (up to 5 years)
Primary	Part B: Proportion of Participants with Peak C-peptide Greater than or Equal to (=) 100 picomole per liter (pmol^L)		From VX-880 infusion to end of study (up to 5 years)
Primary	Part C: Proportion of Participants Free of Severe Hypoglycemic Events With Either a Glycosylated Hemoglobin (HbA1c) <7.0% or a = 1% Reduction in HbA1c From Baseline		At 1 year after VX-880 infusion
Secondary	Part C: Proportion of Participants who are Insulin Independent		At 1 year after VX-880 infusion
Secondary	Part C: Proportion of Participants with Peak C-peptide = 100 pmol^L		From VX-880 infusion to end of study (up to 5 years)
Secondary	Parts C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)		From VX-880 infusion to end of study (up to 5 years)