Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.

August 16, 2019 updated by: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Multicenter, Noncontrol Study of Recombinant Coagulation Factor VIII for Prophylactic Therapy in Subjects With Hemophilia A.

This is a multi-center, open-label, single-arm trial to evaluate the efficacy and safety of Recombinant Coagulation Factor VIII in patients with hemophilia A . Recombinant Coagulation Factor VIII is prophylactic administrated 25 - 35 IU/kg once every other day or three times per week which should be continuous for 24 weeks.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

64

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Anhui
      • Hefei, Anhui, China, 230001
        • Recruiting
        • Anhui Provincial Hospital
        • Contact:
    • Chongqing
      • Chongqing, Chongqing, China, 400010
        • Recruiting
        • The Second Affiliated Hospital of Chongqing Medical University
        • Contact:
        • Principal Investigator:
          • Shifeng Lou
    • Fujian
      • Fuzhou, Fujian, China, 350001
        • Recruiting
        • Fujian Medical University Union Hospital
        • Contact:
        • Principal Investigator:
          • Meijuan Huang
    • Gansu
      • Lanzhou, Gansu, China, 730000
        • Recruiting
        • The First Hospital of Lanzhou University
        • Contact:
        • Principal Investigator:
          • Yaming Xi
    • Guangxi
      • Nanning, Guangxi, China, 530021
        • Recruiting
        • The First Affiliated Hospital of Guangxi Medical University
        • Contact:
        • Principal Investigator:
          • Peng Cheng
    • Henan
      • Zhengzhou, Henan, China, 450003
        • Recruiting
        • Henan Provincial Peoples Hospital
        • Contact:
        • Principal Investigator:
          • Zhen Wang
      • Zhengzhou, Henan, China, 450008
        • Recruiting
        • HeNan Cancer Provincial Hospital
        • Contact:
        • Principal Investigator:
          • Hu Zhou
    • Hunan
      • Changsha, Hunan, China, 410013
        • Recruiting
        • Xiangya Hospital Central South University
        • Contact:
    • Jiangsu
      • Nanjing, Jiangsu, China, 210008
        • Recruiting
        • Nanjing Drum Tower Hospital
        • Contact:
        • Principal Investigator:
          • Rongfu Zhou
    • Jiangxi
      • Nanchang, Jiangxi, China, 330006
        • Recruiting
        • Jiangxi Provincial People's Hospital
        • Contact:
        • Principal Investigator:
          • Chenghao Jin
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Recruiting
        • Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
        • Contact:

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

12 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Hemophilia A.
  2. FVIII:C <1%. 3）12 and 65 years old.

4）Has received FVIII treatment and the treatment exposure days ≥100. 5）Has bleeding treatment records of at least 3 months before randomization. 6）FVIII inhibitor assay results is negative. 7） Subjects should agree to use an adequate method of contraception during the study.

8）Understood and Signed an informed consent form.

Exclusion Criteria:

  1. Has a history or family history of blood coagulation factor VIII inhibitor.
  2. Has other coagulation dysfunction diseases in addition to hemophilia A.
  3. HIV positive.
  4. Plan to receive surgery during the trial.
  5. Has used immunomodulator within one weeks before the first dose，and less than 7 half-life periods.
  6. Known to be allergic to experimental drugs or any excipients.
  7. Severe anemia and need blood transfusion.
  8. Serious liver or kidney damage.
  9. Serious heart disease.
  10. Uncontrollable hypertension.
  11. Has participated in other clinical studies within one month before the first dose.
  12. The researchers believe that it is not suitable for participants.

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Prophylactic therapy
Subjects received Recombinant Human Coagulation FVIII for prophylactic therapy with 25 - 35 IU/kg injection once every other day or three times per week for 6 months.
Drug: Recombinant Human Coagulation FVIII
A kind of Recombinant Coagulation Factor VIII Injection produced by sponsor.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized joint bleeding rate
Time Frame: up to 24 weeks
Annualized joint bleeding rate（AJBR） can be calculated using the following formula: Number of joint bleeding events during efficacy evaluation period/(number of days in treatment period/365.25).
up to 24 weeks
Incremental Recovery of the First Dose
Time Frame: After the first dose on day 1
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection
After the first dose on day 1

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Bleeding Event Treatment Efficacy
Time Frame: up to 24 weeks
The investigator shall evaluate the hemostatic effect after the treatment of every bleeding event of subjects based on a four-point scale(excellent, good, moderate, not relieved).
up to 24 weeks
Monthly Average Number of Bleedings
Time Frame: up to 24 weeks
Number of bleeding events in each month.
up to 24 weeks
The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode
Time Frame: up to 24 weeks
The number and dose of injection of Recombinant Human Coagulation FVIII required per bleeding episode
up to 24 weeks
Incremental Recovery of Duplicated Dose
Time Frame: up to 12 weeks, 24 weeks
Peak activity of FVIII (as Cmax) measured within 1 hour after the end of injection.
up to 12 weeks, 24 weeks
Hemophilia Joint Health Score (HJHS)
Time Frame: Baseline, week 24
The scale assesses the functional status of the six major joints of the elbow, knee, and ankle, including: joint swelling, swelling duration, muscle atrophy, muscle strength, joint friction, joint pain, joint extension, joint flexion Degree decline, overall gait, etc.
Baseline, week 24
Assess the impact of Recombinant Human Coagulation FVIII following Patient Reported Outcome (PRO) EQ-5D
Time Frame: Baseline, week 24
EQ-5D is a general questionnaire designed to measure health status on a scale of 0-100 with the higher value representing a better outcome and record the participants' current health state in 5 domains mobility, selfcare, usual activities, pain, anxiety.
Baseline, week 24

Collaborators and Investigators

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

Sponsor

Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

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 (Actual)

May 15, 2019

Primary Completion (Anticipated)

March 1, 2020

Study Completion (Anticipated)

March 1, 2020

Study Registration Dates

First Submitted

July 16, 2019

First Submitted That Met QC Criteria

August 16, 2019

First Posted (Actual)

August 19, 2019

Study Record Updates

Last Update Posted (Actual)

August 19, 2019

Last Update Submitted That Met QC Criteria

August 16, 2019

Last Verified

August 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CTTQ-NXBYZ-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

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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