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Hemophilia APhase 4NCT02170402

China ADVATE PTP Study

A Phase 4 study testing Octocog alfa (recombinant human coagulation factor VIII) in people with Hemophilia A, run by Baxalta now part of Shire at 11 sites in China.

Completed

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

Official record
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Trial summary

The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs) Read more about this study

Who is it for?
Any age, men only
Conditions the study is for
Hemophilia A
What is being tested?
Octocog alfa (recombinant human coagulation factor VIII)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
82 participants
Where
11 sitesChina
Study dates
26 Jun 2014to 31 May 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 Any age
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Main Inclusion Criteria:

  • Ethnic Chinese
  • is of any age
  • has a documented diagnosis of severe or moderately severe hemophilia A (congenital FVIII deficiency: baseline Factor VIII (FVIII) ≤ 2%)
  • has documented and verified >50 exposure days (EDs) to FVIII (recombinant or plasma derived)
  • is receiving on-demand treatment with FVIII at the time of enrolment in this study
  • has negative history of inhibitor development
  • is HIV negative or HIV positive with stable disease and CD4+ count ≥ 200 cells per mm^3
  • is negative for Hepatitis C virus (HCV); Or participant is HCV positive with chronic stable hepatitis as assessed by investigator

Main Exclusion Criteria:

  • has prior history of hypersensitivity or anaphylaxis associated with receipt of FVIII
  • is diagnosed with other bleeding disorder(s) other than hemophilia A, including but not limited to thrombocytopenia (platelet count < 100000 /mL)
  • has been exposed to an investigational product (IP) within 30 days prior to the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during participation in the study
  • is planned, or likely to have surgery during the study period
  • has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator
  • has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 5 times the upper limit of normal)
  • has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent \& 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
  • is a family member of the investigator or site staff

Trial locations and contacts

11 sites in 1 country.

11 sites in 1 country

China11 sites

  • Beijing Children's Hospital Affiliated to Capital University of Medical Sciences

    Beijing, 100045

  • Cangzhou Central Hospital

    Cangzhou, Hebei, 061001

  • Xiangya Hospital Central South University

    Changsha, Hunan, 410008

  • Peking Union Medical College Hospital

    Dongcheng, Beijing Municipality, 100730

  • Fujian Medical University Union Hospital

    Fuzhou, Fujian, 350001

  • The First Affiliated Hospital of College of Medicine, Zhengjiang University

    Hangzhou, Zhejiang, 310003

  • Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

    Shanghai, 200025

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu, 215006

  • Hospital of Blood Disease, Chinese Academy of Medical Sciences

    Tianjin, 300020

  • Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech/ Wuhan Union Hospital

    Wuhan, Hubei, 430022

  • Tongji Hospital of Tongji Medical College of Hongzhong Science and Techology University

    Wuhan, Hubei, 430030

Other trials in China

About this study

Official title: Study to Evaluate Efficacy and Safety of ADVATE in the Treatment of Previously Treated Patients With Hemophilia A

The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)

Conditions
Hemophilia A
Being tested
Octocog alfa (recombinant human coagulation factor VIII)
Medical subject headings
Hemophilia A

What participants receive

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

  • Octocog alfa (recombinant human coagulation factor VIII)Biological

    Also called ADVATE

    • Part 1: Pharmacokinetic (PK) analysis - Subset of 24 participants
    • Part 2: On-demand treatment regimen
    • Part 3: Prophylaxis treatment regimen
Study groups in NCT02170402
Study groupRoleWhat they receive
Previously Treated Patients (PTPs)ExperimentalPTPs will participate sequentially with: Part 1: Pharmacokinetic parameters of ADVATE measured in subset of 24 participants, consisting of: * 12 adults (>12 years of age) * 12 children (≤12 years of age) Part 2: On-demand treatment with ADVATE for 6 months Part 3: Prophylaxis regimen with ADVATE for 6 months
Receives: Biological: Octocog alfa (recombinant human coagulation factor VIII)

What is being measured

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

  1. Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment

    Computed as:

    {[median ABR on-demand - median ABR prophylaxis]÷[median ABR on-demand]}*100%

    The ABR, will be assumed to have a negative binomial distribution. The 2 treatment regimens (on-demand and prophylaxis) will be compared in terms of mean ABR within a generalized linear model framework (with a logarithmic link function which is the default for the negative binomial distribution), accounting for the fixed effect of study arm and the follow-up time (in years) as an offset. Ratios between treatment means (95% CI) will be estimated within this model.

    Measured: 12 months

Secondary outcomes (12)

  1. Number of units per kg body weight of ADVATE required to resolve a bleeding episode (BE)

    Measured: 12 months

  2. Number of infusions of ADVATE required to resolve a bleeding episode (BE)

    Measured: 12 months

  3. Overall evaluation of efficacy on a four-point scale (Excellent-Good-Fair-Poor)

    Measured: 12 months

  4. Annualized bleeding episode rates (ABR) according to bleed type and bleed etiology summarized by treatment regimen

    Bleed types and etiologies summarized by treatment regimen (prophylaxis, on-demand) including:

    • Joint bleeds
    • Non-joint bleeds
    • Spontaneous bleeds
    • Traumatic bleeds
    • Target joint bleeds

    Measured: 12 months

  5. Inhibitor incidence

    Inhibitor incidence in:

    1. Previously treated patients (PTPs) with previous 51-150 exposure days (EDs) to Factor VIII (FVIII)
    2. PTPs with previous >150 EDs to FVIII

    Measured: 13 months

  6. Adverse events according to relatedness, seriousness, and severity

    Measured: 13 months

  7. Area under the plasma concentration/time curve from time 0 to infinity

    Computed as AUC0-t + Ct/ λz, where t is the time of last quantifiable concentration, Ct is the last quantifiable concentration, and λz is the terminal rate constant

    Measured: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion

  8. Mean Residence Time (MRT)

    Computed as AUMC0-∞ / AUC0-∞ - TI/2, where AUMC0-∞ will be determined in a similar manner as AUC0-∞ and TI represents infusion duration [hour]

    Measured: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion

  9. Clearance (CL)

    Computed as Dose/ AUC0-∞

    Measured: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion

  10. Incremental Recovery (IR) at Cmax

    Computed as: (Cmax - Cpre-infusion)/Dose, where Cmax will be determined as the highest concentration achieved within one hour after infusion

    Measured: Within 30 minutes prior to the start of the infusion, and within 1 hour post-infusion

  11. Elimination phase half-life

    Computed as: ln2/ λz. λz will be estimated from the slope of natural log-linear fitting to latter quantifiable concentrations, with largest adjusted R^2

    Measured: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion

  12. Volume of distribution at steady state (Vss)

    Computed as: CL * MRT

    Measured: Within 30 minutes prior to the start of the infusion through 48 hours post-infusion

Timeline

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

  1. First posted23 Jun 2014 (estimated)
  2. Study start26 Jun 2014
  3. Primary completion31 May 2016
  4. Study completion31 May 2016

Resources

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
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
82 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02170402
Sponsor's protocol ID
061301
Status last verified
Apr 2021
First submitted
20 Jun 2014
Last updated
3 May 2021
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Octocog alfa (recombinant human coagulation factor VIII) in people with Hemophilia A, run by Baxalta now part of Shire at 11 sites in China.

The full registered description is in the About this study section and the official record.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 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 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?

Percentage of reduction in annualized bleed rate (ABR) during prophylactic treatment compared to ABR during on demand treatment (time frame: 12 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 NCT02170402.

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 3 May 2021.

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