Blood Coagulation DisorderNCT03876301
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
An observational study of Blood Coagulation Disorder and Blood Coagulation Disorders, Inherited, run by Spark Therapeutics, Inc. at 11 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- How many people
- 25 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 21 Jan 2019to 2 May 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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
- Adult, Older 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.
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 10Show fewer
- 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.
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
United States6 sites
Emory University
Atlanta, Georgia, 30322
Children's Hospital of Michigan
Detroit, Michigan, 48201
University of Florida
Gainesville, Florida, 32610
Mississippi Center for Advanced Medicine
Madison, Mississippi, 39110
University of California San Francisco
San Francisco, California, 94117
Bloodworks Northwest
Seattle, Washington, 98104
Australia2 sites
The Alfred Hospital
Melbourne, Victoria, 3004
Fiona Stanley Hospital
Murdoch, Western Austrailia, 6150
Canada2 sites
McMaster University / Royal Prince Alfred Hospital
Hamilton, Ontario, L8N3Z5
Providence Hematology/St. Paul's Hosptial
Vancouver, British Columbia, V621Y6
About this study
Official title: A Multi-Center, Observational Study in Males With Hemophilia A
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.
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A, Genetic Diseases, Inborn, Genetic Diseases, X-Linked
- Being tested
- Standard of Care FVIII Replacement therapy
- Keywords
- Factor VIII, Factor VIII Protein, Recombinant
- Medical subject headings
- Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Hematologic Diseases, Hemorrhagic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard of Care FVIII Replacement therapyDrug
There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.
| Study group | Role | What they receive |
|---|---|---|
| Observational Cohort | Adult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200 Receives: Drug: Standard of Care FVIII Replacement therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of bleeding events, annualized
Annualized bleeding rate (ABR)
Measured: 12 months
Secondary outcomes (2)
Dose and total FVIIII consumption
Total FVIII replacement therapy consumption and the corresponding dose
Measured: 12 months
Annualized number of infusions (AIR)
Number of reported infusions over the study period
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2019
- Study start21 Jan 2019
- Primary completion2 May 2023
- Study completion2 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Blood Coagulation DisorderStudies 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
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spark Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03876301
- Sponsor's protocol ID
- SPK-8011-301
- Status last verified
- Jul 2023
- First submitted
- 11 Mar 2019
- Last updated
- 10 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Observational Study in Males With Hemophilia A
- ClinicalTrials.gov ID
- NCT03876301
- Acronym / sponsor ID
- SPK-8011-301
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A, Genetic Diseases, Inborn, Genetic Diseases, X-Linked
- Keywords
- Factor VIII, Factor VIII Protein, Recombinant
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Spark Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Jan 2019 (actual)
- Primary completion
- 2 May 2023 (actual)
- Study completion
- 2 May 2023 (actual)
- First posted
- 15 Mar 2019 (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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational Cohort | – | Drug: Standard of Care FVIII Replacement therapyAdult males with clinically severe hemophilia A, who are negative for neutralizing antibody (NAb) to AAV-Spark200 |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Age groups
- Adult, Older 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.
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (10 listed)
- 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
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 |
|---|---|---|
| Standard of Care FVIII Replacement therapyThere is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy. | Drug | Observational Cohort |
Primary outcome
Number of bleeding events, annualized
Time frame: 12 months
Annualized bleeding rate (ABR)
2 secondary outcomes
Dose and total FVIIII consumption
Time frame: 12 months
Annualized number of infusions (AIR)
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2023.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Blood Coagulation Disorder and Blood Coagulation Disorders, Inherited, run by Spark Therapeutics, Inc. at 11 sites in 4 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 7 inclusion and 10 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: finished, as of the sponsor's last update on 10 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Australia, Canada, Thailand. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 bleeding events, annualized (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Jul 2023.