Hemophilia APhase 4NCT04985682
A Study of ADVATE in People With Hemophilia A in India
A Phase 4 study testing ADVATE in people with Hemophilia A, run by Baxalta now part of Shire at 5 sites in India.
Trial summary
The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ADVATE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 5 sitesIndia
- Study dates
- 14 Jan 2022to 10 Feb 2023
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The participant or legally authorized representative (in case of study participants less than (<) 18 years of age) gave written informed consent to participate in the study.
- Participant of any age with hemophilia A.
- Participant defined as a previously treated patient (PTP):
- Participant aged greater than or equal to (>=) 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 150 exposure doses (EDs).
- Participant aged less than <6 years that has been previously treated with plasma-derived or recombinant FVIII concentrate(s) for a minimum of 50 EDs.
- Participant as negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU) per milliliter (Nijmegen-modified Bethesda assay).
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- Participant is human immunodeficiency virus negative (HIV-); or human immunodeficiency virus positive (HIV+) with stable disease and cluster of differentiation 4 (CD4+) count >=200 cells per cubic millimeter (mm^3), as confirmed by central laboratory at screening.
- Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, anti-body titer will be confirmed by PCR), as confirmed by central laboratory at screening; or hepatitis C virus positive (HCV+) with chronic stable hepatitis.
- Participant is willing and able to comply with the requirements of the protocol.
Who cannot join
- Participant has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII (factor VIII) concentrates.
- Participant has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease (VWD) or thrombocytopenia (platelet count <100,000 per milliliter).
- Participant has received treatment for hemophilia A with non-FVIII products or concentrates (example, emicizumab [Hemlibra®]) in the 6 months prior to screening.
- Participant has severe chronic hepatic dysfunction (example, >=5 times upper limit of normal alanine aminotransferase [ALT], aspartate aminotransferase [AST] or international normalized ratio [INR] >1.5 as confirmed by central laboratory at screening).
- Participant has planned or is likely to have, surgery during the study period.
- Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug or alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance.
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- Participant currently receiving or is scheduled to receive during the course of the study, an immunomodulating drug (example, corticosteroid agents at a dose equivalent to hydrocortisone >10 milligram per day, or α-interferon) other than antiretroviral chemotherapy.
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- Participant is a family member or employee of the investigator.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
India5 sites
St. John's Medical College
Bengaluru, 560034
Amrita Institute of Medical Science & Research Centre
Ernākulam, Kerala, 682041
K J Somaiya Hospital & Research Centre
Mumbai, 400022
All India Institute of Medical Sciences (AIIMS)
New Delhi, 110029
Unique Children's Hospital Pvt. Ltd.
Pune, 411019
About this study
Official title: Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice
The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.
- Conditions
- Hemophilia A
- Being tested
- ADVATE
- Keywords
- Drug Therapy
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADVATEBiological
Antihemophilic factor (AHF) activity expressed in international units (IU) per vial.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A Group | Experimental | Participants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. Receives: Biological: ADVATE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII inhibitor formation) that were at least possibly related to ADVATE were reported.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Secondary outcomes (14)
Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE
An AE was any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Number of participants with non-serious AEs that were at least possibly related to ADVATE were reported.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters
Clinical laboratory parameters included hematology, clinical chemistry, viral serology, factor VIII (FVIII) antigen, FVIII activity, incremental recovery, and FVIII inhibitor. Clinical significance was judged as per Investigator's assessment.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE
ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error was estimated using a generalized linear model (GLM). The total ABR is reported in this outcome measure.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed
ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed sites (example, joint, soft tissue, muscle, other [mouth, gums or nose] are reported in this outcome measure.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed
ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed cause (example, spontaneous, injury, and unknown) are reported in this outcome measure.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Number of ADVATE Infusions Required During Prophylactic Treatment
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment
Body mass adjusted consumption international units per kilograms (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment
Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment
Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes
Overall hemostatic efficacy for treatment of bleeding episodes was rated on 4-point Likert scale as: excellent=full relief of pain and cessation of objective signs of bleeding after a single infusion, no additional infusion is required for the control of bleeding and administration of further infusion to maintain hemostasis would not affect the scoring; good=definite pain relief and/or improvement in signs of bleeding after a single infusion, possibly requires more than 2 infusions for complete resolution and administration of further infusion to maintain hemostasis would not affect the scoring; moderate=probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion, required multiple infusions for complete resolution; none=no improvement of signs or symptoms or conditions worsen.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode
Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.
Measured: Baseline (Day 0) up to end of study (up to 12.9 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2021
- Study start14 Jan 2022
- Primary completion10 Feb 2023
- Study completion10 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04985682
- Sponsor's protocol ID
- TAK-761-4009
- Other IDs
- 2022-004149-11
- Status last verified
- Aug 2023
- First submitted
- 29 Jul 2021
- Last updated
- 7 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 4, Multicenter, Prospective, Interventional, Post-Marketing Study in Hemophilia A Patients in India Receiving ADVATE as On-Demand or Prophylaxis Under Standard Clinical Practice
- ClinicalTrials.gov ID
- NCT04985682
- Acronym / sponsor ID
- TAK-761-4009
- Conditions
- Hemophilia A
- Keywords
- Drug Therapy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Study start
- 14 Jan 2022 (actual)
- Primary completion
- 10 Feb 2023 (actual)
- Study completion
- 10 Feb 2023 (actual)
- First posted
- 2 Aug 2021 (actual)
- Results
- Posted 7 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A Group | Experimental | Biological: ADVATEParticipants with hemophilia A were treated with ADVATE according to a regimen determined by the treating physician at the study site and in accordance with the national product label under standard clinical practice for 6.7 months. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- The participant or legally authorized representative (in case of study participants less than (<) 18 years of age) gave written informed consent to participate in the study.
- Participant of any age with hemophilia A.
- Participant defined as a previously treated patient (PTP):
- Participant as negative history of FVIII inhibitors and negative inhibitor at screening defined as less than 0.6 Bethesda units (BU) per milliliter (Nijmegen-modified Bethesda assay).
- Participant is human immunodeficiency virus negative (HIV-); or human immunodeficiency virus positive (HIV+) with stable disease and cluster of differentiation 4 (CD4+) count >=200 cells per cubic millimeter (mm^3), as confirmed by central laboratory at screening.
Key exclusion criteria (9 listed)
- Participant has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII (factor VIII) concentrates.
- Participant has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease (VWD) or thrombocytopenia (platelet count <100,000 per milliliter).
- Participant has received treatment for hemophilia A with non-FVIII products or concentrates (example, emicizumab [Hemlibra®]) in the 6 months prior to screening.
- Participant has severe chronic hepatic dysfunction (example, >=5 times upper limit of normal alanine aminotransferase [ALT], aspartate aminotransferase [AST] or international normalized ratio [INR] >1.5 as confirmed by central laboratory at screening).
- Participant has planned or is likely to have, surgery during the study period.
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 |
|---|---|---|
| ADVATEAntihemophilic factor (AHF) activity expressed in international units (IU) per vial. | Biological | Hemophilia A Group |
Primary outcome
Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII inhibitor formation) that were at least possibly related to ADVATE were reported.
14 secondary outcomes
Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Number of ADVATE Infusions Required During Prophylactic Treatment
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode
Time frame: Baseline (Day 0) up to end of study (up to 12.9 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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.
Medical Director study director
Takeda
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ADVATE in people with Hemophilia A, run by Baxalta now part of Shire at 5 sites in India.
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 9 inclusion and 9 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: finished, as of the sponsor's last update on 7 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in India. 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?
Yes. Results are posted in the registry (first posted 7 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE (time frame: Baseline (Day 0) up to end of study (up to 12.9 months)). 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 7 Mar 2024.