Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A

October 29, 2014 updated by: Yongjun Fang,MD, Nanjing Medical University

Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A.

Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or defective coagulation factor VIII (FVIII). Prophylaxis is recommended as the standard of care for boys with severe haemophilia by WHO and World Federation Of Hemophilia (WFH). The efficacy and safety of prophylaxis in preventing bleeds and arthropathy in patients with hemophilia has been confirmed in well-designed clinical studies.To keep the factor level above 1%, the standard dosage for patients with severe hemophilia A is 20-40 Units /kg/infusion (average 30 Units /kg) every other day or three times a week. This dosage has a very high consumption of factor, up to 5000-6000 international unit (IU)/kg/year. The high consumption of factor and cost present a major barrier to use the standard prophylaxis in many countries particularly in the developing world.

In China the majority of boys with severe hemophilia A can only pay for on-demand treatment or low-dose prophylaxis. Ao after the affordability of patients was solved and many patients will get more chance to receive standard prophylaxis.

This study is designed to evaluate the Annual Bleeding rate (ABR), joint health outcomes and QoL outcomes in subjects using ADVATE(Recombinant Human Coagulation Factor VIII for injection) standard prophylaxis under the conditions of routine practice.

Study Overview

Status

Unknown

Conditions

Study Type

Interventional

Enrollment (Anticipated)

15

Phase

  • Not Applicable

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Subject has hemophilia A with FVIII≤2 %.
  2. Previously treated patients (PTPs).
  3. Age from 2 to 18 years.

Exclusion Criteria:

  1. Subject has known hypersensitivity to the active substance or any of the excipients.
  2. Subject has known allergic reaction to mouse or hamster proteins.
  3. Subject has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or is scheduled to participate in another clinical study involving another FVIII concentrate or device during the course of this study.
  4. Subject is planned, or likely to have surgery during the study period.
  5. Subject has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator.
  6. Subject has full-blown Acquired Immuno Deficiency Syndrome (AIDS),determined by Cluster Determinant 4+(CD4+) and clinical presentation.
  7. Subject has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 5 times the upper limit of normal).
  8. Subject has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent and persistent international normalized ratio (INR)> 1.4, and/or the presence of splenomegaly and/or significant spider angioma on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices.
  9. The subject in the opinion of the investigator is unable or unwilling to comply with study protocol
  10. Subject is a family member of the investigator or site staff

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ADVATE
The baseline ABR will be assessed from bleeding log and clinic records from preceding year. Subjects will initially be treated standard prophylaxis(20 - 40 IU/Kg body weight every 48 ± 6 hours) with Recombinant Human Coagulation Factor VIII for injection(ADVATE) for 1 year. Subjects must be prescribed ADVATE by the treating physician. Data will be collected over a period of 2 years from the time of study enrollment. Study visits are to coincide with routinely rescheduled and emergency visits. Available data from these visits shall be transcribed onto the case report forms (CRFs).
Subjects will initially be treated standard prophylaxis(20 - 40 IU/Kg body weight 2 times one week) with ADVATE for 1 year.
Other Names:
  • ADVATE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ABR
Time Frame: 3 months
To evaluate the Annual Bleeding rate (ABR) in subjects using standard prophylaxis under the conditions of routine practice.
3 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
incidence of new target joints
Time Frame: 3 months
Incidence of new target joints.
3 months
Pettersson score of joint
Time Frame: 3 months
Status of joint health by X-ray using Pettersson score
3 months
magnetic resonance imaging scoring of joint
Time Frame: 6 months
Status of joint health by magnetic resonance imaging scoring system.
6 months
Hemophilia Joint Health Score (HJHS) of joint
Time Frame: 3 months
Status of joint health using HJHS
3 months
Number of ADAVTE units
Time Frame: 3 months
Number of Recombinant Human Coagulation Factor VIII for injection (ADAVTE) units required for bleed cessation and Number of ADVATE infusions required for bleed cessation
3 months
QoL of patients wiht hemophilia
Time Frame: 3 months
QoL assessment by Canadian Haemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT)
3 months
Inhibitor rate
Time Frame: 3 months
Inhibitor rate monitoring: inhibitor will be tested at entry of study, every 3 months and at end of study.
3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2014

Primary Completion (Anticipated)

December 1, 2016

Study Completion (Anticipated)

December 1, 2016

Study Registration Dates

First Submitted

October 28, 2014

First Submitted That Met QC Criteria

October 29, 2014

First Posted (Estimate)

October 31, 2014

Study Record Updates

Last Update Posted (Estimate)

October 31, 2014

Last Update Submitted That Met QC Criteria

October 29, 2014

Last Verified

October 1, 2014

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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