Clinical trial NCT04786262: A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes
Official title: A Phase 1/2/3 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
Overall status: RECRUITING
Phase: PHASE3
Conditions: Diabetes Mellitus, Type 1
Impaired Hypoglycemic Awareness
Severe Hypoglycemia
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2021-03-08
Enrollment: 52
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
timeFrame: From VX-880 infusion to end of study (up to 5 years)
measure: Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)
timeFrame: 1 year after achieving insulin independence
Secondary outcomes:
measure: Parts B and C: Proportion of Participants who are Insulin Independent
timeFrame: At 1 year after VX-880 infusion
measure: Parts B and C: Proportion of Participants Free from SHEs with a Glycosylated Hemoglobin (HbA1c) less than (<) 7.0%
timeFrame: At 1 year after VX-880 infusion
measure: Parts B and C: Change From Baseline in Glycosylated Hemoglobin (HbA1c)
timeFrame: At 1 year after VX-880 infusion
measure: Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year
timeFrame: From VX-880 infusion to end of study (up to 5 years)
measure: Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
timeFrame: From Signing of informed consent to end of study (up to 5 years)
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Miami
country: United States
facility: UHealth Diabetes Research Institute
state: Florida
city: Chicago
country: United States
facility: Northwestern Organ Transplant Center
state: Illinois
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center Montefiore
state: Pennsylvania
city: Dallas
country: United States
facility: Baylor Scott and White Research Institute
state: Texas
city: Richmond
country: United States
facility: VCU Medical Center, Richmond
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
city: Edmonton
country: Canada
facility: University of Alberta, Edmonton
city: Montreal
country: Canada
facility: McGill University Health Centre
city: Toronto
country: Canada
facility: Toronto General Hospital (TGH)
city: Vancouver
country: Canada
facility: Vancouver General Hospital
city: Lille
country: France
facility: CHU Lille
city: Strasbourg
country: France
facility: Centre de recherche en Biomédecine de Strasbourg
city: Dresden
country: Germany
facility: Dresden Center for Islet Transplantation
city: Milan
country: Italy
facility: IRCCS Ospedale San Raffaele
city: Leiden
country: Netherlands
facility: Leiden University
city: Oslo
country: Norway
facility: Oslo University Hospital
city: Geneve
country: Switzerland
facility: Hopiteaux Universitaires de Geneve
city: Headington
country: United Kingdom
facility: Churchill Hospital
state: Oxford
city: Edinburgh
country: United Kingdom
facility: Royal Infirmary of Edinburgh
city: London
country: United Kingdom
facility: Cardiovascular, Metabolic Medicine and Sciences, King's College London
city: Newcastle Upon Tyne
country: United Kingdom
facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust
Study officials:
References: