Sickle Cell Disease Clinical Trial
Official title:
ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate 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.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, 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. ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. 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.(7)
Status | Recruiting |
Enrollment | 3000 |
Est. completion date | December 2035 |
Est. primary completion date | June 2035 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Participants who meet the following inclusion criteria and none of the exclusion criteria are eligible for enrollment in the base study: Inclusion Criteria: 1. Any age 2. Having a congenital or acquired non-neoplastic hematologic 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. Exclusion Criteria: 1. Does not qualify for inclusion in a cohort 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 Inclusion Criteria: Participants who meet any of the following inclusion criteria are eligible for enrollment into this cohort: 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. Being a known carrier for congenital hemophilia with a factor VIII or factor IX activity greater than or equal to 50% with or without a bleeding phenotype as indicated by an age-adjusted abnormal ISTH Bleeding Assessment Tool score; OR 3. Known congenital hemophilia that have a factor level >50% after receiving vector; OR 4. Acquired hemophilia Exclusion Criteria: None Von Willebrand Disease Cohort Inclusion Criteria: Participants who meet the following inclusion criteria are eligible for enrollment into this cohort: 1. Meeting the definition of VWD or low VWF per most recent international guidelines Exclusion Criteria: None Congenital Platelet Disorders Cohort Inclusion Criteria: Participants who meet the following inclusion criteria are eligible for enrollment into this cohort: 1. Abnormalities of platelet function 1. Glanzmann thrombasthenia (GPIIb or GPIIIa) 2. 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) Exclusion Criteria: Platelet disorders secondary to medications or other substances Rare Disorders Cohort Inclusion Criteria: Participants who meet the following inclusion criteria are eligible for enrollment into this cohort: 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 Exclusion Criteria: None Bleeding NOS Cohort Inclusion Criteria: Participants who meet the following inclusion criteria are eligible for enrollment into this cohort: 1. Have a bleeding phenotype as indicated by an age-adjusted abnormal ISTH Bleeding Assessment Tool score with an unknown diagnosis; OR 2. Connective tissue disorder with bleeding tendency as indicated by an age-adjusted abnormal ISTH Bleeding Assessment Tool score Exclusion Criteria: None Thrombosis/Thrombophilia Cohort Inclusion Criteria Participants who meet the following inclusion criteria are eligible for enrollment into this cohort: 1. Have a prior history of arterial or venous thrombosis 2. Patients with a known congenital or acquired thrombophilia with or without a 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 Exclusion Criteria 1. Acquired thrombophilia secondary to medications (birth control pills or hormone replacement therapy, overweight or obesity, smoking, cancer, pregnancy, surgery, injury, prolonged inactivity/bedrest, heart failure, inflammatory bowel disease, or kidney disease Non-Neoplastic Hematologic Conditions Cohort Inclusion Criteria Participants who meet the following inclusion criteria are eligible for enrollment into this cohort: 1. Having any congenital or acquired non-neoplastic hematologic disorder not included in any other cohort Exclusion Criteria None Previously Untreated Patients Arm Eligibility Criteria Inclusion Criteria Diagnosis of congenital hemophilia A (FVIII <40%) or hemophilia B (FIX <40% or below lower limit for age) 1. Age <18 years at time of enrollment 2. Parent or authorized guardian or legally authorized representative (LAR) can provide informed consent 3. Care established at one of the participating HTCs Exclusion Criteria 1. CFC exposure, fresh frozen plasma (FFP), cryoprecipitate, and single donor platelets >3 EDs 2. Concomitant diagnosis with another bleeding disorder 3. History of confirmed inhibitor INHIBIT Module Eligibility Criteria: Inclusion Criteria 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 exposure, plasma/FFP, cryo, and single donor platelets =3 EDs 4. =5 years of age Exclusion Criteria 1. Concomitant diagnosis with bleeding disorder other than hemophilia A 2. Immune disorder 3. Factor exposure, plasma/FFP, cryo, and single donor platelets >3 EDs 4. Previous history or presence of factor VIII inhibitor ALTUVIIIO® Module Eligibility Criteria: Inclusion criteria: - 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%) - <18 years of age - No history of FVIII inhibitor - Sex at birth of male, female, or intersex - Participants may be exposed to unfractionated blood components, no more than one dose of FVIII concentrate other than efanesoctocog alfa and up to three doses of efanesoctocog alfa prior to enrollment - Potential participants who have a history of bleeding will be eligible for participation if they meet all other inclusion criteria Exclusion criteria: - Not meeting all the inclusion criteria - Any exposure to blood components or FVIII replacement products except as described in the inclusion criteria - History of positive inhibitor testing - History of hypersensitivity reactions associated with efanesoctocog alfa administration - Any concurrent clinically significant major disease that, in the investigator's opinion, would make the participant unsuitable for enrollment. - The presence of any additional inherited bleeding disorder diagnosis - Enrollment in a concurrent clinical interventional drug study - Intake of an Investigational Medicinal Product within three months prior to inclusion in this study - Inability to comply with study requirements - Other, unspecified reasons that, in the investigator's opinion, make the participant unsuitable for enrollment. Hemophilia Natural History Arm Eligibility Criteria: Inclusion Criteria 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. Exclusion Criteria 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. Rebinyn® Module Eligibility Criteria Inclusion Criteria: Participants who meet the following inclusion criteria at the time of screening are eligible to enroll in the study module. Rebinyn® Cohort 1. Has provided signed written consent for the 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. 3. Decision to initiate 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. Rebinyn® RWE Cohort 1. Has provided signed written consent for ATHN Transcends Study before any study-related activities. 2. Currently treated with nonacog beta pegol 3. Any age Exclusion Criteria: Participants who fall into any of the following exclusion criteria at the time of screening are not eligible for enrollment into the study module: Rebinyn® Cohort 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 the neurological assessment/ structured developmental history. Rebinyn® RWE Cohort 1. None Hemophilia Gene Therapy Outcomes Arm Eligibility Criteria: Inclusion Criteria 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. Exclusion Criteria None HEMGENIX® Module Eligibility Criteria Inclusion Criteria: HEMGENIX Cohort • Age 18 years of age or older • Treatment with commercial HEMGENIX • Have provided signed written informed consent within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the treating site. Exclusion Criteria, both cohorts: • Have been treated with etranacogene dezaparvovec in a clinical trial. Congenital Platelet Disorders Arm Eligibility Criteria: Inclusion Criteria 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-GP1ba) 2. Platelet aggregation defect 1. Glanzmann thrombasthenia (Defective integrin aIIbß3 (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) Exclusion Criteria 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%) FIX Prophylaxis Cohort • Age 18 years of age or older • Treatment with FIX prophylaxis therapy • Has provided signed written consent at any time for ATHN Transcends Study Glanzmann Thrombasthenia (GT) Module Eligibility Criteria: Inclusion Criteria 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 Exclusion Criteria None |
Country | Name | City | State |
---|---|---|---|
United States | Akron Children's Hospital - Showers Center for Cancer & Blood Disorders | Akron | Ohio |
United States | Comprehensive Bleeding Disorders Center at Emory University and Children's Healthcare of Atlanta | Atlanta | Georgia |
United States | Emory/Children's Health Care of Atlanta | Atlanta | Georgia |
United States | University of Colorado Denver Hemophilia and Thrombosis Center | Aurora | Colorado |
United States | Johns Hopkins University Hemophilia Treatment Center | Baltimore | Maryland |
United States | Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center | Boston | Massachusetts |
United States | Montefiore Medical Center | Bronx | New York |
United States | Western New York BloodCare | Buffalo | New York |
United States | Comprehensive Hemophilia Treatment Center, University of North Carolina at Chapel Hill | Chapel Hill | North Carolina |
United States | St. Jude Affiliate Clinic at Novant Health Hemby Children's Hospital | Charlotte | North Carolina |
United States | Rush University Medical Center | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center, Hemophilia & Thrombosis Center | Cincinnati | Ohio |
United States | University of Cincinnati Medical Center Hemophilia Treatment Center | Cincinnati | Ohio |
United States | University Hospitals Health System Cleveland | Cleveland | Ohio |
United States | Nationwide Children's Hospital Columbus | Columbus | Ohio |
United States | North Texas Comprehensive Hemophilia Treatment Center | Dallas | Texas |
United States | North Texas Hemophilia and Thrombosis Program - Pediatric Program / Center for Cancer & Blood Disorders | Dallas | Texas |
United States | Dayton Children's Hemostasis and Thrombosis Center | Dayton | Ohio |
United States | Central Michigan Children's Hospital of Michigan | Detroit | Michigan |
United States | Henry Ford Health System Bleeding and Thrombosis Treatment Center | Detroit | Michigan |
United States | Fort Worth Bleeding Disorders Program | Fort Worth | Texas |
United States | University of Florida Hemophilia Treatment Center | Gainesville | Florida |
United States | Hemophilia Outreach Center Green Bay | Green Bay | Wisconsin |
United States | East Carolina University Hemophilia Treatment Center | Greenville | North Carolina |
United States | Gulf States Hemophilia and Thrombophilia Center | Houston | Texas |
United States | Texas Children's Hemophilia & Thrombosis Center/Baylor College of Medicine | Houston | Texas |
United States | Northwell Health Hemostasis and Thrombosis Center at Long Island Jewish and Cohen Children's Medical Center | Hyde Park | New York |
United States | Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana |
United States | Iowa Hemophilia and Thrombosis Center | Iowa City | Iowa |
United States | Children's Mercy Hospital - Kansas City | Kansas City | Missouri |
United States | Cure 4 The Kids Foundation | Las Vegas | Nevada |
United States | Arkansas Center for Bleeding Disorders | Little Rock | Arkansas |
United States | Orthopaedic Institute for Children HTC | Los Angeles | California |
United States | Mississippi Center for Advanced Medicine | Madison | Mississippi |
United States | St. Jude Children's Research Hospital | Memphis | Tennessee |
United States | Comprehensive Center for Bleeding Disorders | Milwaukee | Wisconsin |
United States | Vanderbilt University Medical Center | Nashville | Tennessee |
United States | Yale Hemophilia Treatment Center | New Haven | Connecticut |
United States | Louisiana Center for Bleeding and Clotting Disorders, Tulane University | New Orleans | Louisiana |
United States | Weill Cornell Medical College - New York Presbyterian Hospital | New York | New York |
United States | Newark Beth Israel Medical Center - Hemophilia Center | Newark | New Jersey |
United States | Arnold Palmer Hospital for Children - The Haley Center for Children's Cancer and Blood Disorders | Orlando | Florida |
United States | Bleeding and Clotting Disorders Institute | Peoria | Illinois |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Penn Comprehensive Hemophilia and Thrombophilia Program/Hospital of the University of Pennsylvania | Philadelphia | Pennsylvania |
United States | Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital | Phoenix | Arizona |
United States | Hemophilia Center of Western Pennsylvania | Pittsburgh | Pennsylvania |
United States | Rhode Island Hospital Hemostasis and Thrombosis Center | Providence | Rhode Island |
United States | Hemostasis and Thrombosis Center of Nevada | Reno | Nevada |
United States | American Thrombosis and Hemostasis Network | Rochester | New York |
United States | Mayo Comprehensive Hemophilia Center | Rochester | Minnesota |
United States | University of California at Davis Hemophilia Treatment Center | Sacramento | California |
United States | The John Bouhasin Center for Children with Bleeding Disorders | Saint Louis | Missouri |
United States | Johns Hopkins All Children's Hospital | Saint Petersburg | Florida |
United States | Johns Hopkins All Children's Hospital | Saint Petersburg | Florida |
United States | South Texas Comprehensive Hemophilia and Thrombophilia Treatment Center | San Antonio | Texas |
United States | Loma Linda Hemoglobinopathy and Inherited Bleeding Disorder Program | San Bernardino | California |
United States | Hemophilia & Thrombosis Treatment Center at UC San Diego Health | San Diego | California |
United States | Rady Children's Hospital San Diego | San Diego | California |
United States | Willett Children's Hemophilia Treatment Center at Memorial Health | Savannah | Georgia |
United States | Maine Hemophilia and Thrombosis Center | Scarborough | Maine |
United States | Washington Center for Bleeding Disorders | Seattle | Washington |
United States | Louisiana Center for Advanced Medicine | Slidell | Louisiana |
United States | St. Joseph's Hospital Center for Bleeding & Clotting Disorders | Tampa | Florida |
United States | Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital | Toledo | Ohio |
United States | Children's National Hemophilia Center | Washington | District of Columbia |
United States | Georgetown University | Washington | District of Columbia |
United States | Wake Forest University Health Sciences | Winston-Salem | North Carolina |
Lead Sponsor | Collaborator |
---|---|
American Thrombosis and Hemostasis Network | CSL Behring, Genentech, Inc., Hemab Therapeutics, Hemophilia of Georgia, Inc., Novo Nordisk A/S, Pfizer, Sanofi |
United States,
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Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | To describe real-world effectiveness of therapies by evaluating for Goal attainment | Measured by GOAL-Hem for those participants that opt into this measurement | 15 years | |
Other | To describe real-world effectiveness of therapies by evaluating for Patient Reported Outcomes (PROs) | Measured by the Patient Reported Outcomes Measurement Information System (PROMIS) Profile 29/25/Parent Proxy | 15 years | |
Other | To describe real-world effectiveness of therapies by evaluating treatment adherence | Measured by the Global Adherence Rating (GAR) | 15 years | |
Other | To describe real-world effectiveness of therapies by evaluating health utility | Measured by the EQ-5D-5L | 15 years | |
Primary | To determine the safety of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. | Safety will be measured by those events in the European Safety Surveillance (EUHASS).
Allergic or other acute events Treatment-emergent side effects of therapy Transfusion transmitted infections Inhibitor development Thrombosis Cardiovascular events Malignancies Neurological events Death In addition to the modified EUHASS endpoints, the following events will be collected as adverse events of special interest (AESI): The occurrence of thrombotic microangiopathies, injection site reactions and cases of potential drug-induced liver injury The development of anti-drug antibodies, to be measured and confirmed, if feasible Severe, unanticipated bleeding Hospitalizations Glomerulonephritis Additional safety events of interest (TBD) may be collected. These may be chosen from drug development profiles based on investigational studies, package inserts, and emerging clinical and scientific observations. |
15 years | |
Primary | To describe the safety and tolerability of efanesoctocog alfa in previously untreated patients (PUPs) with hemophilia A without a history of inhibitors. | Safety and tolerability will be measured by Annualized Bleed Rate (ABR), number of doses to treat a bleed, doses during perioperative surgery, and clinical patient reported outcomes (PROs) in this population. | 7 years | |
Secondary | To establish a platform to support study Arms and Modules for participants with bleeding, clotting, other non-neoplastic blood disorders, and connective tissue disorders with bleeding tendency. | For each Arm, a brief set of data elements of interests will be developed and reported for study participants. | 15 years | |
Secondary | To describe medication dosing regimens in the above conditions. | This objective will be evaluated by:
Determining the number of participants who initiate and/or switch treatment with non-factor products and participants' reasons for initiating and/or switching treatment with non-factor products Determining the number of participants who do not initiate treatment with non-factor products Determining the number of participants who switch between different non-factor products and the participants' reasons for switching non-factor products Determining the number of participants who discontinue treatment with non-factor products and participants' reasons for discontinuing treatment with non-factor products |
15 years | |
Secondary | To grow and evolve a biorepository for current and future research through the collection of biospecimens from every person enrolled on this protocol. | All participants will have the option of having specimens drawn (about 5mL each) at baseline to be stored in the ATHN Research Biorepository (ARB). | 15 years | |
Secondary | To describe real-world effectiveness of therapies by evaluating for Health care utilization | Measured by number and type of visits and hospitalizations per year. | 15 years | |
Secondary | To describe bleeding events, changes in overall bleeding, and annualized bleeding rate (ABR) as measured by individual bleeding components. | This objective will be calculated per ISTH Bleeding Assessment Tool (ISTH BAT), and if applicable, a Pictorial Bleeding Assessment Chart (PBAC), for applicable diagnoses. | 15 years |
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