Individualized Prophylaxis for Severe Hemophilia A Children | NCT02116855

Studies
Individualized Prophylaxis for Severe Hemophilia A Children

NCT02116855

Sponsor

Beijing Children's Hospital

Status

Unknown status

Last Update Posted

Apr 17, 2014Estimated

Enrollment

50Estimated

Phase

Not Applicable

Conditions

Hemophilia

Interventions

according to the efficacy of AJBRs

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2014

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Not yet recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2014

Primary Completion Date

Jun 2016Estimated

Completion Date

Not provided

First Submitted Date

Apr 15, 2014

First Submission Date that Met QC Criteria

Apr 15, 2014

First Posted Date

Apr 17, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 15, 2014

Last Update Posted Date

Apr 17, 2014Estimated

Description Module

Brief Summary

A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose regimen in step I, an escalated low dose regimen in step II and a tailored dose regimen based on individual PK profiles in step III. The dose escalation criteria are adjusted according to patterns and frequencies of joint bleeding and assessed in each subject every 3 months. Efficacy of the 3 different dose regimens are measured by the Annualized Joint Bleeding rate (AJBR) as a primary end point and the Hemophilia Joint Health Score (HJHS ) and QoL scores (CHO-KLAT and PedsQoL) , image studies of target joints by Ultrasound, X-ray and MRI examinations, consumption of factor VIII and inhibitor rates as secondary end points.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

50Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
tertiary prophylaxis prophy on Hemophilia A patiets	Experimental	A multi centre 2 year long term tertiary prophylaxis study designed with a three step escalating dose protocol adjusted by individual joint bleeding patterns/frequencies to study the effect and cost saving of 3 low dose /cost prophylaxis regimens in boys with severe hemophilia A and arthropathy in China. Each patient will start treatment with a low dose regimen in step I and be assessed every 3 months according to the escalating criteria.	Other: according to the efficacy of AJBRs

Interventions

Name	Type	Description	Arm Group Labels	Other Names
according to the efficacy of AJBRs	Other		tertiary prophylaxis prophy on Hemophilia A patiets

Measure	Description	Time Frame
AJBRs (annualized Joint bleeding rates)		three months

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Severe hemophilia A with FVIII <1 %
Age from 6 to 10 years
Patients with more than 50 exposure day (ED)
Patients with one or more target joints (a target joint is defined as a joint with 3 or more bleeds in a consecutive 3 months) or disease joints (defined as presence of visible joint swelling and /or limitation of movements and/or joint deformities in the absent of an acute joint bleed )
on-demand treatment more than 12months before the study

Exclusion Criteria:

A history of FVIII inhibitor (titer greater than 0.6 BU) and detectable FVIII inhibitor at screening (titer greater than 0.6 BU)
Chronic renal failure (serum creatinine greater than 2.0 mg /dL)
Chronic Liver disease (ALT greater than 200 U/L))
Patients with clinically documented immunodeficiencies
Patients anticipated to require Major surgery
Patients with competing high risks such as symptomatic HIV infection, Juvenile rheumatoid arthritis, metabolic bone diseases, or other diseases known to mimic or cause joint diseases
Patients live too far from the comprehensive care centre at BCH and had demonstrated previous non compliance to therapies or clinical studies

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

10 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Name	Role	Phone	Extension	Email
Hui R Wu, MD	Contact	8613911383480		runhuiwu@hotmail.com

Name	Affiliation	Role
hui R Wu, MD	Beijing Children's Hospital	Study Chair

Condition Browse Module

MeSH Terms

ID	Term
D006467	Hemophilia A

Ancestor Terms

ID	Term
D025861	Blood Coagulation Disorders, Inherited
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

Browse Leaves

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Browse Branches

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