Hemophilia A With InhibitorATHN 7NCT03619863
ATHN 7: Hemophilia Natural History Study
An observational study of Hemophilia A With Inhibitor and Haemophilia A Without Inhibitor, run by American Thrombosis and Hemostasis Network at 29 sites in United States.
Trial summary
This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor
- How many people
- 395 participants
- Where
- 29 sitesUnited States
- Study dates
- 24 Oct 2018to 31 Mar 2024
- How long will I take part?
- 4 Years
- 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 280 patients with hemophilia who meet the eligibility criteria and receiving care from one of the ATHN-affiliated Hemophilia Treatment Centers (HTC).
Who may be able to join
- Congenital hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility;
- Able to give informed consent (by patient or parent/authorized guardian); and
- Co-enrollment in the ATHNdataset.
Who cannot join
- Presence of any known bleeding disorder other than congenital hemophilia A or B;
- Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and
- Unable or unwilling to comply with the study protocol.
Trial locations and contacts
29 sites in 1 country.
29 sites in 1 country
United States29 sites
Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta
Atlanta, Georgia, 30308
Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center
Boston, Massachusetts, 02114
Rush University Medical Center
Chicago, Illinois, 60612
The Center for Cancer and Blood Disorders, Children's Medical Center of Dallas
Dallas, Texas, 75235
Michigan State University Center for Bleeding and Clotting Disorders
East Lansing, Michigan, 48823
University of Florida Hemophilia Treatment Center
Gainesville, Florida, 32610
Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030
Indiana Hemophilia and Thrombosis Center (IHTC)
Indianapolis, Indiana, 46260
Children's Mercy Hospital - Kansas City
Kansas City, Missouri, 64108
Hemostasis and Thrombosis Center of Nevada
Las Vegas, Nevada, 89178
Orthopaedic Institute for Children HTC
Los Angeles, California, 90007
Orthopedic Institute for Children Hemophilia Program
Los Angeles, California, 90007
St Jude Children's Research Hospital
Memphis, Tennessee, 38105
Comprehensive Center for Bleeding Disorders
Milwaukee, Wisconsin, 53226
Louisiana Center for Bleeding and Clotting Disorders, Tulane University
New Orleans, Louisiana, 70112
Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, 70112
Weill Cornell Medicine
New York, New York, 10065
Newark Beth Israel Medical Center
Newark, New Jersey, 07122
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital
Phoenix, Arizona, 85016
The Hemophilia Center at Oregon Health & Science University
Portland, Oregon, 97239
Hemostasis and Thrombosis Center of Nevada
Reno, Nevada, 89509
Rady Children's Hospital San Diego
San Diego, California, 92123
Maine Hemophilia and Thrombosis Center
Scarborough, Maine, 04074
The John Bouhasin Center for Children with Bleeding Disorders
St Louis, Missouri, 63104
Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital
Toledo, Ohio, 43606
Wake Forest University Health Science
Winston-Salem, North Carolina, 27157
About this study
Official title: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia
This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. The hemophilia treatment center (HTC) physician and participant will decide on the FDA-approved treatment to be used which may include non-factor products, bypassing agents, or clotting factor replacement products. The goal of this research is to study the use of hemophilia treatment products and their outcomes.
Read the detailed description
This non-interventional, minimal risk cohort study will enroll and follow patients with hemophilia A or B as they receive hemophilia treatment for 4 years. This is a pragmatic study of real-world practices across a wide range of patients which will be ongoing as new treatment products receive FDA approval and will be advantageous to the entire hemophilia community. The total study duration is planned for 6 years.
The patients are seen at baseline, annually, and at study exit. Patients will also receive routine quarterly follow-up phone calls from HTC staff to review medical history, bleed events, and product treatment history. Other visits for unplanned events or for the change of treatment product will be scheduled as necessary. All required study visits will be planned to coincide with routine clinical care whenever possible. Co-enrollment in the ATHNdataset by patients is required to participate in the study.
Please note - the treatment regimen will be at the discretion of the patients' hemophilia caregivers. No treatment products are being provided by the study nor will the participants be paid. However, inhibitor titer testing will be provided at no cost to patients by the Center for Disease Control and Prevention (CDC).
The primary objective is to determine the safety of non-factor products, bypassing agents or clotting factor replacement products when used for people with hemophilia with or without inhibitors. Safety will be measured by those events listed in the European Haemophilia Safety Surveillance (EUHASS).
Data collected will include eligibility, demographics, medical history, hemophilia history (genotype and family history), inhibitor history and immune tolerance induction (ITI) treatment regimen (if applicable), co-morbidities at baseline (i.e., HIV, Hepatitis C), detailed treatment product(s) usage, bleeding events, surgical procedures, and EUHASS adverse events and other adverse events of special interest. Data collection will also include patient-reported outcome questionnaires regarding health-related quality of life, treatment use and patient perceptions of treatment products.
Sub-studies
A number of sub-studies are planned with pharmaceutical sponsors to collect information from patients about their specific product use. Participation in these sub-studies (Product Specific Modules) is optional and sub-study visits will be planned to coincide with HTC visits. The modules will collect information from patients about their perception and use of treatment products, physical activity levels and other general health questions. This data will be collected via questionnaire.
Data Collection System
All data collected will be entered into electronic case report forms (eCRFs) within the secure ATHN System by HTC site personnel. All participating study sites will have in place a current, executed Data Use and Business Associate Agreement (DUBAA) with ATHN.
- Conditions
- Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor
- Keywords
- Hemophilia, Non-Factor Products, Bypassing Agents, Clotting Factor Replacement Products
- Medical subject headings
- Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of treatment products used for hemophilia care will be assessed based on the number of reportable European Haemophilia Safety Surveillance (EUHASS) events documented on the ATHN Adverse Event Module Form.
All treatment-related reportable adverse events will be documented on the ATHN Adverse Event Module Form based on EUHASS reportable events which include: death, factor inhibitor development, venous thrombosis, allergic reactions, treatment-emergent side effects, new malignancies, cardiovascular events, and blood-borne infections. Other treatment-related events to be documented on the ATHN Adverse Event Module Form including thrombotic microangiopathies, injection site reactions, drug-induced liver injury and anti-drug antibodies.
Measured: 6 years
Secondary outcomes (5)
Effectiveness of non-factor products, bypassing agents and clotting factor replacement products will be evaluated based on the participant's number of bleeding events reported as the annualized bleeding rate (ABR).
Annualized bleeding rates (bleeds/year) are calculated as the number of bleeding events divided by length of time of the treatment regimen, in years. Participants will report bleeding including spontaneous bleeding, traumatic bleeding, joint related and non-joint bleeding during routine quarterly follow-up with hemophilia care providers. Participants will also have blood loss associated with surgical procedures documented through medical chart review as noted by the surgical care providers. All bleeding data will be gathered and reported.
Measured: 6 years
Dosing regimens for hemophilia treatment products and total amount utilized by the study participant for prophylaxis and treatment of bleeds will be assessed.
The routine quarterly contact by hemophilia care providers with study participants will be used to evaluate their treatment use and will document dosing regimens of hemophilia treatment products, amount of product used, number of participants switching to a different product, number of participants switching between factor and non-factor products, and/or number of participants staying on the current treatment product and the reason for their choice of treatment.
Measured: 6 years
Target joint monitoring
The number and location of target joints at study entry, incidence of target joint development while on study, and the number of target joints that resolve following study enrollment will be documented and analyzed.
The number and location of target joints at study entry, incidence of target joint development while on study, and the number of target joints that resolve following study enrollment will be documented and analyzed.
Measured: 6 years
Efficacy of treatment is rated by health-related outcomes tools: EQ-5D-5L, Patient-Reported Outcomes Measurement Information System (PROMIS), Global Adherence Rating (GAR), and Treatment Satisfaction with Medicines Questionnaire (SATMED-Q).
EuroQol Group's EQ-5D-5L assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Opinions in the 5 categories can be stated as a health profile or converted to a single summary index number. Opinion on overall health is on a scale of 0 to 100 with 100 as best health imagined. PROMIS Profile 29 (adults)/ 25 (peds)/ Parent Proxy 25 assess physical function, anxiety, depression, fatigue, sleep disturbance, ability to participate in social roles/activities, and pain. Questions are ranked on a 5-point Likert Scale and one question on pain has a 11-point rating scale. GAR assesses adherence to the prescribed regimen on a scale of 0 to 10 with 10 always taken as prescribed. SATMED-Q assesses the satisfaction with the prescribed treatment based on a Likert scale and provides a total score for treatment satisfaction by summing all domains: side effects, efficacy, convenience and ease of use, impact of the medicine, medical follow-up/review and overall opinion.
Measured: 6 years
Real world effectiveness of treatment products assessed by the healthcare providers as measure by the number and types of medical visits and/or hospitalizations per year.
Healthcare providers will document the number and types of medical visits/hospitalization per year that are related to hemophilia care and treatment.
Measured: 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2018
- Study start24 Oct 2018
- Primary completion31 May 2023
- Study completion31 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A With InhibitorStudies 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
- 395 participants (actual)
- Target duration
- 4 Years
Sponsor and investigators
- Lead sponsor
- American Thrombosis and Hemostasis Network
- Sponsor type
- Research network
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Tyler Buckner, MD, MSc, Principal investigator, Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical CampusMichael Recht, MD, PhD, Principal investigator, The Hemophilia Center at Oregon Health & Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03619863
- Sponsor's protocol ID
- ATHN 7
- Acronym
- ATHN 7
- Status last verified
- Feb 2025
- First submitted
- 25 Jul 2018
- Last updated
- 12 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Ragni MV. Targeting Antithrombin to Treat Hemophilia. N Engl J Med. 2015 Jul 23;373(4):389-91. doi: 10.1056/NEJMcibr1505657. No abstract available.
PubMed 26200986Gouw SC, van den Berg HM, Fischer K, Auerswald G, Carcao M, Chalmers E, Chambost H, Kurnik K, Liesner R, Petrini P, Platokouki H, Altisent C, Oldenburg J, Nolan B, Garrido RP, Mancuso ME, Rafowicz A, Williams M, Clausen N, Middelburg RA, Ljung R, van der Bom JG; PedNet and Research of Determinants of INhibitor development (RODIN) Study Group. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013 May 16;121(20):4046-55. doi: 10.1182/blood-2012-09-457036. Epub 2013 Apr 3.
PubMed 23553768Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.
PubMed 27223146Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.
PubMed 28691557Carr ME, Tortella BJ. Emerging and future therapies for hemophilia. J Blood Med. 2015 Sep 3;6:245-55. doi: 10.2147/JBM.S42669. eCollection 2015.
PubMed 26366108Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, Hettinger J, Carioto M, Jiang Y, Brodsky J, Prabhala H, Zhang X, Attarwala H, Hutabarat R, Foster D, Milstein S, Charisse K, Kuchimanchi S, Maier MA, Nechev L, Kandasamy P, Kel'in AV, Nair JK, Rajeev KG, Manoharan M, Meyers R, Sorensen B, Simon AR, Dargaud Y, Negrier C, Camire RM, Akinc A. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med. 2015 May;21(5):492-7. doi: 10.1038/nm.3847. Epub 2015 Apr 13.
PubMed 25849132Buckner TW, Carpenter SL, Daoud N, Kempton CL, Lee L, Malec L, McLean TW, Morton P, O'Neill C, Staber JM, Wang M, Croteau SE, Recht M. Safety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study. Haemophilia. 2026 Jan-Feb;32(1):63-70. doi: 10.1111/hae.70151. Epub 2025 Nov 11.
PubMed 41220279
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia
- ClinicalTrials.gov ID
- NCT03619863
- Acronym / sponsor ID
- ATHN 7 / ATHN 7
- Conditions
- Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor
- Keywords
- Hemophilia, Non-Factor Products, Bypassing Agents, Clotting Factor Replacement Products
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 395 participants (actual)
- Lead sponsor
- American Thrombosis and Hemostasis NetworkResearch network
- Collaborators
- Genentech, Inc.
- Study start
- 24 Oct 2018 (actual)
- Primary completion
- 31 May 2023 (actual)
- Study completion
- 31 Mar 2024 (actual)
- First posted
- 8 Aug 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 is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. The hemophilia treatment center (HTC) physician and participant will decide on the FDA-approved treatment to be used which may include non-factor products, bypassing agents, or clotting factor replacement products. The goal of this research is to study the use of hemophilia treatment products and their outcomes.
Detailed description
This non-interventional, minimal risk cohort study will enroll and follow patients with hemophilia A or B as they receive hemophilia treatment for 4 years. This is a pragmatic study of real-world practices across a wide range of patients which will be ongoing as new treatment products receive FDA approval and will be advantageous to the entire hemophilia community. The total study duration is planned for 6 years.
The patients are seen at baseline, annually, and at study exit. Patients will also receive routine quarterly follow-up phone calls from HTC staff to review medical history, bleed events, and product treatment history. Other visits for unplanned events or for the change of treatment product will be scheduled as necessary. All required study visits will be planned to coincide with routine clinical care whenever possible. Co-enrollment in the ATHNdataset by patients is required to participate in the study.
Please note - the treatment regimen will be at the discretion of the patients' hemophilia caregivers. No treatment products are being provided by the study nor will the participants be paid. However, inhibitor titer testing will be provided at no cost to patients by the Center for Disease Control and Prevention (CDC).
The primary objective is to determine the safety of non-factor products, bypassing agents or clotting factor replacement products when used for people with hemophilia with or without inhibitors. Safety will be measured by those events listed in the European Haemophilia Safety Surveillance (EUHASS).
Data collected will include eligibility, demographics, medical history, hemophilia history (genotype and family history), inhibitor history and immune tolerance induction (ITI) treatment regimen (if applicable), co-morbidities at baseline (i.e., HIV, Hepatitis C), detailed treatment product(s) usage, bleeding events, surgical procedures, and EUHASS adverse events and other adverse events of special interest. Data collection will also include patient-reported outcome questionnaires regarding health-related quality of life, treatment use and patient perceptions of treatment products.
Sub-studies
A number of sub-studies are planned with pharmaceutical sponsors to collect information from patients about their specific product use. Participation in these sub-studies (Product Specific Modules) is optional and sub-study visits will be planned to coincide with HTC visits. The modules will collect information from patients about their perception and use of treatment products, physical activity levels and other general health questions. This data will be collected via questionnaire.
Data Collection System
All data collected will be entered into electronic case report forms (eCRFs) within the secure ATHN System by HTC site personnel. All participating study sites will have in place a current, executed Data Use and Business Associate Agreement (DUBAA) with ATHN.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 4 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will enroll approximately 280 patients with hemophilia who meet the eligibility criteria and receiving care from one of the ATHN-affiliated Hemophilia Treatment Centers (HTC).
Key inclusion criteria (3 listed)
- Congenital hemophilia A or B of any severity with or without inhibitors receiving a current therapy, a non-factor product, or for whom use of a non-factor product is a possibility;
- Able to give informed consent (by patient or parent/authorized guardian); and
- Co-enrollment in the ATHNdataset.
Key exclusion criteria (3 listed)
- Presence of any known bleeding disorder other than congenital hemophilia A or B;
- Presence of concurrent hemophilia and a second hemostatic defect (low Von Willebrand Factor (VWF) without Von Willebrand disease (VWD) diagnosis is not excluded); and
- Unable or unwilling to comply with the 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.
Primary outcome
Safety of treatment products used for hemophilia care will be assessed based on the number of reportable European Haemophilia Safety Surveillance (EUHASS) events documented on the ATHN Adverse Event Module Form.
Time frame: 6 years
All treatment-related reportable adverse events will be documented on the ATHN Adverse Event Module Form based on EUHASS reportable events which include: death, factor inhibitor development, venous thrombosis, allergic reactions, treatment-emergent side effects, new malignancies, cardiovascular events, and blood-borne infections. Other treatment-related events to be documented on the ATHN Adverse Event Module Form including thrombotic microangiopathies, injection site reactions, drug-induced liver injury and anti-drug antibodies.
5 secondary outcomes
Effectiveness of non-factor products, bypassing agents and clotting factor replacement products will be evaluated based on the participant's number of bleeding events reported as the annualized bleeding rate (ABR).
Time frame: 6 years
Dosing regimens for hemophilia treatment products and total amount utilized by the study participant for prophylaxis and treatment of bleeds will be assessed.
Time frame: 6 years
Target joint monitoring
Time frame: 6 years
Efficacy of treatment is rated by health-related outcomes tools: EQ-5D-5L, Patient-Reported Outcomes Measurement Information System (PROMIS), Global Adherence Rating (GAR), and Treatment Satisfaction with Medicines Questionnaire (SATMED-Q).
Time frame: 6 years
Real world effectiveness of treatment products assessed by the healthcare providers as measure by the number and types of medical visits and/or hospitalizations per year.
Time frame: 6 years
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 12 Feb 2025.
- Background: PubMed 26200986Ragni MV. Targeting Antithrombin to Treat Hemophilia. N Engl J Med. 2015 Jul 23;373(4):389-91. doi: 10.1056/NEJMcibr1505657. No abstract available.
- Background: PubMed 23553768Gouw SC, van den Berg HM, Fischer K, Auerswald G, Carcao M, Chalmers E, Chambost H, Kurnik K, Liesner R, Petrini P, Platokouki H, Altisent C, Oldenburg J, Nolan B, Garrido RP, Mancuso ME, Rafowicz A, Williams M, Clausen N, Middelburg RA, Ljung R, van der Bom JG; PedNet and Research of Determinants of INhibitor development (RODIN) Study Group. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013 May 16;121(20):4046-55. doi: 10.1182/blood-2012-09-457036. Epub 2013 Apr 3.
- Background: PubMed 27223146Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, Fukazawa N, Yoneyama K, Yoshida H, Nogami K. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.
- Background: PubMed 28691557Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.
- Background: PubMed 26366108Carr ME, Tortella BJ. Emerging and future therapies for hemophilia. J Blood Med. 2015 Sep 3;6:245-55. doi: 10.2147/JBM.S42669. eCollection 2015.
- Background: PubMed 25849132Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, Hettinger J, Carioto M, Jiang Y, Brodsky J, Prabhala H, Zhang X, Attarwala H, Hutabarat R, Foster D, Milstein S, Charisse K, Kuchimanchi S, Maier MA, Nechev L, Kandasamy P, Kel'in AV, Nair JK, Rajeev KG, Manoharan M, Meyers R, Sorensen B, Simon AR, Dargaud Y, Negrier C, Camire RM, Akinc A. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med. 2015 May;21(5):492-7. doi: 10.1038/nm.3847. Epub 2015 Apr 13.
- Derived from this study: PubMed 41220279Buckner TW, Carpenter SL, Daoud N, Kempton CL, Lee L, Malec L, McLean TW, Morton P, O'Neill C, Staber JM, Wang M, Croteau SE, Recht M. Safety and Effectiveness of Emicizumab in People With Haemophilia A Enrolled in the ATHN 7 Haemophilia Natural History Study. Haemophilia. 2026 Jan-Feb;32(1):63-70. doi: 10.1111/hae.70151. Epub 2025 Nov 11.
- Centers for Disease Control accessedLink provided by the sponsor.
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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.
Tyler Buckner, MD, MSc principal investigator
Hemophilia and Thrombosis Center/ University of Colorado Anschutz Medical Campus
Michael Recht, MD, PhD principal investigator
The Hemophilia Center at Oregon Health & Science University
29 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 A With Inhibitor and Haemophilia A Without Inhibitor, run by American Thrombosis and Hemostasis Network at 29 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 3 inclusion and 3 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 12 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 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?
Safety of treatment products used for hemophilia care will be assessed based on the number of reportable European Haemophilia Safety Surveillance (EUHASS) events documented on the ATHN Adverse Event Module Form. (time frame: 6 years). 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 NCT03619863, which also lists 7 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 12 Feb 2025.