Clinical trial NCT05629130: Embolization in Hereditary Coagulopathies
Official title: Embolization in Hereditary Coagulopathies
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Hemophilia
Embolization
Hemarthrosis
Clotting Factor Deficiency
Synovitis
Arthropathy
Lead sponsor: University of Sao Paulo General Hospital
Sponsor class: OTHER
Collaborators: name: Merit Medical Systems, Inc.
First posted: 2022-11-29
Enrollment: 30
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: longitudinal, prospective study.
Primary purpose: TREATMENT

Brief summary: This is a longitudinal, prospective study, which will include 30 subjects with hereditary coagulopathies, with arthropathy, chronic synovitis resulting from hemarthrosis of the elbows, knees and/or ankles followed up at the Centro de Hemofilia HCFMUSP, after approval by the ethics and research committee.

They will undergo imaging tests (X-rays and Magnetic Resonance of knee, elbow, or ankle), physical, pain, quality of life and functional assessments (Hemophilia Joint Health Score, Functional Independence Score in Hemophilia, Perimeter, Test Timed up and Go, 30 second sit and stand test, Haemophilia - Adult - Quality of Life questionnaire (HAEM-A-QoL), Knee Injury and Osteoarthritis Outcome Score, EQ-5D, numerical rating scale for pain and embolization procedure (superselective embolization of target arteries with spherical microparticles Embosphere 100-300 micrometers (Biosphere Medical, Roissy, France), until partial vascular stasis and decharacterization of pathological synovial enhancement.

These evaluations will be performed at baseline, 1, 3, 6, 12, 24, 36, 48 and 60 months after the procedure.

Detailed description: Longitudinal, prospective study, which will include 30 subjects with hereditary coagulopathies, with arthropathy, chronic synovitis resulting from hemarthrosis of the elbows, knees and/or ankles followed up at the Centro de Hemophilia do Hospital das Clinicas, Faculdade de Medicina da Universidade de São Paulo, i.e. HC-FMUSP), after approval by the ethics and research committee and registry at the clinical trials.

Patients will be evaluated by imaging (X-rays and Magnetic Resonance of the affected joint, i.e., knee, elbow, and/or ankle); physical and functional assessments (Hemophilia Joint Health Score, Functional Independence Score in Hemophilia, Perimeter of the affected joint(s), Test Timed up and Go, 30 second sit and stand test, Knee Injury and Osteoarthritis Outcome Score (KOOS); quality of life (Haemophilia - adult - quality of life questionnaires (HAEM-A-QoL), Knee Injury and Osteoarthritis Outcome Score (KOOS), EQ-5D; pain measured by numerical rating scale.

Volunteers will be submitted to embolization procedure (superselective embolization of target arteries with spherical microparticles embosphere 100-300 micrometers (Biosphere Medical, Roissy, France) of the affected joint(s), until partial vascular stasis and decharacterization of pathological synovial enhancement.

These evaluations will be performed at baseline, 1, 3, 6, 12, 24, 36, 48 and 60 months after the procedure.

Eligibility criteria: Inclusion Criteria:

* Patients with hereditary coagulopathies with arthropathy (i.e., chronic synovitis due to hemarthrosis), with X-ray and MRI (documenting synovitis) followed up at the Hemophilia Center HC-FMUSP.

Exclusion Criteria:

* Patients who do not complete the planned assessments
* Patients who do not accept to continue with the study.

Primary outcomes:
measure: Reduction of synovium thickness by embolization
timeFrame: 6 months
Secondary outcomes:
measure: Reduction of synovitis by embolization
timeFrame: 1, 3 12, 24, 36, 48 and 60 months
measure: Inferior limb strength
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Inferior limb strength
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Balance and fall risk
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Quality of life of the patient
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Quality of life of the patient
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Quality of life of the patient
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Functional capacity
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Functional capacity
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Physical capacity
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Pain of the affected joint
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Pain of the affected joint
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Pain of the affected joint
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
measure: Stifness
timeFrame: 1, 3, 6, 12, 24, 36, 48, 60 months
Other outcomes:
Locations:
city: São Paulo
country: Brazil
facility: Instituto de Ortopedia e Traumatologia do Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo
Study officials:
affiliation: IOT-HC-FMUSP (Orthopedics Institute - General Hospital- School of Medicine - University of Sao Paulo
name: Fabiane E FArias, PT
role: PRINCIPAL_INVESTIGATOR
affiliation: Radiology Insititute, General Hospital, School Medicine, University of São Paulo
name: Andre M Assis, MD, PhD
role: STUDY_DIRECTOR
affiliation: Hematology department, General Hospital, School of Medicine, University of Sâo Paulo
name: Paula Villaça, MD, PhD
role: STUDY_DIRECTOR
References: