A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment

NCT02476942 | Completed

• 1 other identifier: BH29768
• Study Type: observational
• Target: 221
• Locations: 12 countries
• Sites: 35

Brief Summary

This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of Bleeds

  Approximately 6 months (from Baseline until study completion)

Secondary Outcomes (3)

• European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients

  Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)

• Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients

  Approximately 6 months (every 4 weeks from Baseline until study completion)

• Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score

  Approximately 6 months (every 4 weeks from Baseline until study completion)

Study Arms (3)

Cohort A: Adults and Adolescents with FVIII Inhibitors

Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

Drug: Bypassing Agents

Cohort B: Children with FVIII Inhibitors

Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

Cohort C: Adults and Adolescents without FVIII Inhibitors

Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed.

Drug: FVIII Replacement

Interventions

Bypassing Agents DRUG

Episodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.

Cohort A: Adults and Adolescents with FVIII Inhibitors

FVIII Replacement DRUG

Episodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.

Cohort C: Adults and Adolescents without FVIII Inhibitors

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia A with or without inhibitors against FVIII under standard-of-care treatment are to be enrolled.

You may qualify if:

• Cohort A: Patients greater than or equal to (\>/=) 12 years of age at time of informed consent
• Cohort A: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, \>/= 5 Bethesda units \[BU\])
• Cohort B: Pediatric patients less than (\<) 12 years of age
• Cohort B: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, \>/=5 BU)
• Cohort C: Patients \>/=12 years of age
• Cohort C: Diagnosis of congenital hemophilia A and FVIII activity \<1 percent (%)
• Cohort C: No prior history of a positive inhibitor against FVIII

You may not qualify if:

• Prior RO5534262 (not applicable if patient agrees that prior RO5534262 will preclude participation in a future investigational RO5534262 study)
• Bleeding disorder other than congenital hemophilia A
• Ongoing (or planned during the study) immune tolerance induction therapy with FVIII or FVIII prophylaxis if currently/previously exposed to an inhibitor
• Previous or concomitant thromboembolic disease
• Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count \<200 cells per microliter (cells/mcL)

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (35)

Santa Monica Oncology Center

Santa Monica, California, 90403, United States

Location

University of Colorado Denver, Children's Hospital

Aurora, Colorado, 80045, United States

Location

Georgetown Uni Medical Center; Lombardi Cancer Center

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

Location

Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Tulane Uni Health Sciences Center

New Orleans, Louisiana, 70112-2699, United States

Location

Children's Hospital of Michigan; Pediatrics

Detroit, Michigan, 48201, United States

Location

Cornell Univ Medical College; Hematology-Oncolog

New York, New York, 10021, United States

Location

Oregon Health & Science Uni ; Dept of Pediatrics

Portland, Oregon, 97201, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia

Seattle, Washington, 98104, United States

Location

Royal Prince Alfred Hospital; Haematology

Camperdown, New South Wales, 2050, Australia

Location

The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit

Melbourne, Victoria, 3004, Australia

Location

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Nanfang Hospital, Southern Medical University

Guangzhou, 510515, China

Location

Tianjin Institute of Hematology & Blood Diseases Hospital

Tianjin, 300020, China

Location

ICIC

San José, 1000, Costa Rica

Location

Universitätsklinikum Bonn (AöR); Inst. für Experimentelle Hämatologie u. Transfusionsmedizin (IHT)

Bonn, 53127, Germany

Location

IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi"

Milan, Lombardy, 20122, Italy

Location

AOU Careggi; SOD Malattie Emorragiche

Florence, Tuscany, 50134, Italy

Location

Nagoya University Hospital

Aichi, 466-8560, Japan

Location

Hyogo College of Medicine Hospital

Hyōgo, 663-8501, Japan

Location

St. Marianna University School of Medicine Hospital

Kanagawa, 216-8511, Japan

Location

Hospital of the University of Occupational and Environmental Health,Japan

Kitakyushu-shi, 807-8556, Japan

Location

Nara Medical University Hospital

Nara, 634-8521, Japan

Location

Tokyo Medical University Hospital

Tokyo, 160-0023, Japan

Location

Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii

Gdansk, 80-952, Poland

Location

SPSK Nr1 Klinika Hematoo&Transpl.Szpiku

Lublin, 20-081, Poland

Location

ALVAMED Lekarskie Gabinety Specjalistyczne

Poznan, 61-828, Poland

Location

Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych

Warsaw, 02-776, Poland

Location

Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center

Johannesburg, 2193, South Africa

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Hospital Universitario la Paz; Servicio de Hematologia

Madrid, 28046, Spain

Location

Hospital Universitario Virgen del Rocio; Servicio de Hematologia

Seville, 41013, Spain

Location

Hospital Universitario la Fe; Servicio de Hematologia

Valencia, 46026, Spain

Location

National Taiwan Uni Hospital

Taipei, 100, Taiwan

Location

Related Publications (1)

• Oldenburg J, Shima M, Kruse-Jarres R, Santagostino E, Mahlangu J, Lehle M, Selak Bienz N, Chebon S, Asikanius E, Trask P, Mancuso ME, Jimenez-Yuste V, von Mackensen S, Levy GG. Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study. Pediatr Blood Cancer. 2020 Oct;67(10):e28474. doi: 10.1002/pbc.28474. Epub 2020 Aug 9.

  PMID: 32776489 DERIVED

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2015
• First Posted: June 22, 2015
• Study Start: May 26, 2015
• Primary Completion: March 31, 2017
• Study Completion: March 31, 2017
• Last Updated: May 30, 2017

Record last verified: 2017-05

Locations

CN (3); IT (2); DE (1); JP (6); ZA (1); TW (1); CO (1); ES (3); AU (2); US (10); PL (4); KR (1)