Haemophilic PseudotumourPhase 4NCT03921294

HemLibra Prophylaxis in Patients With Hemophilic Pseudotumor

A Phase 4 study testing Emicizumab in people with Haemophilic Pseudotumour, run by Indiana Hemophilia &Thrombosis Center, Inc. at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

This is a single arm, phase 4, prospective, open-label, United States single-center study to assess the hemostatic efficacy and safety of Hemlibra (emicizumab) for hemostatic control of hemophilia A patients (baseline FVIII level <40%) with and without inhibitors with hemophilic pseudotumors; secondary outcomes will… Read more about this study

Who is it for?: Any age
Conditions the study is for: Haemophilic Pseudotumour
What is being tested?: Emicizumab
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 1 participants
Where: 1 siteUnited States
Study dates: 15 May 2019to 16 Mar 2022
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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Signed informed consent form from the subject, parent or guardian
Diagnosis of congenital hemophilia A (baseline FVIII level <40%) with or without FVIII inhibitor, either high or low responding, regardless of titer
Diagnosis of a hemophilic pseudotumor confirmed by radiologic assessment such as CT or MRI
Any weight or BMI
Medical documentation of prophylactic or episodic treatment (FVIII or bypassing agent) and the number of bleeding episodes for at least 16 weeks, and up to 6 months if available, prior to entry into the study
Medical documentation of any need for PRBC transfusion or hospitalization for 6 months prior to entry into the study

Show all 19Show fewer

Subjects with a history of an inhibitor should provide documentation of the inhibitor history including date of initial diagnosis of inhibitor, peak titer, and agent utilized for hemostatic control
Subjects with high titer inhibitors or those with low titer inhibitors who do not respond to FVIII must be willing to use rFVIIa as first line therapy for the treatment of breakthrough bleeding events
Medical documentation of ITI therapy for subjects with a history of a FVIII inhibitor and ITI, including current FVIII inhibitor titer
Willingness to discontinue any current prophylactic hemostatic regimen (FVIII or bypassing agent) and/or FVIII ITI therapy for the duration of the study
Subjects receiving FVIII prophylaxis must be willing to discontinue their FVIII prophylactic regimen immediately prior to their second loading dose of Hemlibra (emicizumab)
Subjects receiving bypassing agent prophylaxis must be willing to discontinue their prophylactic regimen at least 24 hours prior to their first loading dose of Hemlibra (emicizumab)
Subjects receiving FVIII ITI therapy must be willing to discontinue ITI immediately prior to their first loading dose of Hemlibra (emicizumab)
Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the health-related questionnaires, activity tracking, and bleed diaries, using systems provided during the study
Adequate hepatic function, defined as total bilirubin ≤1.5 × age-adapted upper limit of normal (ULN) (excluding Gilbert's syndrome) and both AST and ALT ≤3 × age-adapted ULN at the time of screening, and no clinical signs or known laboratory/radiographic evidence consistent with cirrhosis
Subjects must be willing to be vaccinated against HAV and HBV if not previously vaccinated, exposed or immune to HAV or HBV*
Adequate hematologic function, defined as a platelet count ≥100,000/μL and a PT≤1.5 times the ULN at the time of screening
Adequate renal function, defined as serum creatinine ≤2.5 × age-adapted ULN and creatinine clearance ≥30 mL/min by Cockcroft-Gault formula
For women with hemophilia of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods that result in a failure rate of <1% per year during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug

Who cannot join

Inherited or acquired bleeding disorder other than congenital hemophilia A
Lack of a documented diagnosis of hemophilic pseudotumor
Patients who are at high risk for TMA (eg, have a previous medical or family history of TMA), in the Study Investigator's judgment
History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the Study Investigator's judgment
Previous (within the last 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
Other conditions (eg, certain autoimmune diseases) that may currently increase the risk of bleeding or thrombosis

Show all 19Show fewer

History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the Emicizumab injection
Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study
Known HIV infection with CD4 counts <200 cells/μL. HIV infection with CD4 counts ≥200 cells/μL permitted
Use of systemic immunomodulators (eg, interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy
Concomitant disease, condition, significant abnormality on screening evaluations or laboratory tests, or treatment that could interfere with the conduct of the study, or that would, in the opinion of the Study Investigator, pose an additional unacceptable risk in administering study drug to the patient
Receipt of any of the following:
Hemlibra (emicizumab) in a prior investigational study
An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration
A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter
Any other investigational drug currently being administered or planned to be administered
Inability to comply with the study protocol in the opinion of the Study Investigator
Pregnancy or lactation or intention to become pregnant during the study
Women with a positive serum pregnancy test result within 10 days prior to initiation of study drug

Trial locations and contacts

1 site in 1 country.

United States1 site

Indiana Hemophila @Thrombosis Center
Indianapolis, Indiana, 46260

Other trials in United States

About this study

Official title: Prospective, Single-arm, Open-label Use of Hemlibra (Emicizumab) to Treat Hemophilic Pseudotumor

Read the detailed description

This is a single arm, phase 4, prospective, open-label, United States single-center study to assess the hemostatic efficacy and safety of Hemlibra (emicizumab) for hemostatic control of hemophilia A patients, (baseline FVIII level <40%), children and adults, with and without inhibitors with hemophilic pseudotumors; secondary outcomes will assess changes in quality of life and activity level in treated patients.

Hemlibra (emicizumab) will be administered as primary weekly prophylaxis after the enrollment/screening visit is complete (approximately 7-10 days after screening, if laboratory results are available and eligibility is confirmed). If an activity monitoring device is typically utilized by the patient (eg, a Fitbit) then permission will be requested from the patient at screening to access the data for 1 month prior to screening as a baseline comparator for post-treatment activity. The use of an activity-monitoring device is not required by the study.

The enrollment period is 2 years and the study will last a maximum of 4 years; subjects will receive study medication (Hemlibra, emicizumab) for a minimum of 2 years and a maximum of 4 years based upon time of enrollment. Hemlibra (emicizumab) will be administered using the FDA-approved once-weekly dosing regimen for loading dose and prophylactic dose. Breakthrough bleeding events will be recorded and treated with locally available FVIII (eg, pdFVIII or rFVIII) in non-inhibitor subjects and inhibitor subjects with low titer inhibitors (titer<5 BU). The lowest dose of FVIII expected to achieve hemostasis will be utilized for treatment of breakthrough bleeding events in non-inhibitor and low-titer inhibitor patients. Subjects with high-titer inhibitors (titer ≥5 BU) and those with low titer inhibitors who do not respond to FVIII will be required to utilize rFVIIa as first line therapy; aPCC (<100 U/kg/day for preferably no more than 1 day) may only be used upon approval of the Study Investigator and under the supervision of a physician.

The proposed study is seeking to address the following knowledge gaps:

Does weekly prophylactic Hemlibra (emicizumab) reduce the rate of bleeding events in subjects with hemophilia A and pseudotumor, including the rate of hospitalization, anemia and transfusion? Does weekly prophylactic Hemlibra (emicizumab) control the progression of hemophilic pseudotumor? Does weekly prophylactic Hemlibra (emicizumab) result in an increase in QoL and activity level?

Conditions: Haemophilic Pseudotumour
Being tested: Emicizumab

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

EmicizumabDrug
Also called HemLibra
bispecific monoclonal antibody binding to activated Factor IX and Factor X

Study groups in NCT03921294
Study group	Role	What they receive
Single Arm	Experimental	Patients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement. Receives: Drug: Emicizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Hemoglobin
Maintenance or increase of hemoglobin (g/dl) from participants' baseline level based on serial blood tests.
Measured: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Participants' Need for Blood Transfusions or Lack of
Whether or not the patient requires blood transfusions (units of RBCs) due to blood loss secondary to lack of hemostatic efficacy during the duration of study treatment duration.
Measured: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.

Secondary outcomes (13)

Breakthrough Bleeds
Number of breakthrough bleeding events that require hemostatic therapy in addition to Hemlibra prophylaxis
Measured: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Pseudotumor Status
Radiologic evaluation (CT and/or MRI) to evaluate control of progression, stabilization or regression per change in size (measured in cm) of the pseudotumor
Measured: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Patient Quality of Life Based on Haem-A-QOL
The subjective change in quality of life and activity with Hemlibra (emicizumab) prophylaxis will be evaluated during the study. QoL and activity will be assessed using Haem-A-QoL and EQ-5D-5L. Changes in these measures will be determined by changes from the baseline scores compared to follow-up scores.
Measured: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Patient Quality of Life Based on EQ-5D-5L
The subjective change in quality of life and activity with Hemlibra (emicizumab) prophylaxis will be evaluated during the study. QoL and activity will be assessed using Haem-A-QoL and EQ-5D-5L. Changes in these measures will be determined by changes from the baseline scores compared to follow-up scores.
Measured: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Adverse Events
Number of adverse events while on HemLibra (emicizumab) prophylaxis.
Measured: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Serious Adverse Events
Number of SAEs while on HemLibra (emicizumab) prophylaxis
Measured: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Number of Participants With Adverse Events
Number of participants with adverse events while on HemLibra (emicizumab) prophylaxis
Measured: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Anti-Drug Antibodies (ADA)
Development of emicizumab anti-drug antibodies using the ADA assay
Measured: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
ADA and Activated Partial Thromboplastin Time (APTT)
If the patient develops an ADA: the ADA's effect on the patient's APTT
Measured: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
ADA and Factor VIII (FVIII)
If the patient develops an ADA: the ADA's effecton the patient's FVIII assay
Measured: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Planned or Unplanned Surgery
If the patient requires surgery: Whether the procedure(s) was/were planned versus unplanned
Measured: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Hemostatic Agents in Surgery
If the patient requires surgery: Whether hemostatic agents in addition to Hemlibra were required to achieve or maintain hemostasis
Measured: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Blood Loss in Surgery
If the patient requires surgery: Whether blood loss exceeded the estimated/predicted blood loss relative to a patient without hemophilia
Measured: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 May 201916 Mar 2022

First posted19 Apr 2019
Study start15 May 2019
Primary completion16 Mar 2022
Study completion16 Mar 2022

Why it stopped: Global COVID-19 pandemic prevented further study visits or enrollment.

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 (8)Papers the sponsor has linked to this study.
Other trials for Haemophilic PseudotumourStudies 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
Model description: single arm prospective open-label single-center study
Masking: None (open label)
Enrolment: 1 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03921294
Sponsor's protocol ID: RO-IIS-2018-10581
Status last verified: Mar 2024
First submitted: 29 Mar 2019
Last updated: 11 Apr 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: We will share resources and data from this project through collaborative publications in the scientific literature as well as through national, regional and international conference presentations. We will also share our methods and findings in a prompt manner with regional, national and international stakeholders to ensure that findings will be readily available to other researchers and clinicians with clinical or scientific interest in the subject area. Individual participant data that underlie the results reports in publications, reports or presentations (including text, tables, figures and appendices) will be shared after de-identification.

Publications

Ahlberg AK. On the natural history of hemophilic pseudotumor. J Bone Joint Surg Am. 1975 Dec;57(8):1133-6.
PubMed 1202003
Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. No abstract available.
PubMed 25059285
Franchini M, Mannucci PM. Hemophilia A in the third millennium. Blood Rev. 2013 Jul;27(4):179-84. doi: 10.1016/j.blre.2013.06.002. Epub 2013 Jun 28.
PubMed 23815950
Gringeri A, Leissinger C, Cortesi PA, Jo H, Fusco F, Riva S, Antmen B, Berntorp E, Biasoli C, Carpenter S, Kavakli K, Morfini M, Negrier C, Rocino A, Schramm W, Windyga J, Zulfikar B, Mantovani LG. Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study. Haemophilia. 2013 Sep;19(5):736-43. doi: 10.1111/hae.12178. Epub 2013 Jun 4.
PubMed 23731246
Liu SS, White WL, Johnson PC, Gauntt C. Hemophilic pseudotumor of the spinal canal. Case report. J Neurosurg. 1988 Oct;69(4):624-7. doi: 10.3171/jns.1988.69.4.0624.
PubMed 3418398
Magallon M, Monteagudo J, Altisent C, Ibanez A, Rodriguez-Perez A, Riba J, Tusell J, Martin-Villar J. Hemophilic pseudotumor: multicenter experience over a 25-year period. Am J Hematol. 1994 Feb;45(2):103-8. doi: 10.1002/ajh.2830450202.
PubMed 8141115
Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6.
PubMed 22776238
van Ommeren JW, Mooren DW, Veth RP, Novakova IR, van de Kaa CA. Pseudotumor occurring in hemophilia. Arch Orthop Trauma Surg. 2000;120(7-8):476-8. doi: 10.1007/s004029900087.
PubMed 10968546

Study at a glance

From the registry record, last updated by the sponsor on 11 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Prospective, Single-arm, Open-label Use of Hemlibra (Emicizumab) to Treat Hemophilic Pseudotumor
ClinicalTrials.gov ID: NCT03921294
Acronym / sponsor ID: RO-IIS-2018-10581
Conditions: Haemophilic Pseudotumour
Phase: Phase 4
Study type: Interventional
Recruitment status: TerminatedReason stopped: Global COVID-19 pandemic prevented further study visits or enrollment.
Enrolment: 1 participants (actual)
Lead sponsor: Indiana Hemophilia &Thrombosis Center, Inc.University, hospital or other
Collaborators: Genentech, Inc.
Study start: 15 May 2019 (actual)
Primary completion: 16 Mar 2022 (actual)
Study completion: 16 Mar 2022 (actual)
First posted: 19 Apr 2019 (actual)
Results: Posted 20 Mar 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

This is a single arm, phase 4, prospective, open-label, United States single-center study to assess the hemostatic efficacy and safety of Hemlibra (emicizumab) for hemostatic control of hemophilia A patients, (baseline FVIII level <40%), children and adults, with and without inhibitors with hemophilic pseudotumors; secondary outcomes will assess changes in quality of life and activity level in treated patients.

The proposed study is seeking to address the following knowledge gaps:

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: single arm prospective open-label single-center study

Arm	Type	Receives
Single Arm	Experimental	Drug: EmicizumabPatients with hemophilic pseudotumor will be treated with prophylactic emicizumab and assessed for improvement.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (19 listed)

Signed informed consent form from the subject, parent or guardian
Diagnosis of congenital hemophilia A (baseline FVIII level <40%) with or without FVIII inhibitor, either high or low responding, regardless of titer
Diagnosis of a hemophilic pseudotumor confirmed by radiologic assessment such as CT or MRI
Any weight or BMI
Medical documentation of prophylactic or episodic treatment (FVIII or bypassing agent) and the number of bleeding episodes for at least 16 weeks, and up to 6 months if available, prior to entry into the study

Key exclusion criteria (19 listed)

Inherited or acquired bleeding disorder other than congenital hemophilia A
Lack of a documented diagnosis of hemophilic pseudotumor
Patients who are at high risk for TMA (eg, have a previous medical or family history of TMA), in the Study Investigator's judgment
History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the Study Investigator's judgment
Previous (within the last 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease

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
EmicizumabAlso: HemLibrabispecific monoclonal antibody binding to activated Factor IX and Factor X	Drug	Single Arm

Primary outcome

Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Hemoglobin
Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Maintenance or increase of hemoglobin (g/dl) from participants' baseline level based on serial blood tests.
Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Participants' Need for Blood Transfusions or Lack of
Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Whether or not the patient requires blood transfusions (units of RBCs) due to blood loss secondary to lack of hemostatic efficacy during the duration of study treatment duration.

13 secondary outcomes

Breakthrough Bleeds
Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Pseudotumor Status
Time frame: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Patient Quality of Life Based on Haem-A-QOL
Time frame: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Patient Quality of Life Based on EQ-5D-5L
Time frame: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Adverse Events
Time frame: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Serious Adverse Events
Time frame: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Number of Participants With Adverse Events
Time frame: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.
Anti-Drug Antibodies (ADA)
Time frame: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
ADA and Activated Partial Thromboplastin Time (APTT)
Time frame: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
ADA and Factor VIII (FVIII)
Time frame: Every 12 months, for the 2 years and 10 months of the patient's study participation duration.
Planned or Unplanned Surgery
Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Hemostatic Agents in Surgery
Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.
Blood Loss in Surgery
Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.

Results and safety

Results posted to the registry on 20 Mar 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Emicizumab (Single Arm)	1	1

Primary outcome: Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Hemoglobin

Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.. Mean (Full Range). Unit: gm/dL

Measure	Single Arm n = 1
Value	9.5 (8 to 11)

Primary outcome: Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Participants' Need for Blood Transfusions or Lack of

Time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.. Count of participants. Unit: Participants

Measure	Emicizumab (Single Arm) n = 1
Value	0

13 secondary outcomes are reported in the full results.

Adverse events

Time frame: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Single Arm	0 of 1 (0%)	1 of 1 (100%)	0 of 1

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 1202003Ahlberg AK. On the natural history of hemophilic pseudotumor. J Bone Joint Surg Am. 1975 Dec;57(8):1133-6.
Results publication: PubMed 25059285Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014 Nov;12(11):1935-9. doi: 10.1111/jth.12672. Epub 2014 Sep 3. No abstract available.
Results publication: PubMed 23815950Franchini M, Mannucci PM. Hemophilia A in the third millennium. Blood Rev. 2013 Jul;27(4):179-84. doi: 10.1016/j.blre.2013.06.002. Epub 2013 Jun 28.
Results publication: PubMed 23731246Gringeri A, Leissinger C, Cortesi PA, Jo H, Fusco F, Riva S, Antmen B, Berntorp E, Biasoli C, Carpenter S, Kavakli K, Morfini M, Negrier C, Rocino A, Schramm W, Windyga J, Zulfikar B, Mantovani LG. Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study. Haemophilia. 2013 Sep;19(5):736-43. doi: 10.1111/hae.12178. Epub 2013 Jun 4.
Results publication: PubMed 3418398Liu SS, White WL, Johnson PC, Gauntt C. Hemophilic pseudotumor of the spinal canal. Case report. J Neurosurg. 1988 Oct;69(4):624-7. doi: 10.3171/jns.1988.69.4.0624.
Results publication: PubMed 8141115Magallon M, Monteagudo J, Altisent C, Ibanez A, Rodriguez-Perez A, Riba J, Tusell J, Martin-Villar J. Hemophilic pseudotumor: multicenter experience over a 25-year period. Am J Hematol. 1994 Feb;45(2):103-8. doi: 10.1002/ajh.2830450202.
Results publication: PubMed 22776238Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6.
Results publication: PubMed 10968546van Ommeren JW, Mooren DW, Veth RP, Novakova IR, van de Kaa CA. Pseudotumor occurring in hemophilia. Arch Orthop Trauma Surg. 2000;120(7-8):476-8. doi: 10.1007/s004029900087.
Hemophilia Data \& StatisticsLink provided by the sponsor.
What is Hemophilia?Link provided by the sponsor.
WFH Guidelines for the management of hemophiliaLink provided by the sponsor.
WFH report on the annual global survey 2011Link provided by the sponsor.
WFH report on the annual global survey 2013Link provided by the sponsor.
WFH report on the annual global survey 2015Link provided by the sponsor.
Individual participant dataSharing planned: Yes. We will share resources and data from this project through collaborative publications in the scientific literature as well as through national, regional and international conference presentations. We will also share our methods and findings in a prompt manner with regional, national and international stakeholders to ensure that findings will be readily available to other researchers and clinicians with clinical or scientific interest in the subject area. Individual participant data that underlie the results reports in publications, reports or presentations (including text, tables, figures and appendices) will be shared after de-identification.

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.

Amy D Shapiro, MD principal investigator

Indiana Hemophilia &Thrombosis Center, Inc.

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 Emicizumab in people with Haemophilic Pseudotumour, run by Indiana Hemophilia &Thrombosis Center, Inc. at 1 site in United States.

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 19 inclusion and 19 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: stopped early, as of the sponsor's last update on 11 Apr 2024. 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 United States. 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 20 Mar 2024). 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?

Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Hemoglobin (time frame: Every 6 months, for the 2 years and 10 months of the patient's study participation duration.). The study lists 2 primary outcomes in all. 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 NCT03921294, which also lists 8 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 11 Apr 2024.