Hemophilia AOPTIPHASENCT00995046
Individually Tailored Prophylaxis in Patients With Severe Hemophilia A
A study testing FVIII in people with Hemophilia A, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Patients with severe haemophilia A lack clotting factor FVIII and suffer from spontaneous and traumatic bleeds. In the absence of treatment, frequent bleeds in joints lead to severe joint destruction. Read more about this study
- Who is it for?
- Ages 6 to 45 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- FVIII
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteFrance
- Study dates
- Sep 2009to Sep 2012
- Phase
- Interventional
- 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 6 to 45 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Severe haemophilia A (FVIII < 1 IU/dl)
- Currently on prophylactic therapy administered at least 3 times per week with a clinical efficiency
- Age: 6 - 45 years
- Adequate venous access in adults and children i.e. presence of 2 or more good quality peripheral veins, in order to avoid the need for a central venous device. One peripheral vein for FVIII infusions and one other for blood sampling are required.
- Competent in home treatment and infusion therapy (patient or parents)
- Ability of patient or family (for minors) to give informed consent
- Patient affiliated to French Social Insurance System.
Who cannot join
- Age < 6 years and > 45 years
- Hemophilia A with documented history of inhibitor
- Clinically symptomatic liver disease (supported by e.g. diagnosis of cirrhosis, portal hypertension, ascites, PT > 5 seconds above upper limit of normal)
- Platelet count < 100x109/l
- Planned elective surgery within 13 months
- Poor venous access according inclusion criteria
- Presence of a documented target joint
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Optimizing Prophylaxis in Patients With Severe Haemophilia A by Tailoring the Infusions to Individual Patients' Needs Using the Calibrated Automated Thrombin Generation Test
Patients with severe haemophilia A lack clotting factor FVIII and suffer from spontaneous and traumatic bleeds. In the absence of treatment, frequent bleeds in joints lead to severe joint destruction. In 1960s, prophylactic therapy was developed involving the infusion of clotting factor on a regular schedule in order to keep clotting levels sufficiently high to prevent spontaneous bleeding episodes. Prophylaxis is started at an early age before the age of 2 years or after the first joint bleed. The Malmö experience indicates that treatment is most effective when administered in large doses at least 3 times weekly. However, such an intensive treatment in young boys may be very difficult to carry out for home treatment. Currently, there is no international recommendation on prophylactic therapy regimens. Because of the high cost and limited availability of factor concentrates, dosing is an important issue in prophylaxis therapy. It was recently shown that 24 hours after FVIII concentrate administration, in patients presenting similar FVIIII levels, thrombin generation capacity may be significantly different. In addition, independently of the FVIII level, a correlation was found between severe clinical bleeding phenotype and thrombin generating capacity. The aim of the present clinical study is to assess the thrombin generation test as the main surrogate marker to evaluate the coagulating capacity of haemophiliacs on prophylaxis regimen. Optimizing prophylactic therapy to patient's phenotype with no loss of clinical effectiveness can significantly improve patients' quality of life, protect haemophilic children against arthropathy and possibly limit the cost of the prophylaxis therapy.
- Conditions
- Hemophilia A
- Being tested
- FVIII
- Keywords
- Hemophilia A, prophylaxis, thrombin generation test (TGT)
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FVIIIDrug
6 months of prophylaxis treatment administered 3 or 4 times weekly according to patient's initial regimen, (standardized Malmö protocol 25 - 40 IU/kg/infusion). Medical visits will occur at 3-month intervals (+ 5 days) until the end of the study. Weekly, telephone calls to the patients (parents) will also be done.
FVIIIDrug
1 month period where thrombin generating capacity will be evaluated, followed by 6 months of "individually" tailored prophylaxis regimen according to TGT results. Medical visits will occur at 3-month intervals (+ 5 days) until the end of the study. Weekly, telephone calls to the patients (parents) will also be done.
| Study group | Role | What they receive |
|---|---|---|
| usual prophylaxis regimen | Active comparator | All patients will receive their usual prophylaxis regimen during the first 6 months Receives: Drug: FVIII |
| individually tailored prophylaxis regimen | Experimental | All patients will receive an individually tailored prophylaxis regimen in accordance with TGT results during the second 6 month-period. Receives: Drug: FVIII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Consumption of clotting factor concentrate
Measured: 13 months
Secondary outcomes (2)
Number of spontaneous bleeds
Measured: 13 months
Number of spontaneous joint bleeds
Measured: 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2009 (estimated)
- Study startSep 2009
- Primary completionSep 2012
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 Hospices Civils de LyonThe 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 N/A
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yesim Dargaud, MD, PhD, Principal investigator, Hospices Civils de Lyon, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00995046
- Sponsor's protocol ID
- 2007.482
- Acronym
- OPTIPHASE
- Status last verified
- Oct 2009
- First submitted
- 12 Oct 2009
- Last updated
- 14 May 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimizing Prophylaxis in Patients With Severe Haemophilia A by Tailoring the Infusions to Individual Patients' Needs Using the Calibrated Automated Thrombin Generation Test
- ClinicalTrials.gov ID
- NCT00995046
- Acronym / sponsor ID
- OPTIPHASE / 2007.482
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, prophylaxis, thrombin generation test (TGT)
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- Sep 2009
- Primary completion
- Sep 2012 (actual)
- First posted
- 14 Oct 2009 (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.
Patients with severe haemophilia A lack clotting factor FVIII and suffer from spontaneous and traumatic bleeds. In the absence of treatment, frequent bleeds in joints lead to severe joint destruction. In 1960s, prophylactic therapy was developed involving the infusion of clotting factor on a regular schedule in order to keep clotting levels sufficiently high to prevent spontaneous bleeding episodes. Prophylaxis is started at an early age before the age of 2 years or after the first joint bleed. The Malmö experience indicates that treatment is most effective when administered in large doses at least 3 times weekly. However, such an intensive treatment in young boys may be very difficult to carry out for home treatment. Currently, there is no international recommendation on prophylactic therapy regimens. Because of the high cost and limited availability of factor concentrates, dosing is an important issue in prophylaxis therapy. It was recently shown that 24 hours after FVIII concentrate administration, in patients presenting similar FVIIII levels, thrombin generation capacity may be significantly different. In addition, independently of the FVIII level, a correlation was found between severe clinical bleeding phenotype and thrombin generating capacity. The aim of the present clinical study is to assess the thrombin generation test as the main surrogate marker to evaluate the coagulating capacity of haemophiliacs on prophylaxis regimen. Optimizing prophylactic therapy to patient's phenotype with no loss of clinical effectiveness can significantly improve patients' quality of life, protect haemophilic children against arthropathy and possibly limit the cost of the prophylaxis therapy.
Study design
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| usual prophylaxis regimen | Active comparator | Drug: FVIIIAll patients will receive their usual prophylaxis regimen during the first 6 months |
| individually tailored prophylaxis regimen | Experimental | Drug: FVIIIAll patients will receive an individually tailored prophylaxis regimen in accordance with TGT results during the second 6 month-period. |
Who may qualify
- Ages
- 6 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Severe haemophilia A (FVIII < 1 IU/dl)
- Currently on prophylactic therapy administered at least 3 times per week with a clinical efficiency
- Age: 6 - 45 years
- Adequate venous access in adults and children i.e. presence of 2 or more good quality peripheral veins, in order to avoid the need for a central venous device. One peripheral vein for FVIII infusions and one other for blood sampling are required.
- Competent in home treatment and infusion therapy (patient or parents)
Key exclusion criteria (7 listed)
- Age < 6 years and > 45 years
- Hemophilia A with documented history of inhibitor
- Clinically symptomatic liver disease (supported by e.g. diagnosis of cirrhosis, portal hypertension, ascites, PT > 5 seconds above upper limit of normal)
- Platelet count < 100x109/l
- Planned elective surgery within 13 months
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 |
|---|---|---|
| FVIII6 months of prophylaxis treatment administered 3 or 4 times weekly according to patient's initial regimen, (standardized Malmö protocol 25 - 40 IU/kg/infusion). Medical visits will occur at 3-month intervals (+ 5 days) until the end of the study. Weekly, telephone calls to the patients (parents) will also be done. | Drug | usual prophylaxis regimen |
| FVIII1 month period where thrombin generating capacity will be evaluated, followed by 6 months of "individually" tailored prophylaxis regimen according to TGT results. Medical visits will occur at 3-month intervals (+ 5 days) until the end of the study. Weekly, telephone calls to the patients (parents) will also be done. | Drug | individually tailored prophylaxis regimen |
Primary outcome
Consumption of clotting factor concentrate
Time frame: 13 months
2 secondary outcomes
Number of spontaneous bleeds
Time frame: 13 months
Number of spontaneous joint bleeds
Time frame: 13 months
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 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.
Yesim Dargaud, MD, PhD principal investigator
Hospices Civils de Lyon, France
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing FVIII in people with Hemophilia A, run by Hospices Civils de Lyon at 1 site in France.
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 7 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: stopped early, as of the sponsor's last update on 14 May 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 France. 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 was stopped early. Results are not available in the source record.
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?
Consumption of clotting factor concentrate (time frame: 13 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 14 May 2013.