Bleeding and Treatment for Von Willebrand Disease

Recruiting at 16 trial locations

Overseen By Clinical Trials (Australia)

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Hemab ApS

Must not be taking: Anticoagulants, Antiplatelets, NSAIDs

Disqualifiers: Thrombosis, Pregnancy, Surgery, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 12

200 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to gather information about bleeding events, quality of life, and the impact of bleeds on people with Von Willebrand Disease (VWD). Researchers seek to understand bleeding patterns and treatments for different types of VWD, aiding future studies. The trial may suit individuals who frequently experience bleeding episodes, such as bruising or heavy menstrual bleeding, that require treatment.

As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in VWD treatment.

Do I need to stop my current medications to join the trial?

The trial protocol does not clearly specify if you need to stop your current medications. However, if you are taking anticoagulation, antiplatelet, or non-steroidal anti-inflammatory drugs, it might affect your eligibility, so it's best to discuss your medications with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that treatments for Von Willebrand Disease (VWD) have been safe in past studies. For individuals with Type 1 VWD, studies confirm that treatments like Fanhdi® and Alphanate® effectively and safely manage bleeding episodes. One study found that 84% of bleeding treatments worked well for patients with Type 1 VWD.

For Types 2A, 2M, 2N, and Type 3 VWD, research emphasizes the need to understand different bleeding patterns. Although detailed safety data for these types remain less clear, treatments are based on similar methods as those for Type 1, suggesting they are likely safe.

In summary, while treatment specifics can vary depending on the type of VWD, treatments have a history of being safe and well-tolerated in managing the condition. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this trial because it aims to explore bleeding patterns and the effectiveness of treatments in people with Von Willebrand Disease (VWD), specifically those with less common types like Type 2A, 2M, 2N, or Type 3, as well as Type 1 with low levels of VWF. Current treatments for VWD often involve desmopressin or factor replacement therapies, but they may not be as effective for these specific subtypes. By focusing on these groups, the trial seeks to uncover new insights and potentially tailor more effective treatment strategies, offering hope for improved management of bleeding episodes in VWD patients.

What evidence suggests that this trial's treatments could be effective for Von Willebrand Disease?

In this trial, researchers will study participants with Type 1 von Willebrand Disease (VWD) separately from those with Type 2A, Type 2M, Type 2N, or Type 3 VWD. Research has shown that treatments boosting von Willebrand factor (VWF) levels often work well for people with Type 1 VWD, aiding blood clotting. Most patients experience a significant increase in VWF and factor VIII levels, crucial for stopping bleeding. For those with Type 2 and Type 3 VWD, plasma-derived VWF-factor VIII concentrates provide a safe and effective treatment. This approach helps control bleeding in patients with these types of VWD, who often experience more severe symptoms. Overall, these treatments have demonstrated positive results in managing bleeding for different types of VWD. ^{5 6 7 8 9}

Who Is on the Research Team?

VP of Clinical Research

Principal Investigator

Hemab ApS

Are You a Good Fit for This Trial?

Inclusion Criteria

I can understand and agree to the study's requirements.

I understand and agree to follow the study's procedures and rules.

I am 16 years old or older.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Evaluation

Initial assessment of participants' bleeding events, quality of life, and clinical impact

1 week

Observation

Participants are monitored for bleeding events and treatment efficacy with telemedicine check-ins and clinic visits

4-6 months

Telemedicine check-ins every other week, clinic visits every 12 weeks

Optional Extension

Participants may continue in the observation phase for up to a total of 12 months

Up to 12 months

Follow-up

Participants are monitored for safety and effectiveness after the observation period

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Clinical outcomes of patients with VWD, Type 1
• Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3

How Is the Trial Designed?

Group I: VWD Type 2A, Type 2M, Type 2N, or Type 3 Experimental Treatment 1 Intervention

Group II: VWD Type 1 (residual VWF antigen and/or activity less than 30 IU per dL) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Hemab ApS

Lead Sponsor

Trials

6

Recruited

1,100+

PSI CRO

Industry Sponsor

Trials

13

Recruited

2,800+

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6142610/

Advances in the diagnosis and treatment of Von Willebrand ...

VWD is a common and challenging bleeding disorder, given the difficulties in diagnosis and treatment. New options for diagnosis, including use of bleeding ...

2.

ashpublications.org

ashpublications.org/blood/article/123/26/4037/32790/The-bleeding-score-predicts-clinical-outcomes-and

The bleeding score predicts clinical outcomes and ...

Bleeding tendency and efficacy of anti-haemorrhagic treatments in patients with type 1 von Willebrand disease and increased von Willebrand factor clearance.

3.

ccjm.org

ccjm.org/content/91/2/119

von Willebrand disease: A guide for the internist

An adequate increase in VWF antigen, ristocetin cofactor, and factor VIII levels is seen in most cases of type 1 VWD and in many of type 2.

4.

sciencedirect.com

sciencedirect.com/science/article/pii/S2475037924000219

Procedural outcomes in children with mild type 1 von ...

Pediatric patients with mild type 1 VWD can safely undergo procedures using a tailored approach. Bleeding complications were uncommon, with the majority ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06610201

A Study of Bleeding and Treatment in Participants With Von ...

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11403693/

Real-World Efficacy and Safety of Plasma-Derived Von ...

This study confirmed that Fanhdi® and Alphanate® were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries ...

7.

fda.gov

fda.gov/media/188621/download?attachment

September 5, 2025 Clinical Review Memo - VONVENDI

Study 071301 demonstrated that Vonvendi is safe and effective in patients with Type 1 and Type 2 VWD with 100% decrease in sABR in prior OD ...

8.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04052698

NCT04052698 | Clinical Study to Investigate the Efficacy ...

Currently receiving on-demand treatment with a VWF-containing product with at least 1, and an average of ≥2, documented spontaneous BEs per month in the last 6 ...

9.

wilateusa.com

wilateusa.com/vwd/learn-about-wilate/clinical-evidence-safety/

wilate® Clinical Evidence and Safety for VWD Treatment

Successful treatment of bleeding episodes in patients with VWD1, · For all patients studied, 84% of bleeding treatments were successful · For pediatric patients ...