Severe Hemophilia APhase 4CHPSNCT01085344
Canadian Hemophilia Prophylaxis Study
A Phase 4 study testing Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) in people with Severe Hemophilia A, run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy. Read more about this study
- Who is it for?
- Ages 12 to 30 months, men only
- Conditions the study is for
- Severe Hemophilia A
- What is being tested?
- Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteCanada
- Study dates
- 26 Jun 1997to Dec 2014
- 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 12 to 30 months
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Severe hemophilia A (factor level less than 2%).
- Age greater than 1 year and less than or equal to 2.5 years.
- Normal joints using the World Federation of Hemophilia orthopedic scale.
- Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
- Platelet count of > 150,000.
- Informed consent to participate.
Who cannot join
- Three or more clinically determined bleeds into any single elbow, knee or ankle.
- Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
- Family judged to be non-compliant by the local hemophilia clinic director.
- Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.
Read the detailed description
There are 2 specific study objectives. The first is to estimate the incidence of target joint bleeding in patients with severe hemophilia A treated (for primary prophylaxis) with Escalating Dose Prophylactic factor replacement. The second objective is to obtain accurate estimates of the direct and indirect costs associated with this protocol for use in a cost-effectiveness model (comparing Escalating Dose with standard prophylaxis and with intermittent therapy).
- Conditions
- Severe Hemophilia A
- Being tested
- Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)
- Keywords
- hemophilia, prophylaxis, cost effectiveness
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)Biological
Also called Recombinant Factor VIII (antihemophilic agent)
escalating dose prophylaxis
Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)Biological
Also called recombinant factor VIII (antihemophilic agent)
escalating dose
| Study group | Role | What they receive |
|---|---|---|
| Factor VIII | Experimental | escalating dose Factor VIII Receives: Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Developed Target Joint Bleeding
The number of participants who developed target joint bleeding during the study, which was defined as 3 bleeds into any 1 joint within a period of 3 months.
Measured: 6 months
Secondary outcomes (6)
Annualized Bleeding Rate
Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year
Measured: 6 months
Annualized Factor Use
annual factor usage per subject
Measured: 12 months
Number of Patients Who Developed an Inhibitor to FVIII
The number of patients who developed an inhibitor for FVIII, defined as >= 0.5 Bethesda Units
Measured: 6 months
Physical Disability as Measured by the CHAQ
complete the Child Health Assessment Questionnaire (CHAQ) at each 6 month visit. The CHAQ is a validated tool to measure a disability index, with a possible score range of 0-3, where 0 represents no disability and 3 represents maximal disability. The CHAQ is known to have a strong ceiling effect.
The CHAQ was collected at each study visit (i.e. every 6 months for the duration each patient was on study). The reported score represents the median end of study score.
Measured: through study completion, a median of 10 years
Joint Damage as Determined by the Physiotherapy Score
Complete the modified Colarado Physiotherapy Assessment every 6 months at each visit with a score range 0-30 for ankles and knees and 0-26 for elbows), measured at all study visits, which we modified by not assessing crepitus or the ankle joint circumference measurement. For each scale, 0 represents no joint damage, with 26/30 representing maximum possible joint damage. The reported score represents the median end of study score
Measured: through study completion, a median of 10 years
Complications Arising From Indwelling Venous Catheter
collect information on any complications relating to indwelling venous catheters that some subject use.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2010 (estimated)
- Study start26 Jun 1997
- Primary completionDec 2012
- Study completionDec 2014
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Severe 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
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Brian Feldman, The Hospital for Sick Children
- Investigators
- Brian M Feldman, MD, Principal investigator, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01085344
- Sponsor's protocol ID
- 0019970022
- Acronym
- CHPS
- Status last verified
- Nov 2019
- First submitted
- 1 Mar 2010
- Last updated
- 5 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Feldman BM, Rivard GE, Babyn P, Wu JKM, Steele M, Poon MC, Card RT, Israels SJ, Laferriere N, Gill K, Chan AK, Carcao M, Klaassen RJ, Cloutier S, Price VE, Dover S, Blanchette VS. Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet Haematol. 2018 Jun;5(6):e252-e260. doi: 10.1016/S2352-3026(18)30048-6. Epub 2018 May 3.
PubMed 29731369
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
- ClinicalTrials.gov ID
- NCT01085344
- Acronym / sponsor ID
- CHPS / 0019970022
- Conditions
- Severe Hemophilia A
- Keywords
- hemophilia, prophylaxis, cost effectiveness
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- 26 Jun 1997 (actual)
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 11 Mar 2010 (estimated)
- Results
- Posted 5 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.
Detailed description
There are 2 specific study objectives. The first is to estimate the incidence of target joint bleeding in patients with severe hemophilia A treated (for primary prophylaxis) with Escalating Dose Prophylactic factor replacement. The second objective is to obtain accurate estimates of the direct and indirect costs associated with this protocol for use in a cost-effectiveness model (comparing Escalating Dose with standard prophylaxis and with intermittent therapy).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Factor VIII | Experimental | Biological: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)escalating dose Factor VIII |
Who may qualify
- Ages
- 12 to 30 months
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Severe hemophilia A (factor level less than 2%).
- Age greater than 1 year and less than or equal to 2.5 years.
- Normal joints using the World Federation of Hemophilia orthopedic scale.
- Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
- Platelet count of > 150,000.
Key exclusion criteria (4 listed)
- Three or more clinically determined bleeds into any single elbow, knee or ankle.
- Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
- Family judged to be non-compliant by the local hemophilia clinic director.
- Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)
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 |
|---|---|---|
| Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)Also: Recombinant Factor VIII (antihemophilic agent)escalating dose prophylaxis | Biological | Factor VIII |
| Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)Also: recombinant factor VIII (antihemophilic agent)escalating dose | Biological | Factor VIII |
Primary outcome
Number of Participants Who Developed Target Joint Bleeding
Time frame: 6 months
The number of participants who developed target joint bleeding during the study, which was defined as 3 bleeds into any 1 joint within a period of 3 months.
6 secondary outcomes
Annualized Bleeding Rate
Time frame: 6 months
Annualized Factor Use
Time frame: 12 months
Number of Patients Who Developed an Inhibitor to FVIII
Time frame: 6 months
Physical Disability as Measured by the CHAQ
Time frame: through study completion, a median of 10 years
Joint Damage as Determined by the Physiotherapy Score
Time frame: through study completion, a median of 10 years
Complications Arising From Indwelling Venous Catheter
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 29731369Feldman BM, Rivard GE, Babyn P, Wu JKM, Steele M, Poon MC, Card RT, Israels SJ, Laferriere N, Gill K, Chan AK, Carcao M, Klaassen RJ, Cloutier S, Price VE, Dover S, Blanchette VS. Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet Haematol. 2018 Jun;5(6):e252-e260. doi: 10.1016/S2352-3026(18)30048-6. Epub 2018 May 3.
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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.
Brian M Feldman, MD principal investigator
The Hospital for Sick Children
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) in people with Severe Hemophilia A, run by The Hospital for Sick Children at 1 site in Canada.
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 6 inclusion and 4 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 5 Dec 2019. 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 Canada. 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 5 Dec 2019). 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 Who Developed Target Joint Bleeding (time frame: 6 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 NCT01085344, which also lists 1 linked publication.
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 5 Dec 2019.