Hemophilia AGEPHARDNCT02912143
German Pediatric Hemophilia Research Database
An observational study of Hemophilia A and Hemophilia B, run by Goethe University at 1 site in Germany.
Trial summary
The German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed children with hemophilia. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Hemophilia A, Hemophilia B, Children
- What is being tested?
- documentation only
- How many people
- 800 participants
- Where
- 1 siteGermany
- Study dates
- 1 Jan 2017to 31 Dec 2028
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: All children between 0 and 18 yrs diagnosed with hemophilia A or B (FVIII/FIX < 1% to 25%) from 01.01.2017.
Who may be able to join
- hemophilia A or B
- FVIII/FIX <1% to 25%
- informed consent
Who cannot join
- no consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Germany1 site, 1 recruiting
- Recruiting
Goethe University
Frankfurt am Main, Hesse, 60590
Christoph Königs, PhD, +496963018, christoph.koenigs@kgu.de
About this study
The German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed children with hemophilia.
- Conditions
- Hemophilia A, Hemophilia B, Children, Drug Specific Antibodies
- Being tested
- documentation only
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
documentation onlyOther
| Study group | Role | What they receive |
|---|---|---|
| newly diagnosed children with hemophilia | no intervention Receives: Other: documentation only |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with antibody developement to exogenous clotting factors (>0.5 BU)
Blood Test: measurement in Bethesda Units, positive according to local standards, for most labs > 0.5 BU
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2016 (estimated)
- Study start1 Jan 2017
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
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.
- 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
- 800 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Goethe University
- Sponsor type
- University, hospital or other
- Collaborators
- Society for Thrombosis and Haemostasis Research (Germany)
- Responsible party
- Principal investigator, Christoph Königs, Goethe University
- Investigators
- Christoph Königs, PhD, Principal investigator, Goethe University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02912143
- Sponsor's protocol ID
- GEPHARD
- Other IDs
- DRKS00011101
- Acronym
- GEPHARD
- Status last verified
- Mar 2023
- First submitted
- 17 Sep 2016
- Last updated
- 8 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual Patient data will not be shared outside specified collaborating studies (none yet)
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- German Pediatric Hemophilia Research Database
- ClinicalTrials.gov ID
- NCT02912143
- Acronym / sponsor ID
- GEPHARD / GEPHARD
- Conditions
- Hemophilia A, Hemophilia B, Children, Drug Specific Antibodies
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 800 participants (estimated)
- Lead sponsor
- Goethe UniversityUniversity, hospital or other
- Collaborators
- Society for Thrombosis and Haemostasis Research (Germany)
- Study start
- 1 Jan 2017
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 23 Sep 2016 (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 German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed children with hemophilia.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| newly diagnosed children with hemophilia | – | Other: documentation onlyno intervention |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- All children between 0 and 18 yrs diagnosed with hemophilia A or B (FVIII/FIX < 1% to 25%) from 01.01.2017.
Key inclusion criteria (3 listed)
- hemophilia A or B
- FVIII/FIX <1% to 25%
- informed consent
Key exclusion criteria (1 listed)
- no consent
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 |
|---|---|---|
| documentation only | Other | newly diagnosed children with hemophilia |
Primary outcome
Number of patients with antibody developement to exogenous clotting factors (>0.5 BU)
Time frame: 5 years
Blood Test: measurement in Bethesda Units, positive according to local standards, for most labs > 0.5 BU
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2023.
- Individual participant dataSharing planned: No. Individual Patient data will not be shared outside specified collaborating studies (none yet)
Related trials for Hemophilia A
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A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
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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.
Christoph Königs, PhD principal investigator
Goethe University
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 Hemophilia A and Hemophilia B, run by Goethe University at 1 site in Germany.
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 3 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 8 Mar 2023. 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 Germany. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Christoph Königs, PhD (+496963018 ext. 3030, christoph.koenigs@kgu.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with antibody developement to exogenous clotting factors (>0.5 BU) (time frame: 5 years). 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 Mar 2023.