NCT03921294

HemLibra Prophylaxis in Patients With Hemophilic Pseudotumor

Terminated Phase 4 Results posted Last updated 11 April 2024

What this trial tests

Phase 4 trial testing Emicizumab in Haemophilic Pseudotumour in 1 participant. Terminated before completion.

Timeline

15 May 2019

Primary endpoint
16 March 2022

16 March 2022

Quick facts

Lead sponsor	Indiana Hemophilia &Thrombosis Center, Inc.
Phase	Phase 4
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	15 May 2019
Primary completion	16 March 2022
Estimated completion	16 March 2022
Sites	1 location across United States

Drugs / interventions tested

• Emicizumab (EMICIZUMAB) — full drug profile →

Conditions studied

• Haemophilic Pseudotumour — all drugs for Haemophilic Pseudotumour →

Sponsor

Indiana Hemophilia &Thrombosis Center, Inc.

Who can join

Eligibility, any sex, with Haemophilic Pseudotumour. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Hemostatic Efficacy of Prophylactic Weekly Injections of Hemlibra (Emicizumab) Based on Hemoglobin Primary · 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.

Group	Value	95% CI
Single Arm	9.5	8 – 11

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

Group	Value	95% CI
Emicizumab (Single Arm)	0

Breakthrough Bleeds Secondary · Every 6 months, for the 2 years and 10 months of the patient's study participation duration.

Number of breakthrough bleeding events that require hemostatic therapy in addition to Hemlibra prophylaxis

Group	Value	95% CI
Single Arm	14

Adverse Events Secondary · Every 3 months, for the 2 years and 10 months of the patient's study participation duration.

Number of adverse events while on HemLibra (emicizumab) prophylaxis.

Group	Value	95% CI
Single Arm	3

Serious Adverse Events Secondary · Every 3 months, for the 2 years and 10 months of the patient's study participation duration.

Number of SAEs while on HemLibra (emicizumab) prophylaxis

Group	Value	95% CI
Single Arm	0

Number of Participants With Adverse Events Secondary · Every 3 months, for the 2 years and 10 months of the patient's study participation duration.

Number of participants with adverse events while on HemLibra (emicizumab) prophylaxis

Group	Value	95% CI
Single Arm	1

Anti-Drug Antibodies (ADA) Secondary · Every 12 months, for the 2 years and 10 months of the patient's study participation duration.

Development of emicizumab anti-drug antibodies using the ADA assay

Group	Value	95% CI
Single Arm	0

Adverse events — posted to ClinicalTrials.gov

Time frame: Every 3 months, for the 2 years and 10 months of the patient's study participation duration.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Single Arm

Serious: 0/1 (0%)

Deaths: 0/1

Other adverse events (3 terms — click to expand)

Reaction	System	Single Arm
Upper respiratory infection	General disorders	—
Fall with injurty	Musculoskeletal and connective tissue disorders	—
Malaria infection	Infections and infestations	—

Data from ClinicalTrials.gov NCT03921294 adverse events section.

Sponsor's own 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%) with and without inhibitors with hemophilic pseudotumors; secondary outcomes will assess changes in quality of life and activity level in treated patients.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.