The Hemophilia Ultrasound Project

NCT02807753 | Terminated

• 1 other identifier: 20151156
• Study Type: observational
• Target: 18
• Locations: 1 country
• Sites: 4

Brief Summary

To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.

Conditions

Hemophilia A; Hemophilia B

Keywords

Hemophilia; Arthropathy; Pediatric

Outcome Measures

Primary Outcomes (2)

• Prevalence and natural progression of subclinical arthropathy (joint changes) in children with severe hemophilia undergoing prophylaxis using a validated ultrasound (HEAD-US) protocol.

  Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).

  5 years

• Prevalence and natural progression of subclinical arthropathy (joint changes) in children with mild and/or moderate hemophilia using a validated ultrasound (HEAD-US) protocol.

  Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).

  5 years

Secondary Outcomes (1)

• Characteristics of subclinical joint changes (time of presentation, natural evolution, etc.) between patients with severe hemophilia and those with mild/moderate hemophilia.

  5 years

Study Arms (2)

Severe Hemophilia A or B

Medical history, physical exam, vital signs,physical therapist targeted exam and ultrasound joint assessment (ankles and knees) every 6 months. MRI joint assessment (knees and ankles) at End of Trial Visit (Month 60).

Other: Ankle and Knee Ultrasound Joint Assessment; Other: Ankle and Knee Magnetic Resonance Imaging

Mild/Moderate Hemophilia A or B

Medical history, physical exam, vital signs,physical therapist targeted exam and ultrasound joint assessment (ankles and knees) every year. MRI joint assessment (knees and ankles) at End of Trial Visit (Month 60).

Other: Ankle and Knee Ultrasound Joint Assessment; Other: Ankle and Knee Magnetic Resonance Imaging

Interventions

Ankle and Knee Ultrasound Joint Assessment OTHER

Ultrasound Examination of the ankles and knees

Mild/Moderate Hemophilia A or B; Severe Hemophilia A or B

Ankle and Knee Magnetic Resonance Imaging OTHER

Ankle and Knee Magnetic Resonance Imaging

Mild/Moderate Hemophilia A or B; Severe Hemophilia A or B

Eligibility Criteria

• Age: Up to 30 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Mild, Moderate and Severe Hemophilia A \& B patients.

You may qualify if:

• Severe Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of severe hemophilia A or B defined as a factor VIII:C/IX:C of \<1% undergoing prophylaxis regimen with any factor VIII or IX concentrate and without evidence (clinical or by history) of target joint disease.
• Mild/moderate Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of mild hemophilia A or B defined as a factor VIII:C/IX:C of 5-50% and those with diagnosis of moderate hemophilia A or B defined a s a factor VIII:C/IX:C of 1-5% without evidence (clinical or by history) of target joint disease and no history of spontaneous joint bleeds.

You may not qualify if:

• Patients with concomitant Hepatitis B, Hepatitis C and HIV viral infections (because of a recognized arthritogen effect).
• Present or prior history of anti FVIII or IX inhibitors.
• Known inflammatory joint disease.
• Established target joint.

Sponsors & Collaborators

• University of Miami: lead
• Shire: collaborator

Study Sites (4)

University of Florida

Gainesville, Florida, 32610, United States

Location

The University of Miami - Department of Pediatrics

Miami, Florida, 33136, United States

Location

University of Kentucky

Lexington, Kentucky, 40536, United States

Location

Tulane

New Orleans, Louisiana, 70112, United States

Location

Related Publications (7)

• Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia. 2009 Nov;15(6):1219-27. doi: 10.1111/j.1365-2516.2009.02077.x. Epub 2009 Jul 29.

  PMID: 19659939 BACKGROUND

• Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, Ingram JD, Manco-Johnson ML, Funk S, Jacobson L, Valentino LA, Hoots WK, Buchanan GR, DiMichele D, Recht M, Brown D, Leissinger C, Bleak S, Cohen A, Mathew P, Matsunaga A, Medeiros D, Nugent D, Thomas GA, Thompson AA, McRedmond K, Soucie JM, Austin H, Evatt BL. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007 Aug 9;357(6):535-44. doi: 10.1056/NEJMoa067659.

  PMID: 17687129 BACKGROUND

• Martinoli C, Della Casa Alberighi O, Di Minno G, Graziano E, Molinari AC, Pasta G, Russo G, Santagostino E, Tagliaferri A, Tagliafico A, Morfini M. Development and definition of a simplified scanning procedure and scoring method for Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US). Thromb Haemost. 2013 Jun;109(6):1170-9. doi: 10.1160/TH12-11-0874. Epub 2013 Apr 4.

  PMID: 23571706 BACKGROUND

• Sierra Aisa C, Lucia Cuesta JF, Rubio Martinez A, Fernandez Mosteirin N, Iborra Munoz A, Abio Calvete M, Guillen Gomez M, Moreto Quintana A, Rubio Felix D. Comparison of ultrasound and magnetic resonance imaging for diagnosis and follow-up of joint lesions in patients with haemophilia. Haemophilia. 2014 Jan;20(1):e51-7. doi: 10.1111/hae.12268. Epub 2013 Sep 24.

  PMID: 24112687 BACKGROUND

• Di Minno MN, Iervolino S, Soscia E, Tosetto A, Coppola A, Schiavulli M, Marrone E, Ruosi C, Salvatore M, Di Minno G. Magnetic resonance imaging and ultrasound evaluation of "healthy" joints in young subjects with severe haemophilia A. Haemophilia. 2013 May;19(3):e167-73. doi: 10.1111/hae.12107. Epub 2013 Mar 18.

  PMID: 23496145 BACKGROUND

• Doria AS. State-of-the-art imaging techniques for the evaluation of haemophilic arthropathy: present and future. Haemophilia. 2010 Jul;16 Suppl 5:107-14. doi: 10.1111/j.1365-2516.2010.02307.x.

  PMID: 20590865 BACKGROUND

• Muca-Perja M, Riva S, Grochowska B, Mangiafico L, Mago D, Gringeri A. Ultrasonography of haemophilic arthropathy. Haemophilia. 2012 May;18(3):364-8. doi: 10.1111/j.1365-2516.2011.02672.x. Epub 2011 Oct 17.

  PMID: 21999202 BACKGROUND

MeSH Terms

Conditions

Hemophilia A; Hemophilia B; Joint Diseases

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; Genetic Diseases, X-Linked; Musculoskeletal Diseases

Study Officials

• Fernando F. Corrales-Medina, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: June 1, 2016
• First Posted: June 21, 2016
• Study Start: September 16, 2016
• Primary Completion: December 7, 2020
• Study Completion: December 7, 2020
• Last Updated: January 22, 2021

Record last verified: 2021-01

Locations

US (4)