HemophiliaNCT02546622
ATHN 2: Factor Switching Study
An observational study of Hemophilia, run by American Thrombosis and Hemostasis Network at 27 sites in United States.
Trial summary
This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia
- What is being tested?
- Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013 and Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
- How many people
- 310 participants
- Where
- 27 sitesUnited States
- Study dates
- Sep 2015to 30 Jun 2020
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study will enroll approximately 600 patients with hemophilia who meet the eligibility criteria and are receiving care from one of the ATHN-affiliated Hemophilia Treatment Centers (HTC). There will be 2 arms: Arm A (Prospective) will include patients who are switching factor replacement products and will be followed prospectively for up to 1 year. Arm B (Retrospective) will include patients who have switched factor replacement products previously (within the past 50 weeks at the time of enrollment). These patients will be assessed retrospectively and/or followed prospectively for up to 1 year.
Who may be able to join
- Moderate or Severe Congenital Hemophilia A or B (FVIII or FIX clotting activity less than or equal to 5% of normal).
- Able to give informed consent (by patient or parent/authorized guardian).
- Previously treated with plasma-derived or recombinant clotting factor replacement products with at least 50 exposure days (as assessed either from direct clinical records in children under age 5, or by clinical history of dosing in older patients). For Arm B being enrolled retrospectively, this previous treatment must be prior to product switch under study.
- Planning to switch, or recently switched within the previous 50 weeks, to a new brand or type of replacement factor VIII or IX, FDA approved after January 1, 2013.
- Arm B only: Negative inhibitor screen within the last 6 months prior to switching.
- Note: History of prior transient inhibitor or inhibitor eradicated by immune tolerance induction (ITI) are eligible.
Who cannot join
- Presence of any known bleeding disorder other than hemophilia A or B (i.e., patients with concurrent hemophilia and a second hemostatic defect are NOT eligible). Low Von Willebrand Factor (VWF) without VWF diagnosis are not excluded.
- Presence of an active inhibitor >0.6 BU for factor VIII, > 0.4 BU for factor IX at the time of eligibility assessment. Detection of such an inhibitor at the baseline visit prior to dosing with the new product (Arm A), or after dosing with new factor dosing (Arm B), would result in early termination without other study assessments.
- Currently undergoing ITI.
- Immunosuppressive therapy (cyclophosphamide, mycophenolate, IVIG) within 90 days and Rituximab within 6 months; topical steroid treatments and short course steroids for asthma or allergy allowed.
- Previous participation in Phase I, II or III interventional trials of the factor product being switched to.
Trial locations and contacts
27 sites in 1 country.
27 sites in 1 country
United States27 sites
University of Michigan Hemophilia and Coagulation Disorders Program
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta/Emory
Atlanta, Georgia, 30322
University of Colorado Denver Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
Johns Hopkins University Medical Center
Baltimore, Maryland, 21205
Boston Hemophilia Center at Children's Hospital of Boston
Boston, Massachusetts, 02115
UTSW Medical Center at Dallas/Children's Medical Center
Dallas, Texas, 75235
Michigan State University Center for Bleeding and Clotting Disorders
East Lansing, Michigan, 48823
Indiana Hemophilia and Thrombosis Center (IHTC)
Indianapolis, Indiana, 46260
Children's Mercy Hospital
Kansas City, Missouri, 64108
Dartmouth-Hitchcock Comprehensive Hemophilia and Thrombosis Center
Lebanon, New Hampshire, 03756
St Jude Children's Research Hospital
Memphis, Tennessee, 38105
Blood Center of Wisconsin
Milwaukee, Wisconsin, 53201
Yale Hemophilia Treatment Center
New Haven, Connecticut, 06520
Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, 70112
Weill Cornell Medicine
New York, New York, 10065
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
Pennsylvania Comprehensive Hemophilia and Thrombophilia Program / Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
The Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
Oregon Health and Science University
Portland, Oregon, 97239
Mary M. Gooley Hemophilia Center
Rochester, New York, 14621
University of California San Diego (UCSD)
San Diego, California, 92122
Maine Hemophilia and Thrombosis Center
Scarborough, Maine, 04074
Washington Center for Bleeding Disorders Bloodworks Northwest d/b/a Puget Sound Blood Center
Seattle, Washington, 98104
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
St. Joseph's Children's Hospital
Tampa, Florida, 33607
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Factor Replacement Products
This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will be recruited at Hemophilia Treatment Centers (HTC) which are ATHN-affiliates. The primary outcome being studied is the development of inhibitor (i.e., antibodies to factor) at 1 year or 50 exposure days, whichever comes first.
The study will be conducted at approximately 30 HTCs, with a planned enrollment of 600 patients.The entire study duration is projected to be approximately 6 years.
In addition, optional substudies will be included for some products, as "Product-Specific Modules". These will be questionnaires to collect data for subjects receiving selected Factor products. For example, subjects receiving Kovaltry will be approached to participate in the 'Kovaltry Product-Specific Module'; subjects receiving Adynovate will be approached to participate in the 'Adynovate Product-Specific Module'. Questions will be related to product use, perceptions of product use, and other post-marketing consumer data.
Read the detailed description
This non-interventional, minimal risk cohort study will enroll patients with Hemophilia A or B who are planning or have recently switched to a new Factor product. The study will have 2 Arms, prospective and retrospective. The Prospective Arm will enroll patients who plan to switch to a new factor. The Retrospective Arm will enroll patients who have recently switched to a new factor (within the previous 50 weeks). Patient will be seen at baseline and for up to 4 additional visits, and quarterly follow-up by phone. Required study visits will be planned to coincide with routine follow-up visits whenever possible.
Please note that Factor Replacement Products are not being provided by the study.
The primary objective is to assess and characterize the rate of inhibitor development within one (1) year or fifty (50) exposure days, whichever is first, after switching clotting factor replacement products in previously treated patients (PTPs) with hemophilia A or B.
Data collected will include eligibility, demographics, medical history, hemophilia history (clotting history, product history, genotype and family history), inhibitor history, co-morbidities at baseline (i.e., HIV, Hepatitis C.), detailed clotting factor replacement product(s) usage and switching plan, and reasons for switching factor products. Also targeted physical exams will be performed at baseline and during follow-up, and targeted concomitant medication data will be collected. Data collection will also include patient-reported outcome(s) after 1 year, bleeding events, surgeries, laboratory Inhibitor testing and details regarding testing methodology, pharmacokinetic (PK) data (if known), new diagnoses, and co-morbidities (targeted), Safety/Adverse Events using European Union Hemophilia Safety Surveillance (EUHASS) definitions.
This study will evolve to include any newly approved (since January 2013) factors as they come to market. Cohorts will be defined by the brand/type of new clotting factor replacement product approved after January 1, 2013. The current list of specific new Factor VIII replacement products include Eloctate® (Bioverativ) and NovoEight® (NovoNordisk); Factor IX replacement products include Alprolix® (Bioverativ), Rixubis® (Baxalta), and IXinity® (Emergent Biosolutions). Others are both now available and imminent and include: Adynovate®, Idelvion®, Afstyla®, Kovaltry® and Jivi®.
The over-arching rationale for this protocol is that a pragmatic study which is consistent with real world practices across a wide range of patients that is not principally tied to a particular manufacturer or product may be of great advantage to the entire hemophilia community.
Study Duration
- Subjects on prophylaxis will be followed on study for up to 1 year. Each subject will be seen during a study visit or contacted by telephone at least once every 3 months (i.e., quarterly). Patients may participate for multiple 'cycles', if they switch factor products more than once while the study is actively recruiting.
Treatment regimen will be at the discretion of the subject's hemophilia caregivers. No treatment is being provided by the study.
- Substudies A number of substudies are planned with pharmaceutical sponsors to collect information from patients about their products' use. Participation in these optional substudies (product-specific modules) will be planned to coincide with study visits. These modules will collect information from subjects about their perception and use of factor use/treatment, physical activity levels and other general health questions. These data will be collected via questionnaire, primarily via phone.
Concomitant and Excluded Therapies
- Immune tolerance therapy is excluded on study. This includes immunosuppressive treatments used to eradicate inhibitors. Steroid treatments for allergic disorders and asthma, are allowed.
Data Collection System
- All data collected will be entered into electronic case report forms (eCRFs) within the secure ATHN Study Manager system. Subject Identifiers (IDs) will be generated in Clinical Manager.
- Reimbursement will be managed by each participating HTC. Most study centers will reimburse study subjects for travel and parking, but this varies by center.
- Conditions
- Hemophilia
- Being tested
- Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013, Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
- Keywords
- Factor, Coagulation, Inhibitor, HTC, Bleeding
- Medical subject headings
- Hemophilia A, Thrombosis, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013Biological
Also called Eloctate®, NovoEight®, Adynovate®, Nuwiq®
Prophylaxis for prevention of bleeding, various regimens.
Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013Biological
Also called Alprolix®, Rixubis®, IXinity®, Idelvion®
Prophylaxis for prevention of bleeding, various regimens.
| Study group | Role | What they receive |
|---|---|---|
| Arm A Prospective | Patients who are switching to a new Factor VIII and Factor IX Replacement Product for Hemophilia A and B which was FDA approved after January 1, 2013.
These patients will be followed prospectively for up to 1 year. Receives: Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013; Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013 | |
| Arm B Retrospective | Patients who have recently switched to a new Factor VIII and Factor IX Replacement Product for Hemophilia A and B which was FDA approved after January 1, 2013.
Patients must have switched products within the past 50 weeks at the time of enrollment.
These patients will be assessed retrospectively and/or followed prospectively for up to 1 year. Receives: Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013; Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Inhibitor Development
Inhibitor development is the primary outcome. Subjects will be followed closely and tested at baseline, after 10 Exposure Days and 50 Exposure Days, and/or at 1 year. Specimens will be submitted to local laboratories and evaluated for inhibitor titers, and inhibitors will be confirmed by local laboratories and CDC.
Measured: After 50 Exposure Days or 1 Year, whichever comes first
Secondary outcomes (4)
Inhibitor Development
To determine the prospective incidence of inhibitor development after 10 exposure days to a new, novel recombinant factor following a switch from another clotting factor replacement product.
Measured: 10 days
Prevalence of risk factors for inhibitor development
To compare the prevalence of selected risk factors in patients with hemophilia who develop inhibitors following switching to a new product, to those found in subjects who do not develop inhibitors.
Measured: 1 year
Targeted post-marketing approval safety and efficacy data
To collect and summarize targeted post-marketing approval safety and efficacy data for events related to clotting factor replacement products, specifically
- To collect data on bleeding events following switching factor.
- To summarize replacement factor product dosing regimens prescribed to the study population.
Measured: 1 year
Platform for additional substudies
To serve as a platform for product-specific questionnaires in cohorts of patients who switch to a particular product.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2015 (estimated)
- Study startSep 2015
- Primary completion30 Jun 2020
- Study completion30 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for HemophiliaStudies 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
- Other
- Enrolment
- 310 participants (actual)
Sponsor and investigators
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Sponsor type
- Research network
- Collaborators
- Shire, CSL Behring, Bioverativ Therapeutics Inc., Bayer
- Responsible party
- Sponsor
- Investigators
- Ellis J Neufeld, MD, PhD, Principal investigator, St. Jude Children's Research HospitalJanna Journeycake, MD, Principal investigator, Oklahoma Center for Bleeding and Clotting Disorders
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02546622
- Sponsor's protocol ID
- ATHN 2
- Status last verified
- Aug 2020
- First submitted
- 9 Sep 2015
- Last updated
- 9 Dec 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Product-Specific Module data for subjects enrolled in each module will be shared with each sponsor, as appropriate.
Publications
Josephson CD, Abshire T. The new albumin-free recombinant factor VIII concentrates for treatment of hemophilia: do they represent an actual incremental improvement? Clin Adv Hematol Oncol. 2004 Jul;2(7):441-6.
PubMed 16163220Powell JS. Lasting power of new clotting proteins. Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):355-63. doi: 10.1182/asheducation-2014.1.355. Epub 2014 Nov 18.
PubMed 25696879Ragni, MV, Kessler, CM, and Lozier, JN (2009). Clinical aspects and therapy for hemophilia, in Hoffman R, Benz EJ, Shattil, SJ et al eds, Hematology, Basic Principles and Practice, 5th Edition, Churchill Livingstone, Philadelphia, pp 1911-1930.
Abshire TC, Brackmann HH, Scharrer I, Hoots K, Gazengel C, Powell JS, Gorina E, Kellermann E, Vosburgh E. Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy--International Kogenate-FS Study Group. Thromb Haemost. 2000 Jun;83(6):811-6.
PubMed 10896230Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13.
PubMed 24227821
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Factor Replacement Products
- ClinicalTrials.gov ID
- NCT02546622
- Acronym / sponsor ID
- ATHN 2
- Conditions
- Hemophilia
- Keywords
- Factor, Coagulation, Inhibitor, HTC, Bleeding
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 310 participants (actual)
- Lead sponsor
- American Thrombosis and Hemostasis NetworkResearch network
- Collaborators
- Shire, CSL Behring, Bioverativ Therapeutics Inc., Bayer
- Study start
- Sep 2015
- Primary completion
- 30 Jun 2020 (actual)
- Study completion
- 30 Jun 2020 (actual)
- First posted
- 11 Sep 2015 (estimated)
- 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 is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products. The study will follow each patient for up to 1 year. Patients will be recruited at Hemophilia Treatment Centers (HTC) which are ATHN-affiliates. The primary outcome being studied is the development of inhibitor (i.e., antibodies to factor) at 1 year or 50 exposure days, whichever comes first.
The study will be conducted at approximately 30 HTCs, with a planned enrollment of 600 patients.The entire study duration is projected to be approximately 6 years.
In addition, optional substudies will be included for some products, as "Product-Specific Modules". These will be questionnaires to collect data for subjects receiving selected Factor products. For example, subjects receiving Kovaltry will be approached to participate in the 'Kovaltry Product-Specific Module'; subjects receiving Adynovate will be approached to participate in the 'Adynovate Product-Specific Module'. Questions will be related to product use, perceptions of product use, and other post-marketing consumer data.
Detailed description
This non-interventional, minimal risk cohort study will enroll patients with Hemophilia A or B who are planning or have recently switched to a new Factor product. The study will have 2 Arms, prospective and retrospective. The Prospective Arm will enroll patients who plan to switch to a new factor. The Retrospective Arm will enroll patients who have recently switched to a new factor (within the previous 50 weeks). Patient will be seen at baseline and for up to 4 additional visits, and quarterly follow-up by phone. Required study visits will be planned to coincide with routine follow-up visits whenever possible.
Please note that Factor Replacement Products are not being provided by the study.
The primary objective is to assess and characterize the rate of inhibitor development within one (1) year or fifty (50) exposure days, whichever is first, after switching clotting factor replacement products in previously treated patients (PTPs) with hemophilia A or B.
Data collected will include eligibility, demographics, medical history, hemophilia history (clotting history, product history, genotype and family history), inhibitor history, co-morbidities at baseline (i.e., HIV, Hepatitis C.), detailed clotting factor replacement product(s) usage and switching plan, and reasons for switching factor products. Also targeted physical exams will be performed at baseline and during follow-up, and targeted concomitant medication data will be collected. Data collection will also include patient-reported outcome(s) after 1 year, bleeding events, surgeries, laboratory Inhibitor testing and details regarding testing methodology, pharmacokinetic (PK) data (if known), new diagnoses, and co-morbidities (targeted), Safety/Adverse Events using European Union Hemophilia Safety Surveillance (EUHASS) definitions.
This study will evolve to include any newly approved (since January 2013) factors as they come to market. Cohorts will be defined by the brand/type of new clotting factor replacement product approved after January 1, 2013. The current list of specific new Factor VIII replacement products include Eloctate® (Bioverativ) and NovoEight® (NovoNordisk); Factor IX replacement products include Alprolix® (Bioverativ), Rixubis® (Baxalta), and IXinity® (Emergent Biosolutions). Others are both now available and imminent and include: Adynovate®, Idelvion®, Afstyla®, Kovaltry® and Jivi®.
The over-arching rationale for this protocol is that a pragmatic study which is consistent with real world practices across a wide range of patients that is not principally tied to a particular manufacturer or product may be of great advantage to the entire hemophilia community.
Study Duration
- Subjects on prophylaxis will be followed on study for up to 1 year. Each subject will be seen during a study visit or contacted by telephone at least once every 3 months (i.e., quarterly). Patients may participate for multiple 'cycles', if they switch factor products more than once while the study is actively recruiting.
Treatment regimen will be at the discretion of the subject's hemophilia caregivers. No treatment is being provided by the study.
- Substudies A number of substudies are planned with pharmaceutical sponsors to collect information from patients about their products' use. Participation in these optional substudies (product-specific modules) will be planned to coincide with study visits. These modules will collect information from subjects about their perception and use of factor use/treatment, physical activity levels and other general health questions. These data will be collected via questionnaire, primarily via phone.
Concomitant and Excluded Therapies
- Immune tolerance therapy is excluded on study. This includes immunosuppressive treatments used to eradicate inhibitors. Steroid treatments for allergic disorders and asthma, are allowed.
Data Collection System
- All data collected will be entered into electronic case report forms (eCRFs) within the secure ATHN Study Manager system. Subject Identifiers (IDs) will be generated in Clinical Manager.
- Reimbursement will be managed by each participating HTC. Most study centers will reimburse study subjects for travel and parking, but this varies by center.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A Prospective | – | Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013, Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013Patients who are switching to a new Factor VIII and Factor IX Replacement Product for Hemophilia A and B which was FDA approved after January 1, 2013. These patients will be followed prospectively for up to 1 year. |
| Arm B Retrospective | – | Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013, Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013Patients who have recently switched to a new Factor VIII and Factor IX Replacement Product for Hemophilia A and B which was FDA approved after January 1, 2013. Patients must have switched products within the past 50 weeks at the time of enrollment. These patients will be assessed retrospectively and/or followed prospectively for up to 1 year. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will enroll approximately 600 patients with hemophilia who meet the eligibility criteria and are receiving care from one of the ATHN-affiliated Hemophilia Treatment Centers (HTC). There will be 2 arms: Arm A (Prospective) will include patients who are switching factor replacement products and will be followed prospectively for up to 1 year. Arm B (Retrospective) will include patients who have switched factor replacement products previously (within the past 50 weeks at the time of enrollment). These patients will be assessed retrospectively and/or followed prospectively for up to 1 year.
Key inclusion criteria (6 listed)
- Moderate or Severe Congenital Hemophilia A or B (FVIII or FIX clotting activity less than or equal to 5% of normal).
- Able to give informed consent (by patient or parent/authorized guardian).
- Previously treated with plasma-derived or recombinant clotting factor replacement products with at least 50 exposure days (as assessed either from direct clinical records in children under age 5, or by clinical history of dosing in older patients). For Arm B being enrolled retrospectively, this previous treatment must be prior to product switch under study.
- Planning to switch, or recently switched within the previous 50 weeks, to a new brand or type of replacement factor VIII or IX, FDA approved after January 1, 2013.
- Arm B only: Negative inhibitor screen within the last 6 months prior to switching.
Key exclusion criteria (5 listed)
- Presence of any known bleeding disorder other than hemophilia A or B (i.e., patients with concurrent hemophilia and a second hemostatic defect are NOT eligible). Low Von Willebrand Factor (VWF) without VWF diagnosis are not excluded.
- Presence of an active inhibitor >0.6 BU for factor VIII, > 0.4 BU for factor IX at the time of eligibility assessment. Detection of such an inhibitor at the baseline visit prior to dosing with the new product (Arm A), or after dosing with new factor dosing (Arm B), would result in early termination without other study assessments.
- Currently undergoing ITI.
- Immunosuppressive therapy (cyclophosphamide, mycophenolate, IVIG) within 90 days and Rituximab within 6 months; topical steroid treatments and short course steroids for asthma or allergy allowed.
- Previous participation in Phase I, II or III interventional trials of the factor product being switched to.
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 |
|---|---|---|
| Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013Also: Eloctate®, NovoEight®, Adynovate®, Nuwiq®Prophylaxis for prevention of bleeding, various regimens. | Biological | Arm A Prospective, Arm B Retrospective |
| Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013Also: Alprolix®, Rixubis®, IXinity®, Idelvion®Prophylaxis for prevention of bleeding, various regimens. | Biological | Arm A Prospective, Arm B Retrospective |
Primary outcome
Inhibitor Development
Time frame: After 50 Exposure Days or 1 Year, whichever comes first
Inhibitor development is the primary outcome. Subjects will be followed closely and tested at baseline, after 10 Exposure Days and 50 Exposure Days, and/or at 1 year. Specimens will be submitted to local laboratories and evaluated for inhibitor titers, and inhibitors will be confirmed by local laboratories and CDC.
4 secondary outcomes
Inhibitor Development
Time frame: 10 days
Prevalence of risk factors for inhibitor development
Time frame: 1 year
Targeted post-marketing approval safety and efficacy data
Time frame: 1 year
Platform for additional substudies
Time frame: 1 year
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 9 Dec 2021.
- Background: PubMed 16163220Josephson CD, Abshire T. The new albumin-free recombinant factor VIII concentrates for treatment of hemophilia: do they represent an actual incremental improvement? Clin Adv Hematol Oncol. 2004 Jul;2(7):441-6.
- Background: PubMed 25696879Powell JS. Lasting power of new clotting proteins. Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):355-63. doi: 10.1182/asheducation-2014.1.355. Epub 2014 Nov 18.
- BackgroundRagni, MV, Kessler, CM, and Lozier, JN (2009). Clinical aspects and therapy for hemophilia, in Hoffman R, Benz EJ, Shattil, SJ et al eds, Hematology, Basic Principles and Practice, 5th Edition, Churchill Livingstone, Philadelphia, pp 1911-1930.
- Background: PubMed 10896230Abshire TC, Brackmann HH, Scharrer I, Hoots K, Gazengel C, Powell JS, Gorina E, Kellermann E, Vosburgh E. Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy--International Kogenate-FS Study Group. Thromb Haemost. 2000 Jun;83(6):811-6.
- Background: PubMed 24227821Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13.
- Individual participant dataSharing planned: Yes. Product-Specific Module data for subjects enrolled in each module will be shared with each sponsor, as appropriate.
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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.
Ellis J Neufeld, MD, PhD principal investigator
St. Jude Children's Research Hospital
Janna Journeycake, MD principal investigator
Oklahoma Center for Bleeding and Clotting Disorders
27 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia, run by American Thrombosis and Hemostasis Network at 27 sites in United States.
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 6 inclusion and 5 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 9 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States. 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?
Inhibitor Development (time frame: After 50 Exposure Days or 1 Year, whichever comes first). 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 NCT02546622, which also lists 5 linked publications.
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 9 Dec 2021; AzTrials last refreshed its copy on 27 Oct 2026.