Actively Recruiting

All Genders
ID04398628

ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders

Led by American Thrombosis and Hemostasis Network · Updated on 2026-01-12

3000

Participants Needed

71

Research Sites

26 weeks

Total Duration

On this page

Sponsors

A

American Thrombosis and Hemostasis Network

Lead Sponsor

P

Pfizer

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are studying people with various non-cancerous blood disorders to better understand the long-term safety and effectiveness of treatments used for these conditions. This observational study aims to collect consistent and high-quality data over time from patients with congenital or acquired hematologic and connective tissue disorders that cause bleeding or clotting problems. It addresses the need for reliable long-term information beyond initial drug approval trials and includes multiple disease-specific groups for comprehensive research. Participants will join one of several cohorts based on their specific blood disorder, such as hemophilia, von Willebrand disease, congenital platelet disorders, rare bleeding disorders, thrombosis/thrombophilia, or other non-neoplastic hematologic conditions. The study includes several arms and modules focusing on different therapies or patient groups, like previously untreated patients, gene therapy outcomes, and treatment with specific drugs (e.g., ALTUVIIIO® or Hemlibra®). Data collection occurs at enrollment and regularly every six months and annually, with additional data gathered as needed. During the study, participants will undergo various assessments including medical history, treatment records, bleeding event tracking, and biospecimen collection for future research. The study will follow participants for at least 15 years to monitor therapy safety and effectiveness. Researchers will analyze dosing patterns, bleeding rates, and real-world treatment outcomes while maintaining long-term participant follow-up and data quality.

CONDITIONS

Brief Title

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

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Any age
  • Diagnosis of a congenital or acquired blood disorder, bleeding phenotype with abnormal ISTH Bleeding Assessment Tool score and unknown diagnosis, or connective tissue disorder with bleeding tendency
  • Eligible for a currently active disease-specific arm
  • Concurrent enrollment in or current participant of the ATHNdataset
  • Specific criteria per cohort such as hemophilia with factor VIII or IX activity <50%, or congenital platelet disorder with defined platelet function abnormalities
  • For modules, criteria include age limits, specific factor activity levels, treatment history, and consent capability
Not Eligible

You will not qualify if you...

  • Does not qualify for inclusion in a currently active disease-specific arm
  • Unable to give informed consent or assent
  • Unwilling to perform study procedures
  • Platelet disorders secondary to medications or substances
  • Acquired thrombophilia due to medications, obesity, smoking, cancer, pregnancy, surgery, injury, prolonged inactivity, heart failure, inflammatory bowel disease, or kidney disease
  • Presence of bleeding disorders other than those specified for hemophilia arms
  • History of confirmed positive inhibitors for hemophilia modules
  • Concurrent participation in other interventional drug studies
  • Mental incapacity or language barriers precluding cooperation for certain modules
  • History of hypersensitivity to study drugs or related products
  • Other major diseases that may interfere with participation as judged by investigators
  • Previous treatment with investigational products or gene therapy prior to commercial availability for certain modules

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person) for screening and enrollment

Monitoring

Duration - Minimum of 15 years

Participants are followed longitudinally with data collected at enrollment, semi-annually, annually, and as defined by individual study arms and modules to observe the safety and effectiveness of treatments and disease progression.

Visits every 6 months and annually, with additional assessments as needed per assigned cohort or module

Trial Site Locations

Total: 71 locations

1

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

Phoenix, Arizona, United States, 85016

Actively Recruiting

2

Arkansas Center for Bleeding Disorders

Little Rock, Arkansas, United States, 72202

Actively Recruiting

3

Orthopaedic Institute for Children HTC

Los Angeles, California, United States, 90007

Actively Recruiting

4

Childrens Hospital Los Angeles

Los Angeles, California, United States, 90027-6016

Actively Recruiting

5

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States, 94610

Actively Recruiting

6

University of California at Davis Hemophilia Treatment Center

Sacramento, California, United States, 95817

Actively Recruiting

7

Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program

San Bernardino, California, United States, 92408

Actively Recruiting

8

Hemophilia & Thrombosis Treatment Center at UC San Diego Health

San Diego, California, United States, 92121

Actively Recruiting

9

Rady Children's Hospital San Diego

San Diego, California, United States, 92123

Actively Recruiting

10

University of California, San Francisco Hemophilia & Thrombosis Center

San Francisco, California, United States, 94143

Actively Recruiting

11

Connecticut Children's Medical Center

Hartford, Connecticut, United States, 06106

Actively Recruiting

12

Yale Hemophilia Treatment Center

New Haven, Connecticut, United States, 06520

Actively Recruiting

13

Delaware Hemophilia Treatment Center

Wilmington, Delaware, United States, 19801

Actively Recruiting

14

Georgetown University

Washington D.C., District of Columbia, United States, 20007

Actively Recruiting

15

Children's National Hemophilia Center

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

16

University of Florida Hemophilia Treatment Center

Gainesville, Florida, United States, 32610

Actively Recruiting

17

University of Miami Comprehensive Hemophilia Treatment Center

Miami, Florida, United States, 33136

Actively Recruiting

18

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

Orlando, Florida, United States, 32806

Not Yet Recruiting

19

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States, 33701

Actively Recruiting

20

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

Tampa, Florida, United States, 33607

Actively Recruiting

21

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

Atlanta, Georgia, United States, 30308

Actively Recruiting

22

Emory/Children's Health Care of Atlanta

Atlanta, Georgia, United States, 30322

Actively Recruiting

23

Memorial Health University Medical Center

Savannah, Georgia, United States, 31403

Actively Recruiting

24

Rush University Medical Center

Chicago, Illinois, United States, 60612

Actively Recruiting

25

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States, 61664

Actively Recruiting

26

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, United States, 46260

Actively Recruiting

27

Iowa Hemophilia and Thrombosis Center

Iowa City, Iowa, United States, 52242

Actively Recruiting

28

Louisiana Center for Bleeding and Clotting Disorders, Tulane University

New Orleans, Louisiana, United States, 70112

Actively Recruiting

29

Maine Hemophilia and Thrombosis Center

Scarborough, Maine, United States, 04074

Actively Recruiting

30

Johns Hopkins University Hemophilia Treatment Center

Baltimore, Maryland, United States, 21287

Actively Recruiting

31

Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center

Boston, Massachusetts, United States, 02114

Not Yet Recruiting

32

Central Michigan Children's Hospital of Michigan

Detroit, Michigan, United States, 48201

Actively Recruiting

33

Henry Ford Health System Bleeding and Thrombosis Treatment Center

Detroit, Michigan, United States, 48202

Actively Recruiting

34

Mayo Comprehensive Hemophilia Center

Rochester, Minnesota, United States, 55905

Actively Recruiting

35

Children's Mercy Hospital - Kansas City

Kansas City, Missouri, United States, 64108

Actively Recruiting

36

The John Bouhasin Center for Children with Bleeding Disorders

St Louis, Missouri, United States, 63104

Actively Recruiting

37

Cure 4 The Kids Foundation

Las Vegas, Nevada, United States, 89135

Actively Recruiting

38

Hemostasis and Thrombosis Center of Nevada

Reno, Nevada, United States, 89509

Actively Recruiting

39

Newark Beth Israel Medical Center - Hemophilia Center

Newark, New Jersey, United States, 07122

Actively Recruiting

40

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

Albuquerque, New Mexico, United States, 87131

Actively Recruiting

41

Western New York BloodCare

Buffalo, New York, United States, 14202

Actively Recruiting

42

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

Hyde Park, New York, United States, 11040

Actively Recruiting

43

Weill Cornell Medical College - New York Presbyterian Hospital

New York, New York, United States, 10065

Actively Recruiting

44

American Thrombosis and Hemostasis Network

Rochester, New York, United States, 14626

Actively Recruiting

45

Montefiore Medical Center

The Bronx, New York, United States, 10461

Actively Recruiting

46

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

Chapel Hill, North Carolina, United States, 27517

Actively Recruiting

47

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

Charlotte, North Carolina, United States, 28204

Actively Recruiting

48

East Carolina University Hemophilia Treatment Center

Greenville, North Carolina, United States, 27834

Actively Recruiting

49

Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States, 27157

Actively Recruiting

50

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

Akron, Ohio, United States, 44308

Actively Recruiting

51

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

Cincinnati, Ohio, United States, 45229

Actively Recruiting

52

University of Cincinnati Medical Center Hemophilia Treatment Center

Cincinnati, Ohio, United States, 45267

Actively Recruiting

53

University Hospitals Health System Cleveland

Cleveland, Ohio, United States, 44106

Actively Recruiting

54

Nationwide Children's Hospital Columbus

Columbus, Ohio, United States, 43205

Actively Recruiting

55

Dayton Children's Hemostasis and Thrombosis Center

Dayton, Ohio, United States, 45404

Actively Recruiting

56

Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital

Toledo, Ohio, United States, 43606

Actively Recruiting

57

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

58

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

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

59

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States, 15213

Actively Recruiting

60

Rhode Island Hospital Hemostasis and Thrombosis Center

Providence, Rhode Island, United States, 02903

Actively Recruiting

61

St. Jude Children's Research Hospital

Memphis, Tennessee, United States, 38105

Actively Recruiting

62

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37212

Actively Recruiting

63

Children's Blood and Cancer Center of Central Texas

Austin, Texas, United States, 78723

Actively Recruiting

64

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

Dallas, Texas, United States, 75235

Actively Recruiting

65

North Texas Comprehensive Hemophilia Treatment Center

Dallas, Texas, United States, 75390

Actively Recruiting

66

Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, United States, 77030

Actively Recruiting

67

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

Houston, Texas, United States, 77030

Actively Recruiting

68

South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center

San Antonio, Texas, United States, 78229

Actively Recruiting

69

Washington Center for Bleeding Disorders

Seattle, Washington, United States, 98101

Actively Recruiting

70

Hemophilia Outreach Center Green Bay

Green Bay, Wisconsin, United States, 54311

Actively Recruiting

71

Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, United States, 53226

Actively Recruiting

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Research Team

C

Carol Fedor, ND, RN, CCRC

N

Nana Ama Afari-Dwamena, MPH

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

7

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Published Research Related To This Trial

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.

J West, J Wright, D Tuffnell...

https://pubmed.ncbi.nlm.nih.gov/15933313

The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science Workshop on factor VIII inhibitors.

Barbara A Konkle, Michael Recht, members of Working Group 2, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research

https://pubmed.ncbi.nlm.nih.gov/31329362