Hemophilia APhase 3NCT02930317
Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
A Phase 3 study testing Recombinant Factor VIII (50 IU/kg) and Recombinant Factor VIII (On-demand treatment) in people with Hemophilia A, run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. at 4 sites in China.
Trial summary
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years) patients with severe or moderately severe Hemophilia A. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Factor VIII (50 IU/kg) and Recombinant Factor VIII (On-demand treatment)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 4 sitesChina
- Study dates
- Aug 2016to Dec 2017
- Phase
- Phase 3
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of hemophilia A
- Age of 12 Years to 65 Years，Diagnosis of severe （defined as <1% FVIII:C documented in medical records） or moderately severe（defined as 1%-5% FVIII:C documented in medical records） hemophilia A .Subjects who（Age of 18 Years to 65 Years） have received or are currently receiving FVIII products (plasma-derived and/or recombinant FVIII) and have had >150 exposure days (EDs) with a FVIII product；The Callan (Age of 12 Years to 17) have received FVIII products and have had>50 EDs a FVIII product.
- Subjects without a past history of, or current no factor VIII inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer Lower than the laboratory's normal range or <0.6 BU/mL (BU:Bethesda Units ).
- Liver and kidney function in accordance with the standard
- Subjects of childbearing potential should agree to use and utilize an adequate method of contraception throughout treatment and for at least 28 days after study is stopped
- Evidence of a personally or legally acceptable representative (legally acceptable representative is only applicable to Callan subjects) signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study
- The part one of subjects subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures; Subjects must be in a non bleeding state before the administration of rFVIII on Day 1; Subjects should not have received an infusion of any FVIII products for at least 3 days (at least 72 hours) before the administration of rFVIII on Day 1
Who cannot join
- Current FVIII inhibitor or history of FVIII inhibitor (>0.6 BU/mL )
- Diagnosed with any bleeding disorder in addition to hemophilia
- Documented Human Immunodeficiency Virus (HIV)
- Subjects anticipating elective surgery or other invasive procedure within 1 month following study entry
- Treatment with an immunomodulatory within 30 days or 5 half lives preceding Day 1, whichever is longer
- Subjects with known hypersensitivity to the active substance or to any of the excipients of rFVIII. Subjects with a known hypersensitivity to Chinese Human embryonic kidney cell proteins
Show all 11Show fewer
- Subjects with severe anemia requiring blood transfusion
- Subjects with significant hepatic or renal impairment (alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >5 x ULN, or total bilirubin >2 x ULN or serum creatinine >2 x ULN), prothrombin time >1.5 x ULN, platelet count <80,000 μL. History of sensitivity to heparin or heparin induced thrombocytopenia or others thrombocytopenia
- Patients with heart surgery history requires anticoagulation therapy; Subjects with severe heart disease, including myocardial infarction or heart failure Grade 3 or higher(NYHA Classification)
- Blood pressure unable to be controlled ideally(systolic pressure>150 mmHg，diastolic pressure>90 mmHg)
- Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study
Trial locations and contacts
4 sites in 1 country.
Lei Zhang, Doctor contact
China4 sites, 4 recruiting
- Recruiting
Anhui provincial hospital
Hefei, Anhui, 230001
Zimin Sun, Doctor
- Not yet recruiting
Ruijin Hospital Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality, 200025
Xuefeng Wang, Doctor
- Not yet recruiting
Second hospital of Shanxi Medical University
Taiyuan, Shanxi, 030001
Linhua Yang, Doctor
- Recruiting
Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology)
Tianjin, Tianjin Municipality, 300020
Lei Zhang, Doctor
About this study
Official title: An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of rFVIII in the Prevention and Treatment of Bleeding Episodes in Chinese With Hemophilia A
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years) patients with severe or moderately severe Hemophilia A. To determine the pharmacokinetic (PK) parameters of rFVIII.
- Conditions
- Hemophilia A
- Being tested
- Recombinant Factor VIII (50 IU/kg), Recombinant Factor VIII (On-demand treatment)
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Factor VIII (50 IU/kg)Drug
Recombinant Factor VIII (On-demand treatment)Drug
| Study group | Role | What they receive |
|---|---|---|
| Pharmacokinetic parameters | Experimental | Pharmacokinetic parameters of rFVIII measured in subset of 10 participants, consisting of:18 Years to 65 Years. In Part 1 of the study, subjects received a single intravenous infusion of 50 IU/kg rFVIII preceded by a 72 hours washout period. Receives: Drug: Recombinant Factor VIII (50 IU/kg) |
| On-demand treatment | Experimental | On-demand treatment with rFVIII for 6 months age 12 Years to 65 Years. In Parts 2 of the study, subjects received repeat injections of rFVIII either as an on-demand or prophylaxis regimen at a dose and frequency determined by their study doctor. Receives: Drug: Recombinant Factor VIII (On-demand treatment) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recovery rate = (change actual value of rFVIII activity before and after infusion )/(change expected value of rFVIII activity before and after infusion)*100%
Measured: At 15 and 60 minutes after the first infusion
Investigator Hemostatic Efficacy Assessment 6 Hours Post Infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Measured: 6 hours post infusion
Secondary outcomes (7)
The proportion of subjects who achieved the expected effect after the first infusion of the rFVIII
Measured: At 15 minutes after the first infusion
change actual value of rFVIII activity before and after infusion levels
Measured: At 15 and 60 minutes after the first infusion
FVIII Maximum Plasma Concentration
Measured: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Time to Reach Maximum Observed Plasma Concentration
Measured: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Area Under the Plasma Concentration Versus Time Curve From 0 to 48 Hours
Measured: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Terminal Elimination Half-Life (t1/2)
Measured: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Measured: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2016 (estimated)
- Study startAug 2016
- Primary completionOct 2017 (estimated)
- Study completionDec 2017 (estimated)
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 Chia Tai Tianqing Pharmaceutical Group Co., Ltd.The 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02930317
- Sponsor's protocol ID
- CTTQ-NXBYZ
- Status last verified
- Oct 2016
- First submitted
- 9 Sep 2016
- Last updated
- 12 Oct 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of rFVIII in the Prevention and Treatment of Bleeding Episodes in Chinese With Hemophilia A
- ClinicalTrials.gov ID
- NCT02930317
- Acronym / sponsor ID
- CTTQ-NXBYZ
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Aug 2016
- Primary completion
- Oct 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 12 Oct 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.
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years) patients with severe or moderately severe Hemophilia A. To determine the pharmacokinetic (PK) parameters of rFVIII.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pharmacokinetic parameters | Experimental | Drug: Recombinant Factor VIII (50 IU/kg)Pharmacokinetic parameters of rFVIII measured in subset of 10 participants, consisting of:18 Years to 65 Years. In Part 1 of the study, subjects received a single intravenous infusion of 50 IU/kg rFVIII preceded by a 72 hours washout period. |
| On-demand treatment | Experimental | Drug: Recombinant Factor VIII (On-demand treatment)On-demand treatment with rFVIII for 6 months age 12 Years to 65 Years. In Parts 2 of the study, subjects received repeat injections of rFVIII either as an on-demand or prophylaxis regimen at a dose and frequency determined by their study doctor. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of hemophilia A
- Age of 12 Years to 65 Years，Diagnosis of severe （defined as <1% FVIII:C documented in medical records） or moderately severe（defined as 1%-5% FVIII:C documented in medical records） hemophilia A .Subjects who（Age of 18 Years to 65 Years） have received or are currently receiving FVIII products (plasma-derived and/or recombinant FVIII) and have had >150 exposure days (EDs) with a FVIII product；The Callan (Age of 12 Years to 17) have received FVIII products and have had>50 EDs a FVIII product.
- Subjects without a past history of, or current no factor VIII inhibitor. For laboratory-based assessments, any Bethesda inhibitor titer Lower than the laboratory's normal range or <0.6 BU/mL (BU:Bethesda Units ).
- Liver and kidney function in accordance with the standard
- Subjects of childbearing potential should agree to use and utilize an adequate method of contraception throughout treatment and for at least 28 days after study is stopped
Key exclusion criteria (11 listed)
- Current FVIII inhibitor or history of FVIII inhibitor (>0.6 BU/mL )
- Diagnosed with any bleeding disorder in addition to hemophilia
- Documented Human Immunodeficiency Virus (HIV)
- Subjects anticipating elective surgery or other invasive procedure within 1 month following study entry
- Treatment with an immunomodulatory within 30 days or 5 half lives preceding Day 1, whichever is longer
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 |
|---|---|---|
| Recombinant Factor VIII (50 IU/kg) | Drug | Pharmacokinetic parameters |
| Recombinant Factor VIII (On-demand treatment) | Drug | On-demand treatment |
Primary outcome
Recovery rate = (change actual value of rFVIII activity before and after infusion )/(change expected value of rFVIII activity before and after infusion)*100%
Time frame: At 15 and 60 minutes after the first infusion
Investigator Hemostatic Efficacy Assessment 6 Hours Post Infusion
Time frame: 6 hours post infusion
The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
7 secondary outcomes
The proportion of subjects who achieved the expected effect after the first infusion of the rFVIII
Time frame: At 15 minutes after the first infusion
change actual value of rFVIII activity before and after infusion levels
Time frame: At 15 and 60 minutes after the first infusion
FVIII Maximum Plasma Concentration
Time frame: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Time to Reach Maximum Observed Plasma Concentration
Time frame: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Area Under the Plasma Concentration Versus Time Curve From 0 to 48 Hours
Time frame: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Terminal Elimination Half-Life (t1/2)
Time frame: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Time frame: Time Frame: Pre-dose and 0.25, 0.5, 1, 1.5, 2, 3, 6, 9, 24,36,and 48 hours post-dose
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2016.
- Individual participant dataSharing planned: Undecided
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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.
Lei Zhang, Doctor contact
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Factor VIII (50 IU/kg) and Recombinant Factor VIII (On-demand treatment) in people with Hemophilia A, run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. at 4 sites in China.
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 7 inclusion and 11 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: status not confirmed, as of the sponsor's last update on 12 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 4 sites in China. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lei Zhang, Doctor (zlpumc@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recovery rate = (change actual value of rFVIII activity before and after infusion )/(change expected value of rFVIII activity before and after infusion)*100% (time frame: At 15 and 60 minutes after the first infusion). 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?
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 12 Oct 2016; AzTrials last refreshed its copy on 26 Oct 2026.