Rare hemorrhagic disorder due to a constitutional coagulation factors defect

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1Active trials8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Rare hemorrhagic disorder due to a constitutional coagulation factors defect is an umbrella term used to describe a group of inherited bleeding conditions caused by problems with one or more of the proteins (called coagulation factors) that help blood clot properly. These conditions are sometimes called 'rare bleeding disorders' or 'rare coagulation factor deficiencies.' Unlike the more well-known hemophilia A and B, these disorders involve deficiencies in other clotting factors such as factor I (fibrinogen), factor II (prothrombin), factor V, factor VII, factor X, factor XI, factor XIII, or combined factor deficiencies. When one or more of these clotting proteins is missing or does not work correctly, the blood cannot form a stable clot after an injury. This means that even minor cuts, dental procedures, or surgeries can lead to prolonged or excessive bleeding. Internal bleeding into joints, muscles, or organs can also occur, sometimes without any obvious injury. Symptoms vary widely depending on which factor is affected and how severe the deficiency is. Some people experience frequent nosebleeds, easy bruising, heavy menstrual periods, or bleeding gums. Others may have more serious episodes such as bleeding into the brain or gastrointestinal tract. Treatment typically involves replacing the missing clotting factor using plasma-derived or recombinant factor concentrates, fresh frozen plasma, or other clotting agents. With proper management, many people with these disorders can live full and active lives.

Also known as:

Key symptoms:

Prolonged or heavy bleeding after cuts, injuries, or surgeryFrequent or hard-to-stop nosebleedsEasy or unexplained bruisingHeavy or prolonged menstrual periods in womenBleeding gums, especially after dental workBleeding into joints causing pain and swellingBleeding into musclesBlood in the urine or stoolProlonged bleeding after childbirthBleeding in the brain (in severe cases)Umbilical cord bleeding in newbornsDelayed wound healingExcessive bleeding after circumcision

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Oct 2024Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery

CSL Behring — PHASE3

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Rare hemorrhagic disorder due to a constitutional coagulation factors defect.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Phase 31 trial
Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery
Phase 3
Actively Recruiting
PI: Study Director (CSL Behring) · Sites: Basingstoke · Age: 1899 yrs

No specialists are currently listed for Rare hemorrhagic disorder due to a constitutional coagulation factors defect.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Rare hemorrhagic disorder due to a constitutional coagulation factors defect.

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Caregiver Resources

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Mental Health Support

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Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.Which specific clotting factor am I deficient in, and how severe is my deficiency?,What treatment options are available for my specific factor deficiency, and should I be on regular preventive therapy?,What should I do — and who should I call — if I have a serious bleeding episode or a head injury?,Are there activities or medications I should avoid to reduce my bleeding risk?,Should other family members be tested, and what are the chances of passing this condition to my children?,How should I prepare for surgery, dental procedures, or pregnancy?,Is there a hemophilia treatment center near me where I can receive specialized ongoing care?

Common questions about Rare hemorrhagic disorder due to a constitutional coagulation factors defect

What is Rare hemorrhagic disorder due to a constitutional coagulation factors defect?

Rare hemorrhagic disorder due to a constitutional coagulation factors defect is an umbrella term used to describe a group of inherited bleeding conditions caused by problems with one or more of the proteins (called coagulation factors) that help blood clot properly. These conditions are sometimes called 'rare bleeding disorders' or 'rare coagulation factor deficiencies.' Unlike the more well-known hemophilia A and B, these disorders involve deficiencies in other clotting factors such as factor I (fibrinogen), factor II (prothrombin), factor V, factor VII, factor X, factor XI, factor XIII, or c

Are there clinical trials for Rare hemorrhagic disorder due to a constitutional coagulation factors defect?

Yes — 1 recruiting clinical trial is currently listed for Rare hemorrhagic disorder due to a constitutional coagulation factors defect on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.