ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

NCT ID: NCT04398628

Last Updated: 2026-01-12

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 3000 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-09-30
• Study Completion Date: 2035-12-31

Brief Summary

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5

In 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7

With this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8

Detailed Description

This is a longitudinal, natural history observational cohort study being conducted at approximately 150 ATHN-affiliated sites with a target accrual of 3,000 participants. Participants will be followed for a minimum of 15 years on an assigned arm within a cohort; however, arm or module participation may last longer, and participants will continue participation in the arm or module for its duration. Harmonized data elements will be collected at the time of enrollment, semi-annually (every 6 months), annually, ad hoc, and as defined by the terms of individual arms and modules. Data will be collected for participants enrolled in cohort-specific arms and modules.

Each participant will be assigned to a single cohort: Hemophilia, von Willebrand Disease, Congenital Platelet Disorders, Rare Disorders, Bleeding Not Otherwise Specified (NOS), Thrombosis/Thrombophilia, or Non-Neoplastic Hematologic Conditions.

Study arms and study modules are developed to advance the exploration of blood disorders disease specific insights by ATHN and its partners. Arms may branch off into product-specific data collection via Modules to be collected during the study, in conjunction with planned study assessments.

ATHN Transcends

Co- Principal Investigators:

Tammuella Chrisentery-Singleton, MD Ochsner Clinic Foundation American Thrombosis and Hemostasis Network

Michael Recht, MD, PhD, MBA Yale University School of Medicine National Bleeding Disorders Foundation

PUPs Arm

Principal Investigator:

Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy Hospital

ALTUVIIO Module

Principal Investigator:

Shannon Carpenter, MD, MS University of Missouri Kansas City School of Medicine Children's Mercy Hospital

INHIBIT Module

Principal Investigator:

Nicoletta Machin DO, MS Hemophilia Center of Western Pennsylvania University of Pittsburgh Medical Center

Hemophilia Natural History Arm

Principal Investigator:

Fernando Corrales-Medina, MD, FAAP University of Miami-Comprehensive Hemophilia Treatment Center University of Miami-Miller School of Medicine

Rebinyn Module

Co-Principal Investigators:

Lauren Amos, MD University of Missouri Kansas City School of Medicine Children's Mercy Hospital

Guy Young, MD University of Southern California Children's Hospital Los Angeles

Distress Module

Principal Investigator:

Tammuella Chrisentery-Singleton, MD Ochsner Clinic Foundation American Thrombosis and Hemostasis Network

Hemlibra Module

Principal Investigator:

Fernando Corrales-Medina, MD, FAAP University of Miami-Comprehensive Hemophilia Treatment Center University of Miami-Miller School of Medicine

Hemophilia Gene Therapy Outcomes Arm:

Co-Principal Investigators:

Janice M. Staber, MD Iowa Hemophilia and Thrombosis Center University of Iowa Stead Family Children's Hospital

Ulrike M. Reiss, MD Hemophilia Treatment Center St. Jude's Children's Research Hospital

HEMGENIX Module

Co-Principal Investigators:

Janice M. Staber, MD Iowa Hemophilia and Thrombosis Center University of Iowa Stead Family Children's Hospital

Ulrike M. Reiss, MD Hemophilia Treatment Center, St. Jude's Children's Research Hospital

Severe VWD Natural History Arm:

Co-Principal Investigators:

Robert F. Sidonio, Jr., MD, MSc Aflac Cancer and Blood Disorders Center, Hemophilia of Georgia Center for Bleeding and Clotting Disorders

Angela C. Weyand, MD C.S. Mott Children's Hospital, University of Michigan Medical School, Ann Arbor

Congenital Platelet Disorders Natural History Arm:

Principal Investigator Sanjay Ahuja, MD Innovative Hematology, Indiana Hemophilia & Thrombosis Center

Glanzmann Thrombasthenia Module:

Co-Principal Investigators:

Divya Citla-Sridhar, MD University of Arkansas for Medical Sciences Arkansas Children's Hospital

Meera Chitlur, MD Central Michigan University, Children's Hospital of Michigan

Hemophilia Cohort

This cohort includes three Arms and six Modules:

Previously Untreated Patients (PUPs) Arm This is a pediatric focused Arm of PUPs with hemophilia A or B of any severity.

Efanestoctocog alfa (ALTUVIIIO®) Module The purpose is to investigate the safety, tolerability, and effectiveness of efanesoctocog alfa (ALTUVIIIO®) in PUPs with severe hemophilia A.

INHIBIT Module This is an observational study assessing the rate of inhibitor formation in young children with severe hemophilia A in the current treatment era.

Hemophilia Natural History Arm This Arm is investigating the safety, effectiveness, and practice of treatment for people with hemophilia.

Emicizumab (Hemlibra®) Module All participants treated with Hemlibra® are eligible to participate.

Distress Module Participants with Congenital Hemophilia A or B, 18 years of age or older, will be followed longitudinally for 2 years or from time of enrollment for a total planned study duration of 3 years

Nonacog beta pegol (Rebinyn®) Module The Rebinyn® Module is a prospective study in hemophilia B participants without inhibitors.

Hemophilia Gene Therapy Outcomes Arm This Arm is investigating the safety and effectiveness of gene therapy in people with hemophilia.

Etranacogene dezaparvovec (HEMGENIX®) Module This is an observational study to characterize the effectiveness and safety of HEMGENIX® in participants with hemophilia B.

Congenital Platelet Disorders (CPD) Natural History Arm:

The CPD Arm is investigating the natural history of the safety and efficacy of hemostatic therapies (such as platelet transfusions, desmopressin, antifibrinolytics, recombinant factor VIIa) in the prevention or treatment of bleeding events (on demand, surgery, prophylaxis) in adult and pediatric participants with inherited congenital platelet disorders..

Glanzmann Thrombasthenia (GT) Module:

This Module is a study of bleeding symptoms, treatments, and treatment outcomes in patients with Glanzmann thrombasthenia.

Von Willebrand Disease Cohort No arms or modules open at this time.

Rare Disorders Cohort No arms or modules open at this time.

Bleeding NOS No arms or modules open at this time.

Thrombosis/Thrombophilia No arms or modules open at this time.

Non-Neoplastic Hematologic Conditions No arms or modules open at this time.

Conditions

Hematologic Disorder; Bleeding Disorder; Connective Tissue Disorder; Hemophilia; Thrombosis; Von Willebrand Diseases; Thrombophilia; Rare Bleeding Disorder; Platelet Disorder; Factor IX Deficiency; Factor VIII Deficiency; Thalassemia; Sickle Cell Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Hemophilia

This cohort includes three Arms and six Modules:

Previously Untreated Patients (PUPs) Arm

Efanestoctocog alfa (ALTUVIIIO®) Module

INHIBIT Module

Hemophilia Natural History Arm

Emicizumab (Hemlibra®) Module

Nonacog beta pegol (Rebinyn®)Module

Distress Module

Hemophilia Gene Therapy Outcomes Arm

Etranacogene dezaparvovec (HEMGENIX®) Module

No interventions assigned to this group

Congenital Platelet Disorders

This cohort includes one Arm and Module:

Congenital Platelet Disorders (CPD) Natural History Arm Glanzmann Thrombasthenia (GT) Module

No interventions assigned to this group

Von Willebrand Disease

No arms or modules

No interventions assigned to this group

Rare Disorders

No arms or modules

No interventions assigned to this group

Bleeding NOS

No arms or modules

No interventions assigned to this group

Thrombosis/Thrombophilia

No arms or modules

No interventions assigned to this group

Non-Neoplastic Hematologic Conditions

No arms or modules

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

1. Any age

2. Having a congenital or acquired blood disorder; or

3. Having a bleeding phenotype as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; or

4. Connective tissue disorder with bleeding tendency as indicated by an age adjusted abnormal ISTH Bleeding Assessment Tool score.

5. Eligible for a currently active disease-specific arm.

6. Concurrent enrollment in the ATHNdataset or current ATHNdataset participant.

1. Factor VIII or factor IX activity <50%, without another explanation for low clotting factor other than congenital hemophilia or being a known carrier for congenital hemophilia; OR

2. Carrier for congenital hemophilia with a factor VIII >=50% or factor IX activity >=50% with or without a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years OR

3. Known congenital hemophilia that have a factor level >50% after receiving vector, OR 4. Acquired hemophilia.

1. Meeting the definition of VWD or low VWF per most recent international guidelines

1. Abnormalities of platelet function a. Glanzmann thrombasthenia (GPIIb or GPIIIa) b. Bernard-Soulier syndrome (GPIbalpha, GPIbbeta, or GPIX)

2. Abnormalities of platelet granules

3. Abnormalities of platelet signal transduction

4. Abnormalities of platelet secretion

5. Collagen Receptor Defect

6. ADP Receptor Defect

7. Thromboxane Receptor Defect

8. Giant Platelet Disorder

9. Abnormalities in platelet aggregation testing due to another or unknown cause (not drug related)

1. Have an established Rare Coagulation Disorder (RCD) diagnosis of one of the following:

1. PAI-1 deficiency

2. Factor I, II, V, VII, X, XI, XIII deficiencies

3. Combined FV and FVIII deficiency

4. Plasminogen deficiency

5. Decreased tissue plasminogen activator

6. Afibrinogenemia/hypofibrinogenemia/dysfibrinogenemia

7. Thrombotic Thrombocytopenia Purpura or Congenital Hemolytic Uremic Syndrome

8. Wiskott-Aldrich

9. Methylenetetrahydrofolate Reductase Deficiency

1. Have a bleeding phenotype as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years with an unknown diagnosis, OR

2. Connective tissue disorder with bleeding tendency as indicated by an ISTH Bleeding Assessment Tool score of ≥4 for adult males, ≥6 for adult females, or ≥3 for children younger than 18 years.

1. Have a prior history of arterial or venous thrombosis. 2. Participants with a known congenital or acquired thrombophilia with or without thrombosis.

a. Common congenital thrombophilias: i. Protein C deficiency ii. Protein S deficiency iii. Antithrombin deficiency iv. Factor V Leiden v. Prothrombin gene mutation b. Rare genetic factors i. Hyperhomocysteinemia c. Indeterminate genetic factors i. Elevated factor VIII ii. Elevated factor IX iii. Elevated factor XI iv. Elevated lipoprotein (a) d. Acquired thrombophilias i. Lupus anticoagulant ii. Anti-cardiolipin antibodies/Beta2 glycoprotein antibodies iii. Antiphospholipid syndrome

1. Having any congenital or acquired non-neoplastic hematologic disorder not included in any other cohort

1. Diagnosis of congenital hemophilia A (FVIII <40%) or hemophilia B (FIX <40% or below lower limit for age)

2. Age <18 years at time of enrollment

3. Parent or authorized guardian or legally authorized representative (LAR) can provide informed consent

4. Care established at one of the ATHN Transcends participating HTCs

5. Clotting Factor Concentrate (CFC) exposure, fresh frozen plasma (FFP), cryoprecipitate, and single donor platelets <3 exposure days (ED)

1. Diagnosis of severe factor VIII deficiency with baseline factor VIII level <1% 2. Initiating or plan to initiate prophylaxis with emicizumab or factor replacement 3. Factor concentrate exposure, Fresh Frozen Plasma (FFP), cryoprecipitate, and single donor platelets ≤3 EDs 4. ≤5 years of age

1. Ability of the potential participant's legally authorized representative (e.g., their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulation.

2. People with severe HA with a baseline FVIII activity of less than 1%. (While inclusion for participation in ATHN Transcends lists <5% FVIII activity, this proposed module will limit enrollment to people with FVIII activity levels of <1%.) Other severities may be included per ATHN Transcends PI approval.

3. <18 years of age.

4. No history of a confirmed, positive FVIII inhibitor.

5. Sex assigned at birth of male, female, or intersex.

6. Participants should have no more than three (3) exposure days of blood products (fresh frozen plasma, cryoprecipitate, or platelets), no more than three (3) doses of any FVIII concentrate other than efanesoctocog alfa, and up to three (3) doses of efanesoctocog alfa prior to enrollment.

3. History of positive inhibitor testing.

4. History of hypersensitivity reactions associated with efanesoctocog alfa administration.

5. Other coagulation disorder(s) in addition to Hemophilia A.

6. Any concurrent clinically significant major disease such as cancer that, in the opinion of the investigator, would make the participant unsuitable for enrollment.

7. Concurrent systemic treatment with chemotherapy and/or other immunosuppressant medications. Use of corticosteroids for the treatment of asthma or management of acute allergic or otherwise life-threatening episodes is allowed except for systemic corticosteroid treatment given to children daily or on an alternate day schedule at > 2 mg/kg/day of prednisone or its equivalent or > 20 mg/day if the duration is longer than 14 days.

8. Enrollment in a concurrent clinical interventional drug study.

9. Intake of an Investigational Medicinal Product within three (3) months prior to inclusion in this study.

10. Inability to comply with study requirements.

11. Other, unspecified reasons that, in the investigator's opinion, make the participant unsuitable for enrollment.

Hemophilia Natural History Arm

1. Congenital or acquired 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, OR

2. Females of any age, with confirmed congenital hemophilia A or B carrier status with genetic mutational analysis and any factor level.

1. Has provided signed written consent for the nonacog beta pegol (Rebinyn®)Module before any study-related activities.

2. Male participants, at any age with hemophilia B, naïve or minimally exposed (up to 3 EDs) to nonacog beta pegol treatment at time of study enrollment. Additional doses may be allowable per ATHN Transcends PI approval.

3. Decision to initiate continuous prophylaxis treatment with commercially available nonacog beta pegol has been made by the participant(s)/Legally Authorized Representative(s) (LAR(s)) and the treating physician before and independently from the decision to include the participant in this study.

1. Participant currently treated with emicizumab (Hemlibra®)

2. Currently enrolled in the Hemophilia Natural History Arm of ATHN Transcends

1. 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

2. Age 18 years of age or older

3. English speaking

1. Hemophilia A or B of any severity with or without inhibitors having received or will receive a hemophilia gene transfer product in the next 6 months.

2. Age 18 years and older.

3. Able to give informed consent.

Etranacogene dezaparvovec (HEMGENIX®) Cohort

1. Age 18 years of age or older

2. Treatment with commercial etranacogene dezaparvovec (HEMGENIX®)

3. Have provided signed written informed consent within 3 months before or within 6 months after etranacogene dezaparvovec (HEMGENIX®) treatment, or within 6 months of when the study is initiated at the treating site.

FIX Prophylaxis Cohort

1. Age 18 years of age or older

2. Treatment with FIX prophylaxis therapy

3. Has provided signed written consent at any time for ATHN Transcends Study

1. Platelet adhesion defect

1. Bernard Soulier syndrome (Defective GPIb-IX-V receptor, impaired adhesion to vWF)

2. Velocardio-facial syndrome/DiGeorge syndrome (Defective GPIb-IX-V receptor)

3. Platelet type vWD (Defective GPIb-IX-V, gain of function interaction between vWF-GP1bα)

2. Platelet aggregation defect

1. Glanzmann thrombasthenia (Defective integrin αIIbβ3 (GPIIb/IIIa)

2. Platelet aggregation defect, NOS

3. Agonist receptor defects

1. Epinephrine

2. ADP

3. Collagen

4. Thromboxane A2

4. Platelet signaling defects

1. Cyclooxygenase deficiency (PTGS1 mutation)

2. Phospholipase A2 deficiency

3. Thromboxane synthase deficiency (TBXAS1 mutation)

4. G protein activation defect (GNAS mutation)

5. Scott syndrome (defect in phosphatidyl serine translocation)

5. Platelet Granule disorders

1. Dense granule storage pool disorder

• Hermansky Pudlak syndrome
• Chediak Higashi syndrome
• Griscelli syndrome

2. Alpha granule storage pool disorder

• Grey platelet syndrome
• Arthrogryposis-Renal Dysfunction-Cholestasis (ARC) syndrome
• Quebec platelet disorder
• Paris-Trousseau syndrome

3. Combined alpha delta granule deficiency

6. Platelet cytoskeletal structure defects

1. Wiskott Aldrich syndrome

2. MYH9 associated disorders (myosin heavy chain)

• May Hegglin syndrome
• Fechtner syndrome
• Sebastian syndrome
• Epstein syndrome

3. Other mutations

• FLNA mutations (Filamin)
• DIAPH1 (Actin and microtubules)
• ACTN1 (alpha actinin)
• TPM4 (tropomyosin)
• TUBB1 (beta tubulin)

7. Other Congenital thrombocytopenias

1. Familial platelet disorders and predisposition to AML (RUNX1)

2. X linked thrombocytopenia with dyserythropoiesis (GATA1)

3. Congenital amegakaryocytic thrombocytopenia (MPL)

1. Participant has signed the informed consent/assent form

2. Participant has flow cytometry or aggregometry or genetics confirmed GT

3. Participant is willing to perform study procedures, including daily bleed tracking for 3 months and further if requested

4. Participants are 2 years or older at time of consent

Exclusion Criteria

1. Does not qualify for inclusion in a currently activedisease-specific arm; participants may be eligible to enroll as future cohorts and arms are activated; 2. Unable to give informed consent or assent 3. Unwilling to perform study procedures

Cohort Participant Selection

Each participant is to be enrolled in the cohort for which they qualify as defined below.

Hemophilia Cohort

None

Von Willebrand Disease Cohort

None

Congenital Platelet Disorders Cohort

1. Platelet disorders secondary to medications or other substances

Rare Disorders Cohort

None

Bleeding NOS Cohort

None

Thrombosis/Thrombophilia Cohort

Non-Neoplastic Hematologic Conditions Cohort

Arm/Module Participant Selection

Previously Untreated Patients Arm

1. Concomitant diagnosis with another bleeding disorder

2. History of a confirmed, positive inhibitor

INHIBIT Module

1. Concomitant diagnosis with bleeding disorder other than hemophilia A

2. Immune disorder

3. Previous history or presence of factor VIII inhibitor. A confirmed, positive inhibitor is defined as two consecutive positive inhibitor titers (≥ 0.6 BU) that result in changes in treatment recommendations.

Efanesoctocog alfa (ALTUVIIIO®) Module

1. Presence of any known bleeding disorder other than congenital hemophilia A or B

2. Presence of concurrent hemophilia and a second hemostatic defect (low von Willebrand Factor (vWF) without vWD diagnosis is not excluded)

3. Unable or unwilling to comply with the study arm protocol.

Nonacog beta pegol (Rebinyn®) Module

1. Previous participation in this study. Participation is defined as having given informed consent in this study.

2. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation, including a diagnosis or suspicion of attention deficit hyperactivity disorder (ADHD) or autism spectrum disorder (ASD) per the discretion of the Principal Investigator.

3. Known or suspected hypersensitivity to nonacog beta pegol or related products.

4. Clinical suspicion or presence of FIX inhibitor at time of inclusion.

5. Inability or unwillingness to undergo neurological assessment/structured developmental history.

Emicizumab (Hemlibra®) Module

1. Unable or unwilling to comply with the protocol

Distress Module

1. Presence of any known bleeding disorder other than congenital hemophilia A or B;

2. Presence of concurrent hemophilia and a second hemostatic defect (low von Willebrand Factor (vWF) without vWD diagnosis is not excluded); and

3. Unable or unwilling to comply with the study arm protocol

Hemophilia Gene Therapy Outcomes Arm

Etranacogene dezaparvovec (HEMGENIX®) Module

1. Have been treated with etranacogene dezaparvovec in a clinical trial prior to commercial availability. These patients are still eligible for enrollment in the Gene Therapy Outcomes Arm, and their data may be collected for separate analysis.

Congenital Platelet Disorders Arm

1. Diagnosis of von Willebrand Disease (Meeting the definition of vWD or low vWF per most recent international guidelines)

2. Diagnosis of Hemophilia A or Hemophilia B (Factor VIII or IX ≤ 40%)

Glanzmann Thrombasthenia (GT) Module

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: collaborator

Hemophilia of Georgia, Inc.

OTHER

Sponsor Role: collaborator

Genentech, Inc.

INDUSTRY

Sponsor Role: collaborator

CSL Behring

INDUSTRY

Sponsor Role: collaborator

Sanofi

INDUSTRY

Sponsor Role: collaborator

Novo Nordisk A/S

INDUSTRY

Sponsor Role: collaborator

Hemab ApS

INDUSTRY

Sponsor Role: collaborator

American Thrombosis and Hemostasis Network

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael Recht, MD, PhD, MBA

Role: PRINCIPAL_INVESTIGATOR

Yale University School of Medicine & National Bleeding Disorders Foundation

Tammuella Chrisentery-Singleton, MD

Role: PRINCIPAL_INVESTIGATOR

ATHN, Ochsner Clinic Foundation

Locations

Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status: RECRUITING

Arkansas Center for Bleeding Disorders

Little Rock, Arkansas, United States

Site Status: RECRUITING

Orthopaedic Institute for Children HTC

Los Angeles, California, United States

Site Status: RECRUITING

Childrens Hospital Los Angeles

Los Angeles, California, United States

Site Status: RECRUITING

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Site Status: RECRUITING

University of California at Davis Hemophilia Treatment Center

Sacramento, California, United States

Site Status: RECRUITING

Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program

San Bernardino, California, United States

Site Status: RECRUITING

Hemophilia & Thrombosis Treatment Center at UC San Diego Health

San Diego, California, United States

Site Status: RECRUITING

Rady Children's Hospital San Diego

San Diego, California, United States

Site Status: RECRUITING

University of California, San Francisco Hemophilia & Thrombosis Center

San Francisco, California, United States

Site Status: RECRUITING

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Site Status: RECRUITING

Yale Hemophilia Treatment Center

New Haven, Connecticut, United States

Site Status: RECRUITING

Delaware Hemophilia Treatment Center

Wilmington, Delaware, United States

Site Status: RECRUITING

Georgetown University

Washington D.C., District of Columbia, United States

Site Status: RECRUITING

Children's National Hemophilia Center

Washington D.C., District of Columbia, United States

Site Status: RECRUITING

University of Florida Hemophilia Treatment Center

Gainesville, Florida, United States

Site Status: RECRUITING

University of Miami Comprehensive Hemophilia Treatment Center

Miami, Florida, United States

Site Status: RECRUITING

Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders

Orlando, Florida, United States

Site Status: NOT_YET_RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status: RECRUITING

St. Joseph's Hospital Center for Bleeding & Clotting Disorders

Tampa, Florida, United States

Site Status: RECRUITING

Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status: RECRUITING

Emory/Children's Health Care of Atlanta

Atlanta, Georgia, United States

Site Status: RECRUITING

Memorial Health University Medical Center

Savannah, Georgia, United States

Site Status: RECRUITING

Rush University Medical Center

Chicago, Illinois, United States

Site Status: RECRUITING

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

Site Status: RECRUITING

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, United States

Site Status: RECRUITING

Iowa Hemophilia and Thrombosis Center

Iowa City, Iowa, United States

Site Status: RECRUITING

Louisiana Center for Bleeding and Clotting Disorders, Tulane University

New Orleans, Louisiana, United States

Site Status: RECRUITING

Maine Hemophilia and Thrombosis Center

Scarborough, Maine, United States

Site Status: RECRUITING

Johns Hopkins University Hemophilia Treatment Center

Baltimore, Maryland, United States

Site Status: RECRUITING

Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center

Boston, Massachusetts, United States

Site Status: NOT_YET_RECRUITING

Central Michigan Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status: RECRUITING

Henry Ford Health System Bleeding and Thrombosis Treatment Center

Detroit, Michigan, United States

Site Status: RECRUITING

Mayo Comprehensive Hemophilia Center

Rochester, Minnesota, United States

Site Status: RECRUITING

Children's Mercy Hospital - Kansas City

Kansas City, Missouri, United States

Site Status: RECRUITING

The John Bouhasin Center for Children with Bleeding Disorders

St Louis, Missouri, United States

Site Status: RECRUITING

Cure 4 The Kids Foundation

Las Vegas, Nevada, United States

Site Status: RECRUITING

Hemostasis and Thrombosis Center of Nevada

Reno, Nevada, United States

Site Status: RECRUITING

Newark Beth Israel Medical Center - Hemophilia Center

Newark, New Jersey, United States

Site Status: RECRUITING

University of New Mexico Ted R. Montoya Hemophilia & Thrombosis Program

Albuquerque, New Mexico, United States

Site Status: RECRUITING

Western New York BloodCare

Buffalo, New York, United States

Site Status: RECRUITING

Northwell Health Hemostasis and Thrombosis Center at Long Island Jewish and Cohen Children's Medical Center

Hyde Park, New York, United States

Site Status: RECRUITING

Weill Cornell Medical College - New York Presbyterian Hospital

New York, New York, United States

Site Status: RECRUITING

American Thrombosis and Hemostasis Network

Rochester, New York, United States

Site Status: RECRUITING

Montefiore Medical Center

The Bronx, New York, United States

Site Status: RECRUITING

Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status: RECRUITING

St. Jude Affiliate Clinic at Novant Health Hemby Children's Hospital

Charlotte, North Carolina, United States

Site Status: RECRUITING

East Carolina University Hemophilia Treatment Center

Greenville, North Carolina, United States

Site Status: RECRUITING

Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States

Site Status: RECRUITING

Akron Children's Hospital - Showers Center for Cancer & Blood Disorders

Akron, Ohio, United States

Site Status: RECRUITING

Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center

Cincinnati, Ohio, United States

Site Status: RECRUITING

University of Cincinnati Medical Center Hemophilia Treatment Center

Cincinnati, Ohio, United States

Site Status: RECRUITING

University Hospitals Health System Cleveland

Cleveland, Ohio, United States

Site Status: RECRUITING

Nationwide Children's Hospital Columbus

Columbus, Ohio, United States

Site Status: RECRUITING

Dayton Children's Hemostasis and Thrombosis Center

Dayton, Ohio, United States

Site Status: RECRUITING

Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital

Toledo, Ohio, United States

Site Status: RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status: RECRUITING

Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status: RECRUITING

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Site Status: RECRUITING

Rhode Island Hospital Hemostasis and Thrombosis Center

Providence, Rhode Island, United States

Site Status: RECRUITING

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status: RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status: RECRUITING

Children's Blood and Cancer Center of Central Texas

Austin, Texas, United States

Site Status: RECRUITING

North Texas Hemophilia and Thrombosis Program - Pediatric Program / Center for Cancer & Blood Disorders

Dallas, Texas, United States

Site Status: RECRUITING

North Texas Comprehensive Hemophilia Treatment Center

Dallas, Texas, United States

Site Status: RECRUITING

Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, United States

Site Status: RECRUITING

Texas Children's Hemophilia & Thrombosis Center/Baylor College of Medicine

Houston, Texas, United States

Site Status: RECRUITING

South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center

San Antonio, Texas, United States

Site Status: RECRUITING

Washington Center for Bleeding Disorders

Seattle, Washington, United States

Site Status: RECRUITING

Hemophilia Outreach Center Green Bay

Green Bay, Wisconsin, United States

Site Status: RECRUITING

Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Carol Fedor, ND, RN, CCRC

Role: CONTACT

cfedor@athn.org

800-360-2846 ext. 122

Nana Ama Afari-Dwamena, MPH

Role: CONTACT

nafaridwamena@athn.org

800-360-2846 ext. 118

Facility Contacts

Erica Sieber

Role: primary

esieber@phoenixchildrens.com

Rachel Kelley

Role: primary

KelleyRachelM@uams.edu

501-364-1494

Christopher Chan

Role: primary

christopherchan@mednet.ucla.edu

213-742-1402

Christina Le

Role: primary

chle@chla.usc.edu

Susannah Lim

Role: primary

Susannah.Lim@ucsf.edu

Celynn Knight

Role: primary

ceknight@ucdavis.edu

Rosa Rivas

Role: primary

909-651-1910

Isabel Chang

Role: primary

i3chang@health.ucsd.edu

Jacqueline Limjoco, RN, BSN

Role: primary

jlimjoco1@rchsd.org

Sachiko Suzuki, MPH

Role: primary

Sachiko.suzuki@ucsf.edu

Dayna Kennedy

Role: primary

dkennedy@connecticutchildrens.org

Lia Louizos

Role: primary

E.Louizos@yale.edu

Nicole Wittmeyer

Role: primary

Nicole.Wittmeyer@nemours.org

Helena Jacobs

Role: primary

jacobsh@georgetown.edu

Michaela Ramandanes

Role: primary

mramandane@childrensnational.org

202-476-3622

Mona Huq

Role: primary

monahuq@peds.ufl.edu

352-273-9120

Leandro Pisani

Role: primary

LFP34@miami.edu

Stephanie Sharon, BSN, RN

Role: primary

stephanie.sharon@orlandohealth.com

Dawn Harrison, FNP

Role: primary

dharr172@jhmi.edu

Cindy Manis

Role: primary

cindy.manis@baycare.org

Gina Aulisio

Role: primary

gina.aulisio@emory.edu

404-778-7062

Simone Henry

Role: primary

shenry@emory.edu

Rebekah Hagan

Role: primary

rebekah.hagan@hcahealthcare.com

Hinal Patel

Role: primary

hinal_r_patel@rush.edu

Sarah Malik

Role: primary

sarawm@ilbcdi.org

Nancy Hoard

Role: primary

nhoard@ihtc.org

Alison Currie

Role: primary

alison-currie@uiowa.edu

319-356-4277

Melody Benton

Role: primary

mbenton@tulane.edu

Dana Grass

Role: primary

Dana.Grass@mainehealth.org

Kimberly Jones

Role: primary

kjones62@jhmi.edu

Carmen Zhou

Role: primary

czhou12@mgh.harvard.edu

Negin Salehi

Role: primary

saleh1n@cmich.edu

313-966-8393

Mary Mueller

Role: primary

lmuelle1@hfhs.org

313-725-7791

Jasmine Sexton

Role: primary

Sexton.Jasmine@mayo.edu

Anna Wiseman

Role: primary

amwiseman@cmh.edu

Gina Martin

Role: primary

gina.martin@health.slu.edu

Giany Beltron

Role: primary

gbeltran@cure4thekids.org

Lisa Cervantes

Role: primary

lisa.cervantes@htcnv.org

Arjun Gadhiya

Role: primary

Arjun.Gadhiya@rwjbh.org

973-926-3136

Jessica Salazar

Role: primary

jtsalazar@salud.unm.edu

Michelle Acosta

Role: primary

macosta@wnybloodcare.org

Mabel Origho

Role: primary

morigho@northwell.edu

Ilene Goldberg

Role: primary

igoldber@med.cornell.edu

212-746-3403

Nana Afari-Dwamena

Role: primary

nafaridwamena@athn.org

Carol Fedor, ND, RN

Role: backup

cfedor@athn.org

Ariel Levy

Role: primary

arlevy@montefiore.org

Ethan Meadows

Role: primary

Ethan_meadows@med.unc.edu

AQesha Ritzie-Spinks

Role: primary

aritziespinks@novanthealth.org

Danielle McCloskey

Role: primary

mccloskeyd19@ecu.edu

Debra Holder

Role: primary

djholde@wakehealth.edu

336-716-9551

Valisha Watkins

Role: primary

vwatkins1@akronchildrens.org

Kenzie Nolte

Role: primary

mackenzie.nolte@cchmc.org

Jill Roeder

Role: primary

roederjr@ucmail.uc.edu

George Lucas

Role: primary

George.Lucas@UHhospitals.org

Elliot Smith

Role: primary

elliot.smith@cchmc.org

Bethany Linegang

Role: primary

LinegangB@childrensdayton.org

Patricia Ahrens

Role: primary

patricia.ahrens@promedica.org

419-291-2210

Praharsha Konde

Role: primary

kondep@chop.edu

Karen Panckeri

Role: primary

Karen.Panckeri@uphs.upenn.edu

215-614-0506

Deborah Vehec

Role: primary

dvehec@vitalant.org

412-209-7564

Asher Dillman

Role: primary

ADillmann@Lifespan.org

Margie King

Role: primary

margaret.king@stjude.org

Stephanie Minogue

Role: primary

stephanie.minogue@vumc.org

Rhea Robinson

Role: primary

rmrobinson@ascension.org

Anna Winborn

Role: primary

ANNA.WINBORN@childrens.com

Kasia Harrah

Role: primary

kasia.harrah@utsouthwestern.edu

Katherine Addy

Role: primary

Katherine.E.Addy@uth.tmc.edu

713-500-8352

Janine Starks

Role: primary

jxstarks@texaschildrens.org

832-824-4969

Shawn Lade

Role: primary

lade@uthscsa.edu

Sophia Beyer

Role: primary

sophia.beyer@wacbd.org

Andrea Miller

Role: primary

Andream@hocgb.org

920-965-0606

Karen Stephany

Role: primary

kstephany@versiti.org

References

Weijer C, Freedman B, Fuks A, Robbins J, Shapiro S, Skrutkowska M. What difference does it make to be treated in a clinical trial? A pilot study. Clin Invest Med. 1996 Jun;19(3):179-83.

Reference Type: BACKGROUND

PMID: 8724821 (View on PubMed)

Braunholtz DA, Edwards SJ, Lilford RJ. Are randomized clinical trials good for us (in the short term)? Evidence for a "trial effect". J Clin Epidemiol. 2001 Mar;54(3):217-24. doi: 10.1016/s0895-4356(00)00305-x.

Reference Type: BACKGROUND

PMID: 11223318 (View on PubMed)

West J, Wright J, Tuffnell D, Jankowicz D, West R. Do clinical trials improve quality of care? A comparison of clinical processes and outcomes in patients in a clinical trial and similar patients outside a trial where both groups are managed according to a strict protocol. Qual Saf Health Care. 2005 Jun;14(3):175-8. doi: 10.1136/qshc.2004.011478.

Reference Type: BACKGROUND

PMID: 15933313 (View on PubMed)

Unger JM, Barlow WE, Martin DP, Ramsey SD, Leblanc M, Etzioni R, Hershman DL. Comparison of survival outcomes among cancer patients treated in and out of clinical trials. J Natl Cancer Inst. 2014 Mar;106(3):dju002. doi: 10.1093/jnci/dju002. Epub 2014 Mar 13.

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PMID: 24627276 (View on PubMed)

https://www.fda.gov/drugs/resources-information-approved-drugs/hematologyoncology-cancer-approvals-safety-notifications. Accessed 04 Jul 2019

Reference Type: BACKGROUND

https://www.clinicaltrials.gov/ct2/results?cond=Hematologic+Diseases&term=&cntry=&state=&city=&dist=. Accessed 04 Jul 2019

Reference Type: BACKGROUND

Konkle BA, Recht M; members of Working Group 2, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research. The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors. Haemophilia. 2019 Jul;25(4):590-594. doi: 10.1111/hae.13772.

Reference Type: BACKGROUND

PMID: 31329362 (View on PubMed)

Iorio A, Keepanasseril A, Foster G, Navarro-Ruan T, McEneny-King A, Edginton AN, Thabane L; WAPPS-Hemo co-investigator network. Development of a Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo): Study Protocol. JMIR Res Protoc. 2016 Dec 15;5(4):e239. doi: 10.2196/resprot.6558.

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PMID: 27977390 (View on PubMed)

Other Identifiers

ATHN Transcends

Identifier Type: -

Identifier Source: org_study_id