Health Trials

Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

ConditionsVon Willebrand Disease, Type 3Concomitant VWD and Hemophilia
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age0-90
SponsorBleeding and Clotting Disorders Institute Peoria, Illinois

About this trial

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Signed informed consent

Age 0 and older (infants weighing ≥3 kg)

ability to comply with protocol in investigators judgement

diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding </= 20 U/dl or variant VWD confirmed by genetic mutation and VWF ag, activity or CB < 50 U/dl based on historical medical records of study site.

Disqualifiers

Patients and/or infants weighing < 3 kg.

Patients with low VWF or non-severe VWD (ie.not meeting the above criteria)

Other concomitant bleeding disorders including coagulopathy from liver cirrhosis.

Current treatment with emicizumab or emicizumab therapy in the previous 18 months.

Trial design

Design model

Single group

Treatments tested in this trial

  • Emicizumab

    Drug

    Subcutaneous injection of emicizumab for prophylaxis

Treatment groups

40 Participants
are divided into 1 treatment group
Group A: Open Label EmicizumabOther 1 intervention

Trial outcomes

Primary outcomes

1

Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A

Establish bleed occurrence during treatment evaluated through descriptive statistical analysis to determine proof of principle

Time frame
18 months
2

Emicizumab is safe for prophylaxis in severe VWD & concomitant VWD/Hemophilia A

Collection of AE's, hypersensitivity reactions, thrombotic events during treatment

Time frame
18 months

Secondary outcomes

1

Reduced treatment burden vs VWF/FVIII prophylaxis

\# of infusions and methods of infusions collected during study

Time frame
18 months
2

Decreased bleed occurrence

Evaluate historical ABR with on demand or prophy prior to study treatment

Time frame
12 months
3

Diminish bleed severity

Collection of bleed data prior to study entry and throughout study treatment

Time frame
18 months

Other outcomes

1

Improve health related QOL in study participants

Collection of HRQOL PRO's

Time frame
18 months
2

Reduce product use for spontaneous or traumatic bleeds

Bleed \& treatment logs collecting product use prior to study entry and throughout study treatment

Time frame
18 months
3

Reduce product use during surgery

Bleed \& treatment logs collect information for any surgical procedure while on study, blood loss, hemostatic efficacy and concentrate consumption.

Time frame
18 months
4

Reduce self-reported treatment burden for HMB

Bleed \& treatment logs collect treatment information prior to and during study for HMB including antifibrinolytics, concentrates and hormonal therapies.

Time frame
18 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Bleeding and Clotting Disorders Institute Peoria, Illinois

Lead sponsor

Genentech, Inc.

Collaborator

Source ClinicalTrials.gov
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