Alpha1-Antitrypsin Deficiency Clinical Trial
Official title:
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
| Verified date | May 2022 |
| Source | Vertex Pharmaceuticals Incorporated |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
| Status | Completed |
| Enrollment | 44 |
| Est. completion date | May 4, 2021 |
| Est. primary completion date | May 4, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 80 Years |
| Eligibility | Key Inclusion Criteria: - Subjects must have a PiZZ genotype confirmed at screening - Plasma AAT levels indicating severe deficiency at screening Key Exclusion Criteria: - History of a medical condition that could negatively impact the ability to complete the study - Solid organ, or hematological transplantation or is currently on a transplant list - History of use of gene therapy or RNAi therapy at any time previously Other protocol defined Inclusion/Exclusion criteria may apply |
| Country | Name | City | State |
|---|---|---|---|
| Canada | Queen Elizabeth II Health Sciences Center | Halifax | |
| Canada | Inspiration Research Ltd | Toronto | |
| Germany | University Hospital RWTH Aachen | Aachen | |
| Germany | Universitätsklinikum Essen | Essen | |
| Germany | Medizinische Hochschule Hannover | Hannover | |
| Ireland | Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital | Beaumont | |
| Sweden | Skanes Universitetssjukhus, Malmo | Malmö | |
| United Kingdom | Staploe Medical Centre | Soham Ely | |
| United States | University of Alabama at Birmingham | Birmingham | Alabama |
| United States | University of North Carolina Medical Center | Chapel Hill | North Carolina |
| United States | Medical University of South Carolina | Charleston | South Carolina |
| United States | University of Cincinnati | Cincinnati | Ohio |
| United States | National Jewish Health | Denver | Colorado |
| United States | Inova Fairfax Medical Campus | Falls Church | Virginia |
| United States | University of Florida | Gainesville | Florida |
| United States | Blessing Corporate Services, Inc., dba Blessing Health System | Hannibal | Missouri |
| United States | Renovatio Clinical | Houston | Texas |
| United States | The University of Iowa Hospitals and Clinics | Iowa City | Iowa |
| United States | University of Kansas Medical Center | Kansas City | Kansas |
| United States | David Geffen School of Medicine at UCLA | Los Angeles | California |
| United States | University of Miami Miller School of Medicine | Miami | Florida |
| United States | University of Minnesota | Minneapolis | Minnesota |
| United States | Central Florida Pulmonary | Orlando | Florida |
| United States | Temple University Hospital | Philadelphia | Pennsylvania |
| United States | Oregon Health & Science University | Portland | Oregon |
| United States | University of California Davis Medical Center | Sacramento | California |
| United States | Cardinal Glennon Children's Hospital - St. Louis University | Saint Louis | Missouri |
| United States | University of Utah Health | Salt Lake City | Utah |
| United States | The University of Texas Health Science Center at Tyler | Tyler | Texas |
| United States | Wake Forest University Baptist Medical Center | Winston-Salem | North Carolina |
| Lead Sponsor | Collaborator |
|---|---|
| Vertex Pharmaceuticals Incorporated |
United States, Canada, Germany, Ireland, Sweden, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels | From Baseline at Day 28 | ||
| Primary | Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 8 | ||
| Secondary | Change in Plasma Antigenic AAT Levels | From Baseline at Day 28 | ||
| Secondary | Observed Pre-dose Plasma Concentration (Ctrough) of VX-864 | Pre-dose at Day 7, Day 14, Day 21 and Day 28 |
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