Hemophilia A With InhibitorNCT03372993
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven
An observational study of Hemophilia A With Inhibitor and Hemophilia B With Inhibitor, run by AryoGen Pharmed Co. at 2 sites in Iran.
Trial summary
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency
- How many people
- 200 participants
- Where
- 2 sitesIran
- Study dates
- 13 Jun 2016to 15 Apr 2021
- Phase
- Observational
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Hemophilia A or B with inhibitors, Factor VII Deficiency, Glanzmann's thrombasthenia, who have received one or more dose of AryoSeven in the daily practice and are negative at immunogenicity testing (antibodies neutralizing FVII).
Who may be able to join
- Patients with Hemophilia A or B with inhibitors, Factor VII Deficiency, Glanzmann's thrombasthenia
- Treated with AryoSeven in the daily practice,
- Signed informed consent (parents for children).
- an immunogenicity test negative, performed at the earliest time after NovoSeven was no longer available in Iran. Patients who do not have a previous negative immunogenicity test should be enrolled if negative at immunogenicity test performed during screening for this study.
Who cannot join
- Patients who received NovoSeven for any indication at any time before inclusion in the study, until an immunogenicity test excluded the development of immunogenicity related to NovoSeven.
- Parallel participation in another experimental drug trial.
- Parallel participation in another marketed drug trial (except for AryoSeven) that may affect the immunogenicity endpoint of the study.
Trial locations and contacts
2 sites in 1 country.
Iran2 sites
Iranian Blood Transfusion Organization (IBTO)
Tehran
MAHTA (Iranian Hemophilia and Thrombophilia Association)
Tehran
About this study
Official title: A Post-Marketing, Observational Study in Iran to Determine the Immunogenicity Status in Patients Who Have Received rFVIIa AryoSeven.
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
Read the detailed description
This is a Non-interventional, observational, prospective evaluation of immunogenicity (neutralizing antibodies toward FVII) of patients with Hemophilia A or B with inhibitors, Factor VII Deficiency, Glanzmann's thrombasthenia, who have received one or more dose of AryoSeven in the daily practice. This study is conducted in Iran where AryoSeven is available on the market since 21-8-2012 and NovoSeven is no longer available since 2013. Patients will be retrieved from the Iranian Registry of Hemophilia patients of MATHA (Iranian Hemophilia and Thrombophilia Association, Tehran, Iran). The Registry Patient Screener will sequentially review patients in the Registry to identify patients who have received treatment with AryoSeven, in reverse chronological order of inclusion in the registry, starting from a date 6 months prior to the date of this study and until when the sample size to enroll (200 patients) is completed.
Patients identified will be called for a visit (Screening visit) for providing informed consent, interview, and blood sampling collection for prospective immunogenicity testing. Retrospective data collection, including previous immunogenicity status and the number of exposure days, will be performed on hospital records, patient diary. Immunogenicity will be tested by a centralized laboratory in Iran using the PT-based Bethesda assay.
Patients will be enrolled if they had an immunogenicity test negative, performed at the earliest time after NovoSeven was no longer available in Iran. Patients who do not have a previous negative immunogenicity test will be enrolled if negative at immunogenicity test performed during screening for this study.
Patients enrolled will be followed for at least 12 months for immunogenicity and Adverse Drug Reactions.
Patients who have a positive previous immunogenicity test or results positive at immunogenicity test performed at Screening (patients without a previous immunogenicity test), will not be enrolled, but registered and followed for 12 months (or longer) to collect data on the natural history of their disease.
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency, Glanzmann's Thrombasthenia
- Medical subject headings
- Factor VII Deficiency, Thrombasthenia
What is being measured
The primary outcome is the main question the study is designed to answer.
Immunogenicity (neutralising antibodies toward FVII)
Immunogenicity will be assessed with the PT based Bethesda assay. In case of positive results, an inhibitor retesting using a second separately drawn sample as confirmatory measurement will be performed by a different specialised laboratory.
Measured: On plasma samples obtained 12 months after patient inclusion.
Secondary outcomes (1)
Adverse Drug Reactions
Adverse Drug Reactions related to the administration of AryoSeven reported by patients.
Measured: Adverse Drug Reactions will be monitored throughout the trial, from patient inclusion up to 12 months follow-up.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2017
- Study start13 Jun 2016
- Primary completionMar 2021
- Study completion15 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- AryoGen Pharmed Co.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mohammad Faranoush, MD, Study director, Hazrat Rasool Akram Hospital, MAHAK Children's Hospital, Iran University of Medical Sciences, Tehran
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03372993
- Sponsor's protocol ID
- ARY2016-01
- Status last verified
- Jul 2020
- First submitted
- 5 Dec 2017
- Last updated
- 15 Feb 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Post-Marketing, Observational Study in Iran to Determine the Immunogenicity Status in Patients Who Have Received rFVIIa AryoSeven.
- ClinicalTrials.gov ID
- NCT03372993
- Acronym / sponsor ID
- ARY2016-01
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency, Glanzmann's Thrombasthenia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- AryoGen Pharmed Co.Industry (pharma, biotech, devices)
- Study start
- 13 Jun 2016 (actual)
- Primary completion
- Mar 2021 (actual)
- Study completion
- 15 Apr 2021 (actual)
- First posted
- 14 Dec 2017 (actual)
- 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.
This study evaluates the immunogenicity of the biosimilar rFVIIa (AryoSeven) in subjects receiving AryoSeven in real-life clinical practice.
Detailed description
This is a Non-interventional, observational, prospective evaluation of immunogenicity (neutralizing antibodies toward FVII) of patients with Hemophilia A or B with inhibitors, Factor VII Deficiency, Glanzmann's thrombasthenia, who have received one or more dose of AryoSeven in the daily practice. This study is conducted in Iran where AryoSeven is available on the market since 21-8-2012 and NovoSeven is no longer available since 2013. Patients will be retrieved from the Iranian Registry of Hemophilia patients of MATHA (Iranian Hemophilia and Thrombophilia Association, Tehran, Iran). The Registry Patient Screener will sequentially review patients in the Registry to identify patients who have received treatment with AryoSeven, in reverse chronological order of inclusion in the registry, starting from a date 6 months prior to the date of this study and until when the sample size to enroll (200 patients) is completed.
Patients identified will be called for a visit (Screening visit) for providing informed consent, interview, and blood sampling collection for prospective immunogenicity testing. Retrospective data collection, including previous immunogenicity status and the number of exposure days, will be performed on hospital records, patient diary. Immunogenicity will be tested by a centralized laboratory in Iran using the PT-based Bethesda assay.
Patients will be enrolled if they had an immunogenicity test negative, performed at the earliest time after NovoSeven was no longer available in Iran. Patients who do not have a previous negative immunogenicity test will be enrolled if negative at immunogenicity test performed during screening for this study.
Patients enrolled will be followed for at least 12 months for immunogenicity and Adverse Drug Reactions.
Patients who have a positive previous immunogenicity test or results positive at immunogenicity test performed at Screening (patients without a previous immunogenicity test), will not be enrolled, but registered and followed for 12 months (or longer) to collect data on the natural history of their disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Hemophilia A or B with inhibitors, Factor VII Deficiency, Glanzmann's thrombasthenia, who have received one or more dose of AryoSeven in the daily practice and are negative at immunogenicity testing (antibodies neutralizing FVII).
Key inclusion criteria (4 listed)
- Patients with Hemophilia A or B with inhibitors, Factor VII Deficiency, Glanzmann's thrombasthenia
- Treated with AryoSeven in the daily practice,
- Signed informed consent (parents for children).
- an immunogenicity test negative, performed at the earliest time after NovoSeven was no longer available in Iran. Patients who do not have a previous negative immunogenicity test should be enrolled if negative at immunogenicity test performed during screening for this study.
Key exclusion criteria (3 listed)
- Patients who received NovoSeven for any indication at any time before inclusion in the study, until an immunogenicity test excluded the development of immunogenicity related to NovoSeven.
- Parallel participation in another experimental drug trial.
- Parallel participation in another marketed drug trial (except for AryoSeven) that may affect the immunogenicity endpoint of the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Immunogenicity (neutralising antibodies toward FVII)
Time frame: On plasma samples obtained 12 months after patient inclusion.
Immunogenicity will be assessed with the PT based Bethesda assay. In case of positive results, an inhibitor retesting using a second separately drawn sample as confirmatory measurement will be performed by a different specialised laboratory.
1 secondary outcome
Adverse Drug Reactions
Time frame: Adverse Drug Reactions will be monitored throughout the trial, from patient inclusion up to 12 months follow-up.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2022.
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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.
Mohammad Faranoush, MD study director
Hazrat Rasool Akram Hospital, MAHAK Children's Hospital, Iran University of Medical Sciences, Tehran
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A With Inhibitor and Hemophilia B With Inhibitor, run by AryoGen Pharmed Co. at 2 sites in Iran.
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 4 inclusion and 3 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 15 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Iran. 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.
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?
Immunogenicity (neutralising antibodies toward FVII) (time frame: On plasma samples obtained 12 months after patient inclusion.). 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 15 Feb 2022.