Blood Coagulation DisordersPhase 3NCT00000582
Cooperative Study of Factor VIII Inhibitors
A Phase 3 study testing factor ix in people with Blood Coagulation Disorders and Hematologic Diseases, run by National Heart, Lung, and Blood Institute (NHLBI).
Trial summary
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of hemophiliac patients who had inhibitors to Factor VIII. Read more about this study
- Who is it for?
- Ages 18 to 75 years, men only
- Conditions the study is for
- Blood Coagulation Disorders, Hematologic Diseases, Hemophilia A
- What is being tested?
- factor ix
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Jul 1978to Jul 1979
- Phase
- Phase 3
- 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 18 to 75 years
- Men only
- No healthy volunteers
- Adult, Older adult
Males with hemophilia, not stratified as to ethnic group.
About this study
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of hemophiliac patients who had inhibitors to Factor VIII.
Read the detailed description
BACKGROUND:
Despite major advances in the treatment of patients with hemophilia, a serious remaining challenge was presented by the occurrence of circulating inhibitors to Factor VIII. Because of lack of information on the natural course of patients with Factor VIII inhibitors, the relative efficacy of various modes of therapy was not established. The Division of Blood Diseases and Resources decided to sponsor a clinical investigation which would evaluate populations of hemophilia patients for Factor VIII inhibitors, follow up these patients to provide information on the natural history of the inhibitor in the hemophilia patients, and make available a reference center to monitor results and attain uniformity.
Treatment of a patient with a severe inhibitor and consequent bleeding remained a problem. Management included protracted treatment with Factor VIII, use of immunosuppressive agents and prothrombin complex (or Factor IX) concentrates. The rationale for Factor IX was that it bypassed the defect in Factor VIII caused by the inhibitor. This method of therapy attracted wide popularity, but the success was greatly debated. It was intended at the very outset of the Factor VIII study that therapeutic trials involving patients with inhibitors would not be a prime function, but that such studies would be monitored if necessary. A controlled trial of Factor IX concentrates therapy was strongly advised by the DBDR Advisory Committee. Accordingly, during fiscal year 1978, a protocol for a double-blind control study was developed by the Factor VIII inhibitor group. The trial began in the spring of 1978, and the intervention terminated about one year later.
DESIGN NARRATIVE:
Double-blind study; patients served as their own controls. A total of 51 patients each received a single large dose of Konyne, Proplex, or diluted albumin (as a control). Joint bleeding of the elbow, knee, and ankle was evaluated six hours after each dose.
- Being tested
- factor ix
- Medical subject headings
- Blood Coagulation Disorders, Hematologic Diseases, Hemophilia A, Hemorrhagic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
factor ixDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 1999 (estimated)
- Study startJul 1978
- Study completionJul 1979
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Blood Coagulation DisordersStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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 3
- Primary purpose
- Treatment
- Masking
- Double blind
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Investigators
- Louis Aledort, Icahn School of Medicine at Mount SinaiJ. Edson, University of MinnesotaM. Eyster, Milton S. Hershey Medical CenterScott Goodnight, University of Oregon Health Sciences CenterWilliam Hathaway, University of Colorado Medical CenterJack Lazerson, Milwaukee Children's HospitalPeter Levine, Memorial hospitalJeanne Lusher, Wayne State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00000582
- Sponsor's protocol ID
- 302
- Status last verified
- Apr 2012
- First submitted
- 27 Oct 1999
- Last updated
- 27 Apr 2012
- Results posted
- No
Publications
Eyster ME, Gill FM, Blatt PM, Hilgartner MW, Ballard JO, Kinney TR. Central nervous system bleeding in hemophiliacs. Blood. 1978 Jun;51(6):1179-88. No abstract available.
PubMed 647123Lusher JM, McMillan CW. Severe factor VIII and factor IX deficiency in females. Am J Med. 1978 Oct;65(4):637-48. doi: 10.1016/0002-9343(78)90852-5.
PubMed 309282Kasper CK. Effect of prothrombin complex concentrates on factor VIII inhibitor levels. Blood. 1979 Dec;54(6):1358-68.
PubMed 508942Lusher JM, Shapiro SS, Palascak JE, Rao AV, Levine PH, Blatt PM. Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. N Engl J Med. 1980 Aug 21;303(8):421-5. doi: 10.1056/NEJM198008213030803.
PubMed 6771653Gerety RJ, Eyster ME, Tabor E, Drucker JA, Lusch CJ, Prager D, Rice SA, Bowman HS. Hepatitis B virus, hepatitis A virus and persistently elevated aminotransferases in hemophiliacs. J Med Virol. 1980;6(2):111-8. doi: 10.1002/jmv.1890060203.
PubMed 6264019
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cooperative Study of Factor VIII Inhibitors
- ClinicalTrials.gov ID
- NCT00000582
- Acronym / sponsor ID
- 302
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- Jul 1978
- Study completion
- Jul 1979 (actual)
- First posted
- 28 Oct 1999 (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.
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of hemophiliac patients who had inhibitors to Factor VIII.
Detailed description
BACKGROUND:
Despite major advances in the treatment of patients with hemophilia, a serious remaining challenge was presented by the occurrence of circulating inhibitors to Factor VIII. Because of lack of information on the natural course of patients with Factor VIII inhibitors, the relative efficacy of various modes of therapy was not established. The Division of Blood Diseases and Resources decided to sponsor a clinical investigation which would evaluate populations of hemophilia patients for Factor VIII inhibitors, follow up these patients to provide information on the natural history of the inhibitor in the hemophilia patients, and make available a reference center to monitor results and attain uniformity.
Treatment of a patient with a severe inhibitor and consequent bleeding remained a problem. Management included protracted treatment with Factor VIII, use of immunosuppressive agents and prothrombin complex (or Factor IX) concentrates. The rationale for Factor IX was that it bypassed the defect in Factor VIII caused by the inhibitor. This method of therapy attracted wide popularity, but the success was greatly debated. It was intended at the very outset of the Factor VIII study that therapeutic trials involving patients with inhibitors would not be a prime function, but that such studies would be monitored if necessary. A controlled trial of Factor IX concentrates therapy was strongly advised by the DBDR Advisory Committee. Accordingly, during fiscal year 1978, a protocol for a double-blind control study was developed by the Factor VIII inhibitor group. The trial began in the spring of 1978, and the intervention terminated about one year later.
DESIGN NARRATIVE:
Double-blind study; patients served as their own controls. A total of 51 patients each received a single large dose of Konyne, Proplex, or diluted albumin (as a control). Joint bleeding of the elbow, knee, and ankle was evaluated six hours after each dose.
Study design
- Primary purpose
- Treatment
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 75 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- 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 |
|---|---|---|
| factor ix | 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 27 Apr 2012.
- Background: PubMed 647123Eyster ME, Gill FM, Blatt PM, Hilgartner MW, Ballard JO, Kinney TR. Central nervous system bleeding in hemophiliacs. Blood. 1978 Jun;51(6):1179-88. No abstract available.
- Background: PubMed 309282Lusher JM, McMillan CW. Severe factor VIII and factor IX deficiency in females. Am J Med. 1978 Oct;65(4):637-48. doi: 10.1016/0002-9343(78)90852-5.
- Background: PubMed 508942Kasper CK. Effect of prothrombin complex concentrates on factor VIII inhibitor levels. Blood. 1979 Dec;54(6):1358-68.
- Background: PubMed 6771653Lusher JM, Shapiro SS, Palascak JE, Rao AV, Levine PH, Blatt PM. Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. N Engl J Med. 1980 Aug 21;303(8):421-5. doi: 10.1056/NEJM198008213030803.
- Background: PubMed 6264019Gerety RJ, Eyster ME, Tabor E, Drucker JA, Lusch CJ, Prager D, Rice SA, Bowman HS. Hepatitis B virus, hepatitis A virus and persistently elevated aminotransferases in hemophiliacs. J Med Virol. 1980;6(2):111-8. doi: 10.1002/jmv.1890060203.
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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.
Louis Aledort
Icahn School of Medicine at Mount Sinai
J. Edson
University of Minnesota
M. Eyster
Milton S. Hershey Medical Center
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing factor ix in people with Blood Coagulation Disorders and Hematologic Diseases, run by National Heart, Lung, and Blood Institute (NHLBI).
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 27 Apr 2012. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
On the ClinicalTrials.gov record for NCT00000582, which also lists 5 linked publications.
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 27 Apr 2012.