Severe Haemophilia A With InhibitorsNCT02018393
Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients With Haemophilia A and Inhibitors
An observational study of Severe Haemophilia A With Inhibitors, run by Hospital Universitario La Paz at 1 site in Spain.
Trial summary
This study aims to evaluate the immunomodulatory effect of FEIBA® in patients with severe haemophilia A and inhibitors. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Severe Haemophilia A With Inhibitors
- Type of treatment
- Injection or infusion
- How many people
- 20 participants
- Where
- 1 siteSpain
- Study dates
- Jan 2014to Dec 2014
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Patients with severe haemophilia A and inhibitors treated with FEIBA® (on demand or prophylaxis) for the control of bleedings.
Who may be able to join
- The subject has signed and dated the Informed Consent form for participation in this study.
- Age ≥ 18 years old.
- Patient with severe haemophilia A (FVIII <1%) and high-responding inhibitor (titre >5 UB) at some point in their life, currently on therapy with FEIBA® to control bleeding.
- Group 1: Patients in prophylaxis with FEIBA®: The patient has been on prophylaxis regimen with FEIBA® for at least 6 months prior to the study visit. Group 2: Patients on-demand regimen with FEIBA®: The patient has been under on-demand treatment with FEIBA® for at 6 months prior to the study visit.
Who cannot join
- The presence of any inflammatory condition at the time of the study visit or the previous 30 days that, according to the medical criterion, would affect the study objectives.
- The patient is under immune tolerance treatment or has been at any time during the 30 days prior to the study visit.
- The administration of any anti-inflammatory or immunosuppressive drug 15 days before the study visit.
- Levels of CD4 <200/l regardless of the HIV status.
- Altered hepatic or renal function defined by the presence of abnormal levels of alanine aminotransferase (ALAT) or serum creatinine, respectively.
- Administration of any haemostatic treatment to control bleeding within the 5 days prior to the study visit.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients
This study aims to evaluate the immunomodulatory effect of FEIBA® in patients with severe haemophilia A and inhibitors.
Read the detailed description
This is an observational, multicentric, case-control and one-single-visit study of patients with severe haemophilia A and inhibitors against FVIII in therapy with FEIBA®.
Two subject groups will be included: Group 1 (cases) will be composed of patients with severe haemophilia A and inhibitors in prophylaxis with FEIBA®; Group 2 (controls) will be composed of patients with severe haemophilia A and inhibitors on-demand treatment with FEIBA®.
- Conditions
- Severe Haemophilia A With Inhibitors
- Keywords
- FEIBA®
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Prophylaxis group | Prophylaxis with FEIBA is defined in this study as the regular infusion of FEIBA for the prevention of bleeding at a dose of ≥50 UF/kg on at least three non-consecutive days a week. Patients must have been on this modality for at least 6 months prior to the study visit | |
| On demand group | On-demand Treatment is defined as the administration of FEIBA only to control bleeding. Patients must have been on this modality for at least 6 months prior to the study visit. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of plasma levels of anti-FVIII antibodies (neutralising and non-neutralising)
Measured: At inclusion
Secondary outcomes (1)
Measurement of the serum levels of inflammatory cytokines
Measured: At inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2013 (estimated)
- Study startJan 2014
- Primary completionJun 2014 (estimated)
- Study completionDec 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Haemophilia A With InhibitorsStudies 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
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospital Universitario La Paz
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Victor Jimenez-Yuste, Hospital Universitario La Paz
- Investigators
- Victor Jimenez-Yuste, Medical doctor, PhD, Principal investigator, Hospital Universitario La Paz
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02018393
- Sponsor's protocol ID
- PI-1405
- Status last verified
- Dec 2013
- First submitted
- 17 Dec 2013
- Last updated
- 23 Dec 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients
- ClinicalTrials.gov ID
- NCT02018393
- Acronym / sponsor ID
- PI-1405
- Conditions
- Severe Haemophilia A With Inhibitors
- Keywords
- FEIBA®
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Hospital Universitario La PazUniversity, hospital or other
- Study start
- Jan 2014
- Primary completion
- Jun 2014 (estimated)
- Study completion
- Dec 2014 (estimated)
- First posted
- 23 Dec 2013 (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.
This study aims to evaluate the immunomodulatory effect of FEIBA® in patients with severe haemophilia A and inhibitors.
Detailed description
This is an observational, multicentric, case-control and one-single-visit study of patients with severe haemophilia A and inhibitors against FVIII in therapy with FEIBA®.
Two subject groups will be included: Group 1 (cases) will be composed of patients with severe haemophilia A and inhibitors in prophylaxis with FEIBA®; Group 2 (controls) will be composed of patients with severe haemophilia A and inhibitors on-demand treatment with FEIBA®.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prophylaxis group | – | –Prophylaxis with FEIBA is defined in this study as the regular infusion of FEIBA for the prevention of bleeding at a dose of ≥50 UF/kg on at least three non-consecutive days a week. Patients must have been on this modality for at least 6 months prior to the study visit |
| On demand group | – | –On-demand Treatment is defined as the administration of FEIBA only to control bleeding. Patients must have been on this modality for at least 6 months prior to the study visit. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with severe haemophilia A and inhibitors treated with FEIBA® (on demand or prophylaxis) for the control of bleedings.
Key inclusion criteria (4 listed)
- The subject has signed and dated the Informed Consent form for participation in this study.
- Age ≥ 18 years old.
- Patient with severe haemophilia A (FVIII <1%) and high-responding inhibitor (titre >5 UB) at some point in their life, currently on therapy with FEIBA® to control bleeding.
- Group 1: Patients in prophylaxis with FEIBA®: The patient has been on prophylaxis regimen with FEIBA® for at least 6 months prior to the study visit. Group 2: Patients on-demand regimen with FEIBA®: The patient has been under on-demand treatment with FEIBA® for at 6 months prior to the study visit.
Key exclusion criteria (6 listed)
- The presence of any inflammatory condition at the time of the study visit or the previous 30 days that, according to the medical criterion, would affect the study objectives.
- The patient is under immune tolerance treatment or has been at any time during the 30 days prior to the study visit.
- The administration of any anti-inflammatory or immunosuppressive drug 15 days before the study visit.
- Levels of CD4 <200/l regardless of the HIV status.
- Altered hepatic or renal function defined by the presence of abnormal levels of alanine aminotransferase (ALAT) or serum creatinine, respectively.
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
Measurement of plasma levels of anti-FVIII antibodies (neutralising and non-neutralising)
Time frame: At inclusion
1 secondary outcome
Measurement of the serum levels of inflammatory cytokines
Time frame: At inclusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2013.
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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.
Victor Jimenez-Yuste, Medical doctor, PhD principal investigator
Hospital Universitario La Paz
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Severe Haemophilia A With Inhibitors, run by Hospital Universitario La Paz at 1 site in Spain.
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 6 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 23 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ihosvany Fernandez-Bello, Pharmacist (+34669089737, ihosvanyf@yahoo.es), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurement of plasma levels of anti-FVIII antibodies (neutralising and non-neutralising) (time frame: At 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 23 Dec 2013.