SAAVY: A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A

• Sponsor: BioMarin Pharmaceutical (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04560933
• Collaborator: (none)
• Enrollment: 1,000
• Location: 1
• Anticipated Duration (Months): 21.1
• Patients Per Site Per Month: 47.3

Study Details

Study Description

Brief Summary

To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A	Procedure: Blood sample collection

Detailed Description

This is a single-center, decentralized, patient-centered, prospective, observational study utilizing biospecimen samples collected from hemophilia A subjects across the United States to evaluate and characterize seroprevalence and the rate of seroconversion of antibodies against AAV serotypes and exploratory vectors, and to investigate the associated factors that may influence the vector titers.

Relevant medical findings will also be collected from the subject, as well as symptoms related to hemophilia A. The collection of medical history may include major illnesses, diagnoses, and surgeries.

Study Design

• Study Type: Observational
• Anticipated Enrollment: 1000 participants
• Observational Model: Ecologic or Community
• Time Perspective: Prospective
• Official Title: A Prospective, Observational Study Evaluating Seroprevalence and Rate of Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes and Exploratory Vectors in Subjects With Hemophilia A in the United States
• Actual Study Start Date: Aug 25, 2020
• Anticipated Primary Completion Date: Feb 25, 2022
• Anticipated Study Completion Date: May 30, 2022

Outcome Measures

Primary Outcome Measures

1. Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A [Baseline]

   To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline

2. Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A [Change from baseline through Week 12]

Secondary Outcome Measures

1. To describe and characterize AAV vector titer values in subjects with Hemophilia A [Change from baseline through Week 12]

   To describe and characterize AAV vector titer values in subjects with Hemophilia A

2. To describe and characterize AAV vector titer values in subjects with Hemophilia A [Change from baseline through Week 24]

   To describe and characterize AAV vector titer values in subjects with Hemophilia A

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All

Inclusion Criteria:

• Subjects diagnosed with Hemophilia A

• Subjects aged 18years or over at time of entry

Exclusion Criteria:

• Currently participating in an interventional study of any investigational product, device or procedure.

• Subjects who have been previously treated with AAV vector gene therapy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Covance Inc	Madison	Wisconsin	United States	53704

Sponsors and Collaborators

• BioMarin Pharmaceutical

Investigators

• Principal Investigator: Stephen Flach, MD, Covance

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: BioMarin Pharmaceutical
• ClinicalTrials.gov Identifier: NCT04560933
• Other Study ID Numbers: 270-701
• First Posted: Sep 23, 2020
• Last Update Posted: Sep 23, 2020
• Last Verified: Sep 1, 2020

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Hemophilia A

Study Results

No Results Posted as of Sep 23, 2020