HaemophiliaHOLISTICNCT02198430

Multidisciplinary Evaluation of Patients With Hemophilia

An observational study of Haemophilia, run by Universidad de Murcia at 1 site in Spain.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

Multidisciplinary assessment of the physical, functional and psychosocial alterations in patients with hemophilia around the country. The aims of this study are: Descriptive study of joint involvement in hemophilia patients with and without a history of hemarthrosis. Read more about this study

Who is it for?: Ages 8 to 60 years, men only
Conditions the study is for: Haemophilia
How many people: 104 participants
Where: 1 siteSpain
Study dates: May 2014to Feb 2016
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 8 to 60 years
Men only
No healthy volunteers
Child, Adult

Study population: Patients with haemophilia attending hemophilia shelters organized by the Federación Española de Hemofilia (Fedhemo) in the Centro de Formación Permanente en Hemofilia La Charca (Murcia, Spain).

Who may be able to join

Patients with hemophilia A and B.
Patients of all ages (pediatric, adolescents, youth and adults).
With or without joint involvement clinically diagnosed, and with or without a previous history of hemarthrosis joint loading.
Patients with or without inhibitors.

Who cannot join

Patients without prior walking capacity.
Patients diagnosed with other congenital coagulopathy (von Willebrand disease, etc..).
Patients with neurological or cognitive impairments that prevent understanding the questionnaires and physical tests.

Trial locations and contacts

1 site in 1 country.

Spain1 site

Universidad de Murcia
Murcia, Murcia, 30100

Other trials in Spain

About this study

Official title: Multidisciplinary Assessment of the Physical, Functional and Psychosocial Alterations in Patients With Hemophilia. An Observational Study.

Multidisciplinary assessment of the physical, functional and psychosocial alterations in patients with hemophilia around the country. The aims of this study are:

Descriptive study of joint involvement in hemophilia patients with and without a history of hemarthrosis.
Descriptive study of the alterations of periarticular muscle strength as a result of acute processes or the development of chronic articular sequelae.
Descriptive study of biomechanical changes in lower limb joints and their impact on gait in patients with hemophilia.
Descriptive study of the joint space, regarding hemarthrosis and synovitis, intraarticular by ultrasonography.
Descriptive study of the relationship between skeletal muscle pathology in patients with hemophilia and their perceptions of it and their quality of life.

Read the detailed description

Observational study that will allow us:

Analyze and evaluate the skeletal muscle of patients with hemophilia involvement.

Describe the periarticular muscular deficit, depending on the degree of injury, age, presence of inhibitors and the development of physical activity.

Identify the relationship between the functional deficit by clinical assessment performed with the use of validated scales measure force production with patients.

Observe the presence of joint bleeds in children with hemophilia and synovial hypertrophy developing therein.

Detect biomechanical abnormalities of the lower limbs and their impact on gait in patients with hemophilia.

Assess the factors that influence the perception of illness and quality of life of patients with hemophilia.

Conditions: Haemophilia
Keywords: Haemophilia, Arthropathy, Range of motion, Strength, Proprioception, Gait, Quality of Life.
Medical subject headings: Hemophilia A, Joint Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02198430
Study group	Role	What they receive
Patients		Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.
Control group		Children without hemophilia

What is being measured

The primary outcome is the main question the study is designed to answer.

Assess the Joint Damage
Measurement with Haemophilia Joint Health Score 2.1 (HJHS)
Measured: Screening visit
Assess the Perception of Quality of Life
Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).
Measured: Screening visit

Secondary outcomes (2)

Assessment of Clinical Patient Variables
Hemophilia type measuring (A or B)
Measured: Screening visit
Measure of Weight
Measure of weight
Measured: Screening visit (pretreatment assessment)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2014Feb 2016

First posted23 Jul 2014 (estimated)
Study startMay 2014
Primary completionNov 2015
Study completionFeb 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for HaemophiliaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 104 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02198430
Sponsor's protocol ID: Hemo-HOLISTIC
Other IDs: HOLISTIC
Acronym: HOLISTIC
Status last verified: Jun 2016
First submitted: 22 Jul 2014
Last updated: 20 Oct 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

All Haemophilia trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Multidisciplinary Assessment of the Physical, Functional and Psychosocial Alterations in Patients With Hemophilia. An Observational Study.
ClinicalTrials.gov ID: NCT02198430
Acronym / sponsor ID: HOLISTIC / Hemo-HOLISTIC
Conditions: Haemophilia
Keywords: Haemophilia, Arthropathy, Range of motion, Strength, Proprioception, Gait, Quality of Life.
Study type: Observational
Recruitment status: Completed
Enrolment: 104 participants (actual)
Lead sponsor: Universidad de MurciaUniversity, hospital or other
Study start: May 2014
Primary completion: Nov 2015 (actual)
Study completion: Feb 2016 (actual)
First posted: 23 Jul 2014 (estimated)
Results: Posted 20 Oct 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Multidisciplinary assessment of the physical, functional and psychosocial alterations in patients with hemophilia around the country. The aims of this study are:

Descriptive study of joint involvement in hemophilia patients with and without a history of hemarthrosis.
Descriptive study of the alterations of periarticular muscle strength as a result of acute processes or the development of chronic articular sequelae.
Descriptive study of biomechanical changes in lower limb joints and their impact on gait in patients with hemophilia.
Descriptive study of the joint space, regarding hemarthrosis and synovitis, intraarticular by ultrasonography.
Descriptive study of the relationship between skeletal muscle pathology in patients with hemophilia and their perceptions of it and their quality of life.

Detailed description

Observational study that will allow us:

Analyze and evaluate the skeletal muscle of patients with hemophilia involvement.

Describe the periarticular muscular deficit, depending on the degree of injury, age, presence of inhibitors and the development of physical activity.

Identify the relationship between the functional deficit by clinical assessment performed with the use of validated scales measure force production with patients.

Observe the presence of joint bleeds in children with hemophilia and synovial hypertrophy developing therein.

Detect biomechanical abnormalities of the lower limbs and their impact on gait in patients with hemophilia.

Assess the factors that influence the perception of illness and quality of life of patients with hemophilia.

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 2

Arm	Type	Receives
Patients	–	–Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.
Control group	–	–Children without hemophilia

Who may qualify

Ages: 8 to 60 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child, Adult
Study population: Patients with haemophilia attending hemophilia shelters organized by the Federación Española de Hemofilia (Fedhemo) in the Centro de Formación Permanente en Hemofilia La Charca (Murcia, Spain).

Key inclusion criteria (4 listed)

Patients with hemophilia A and B.
Patients of all ages (pediatric, adolescents, youth and adults).
With or without joint involvement clinically diagnosed, and with or without a previous history of hemarthrosis joint loading.
Patients with or without inhibitors.

Key exclusion criteria (3 listed)

Patients without prior walking capacity.
Patients diagnosed with other congenital coagulopathy (von Willebrand disease, etc..).
Patients with neurological or cognitive impairments that prevent understanding the questionnaires and physical tests.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

Assess the Joint Damage
Time frame: Screening visit
Measurement with Haemophilia Joint Health Score 2.1 (HJHS)
Assess the Perception of Quality of Life
Time frame: Screening visit
Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).

2 secondary outcomes

Assessment of Clinical Patient Variables
Time frame: Screening visit
Measure of Weight
Time frame: Screening visit (pretreatment assessment)

Results and safety

Results posted to the registry on 20 Oct 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Patients	53	53
Control Group	51	51

Primary outcome: Assess the Joint Damage

Time frame: Screening visit. Mean (Standard Deviation). Unit: points

Measure	Patients n = 53	Control Group n = 51
Value	0.36 (0.84)	0 (0)

Population analysed: Haemophilia Joint Health Score assesses joint health in patients with hemophilia. It consists of eight dimensions: swelling, muscular atrophy, crepitation and range of motion, joint pain, strength, motion and axial alignment. The score range is from 0 to 24 points (a score of 0 indicates no joint damage. The higher the score, the higher).

Primary outcome: Assess the Perception of Quality of Life

Time frame: Screening visit. Mean (Standard Deviation). Unit: points

Measure	Patients n = 53	Control Group n = 51
Value	38.32 (4.72)	37.55 (5.91)

Population analysed: The score ranges from 0 (poor QoL) to 100 points (good perception of QoL).

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Serious and Other (Not Including Serious) Adverse Events were not collected/assessed. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Patients	0 of 0	0 of 0	Not reported
Control Group	0 of 0	0 of 0	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Haemophilia

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Rubén Cuesta-Barriuso, PhD principal investigator

Catholic University San Antonio of Murcia

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Haemophilia, run by Universidad de Murcia at 1 site in Spain.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Assess the Joint Damage (time frame: Screening visit). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02198430.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2016.