Hemophilia BPhase 1/2NCT00004801
Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency
A Phase 1/2 study testing monoclonal factor IX replacement therapy in people with Hemophilia B and Factor IX Deficiency, run by National Center for Research Resources (NCRR).
Trial summary
OBJECTIVES: Assess the safety and long-term efficacy of monoclonal factor IX concentrate in patients with factor IX deficiency. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia B, Factor IX Deficiency
- What is being tested?
- monoclonal factor IX replacement therapy
- How many people
- 3 participants
- Where
- No sites listed yet
- Study dates
- Mar 1992to not given
- Phase
- Phase 1/2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
PROTOCOL ENTRY CRITERIA:
Patients with factor IX deficiency.
About this study
OBJECTIVES:
Assess the safety and long-term efficacy of monoclonal factor IX concentrate in patients with factor IX deficiency.
Read the detailed description
PROTOCOL OUTLINE:
Patients receive monoclonal factor IX replacement therapy as inpatients. Patients with a satisfactory response receive further therapy as needed for bleeding for up to 36 months at home.
- Conditions
- Hemophilia B, Factor IX Deficiency
- Being tested
- monoclonal factor IX replacement therapy
- Keywords
- hematologic disorders, rare disease
- Medical subject headings
- Hemophilia B, Hematologic Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
monoclonal factor IX replacement therapyDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startMar 1992
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies 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 1/2
- Primary purpose
- Treatment
- Enrolment
- 3 participants
Sponsor and investigators
- Lead sponsor
- National Center for Research Resources (NCRR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- University of North Carolina
- Investigators
- Gilbert C. White, Study chair, University of North Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004801
- Sponsor's protocol ID
- 199/11956
- Other IDs
- UNCCH-621
- Status last verified
- Jan 1998
- First submitted
- 24 Feb 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency
- ClinicalTrials.gov ID
- NCT00004801
- Acronym / sponsor ID
- 199/11956
- Conditions
- Hemophilia B, Factor IX Deficiency
- Keywords
- hematologic disorders, rare disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants
- Lead sponsor
- National Center for Research Resources (NCRR)US National Institutes of Health
- Collaborators
- University of North Carolina
- Study start
- Mar 1992
- First posted
- 25 Feb 2000 (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.
OBJECTIVES:
Assess the safety and long-term efficacy of monoclonal factor IX concentrate in patients with factor IX deficiency.
Detailed description
PROTOCOL OUTLINE:
Patients receive monoclonal factor IX replacement therapy as inpatients. Patients with a satisfactory response receive further therapy as needed for bleeding for up to 36 months at home.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| monoclonal factor IX replacement therapy | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
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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.
Gilbert C. White study chair
University of North Carolina
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing monoclonal factor IX replacement therapy in people with Hemophilia B and Factor IX Deficiency, run by National Center for Research Resources (NCRR).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 24 Jun 2005. Status can change between updates, so confirm with the study contact before making plans.
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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
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 24 Jun 2005.