Lead-in Study to Collect Prospective Efficacy and Safety... | NCT03876301

Studies
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

NCT03876301

Sponsor

Spark Therapeutics, Inc.

Status

Completed

Last Update Posted

Jul 10, 2023Actual

Enrollment

25Actual

Phase

Not provided

Conditions

Blood Coagulation Disorder
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Hemophilia A
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders
Factor VIII Deficiency

Interventions

Standard of Care FVIII Replacement therapy

Countries

United States
Australia
Canada
Thailand

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Jul 2023

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 21, 2019Actual

Primary Completion Date

May 2, 2023Actual

Completion Date

May 2, 2023Actual

First Submitted Date

Mar 11, 2019

First Submission Date that Met QC Criteria

Mar 13, 2019

First Posted Date

Mar 15, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 6, 2023

Last Update Posted Date

Jul 10, 2023Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

25Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Observational Cohort		Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200	Drug: Standard of Care FVIII Replacement therapy

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Standard of Care FVIII Replacement therapy	Drug	There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of bleeding events, annualized	Annualized bleeding rate (ABR)	12 months

Secondary Outcomes

Measure	Description	Time Frame
Dose and total FVIIII consumption	Total FVIII replacement therapy consumption and the corresponding dose	12 months
Annualized number of infusions (AIR)	Number of reported infusions over the study period

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Able and willing to provide written informed consent.
Males ≥18 years of age.
Clinically severe hemophilia A
Previous exposure to FVIII therapy
No prior history of hypersensitivity or anaphylaxis associated with an FVIII or intravenous immunoglobulin administration.
No measurable inhibitor against FVIII
Willing to participate and receive treatment in a future Spark hemophilia A gene therapy study.

Exclusion Criteria:

Documented active hepatitis B or C within the past 12 months of Screening
Currently on antiviral therapy to treat hepatitis B or C;
Documented significant liver disease within the past 6 months of Screening
Have serological evidence of HIV-1 or HIV-2
Anti-AAV-Spark 200 neutralizing titers ≥1:1
Previously received SPK-8011;
Previously dosed with any investigational or approved gene therapy product at any time or treated with an investigational drug within the last 12 weeks;
Planned surgical procedure in the next 12 months requiring FVIII prophylactic treatment.
Any history of chronic infection or other chronic disease, concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) including active malignancy, except for non-melanoma skin cancer, any other condition or any other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the participant unsuitable for participation and dosing in a future clinical study for Spark's hemophilia A gene therapy.
Unable or unwilling to comply with the schedule of visits and/or study assessments described in the protocol.

Accepts Healthy Volunteers

Not provided

Sex

Male

Sex/Gender Based

Yes

Sex/Gender Description

Genetically male

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Adult males with clinically severe hemophilia A (i.e., ≤2% IU/dL FVIII activity level), who meet eligibility criteria for neutralizing antibody (NAb) to AAV-Spark200.

Sampling Method

Non-Probability Sample

Facility	Status	City	State	ZIP	Country	Contacts
University of California San Francisco		San Francisco	California	94117	United States
University of Florida

ID	Term
D006425	Hemic and Lymphatic Diseases
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ID	Term
D001778	Blood Coagulation Disorders
D025861	Blood Coagulation Disorders, Inherited
D020147	Coagulation Protein Disorders
D006467	Hemophilia A
D030342	Genetic Diseases, Inborn
D040181	Genetic Diseases, X-Linked
D006402	Hematologic Diseases
D006474	Hemorrhagic Disorders