Hemophilia ANCT03432520
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
An observational study of Hemophilia A, run by Spark Therapeutics, Inc. at 12 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- SPK-8011 and SPK-8016
- How many people
- 29 participants
- Where
- 12 sitesin 3 countries
- Study dates
- 14 Aug 2018to Dec 2032
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Males with hemophilia A, who received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored study.
Who may be able to join
- 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.
Who cannot join
- Participants are excluded from the study if any of the following criteria apply:
- 1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
Trial locations and contacts
12 sites in 3 countries.
12 sites in 3 countries
United States9 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Pennsylvania State University Milton S. Hershey Medical Center
Hershey, Pennsylvania, 10733
Truman Medical Centers
Kansas City, Missouri, 64108
Mississippi Center for Advanced Medicine
Madison, Mississippi, 39110
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
Australia2 sites
The Alfred Hospital & Monash Medical Centre
Melbourne, Victoria
Royal Prince Alfred Hospital Department of Cell & Molecular Therapies
Sydney, New South Wales, 2050
About this study
Official title: A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
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.
Read the detailed description
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).
- Conditions
- Hemophilia A
- Being tested
- SPK-8011, SPK-8016
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Up to 9 years
Number of Participants with Adverse Events of Special Interest (AESIs)
Measured: Up to 9 years
Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development
Measured: Up to 9 years
Number of Participants with Clinical Laboratory Abnormalities
Measured: Up to 9 years
Number of Participants with Vital Sign Abnormalities
Measured: Up to 9 years
Number of Participants with Physical Exam Abnormalities
Measured: Up to 9 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2018
- Study start14 Aug 2018
- Primary completionDec 2032 (estimated)
- Study completionDec 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spark Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Director, Study director, Spark Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03432520
- Sponsor's protocol ID
- SPK-8011/8016-LTFU
- Status last verified
- Nov 2024
- First submitted
- 24 Jan 2018
- Last updated
- 29 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 34788507
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia A
- ClinicalTrials.gov ID
- NCT03432520
- Acronym / sponsor ID
- SPK-8011/8016-LTFU
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Spark Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Aug 2018 (actual)
- Primary completion
- Dec 2032 (estimated)
- Study completion
- Dec 2032 (estimated)
- First posted
- 14 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This study will follow patients with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any prior Spark-sponsored SPK-8011 or SPK-8016 study. Participants will be followed for up to a total of 10 years post infusion (including the time on the dosing study).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Males with hemophilia A, who received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored study.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (2 listed)
- 1.Unable or unwilling to comply with the schedule of visits and study assessments as described in this study protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SPK-8011Observational long-term safety follow-up study of participants previously treated with SPK-8011 in any Spark-sponsored SPK-8011 study | Genetic | – |
| SPK-8016Observational long-term safety follow-up study of participants previously treated with SPK-8016 in any Spark-sponsored SPK-8016 study | Genetic | – |
Primary outcome
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to 9 years
Number of Participants with Adverse Events of Special Interest (AESIs)
Time frame: Up to 9 years
Incidence of Coagulation Factor VIII (FVIII) Inhibitor Development
Time frame: Up to 9 years
Number of Participants with Clinical Laboratory Abnormalities
Time frame: Up to 9 years
Number of Participants with Vital Sign Abnormalities
Time frame: Up to 9 years
Number of Participants with Physical Exam Abnormalities
Time frame: Up to 9 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2024.
- Derived from this study: PubMed 34788507George 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Director study director
Spark Therapeutics, Inc.
12 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Spark Therapeutics, Inc. at 12 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 29 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Australia, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to 9 years). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03432520, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2024.