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Von Willebrand Disease (VWD)VWD-ASSIUTNCT07410130

Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate

An observational study of Von Willebrand Disease (VWD) and Inherited Bleeding Disorders in Children, run by Assiut University at 1 site in Egypt.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting. Read more about this study

Who is it for?
Ages 0 to 18 years
What is being tested?
Tranexamic Acid and Von Willebrand Factor-Containing Concentrates
How many people
25 participants
Where
1 siteEgypt
Study dates
Mar 2026to Dec 2026
Phase
Observational
Are there results?
Not yet: the study is under way

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 0 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Study population: This study includes pediatric patients aged 0 to 18 years with suspected or confirmed von Willebrand disease (VWD) who are residents of Assiut Governorate or are receiving medical care at Assiut University Children's Hospital. The study population represents children evaluated for bleeding symptoms or referred for assessment of possible inherited bleeding disorders within a tertiary pediatric healthcare setting. Eligible participants are identified based on clinical presentation suggestive of VWD, such as recurrent epistaxis, easy bruising, mucocutaneous bleeding, prolonged bleeding following trauma or surgical procedures, and heavy menstrual bleeding in adolescent females. Both newly evaluated patients and previously diagnosed cases with accessible medical records are included to allow comprehensive assessment of disease frequency, clinical spectrum, and management outcomes. All participants undergo standardized clinical evaluation, including detailed medical history, family history

Who may be able to join

  • Age 0-18 years.
  • Residents of Assiut Governorate or receiving care at Assiut University Children's Hospital.
  • Suspected or confirmed von Willebrand disease (VWD) based on clinical bleeding symptoms or referral for evaluation.
  • Patients diagnosed with VWD using standard laboratory tests, including:
  • VWF antigen (VWF:Ag).
  • VWF ristocetin cofactor activity (VWF:RCo).
  • Factor VIII activity.

Who cannot join

  • Other inherited bleeding disorders, such as:
  • Hemophilia A or B.
  • Rare coagulation factor deficiencies (e.g., factors I, V, VII, X, XI deficiency).
  • Platelet function disorders.
  • Acquired bleeding disorders, including:
  • Liver disease.
Show all 12Show fewer
  • Renal insufficiency.
  • Vitamin K deficiency.
  • Disseminated intravascular coagulation (DIC).
  • Use of medications that may interfere with coagulation testing (e.g., anticoagulants, antiplatelet drugs).
  • Incomplete clinical or laboratory data (for retrospective cases).
  • Refusal of consent for participation (for prospective cases).

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Egypt1 site
  • Assiut university

    Asyut

    Assiut University, 08822080150, vp_grad@aun.edu.eg

Other trials in Egypt

About this study

Official title: Clinical Spectrum of Von Willebrand Disease Among Children: Frequency, Management, and Outcomes in Assiut Governorate

Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting. Children with VWD may experience frequent nosebleeds, easy bruising, prolonged bleeding after injuries or surgeries, and, in adolescent girls, heavy menstrual bleeding. The severity of symptoms varies widely depending on the type of the disease and the level of the clotting factor.

Despite its clinical importance, data about the frequency, clinical presentation, and treatment outcomes of von Willebrand disease among children in Upper Egypt are limited. Early recognition and appropriate management are crucial to prevent complications, reduce hospital visits, and improve quality of life.

Read more

This observational study aims to assess the frequency of von Willebrand disease among children attending Assiut University Children's Hospital, describe the different disease subtypes, and evaluate the clinical bleeding patterns and management strategies used in routine practice. The study will include children aged 0-18 years with suspected or confirmed VWD.

Information will be collected from medical records and clinical evaluations, including bleeding symptoms, laboratory test results, disease classification, and treatment approaches. The results of this study are expected to improve understanding of von Willebrand disease in children in this region and support better diagnostic and therapeutic planning for affected patients.

Read the detailed description

Von Willebrand disease (VWD) is the most common inherited bleeding disorder worldwide and represents a significant cause of mucocutaneous bleeding in children. It results from quantitative or qualitative defects of von Willebrand factor (VWF), a glycoprotein that plays a key role in platelet adhesion and stabilization of factor VIII. VWD is classified into three main types according to International Society on Thrombosis and Hemostasis (ISTH) criteria: type 1 (partial quantitative deficiency), type 2 (qualitative defects with several subtypes), and type 3 (severe quantitative deficiency).

The clinical presentation of VWD in children is highly variable and may include epistaxis, easy bruising, gingival bleeding, prolonged bleeding after minor trauma or surgery, and menorrhagia in adolescent females. The severity and frequency of bleeding episodes are influenced by disease subtype, VWF levels, age, and associated conditions. In pediatric populations, diagnosis may be delayed or missed due to mild symptoms, age-related physiological variations in VWF levels, and limited awareness.

This study is designed as a descriptive retrospective-prospective observational study conducted at Assiut University Children's Hospital. The target population includes children aged 0-18 years with suspected or confirmed von Willebrand disease who are residents of Assiut Governorate or receive care at the study center. Patients with other inherited or acquired bleeding disorders will be excluded.

During the retrospective phase, medical records of previously diagnosed VWD patients will be reviewed to collect demographic data, family history, clinical presentation, laboratory findings, disease subtype, treatment received, and documented outcomes. In the prospective phase, children presenting with bleeding symptoms suggestive of VWD will undergo standardized clinical assessment, including a structured bleeding questionnaire and physical examination, followed by laboratory evaluation.

Laboratory investigations will include complete blood count, coagulation profile, VWF antigen level, VWF activity, and factor VIII activity, with additional specialized testing when available. Disease classification will be performed according to ISTH guidelines.

Management data will be collected to describe current treatment practices, including on-demand therapy during bleeding episodes and prophylactic therapy for patients with recurrent or severe bleeding. Treatment response, frequency of bleeding episodes, need for hospital visits, and occurrence of adverse events will be monitored during follow-up.

The primary outcomes of the study include the frequency of von Willebrand disease among the investigated pediatric population, distribution of disease subtypes, and characterization of clinical bleeding patterns. Secondary outcomes include assessment of treatment modalities, recurrence of bleeding episodes, and short-term clinical outcomes.

By providing comprehensive data on the clinical spectrum and management of VWD among children in Assiut Governorate, this study aims to enhance early diagnosis, guide evidence-based management strategies, and improve overall patient care in this setting.

Being tested
Tranexamic Acid, Von Willebrand Factor-Containing Concentrates
Keywords
Von Willebrand Disease, VWD, Pediatric bleeding disorders, Inherited coagulation disorders, Von willebrand factor, Mucocutaneous bleeding, Epistaxis in children, Menorrhagia, Bleeding assessment tool, VWD antigen, VWD activity, Hemostasis, Pediatric hematology, Factor VIII, Observational study
Medical subject headings
von Willebrand Diseases, Blood Coagulation Disorders, Inherited, Menorrhagia, Hemophilia A

What participants receive

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

  • Tranexamic AcidDrug

    Tranexamic acid is used as an antifibrinolytic agent for the management of mucocutaneous bleeding episodes in children with von Willebrand disease, according to standard clinical practice.

  • Von Willebrand Factor-Containing ConcentratesDrug

    Plasma-derived von Willebrand factor/factor VIII concentrates are administered either on-demand during bleeding episodes or as regular prophylactic therapy in patients with recurrent or severe bleeding, based on clinical need.

Study groups in NCT07410130
Study groupRoleWhat they receive
On-Demand Therapy GroupThis cohort includes children with confirmed von Willebrand disease who receive treatment only during active bleeding episodes or prior to invasive procedures. Management is based on clinical indication and routine care practices, without scheduled prophylactic therapy. Bleeding frequency, treatment response, and short-term outcomes are documented during follow-up.
Receives: Drug: Tranexamic Acid; Drug: Von Willebrand Factor-Containing Concentrates
Prophylaxis Therapy GroupThis cohort includes children with von Willebrand disease who experience recurrent, severe, or clinically significant bleeding and therefore receive regular prophylactic treatment with von Willebrand factor-containing concentrates. Patients are followed prospectively to assess bleeding frequency, treatment effectiveness, and clinical outcomes under scheduled preventive therapy.
Receives: Drug: Tranexamic Acid; Drug: Von Willebrand Factor-Containing Concentrates

What is being measured

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

  1. Frequency of Von Willebrand Disease Among Investigated Children

    The proportion of children diagnosed with von Willebrand disease among those evaluated for suspected bleeding disorders at Assiut University Children's Hospital.

    Measured: at enrollment

  2. Distribution of Von Willebrand Disease Subtypes

    Classification and relative frequency of von Willebrand disease types (Type 1, Type 2, and Type 3) according to ISTH diagnostic criteria based on laboratory findings.

    Measured: Within 2 weeks of enrollment

  3. Clinical Bleeding Patterns in Children With Von Willebrand Disease

    Assessment of bleeding manifestations, including epistaxis, bruising, mucosal bleeding, postsurgical bleeding, and menorrhagia, using standardized clinical evaluation and bleeding assessment tools.

    Measured: at enrollment.

Timeline

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

  1. First posted13 Feb 2026
  2. Study startMar 2026 (estimated)
  3. Primary completionSep 2026 (estimated)
  4. Study completionDec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Other
Enrolment
25 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07410130
Sponsor's protocol ID
AUN-PED-VWD-2026-01
Acronym
VWD-ASSIUT
Status last verified
Feb 2026
First submitted
29 Jan 2026
Last updated
13 Feb 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Ahmed EH, et al. Screening of Von Willebrand Disease Among Children with Severe or Recurrent Epistaxis. Egyptian Journal, 2025.

  • Brauchli YB, Wais T, Gratwohl A, Heim D, Schipf A, Diebold J, Krahenbuhl S. Fatal myocardial infarction during nilotinib treatment in a 60-year-old male patient. Acta Oncol. 2010 May;49(4):523-5. doi: 10.3109/02841861003691952. No abstract available.

    PubMed 20307244
  • Jaffe JJ, Chrin LR. Thymidylate synthetase activity in normal and brugia pahangi-infected aedes aegypti. Biochem Pharmacol. 1979 Jun 15;28(12):1831-5. doi: 10.1016/0006-2952(79)90633-6. No abstract available.

    PubMed 454454
  • Sharma R, Haberichter SL. New advances in the diagnosis of von Willebrand disease. Hematology Am Soc Hematol Educ Program. 2019 Dec 6;2019(1):596-600. doi: 10.1182/hematology.2019000064.

    PubMed 31808831
  • Shui M, D'Angelo L, Croteau SE. Low von Willebrand factor in pediatric patients: Retrospective analysis of 293 cases informs diagnostic and therapeutic decision making. Pediatr Blood Cancer. 2020 Sep;67(9):e28497. doi: 10.1002/pbc.28497. Epub 2020 Jun 23.

    PubMed 32573918
  • Abe K, Dupervil B, O'Brien SH, Oakley M, Kulkarni R, Gill JC, Byams V, Soucie MJ; US Hemophilia Treatment Center Network. Higher rates of bleeding and use of treatment products among young boys compared to girls with von Willebrand disease. Am J Hematol. 2020 Jan;95(1):10-17. doi: 10.1002/ajh.25656. Epub 2019 Oct 29.

    PubMed 31612544
  • Sanders YV, Fijnvandraat K, Boender J, Mauser-Bunschoten EP, van der Bom JG, de Meris J, Smiers FJ, Granzen B, Brons P, Tamminga RY, Cnossen MH, Leebeek FW; WiN Study Group. Bleeding spectrum in children with moderate or severe von Willebrand disease: Relevance of pediatric-specific bleeding. Am J Hematol. 2015 Dec;90(12):1142-8. doi: 10.1002/ajh.24195. Epub 2015 Nov 17.

    PubMed 26375306
  • Halimeh S, Krumpel A, Rott H, Bogdanova N, Budde U, Manner D, Faeser B, Mesters R, Nowak-Gottl U. Long-term secondary prophylaxis in children, adolescents and young adults with von Willebrand disease. Results of a cohort study. Thromb Haemost. 2011 Apr;105(4):597-604. doi: 10.1160/TH10-09-0616. Epub 2011 Feb 8.

    PubMed 21301780

Questions about this trial

What is this clinical trial studying?

An observational study of Von Willebrand Disease (VWD) and Inherited Bleeding Disorders in Children, run by Assiut University at 1 site in Egypt.

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 7 inclusion and 12 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: opening soon, as of the sponsor's last update on 13 Feb 2026. 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 Egypt. 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.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Ali Maher Maher (01141914839, Ali.15235776@med.aun.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency of Von Willebrand Disease Among Investigated Children (time frame: at enrollment). The study lists 3 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 NCT07410130, 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 13 Feb 2026.

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