NCT03432520

Brief Summary

This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
29

participants targeted

Target at below P25 for all trials

Timeline
80mo left

Started Aug 2018

Longer than P75 for all trials

Geographic Reach
3 countries

12 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress54%
Aug 2018Dec 2032

First Submitted

Initial submission to the registry

January 24, 2018

Completed
21 days until next milestone

First Posted

Study publicly available on registry

February 14, 2018

Completed
6 months until next milestone

Study Start

First participant enrolled

August 14, 2018

Completed
14.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2032

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2032

Last Updated

November 29, 2024

Status Verified

November 1, 2024

Enrollment Period

14.3 years

First QC Date

January 24, 2018

Last Update Submit

November 26, 2024

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (6)

  • Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Up to 9 years

  • Number of Participants with Adverse Events of Special Interest (AESIs)

    Up to 9 years

  • Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development

    Up to 9 years

  • Number of Participants with Clinical Laboratory Abnormalities

    Up to 9 years

  • Number of Participants with Vital Sign Abnormalities

    Up to 9 years

  • Number of Participants with Physical Exam Abnormalities

    Up to 9 years

Interventions

SPK-8011GENETIC

Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study

SPK-8016GENETIC

Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study

Eligibility Criteria

Age18 Years+
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsGenetic males
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Males with hemophilia A, who received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored study.

You may qualify if:

  • Participants are eligible to be included in the study only if all of the following criteria apply:
  • Have received a single intravenous administration of SPK-8011 or SPK-8016 in either Study SPK-8011-101 or SPK-8016-101, respectively.
  • Understand the purpose and risks of the study and provide signed and dated informed consent before undergoing any study-specific procedures.

You may not qualify if:

  • Participants are excluded from the study if any of the following criteria apply:
  • Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Mississippi Center for Advanced Medicine

Madison, Mississippi, 39110, United States

Location

Truman Medical Centers

Kansas City, Missouri, 64108, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Pennsylvania State University Milton S. Hershey Medical Center

Hershey, Pennsylvania, 10733, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Royal Prince Alfred Hospital Department of Cell & Molecular Therapies

Sydney, New South Wales, 2050, Australia

Location

The Alfred Hospital & Monash Medical Centre

Melbourne, Victoria, Australia

Location

St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

Location

Related Publications (1)

  • George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205.

    PMID: 34788507DERIVED

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Clinical Trial Director

    Spark Therapeutics, Inc.

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 24, 2018

First Posted

February 14, 2018

Study Start

August 14, 2018

Primary Completion (Estimated)

December 1, 2032

Study Completion (Estimated)

December 1, 2032

Last Updated

November 29, 2024

Record last verified: 2024-11

Locations

AU(2)CA(1)US(9)