Hemophilia A With InhibitorsPhase 2ENJOIHNCT01105546
rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors
A Phase 2 study testing recombinant activated factor VII in people with Hemophilia A With Inhibitors, run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 15 sites in 6 countries.
Trial summary
The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors. Read more about this study
- Who is it for?
- Ages Up to 8 years, men only
- Conditions the study is for
- Hemophilia A With Inhibitors
- What is being tested?
- recombinant activated factor VII
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 15 sitesin 6 countries
- Study dates
- Dec 2010to Apr 2014
- Phase
- Phase 2
- 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 Up to 8 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Patients with hemophilia A who have been treated with factor VIII on demand or on prophylaxis and who have developed inhibitors to factor VIII
- ≤ 2 years from the time of first inhibitor detection.
- High-responding inhibitors (historical peak > 5 BU/mL)and known anamnestic response in case of negative inhibitor titre.
- Candidates to start daily ITI with FVIII doses ranging from 50 IU/Kg/day to 200 IU/Kg/day
- Maximal two bleedings in the same joint within the last 6 months before entering the study or maximal six joint bleeds in the same joint within 2 years
- Adequate venous access for daily infusion and capable (caregiver) of reconstituting and injecting the study drug
- Informed consent by parents or legal guardians.
Who cannot join
- ITI already started
- Known or suspected hypersensitivity to the active substance or to any of the excipients of the study drug
- Administration of any investigational product within 30 days prior to randomisation
- Other coagulation disorders than congenital hemophilia A.
- Family history of thrombosis at an early age (< 40 years), known thrombophilia, any previous thrombosis including catheter-related deep vein thrombosis, previous neonatal thrombosis.
- Known pseudo tumours
Show all 9Show fewer
- Known severe liver disease
- Platelet count < 50,000 platelets/µL at screening
- Surgery within one month or planned major and/or orthopaedic surgery.
Trial locations and contacts
15 sites in 6 countries.
15 sites in 6 countries
United States3 sites
Emory University
Atlanta, Georgia, 30322
Children's Mercy Hospital
Kansas City, Missouri, 64108
Children's Hospital Los Angeles
Los Angeles, California, 90027
Germany3 sites
Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center
Berlin, Germany, 10249
Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik
Bremen, 28205
Klinikum der Johann Wolfgang Goethe-Universitat
Frankfurt/M, 60590
Italy3 sites
Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento
Florence, 50134
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Italy, 20122
Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco
Naples, 80144
Spain3 sites
Unitat Hemofilia, Hospital Vall d'Hebron
Barcelona, Spain, 08035
Centro de Hemofilia, Hospital Universitario La Paz
Madrid, Spain, 28046
Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe
Valencia, Spain, 46009
Romania2 sites
National Institute for Transfusional Hematology
Bucharest, Romania
Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy
Timișoara, Romania, 300011
France1 site
Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital
Lyon, 69003
About this study
Official title: An Investigator-initiated Study on rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors - European Initiative to Prevent Joint Damage in Hemophilia A Children With Inhibitors
The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors.
Read the detailed description
This is a multicentre, randomised, controlled study designed to gain evidence of the advantage of the prophylactic, daily treatment with recombinant activated FVII as compared to the conventional on demand therapy in reducing the bleeding frequency and preserving the orthopaedic status in hemophilic children with inhibitors.
- Conditions
- Hemophilia A With Inhibitors
- Being tested
- recombinant activated factor VII
- Keywords
- hemophilia A, inhibitors, prophylaxis, immune tolerance induction
- 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 activated factor VIIDrug
Also called NovoSeven
90 µg/kg/day i.v.
recombinant activated factor VIIDrug
Also called NovoSeven
treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution
| Study group | Role | What they receive |
|---|---|---|
| prophylaxis | Experimental | prophylaxis with recombinant activated FVII 90 µg/kg/day i.v. Receives: Drug: recombinant activated factor VII |
| on demand treatment | Active comparator | treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution Receives: Drug: recombinant activated factor VII |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total number of joint bleeds.
Measured: 18 months
Secondary outcomes (2)
Joint status evaluated by the Hemophilia Joint Health Score
Measured: 18 months
Number of adverse events and serious adverse events.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2010 (estimated)
- Study startDec 2010
- Primary completionFeb 2014 (estimated)
- Study completionApr 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorsStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Elena Santagostino, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
- Investigators
- Elena Santagostino, MD, PhD, Principal investigator, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01105546
- Sponsor's protocol ID
- ENJOIH 01
- Other IDs
- IND 14503
- Acronym
- ENJOIH
- Status last verified
- Apr 2013
- First submitted
- 15 Apr 2010
- Last updated
- 8 Apr 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator-initiated Study on rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors - European Initiative to Prevent Joint Damage in Hemophilia A Children With Inhibitors
- ClinicalTrials.gov ID
- NCT01105546
- Acronym / sponsor ID
- ENJOIH / ENJOIH 01
- Conditions
- Hemophilia A With Inhibitors
- Keywords
- hemophilia A, inhibitors, prophylaxis, immune tolerance induction
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoUniversity, hospital or other
- Study start
- Dec 2010
- Primary completion
- Feb 2014 (estimated)
- Study completion
- Apr 2014 (estimated)
- First posted
- 16 Apr 2010 (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.
The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors.
Detailed description
This is a multicentre, randomised, controlled study designed to gain evidence of the advantage of the prophylactic, daily treatment with recombinant activated FVII as compared to the conventional on demand therapy in reducing the bleeding frequency and preserving the orthopaedic status in hemophilic children with inhibitors.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| prophylaxis | Experimental | Drug: recombinant activated factor VIIprophylaxis with recombinant activated FVII 90 µg/kg/day i.v. |
| on demand treatment | Active comparator | Drug: recombinant activated factor VIItreatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution |
Who may qualify
- Ages
- Up to 8 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Patients with hemophilia A who have been treated with factor VIII on demand or on prophylaxis and who have developed inhibitors to factor VIII
- ≤ 2 years from the time of first inhibitor detection.
- High-responding inhibitors (historical peak > 5 BU/mL)and known anamnestic response in case of negative inhibitor titre.
- Candidates to start daily ITI with FVIII doses ranging from 50 IU/Kg/day to 200 IU/Kg/day
- Maximal two bleedings in the same joint within the last 6 months before entering the study or maximal six joint bleeds in the same joint within 2 years
Key exclusion criteria (9 listed)
- ITI already started
- Known or suspected hypersensitivity to the active substance or to any of the excipients of the study drug
- Administration of any investigational product within 30 days prior to randomisation
- Other coagulation disorders than congenital hemophilia A.
- Family history of thrombosis at an early age (< 40 years), known thrombophilia, any previous thrombosis including catheter-related deep vein thrombosis, previous neonatal thrombosis.
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 activated factor VIIAlso: NovoSeven90 µg/kg/day i.v. | Drug | prophylaxis |
| recombinant activated factor VIIAlso: NovoSeventreatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution | Drug | on demand treatment |
Primary outcome
Total number of joint bleeds.
Time frame: 18 months
2 secondary outcomes
Joint status evaluated by the Hemophilia Joint Health Score
Time frame: 18 months
Number of adverse events and serious adverse events.
Time frame: 18 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 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.
Elena Santagostino, MD, PhD principal investigator
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
15 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing recombinant activated factor VII in people with Hemophilia A With Inhibitors, run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 15 sites in 6 countries.
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 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: status not confirmed, as of the sponsor's last update on 8 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Germany, Italy, Spain, Romania and 1 more country. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
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?
Total number of joint bleeds. (time frame: 18 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 8 Apr 2013.