Gait Examination in Patients With Hemophilia in Austria

Completed

• Conditions: Hemophilia
• Interventions: Diagnostic Test: 3-d gait examination (level walking); Diagnostic Test: Anthropometrics and body composition assessment; Diagnostic Test: Biomarkers; Diagnostic Test: Clinical examination; Diagnostic Test: Habitual physical activity tracking (accelerometer based)

• Registration Number: NCT03541811
• Lead Sponsor: FH Campus Wien, University of Applied Sciences

Brief Summary

Hemophilia is an inherited disease caused by deficiency in varying degrees of clotting factors VIII and IX. Depending on the percentage of clotting factor in the blood the disease is categorized as "severe" (\<1%, characterized by spontaneous bleedings), "moderate" (1-5%) and "mild" (\>5%). If untreated, recurrent bleeding into the synovial joints often results in irreversible damage due to destruction of the cartilages and progressive joint impairment.

3d-gait analysis has been demonstrated as valid method to assess abnormal gait patterns and to monitor disease progression in patients with hemophilia (PWH). Furthermore, its outcomes facilitate the design of individually tailored therapeutic programs. In contrast to radiological examinations, 3d-gait analyses take place under weight-bearing conditions, which is a relevant issue in terms of weight-induced pain.

This study aims to explore the applicability of 3-d gait analysis as biomarker (gait deviation index) for functional impairments in PWH. Besides 3-d gait scores, secondary endpoints such as biomarkers reflecting cartilage damage and a laterality-ratio of leg muscle mass (in the case of one-sided target joints) will be tested for their ability to detect functional impairments in young adults with hemophilia.

Based on sample size calculation, 24 subjects aged 16-49 years, able to walk without aids or assistance will be included in each of the two groups: control (healthy, male), PWH (severe or moderate, treated prophylactically). Subjects suffering from functional impairments caused by other conditions than hemophilia, patients with bleedings within 30 days prior to the examination, PWH treated with immune-tolerance therapy and/or not successfully treated present or past high-titer factor VIII or FIX inhibitor will be excluded.

Subjects will pass through a set of examinations (medical history, clinical examination, 3d-gait analysis, anthropometrics, body composition analysis, venipuncture, and urine sampling) and carry an accelerometer device for seven consecutive days.

Confounder adjusted group differences will be assessed by ANCOVA with contrasts and Bonferroni correction. Correlations between the applied examination approaches will be assessed.

An evidence based health promotion program including follow-up examinations, physical activity promotion, and tailored physiotherapy are being envisaged as a follow-up project (JOSEPHA phase 2).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-04-03
• Study First Submitted QC: 2018-05-29
• Study First Posted: 2018-05-31
• Study Start: 2018-07-02
• Primary Completion: 2019-07-01
• Study Completion: 2019-07-01
• Status Verified: 2019-08
• Last Update Submitted: 2019-08-06
• Last Update Posted: 2019-08-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 42

Inclusion Criteria

Not provided

Exclusion Criteria

• Subjects suffering from functional impairments caused by other conditions than hemophilia
• PWH with joint bleedings within 30 days prior to the examination (applies for test group only)
• PWH treated with immune-tolerance therapy and/or not successfully treated present or past high-titer factor VIII or FIX inhibitor (applies for test group only)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
peer-matched healthy control	Biomarkers	not applicable (no intervention administered)
patients with hemophilia (16-45y)	Anthropometrics and body composition assessment	not applicable (no intervention administered)
patients with hemophilia (16-45y)	3-d gait examination (level walking)	not applicable (no intervention administered)
peer-matched healthy control	Anthropometrics and body composition assessment	not applicable (no intervention administered)
patients with hemophilia (16-45y)	Biomarkers	not applicable (no intervention administered)
patients with hemophilia (16-45y)	Habitual physical activity tracking (accelerometer based)	not applicable (no intervention administered)
peer-matched healthy control	3-d gait examination (level walking)	not applicable (no intervention administered)
patients with hemophilia (16-45y)	Clinical examination	not applicable (no intervention administered)
peer-matched healthy control	Clinical examination	not applicable (no intervention administered)
peer-matched healthy control	Habitual physical activity tracking (accelerometer based)	not applicable (no intervention administered)

Primary Outcome Measures

Name	Time	Method
Gait Deviation Index (GDI)	May 2018 - Oct 2018 (anticipated)	Score expressing overall gait physiology; resulting from 3-d gait analysis \[dimensionless\]

Secondary Outcome Measures

Name	Time	Method
Walking velocity	May 2018 - Oct 2018 (anticipated)	\[m sec-1\]
Walking cadence	May 2018 - Oct 2018 (anticipated)	\[steps min-1\]
Kinematic gait parameters	May 2018 - Oct 2018 (anticipated)	Functional range of motion Hip, Knee, Ankle \[°\]
Background factors (level of education)	May 2018 - Oct 2018 (anticipated)	International Standard Classification of Education (ISCED)
Step length	May 2018 - Oct 2018 (anticipated)	\[m\]
Stance phase duration	May 2018 - Oct 2018 (anticipated)	\[% gait cycle\]
Swing phase duration	May 2018 - Oct 2018 (anticipated)	\[% gait cycle\]
Body composition analysis	May 2018 - Oct 2018 (anticipated)	Bioelectrical impedance derived body fat rate \[kg, %\], fat-free mass \[kg, %\]
Background factors (type and history of substitutional therapy )	May 2018 - Oct 2018 (anticipated)	Type and history of substitutional therapy
Background factors (history of joint bleedings)	May 2018 - Oct 2018 (anticipated)	annualized bleeding rate
Biomarkers measured from serum and urine	May 2018 - Oct 2018 (anticipated)	sCOMP, sC1,2C, sCS846, uCTX-II, Vit D, CRP
Background factors (age)	May 2018 - Oct 2018 (anticipated)	\[years\]
Background factors (type and severity of hemophilia)	May 2018 - Oct 2018 (anticipated)	Type and severity of hemophilia
Hemophilia joint health score (HJHS)	May 2018 - Oct 2018 (anticipated)	Clinical score \[dimensionless\]
Background factors (inhibitors & immune-tolerance therapy)	May 2018 - Oct 2018 (anticipated)	Inhibitors \& immune-tolerance therapy history

Trial Locations

Locations (1)

FH Campus Wien - University of Applied Sciences; 🇦🇹 Vienna, Austria