Hemophilia APhase 4NCT02727647

Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients

A Phase 4 study testing FVIII in people with Hemophilia A, run by Chulalongkorn University at 1 site in Thailand.

Completed

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

Trial summary

Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of hemophilia treatment in term of breakthrough bleeding, number of hospital stay and day-off… Read more about this study

Who is it for?: Ages 6 months to 20 yearsHealthy volunteers can take part
Conditions the study is for: Hemophilia A
What is being tested?: FVIII
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 16 participants
Where: 1 siteThailand
Study dates: May 2015to Mar 2016
Phase: Phase 4
Are there results?: None posted in the registry

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 6 months to 20 years
All sexes
Healthy volunteers accepted
Child, Adult

Who may be able to join

Hemophilia patients with moderate (factor level 1-3%) or severe (factor level <1%) severity

Who cannot join

Platelet count less than 100,000 /mm3 or other bleeding tendency
Hemophilia patients who have FVIII inhibitor >0.6 BU (modified Nijmogen method)
Hemophilia patients who have no bleeding symptoms

Trial locations and contacts

1 site in 1 country.

Thailand1 site

Division of Hemato/oncology, Department of Pediatrics, Faculty of medicine,Chulalongkorn U
Bangkok, Pathumwan, 10330

Other trials in Thailand

About this study

Study design: Single center clinical trials

Concise methodology:

Study Population: Children with hemophilia A who received treatment at KCMH from May 2015 to March 2016 will be enrolled in this study. The consent will be obtained before the study.
Observation and measurement:
History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF)
Measurement:
Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment score
Data analysis: The p-value of less than 0.05 will be considered statistically significant. Mann-Whitney test will be used to test correlation of these variables (CBC, Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L quality of life assessment score) Sample size: 16 patients

Potential impacts:

The outcomes of different factor VIII concentrate dose between 15-20 U/kg/dose 2 times/week and 35-40 U/kg/dose 1 time/week will be revealed. These outcomes include number of breakthrough bleeding, number of hospital stay, day-off from school, joint health and quality of life. The result of this study will guide further study on optimal dose and duration of factor VIII treatment of hemophilia A patients in the future.

Conditions: Hemophilia A
Being tested: FVIII
Keywords: Moderate hemophilia A, Severe hemophilia A, Intermediate dose prophylaxis, Low dose prophylaxis
Medical subject headings: Hemophilia A

What participants receive

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

FVIIIDrug
Also called Hemofil-M
FVIII concentration 35-40 U/kg/dose 1 time/week for 5 months
FVIIIDrug
Also called Hemofil-M
FVIII concentration 15-20 U/kg/dose 1 time/week for 5 months

Study groups in NCT02727647
Study group	Role	What they receive
Arm 1	Experimental	FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months Receives: Drug: FVIII
Arm 2	Experimental	FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months Receives: Drug: FVIII

What is being measured

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

Annualized bleeding rate per year
Measured: 10 months

Secondary outcomes (5)

Hemophilia joint health score
Measured: 10 months
Amount of FVIII use
Measured: 10 months
Quality of life score
EQ-5D-5L (Thailand version)
Measured: 10 months
Number of hospital stays
Measured: 10 months
Number of school days loss
Measured: 10 months

Timeline

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

May 2015Mar 2016

First posted4 Apr 2016 (estimated)
Study startMay 2015
Primary completionMar 2016
Study completionMar 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Hemophilia AStudies of the same condition, open and under way.
More trials run by Chulalongkorn UniversityThe sponsor's other registered studies.
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: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02727647
Sponsor's protocol ID: 3125056
Status last verified: Oct 2015
First submitted: 30 Mar 2016
Last updated: 4 Apr 2016
Results posted: No
Data monitoring committee: No
Individual data sharing: 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 Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 4 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
ClinicalTrials.gov ID: NCT02727647
Acronym / sponsor ID: 3125056
Conditions: Hemophilia A
Keywords: Moderate hemophilia A, Severe hemophilia A, Intermediate dose prophylaxis, Low dose prophylaxis
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: Chulalongkorn UniversityUniversity, hospital or other
Study start: May 2015
Primary completion: Mar 2016 (actual)
Study completion: Mar 2016 (actual)
First posted: 4 Apr 2016 (estimated)
Results: Not posted in the source record

Clinical summary

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

Study design: Single center clinical trials

Concise methodology:

Study Population: Children with hemophilia A who received treatment at KCMH from May 2015 to March 2016 will be enrolled in this study. The consent will be obtained before the study.
Observation and measurement:
History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF)
Measurement:
Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment score
Data analysis: The p-value of less than 0.05 will be considered statistically significant. Mann-Whitney test will be used to test correlation of these variables (CBC, Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L quality of life assessment score) Sample size: 16 patients

Potential impacts:

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Arm 1	Experimental	Drug: FVIIIFVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months
Arm 2	Experimental	Drug: FVIIIFVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months

Who may qualify

Ages: 6 months to 20 years
Sex: All
Healthy volunteers: Accepted
Age groups: Child, Adult

Key inclusion criteria (1 listed)

Hemophilia patients with moderate (factor level 1-3%) or severe (factor level <1%) severity

Key exclusion criteria (3 listed)

Platelet count less than 100,000 /mm3 or other bleeding tendency
Hemophilia patients who have FVIII inhibitor >0.6 BU (modified Nijmogen method)
Hemophilia patients who have no bleeding symptoms

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

Interventions

Intervention	Type	Arms
FVIIIAlso: Hemofil-MFVIII concentration 35-40 U/kg/dose 1 time/week for 5 months	Drug	Arm 1
FVIIIAlso: Hemofil-MFVIII concentration 15-20 U/kg/dose 1 time/week for 5 months	Drug	Arm 2

Primary outcome

Annualized bleeding rate per year
Time frame: 10 months

5 secondary outcomes

Hemophilia joint health score
Time frame: 10 months
Amount of FVIII use
Time frame: 10 months
Quality of life score
Time frame: 10 months
Number of hospital stays
Time frame: 10 months
Number of school days loss
Time frame: 10 months

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2016.
Individual participant dataSharing planned: No

Related trials for Hemophilia A

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.

Darintr Sosothikul, MD principal investigator

Chulalongkorn University

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

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing FVIII in people with Hemophilia A, run by Chulalongkorn University at 1 site in Thailand.

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 1 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 4 Apr 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 Thailand. 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.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Annualized bleeding rate per year (time frame: 10 months). 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 NCT02727647.

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 4 Apr 2016.