Clinical trial NCT04131920: Handheld Ultrasound (HHUS) for Home Use in Hemophilia
Official title: Handheld Ultrasound (HHUS) for Home Use in Hemophilia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Joint Bleed
Hemophilia A
Keywords: Joint Bleed
Hemophilia A
Handheld Ultrasound
Lead sponsor: Washington Institute for Coagulation
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2019-10-18
Enrollment: 10
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Males patients at Hemophilia Treatment Centers with severe Hemophilia A

Brief summary: This is a study in 3 phases that will assess the feasibility and utility of handheld home ultrasound (HHUS) in a patient's home to assess whether a painful episode in the elbow, knee or ankle is a bleed or not.

Detailed description: This is a study in 3 phases that will assess the feasibility and utility of handheld home ultrasound (HHUS) in a patient's home to assess whether a painful episode in the elbow, knee or ankle is a bleed or not.

Phase 1 will assess whether 10 subjects with severe hemophilia A can be trained to use HHUS in clinic to identify basic joint structures of the elbow, knee and ankle. It will then assess whether these subjects can identify these structures at home and transmit the images life via tele-ultrasound.

Phase 2 will assess whether the determination of "bleed" vs. "non-bleed" made at home via HHUS can be confirmed/validated with a standard high resolution ultrasound machine in clinic.

Phase 3 will then utilize HHUS during the EmiMSKUS study over 3 years to more objectively identify patient reported bleeding during the study.

Eligibility criteria: Inclusion Criteria:

* Severe hemophilia A
* Male
* Over 18 years of age
* Able to read, write and understand English
* Willing to come to the center for an 8 hour training session
* Lives in proximity of the HTC and is willing to come in for acute joint episodes in phase 2
* Willing and able to follow study procedures
* Willing and able to keep HHUS at a safe place
* Have the dexterity to operate a HHUS unit

Exclusion Criteria:

* Unable to follow study instructions
* Physically or mentally unable to operate a HHUS unit

Primary outcomes:
measure: Outcome 1.1: Participant understanding of basic ultrasound transducer concepts and ability to perform basic positioning maneuvers.
timeFrame: 3 months
measure: Outcome 1.2: Participating subjects are able to perform basic transducer positions and identify basic structures of the elbow, knee and ankle.
timeFrame: 3 months
measure: Outcome 1.3: Participating subjects can perform what they learned during their training at the HTC in outcome 1.1 and outcome 1.2 at home and transmit images to the HTC.
timeFrame: 3 months
measure: Outcome 2.1: Subjects will be able to attain and transmit images of their joint spaces to the HTC via telemedicine utilizing the HHUS technology they learned in phase 1 and React App.
timeFrame: 6 months
measure: Outcome 2.2: Accuracy of the assignment of "bleed" vs. "non-bleed" will be determined on the stationary GE LS8 Logiq Pro in the HTC.
timeFrame: 6 months
measure: Outcome 3.1: To establish how accurate patient reported bleeding is compared to HHUS utilizing tele-ultrasound.
timeFrame: 3 years
measure: Outcome 3.2: To establish more accurate bleeding data for the EmiMSKUS study.
timeFrame: 3 years
Secondary outcomes:
Other outcomes:
Locations:
city: Seattle
country: United States
facility: Washington Center for Bleeding Disorders at Washington Institute for Coagulation
state: Washington
Study officials:
affiliation: Washington Institute for Coagulation
name: Rebecca Kruse-Jarres, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References:
citation: Forsyth AL, Giangrande P, Hay CR, Kenet G, Kessler CM, Knobl PN, Llinas A, Santagostino E, Young G. Difficult clinical challenges in haemophilia: international experiential perspectives. Haemophilia. 2012 Jul;18 Suppl 5:39-45. doi: 10.1111/j.1365-2516.2012.02887.x.
citation: 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.
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citation: Leissinger C, Gringeri A, Antmen B, Berntorp E, Biasoli C, Carpenter S, Cortesi P, Jo H, Kavakli K, Lassila R, Morfini M, Negrier C, Rocino A, Schramm W, Serban M, Uscatescu MV, Windyga J, Zulfikar B, Mantovani L. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med. 2011 Nov 3;365(18):1684-92. doi: 10.1056/NEJMoa1104435.
citation: Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.
citation: Mahlangu et al. Emicizumab prophylaxis administered once-weekly or every two weeks provides effective bleed prevention in persons with haemophilia A without inhibitors - Results from the phase III HAVEN 3 study . Oral presented at the World Federation of Hemophilia Meeting in Glasgow, May 21, 2018.
citation: Ceponis A, Wong-Sefidan I, Glass CS, von Drygalski A. Rapid musculoskeletal ultrasound for painful episodes in adult haemophilia patients. Haemophilia. 2013 Sep;19(5):790-8. doi: 10.1111/hae.12175. Epub 2013 May 15.
citation: Zhou JY, Rappazzo KC, Volland L, Barnes RFW, Brackman M, Steiner B, Kruse-Jarres R, Quon DV, Bailey C, Chang EY, von Drygalski A. Pocket handheld ultrasound for evaluation of the bleeding haemophilic joint: A novel and reliable way to recognize joint effusions. Haemophilia. 2018 Mar;24(2):e77-e80. doi: 10.1111/hae.13429. Epub 2018 Feb 12. No abstract available.