Is NCT05500807 recruiting in Kansas City, MO?

Yes, NCT05500807 is currently recruiting participants at its Kansas City, MO location. This von willebrand disease, type 3 clinical trial is actively enrolling qualified participants.

How do I join the von willebrand disease, type 3 clinical trial in Kansas City?

To join this clinical trial in Kansas City, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Kansas City clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Von Willebrand Disease, Type 3 Clinical Trial in Kansas City, MO

About This Von Willebrand Disease, Type 3 Study in Kansas City

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.

Sponsor: Bleeding and Clotting Disorders Institute Peoria, Illinois

Who Can Participate

Inclusion Criteria

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.

diagnosis of VWD/hemophilia A defined as VWF:ag, activity or CB \<50 U/dl, and mild moderate or severe hemophilia A(defined by ISTH criteria) based on historical medical records of the study site.

plan to be adherent to emicizumab prophylaxis during the study

Patient's bleeding phenotype necessitating prophylaxis per treating provider recommendations.

Patient on current prophylaxis for VWD or VWD/hemophilia A may enroll if they are currently on a non-emicizumab agent, and if it has been \> 18 months since last off-label dose of emicizumab, and are willing to discontinue current prophylaxis.

For menstruating individuals: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of \< 1% per year during the study period. A menstruating individual is considered to be of childbearing potential if they are post-menarchal, have not reached a postmenopausal state (12 continuous months of amenorrhea with no identified cause other than menopause), and have not undergone surgical sterilization (removal of ovaries and/or uterus). Examples of highly effective contraceptive methods with a failure rate of \< 1% per year include proper use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone-releasing intrauterine devices, and copper intrauterine devices. The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post-ovulation methods) and withdrawal are not acceptable methods of contraception.

Exclusion Criteria

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.

Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or current signs of thromboembolic disease

Other conditions (e.g., certain autoimmune diseases, including, but not limited to diseases such as systemic lupus erythematosus, inflammatory bowel disease, and antiphospholipid syndrome) that may increase the risk of bleeding or thrombosis

Patients who are at high risk for thrombotic microangiopathy (TMA; e.g., have a previous medical or family history of TMA), in the investigator's judgment

Would refuse treatment with blood or blood products, if necessary.

Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study

Treatment with any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Study Day 1 A non-hemophilia-related investigational drug within the last 30 days or 5 halflives- before Study Day 1, whichever is longer An investigational drug concurrently

History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection

Pregnant or lactating, or intending to become pregnant during the study

Women of childbearing potential must have a negative serum pregnancy test result within 7 days before Study Day 1

Illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment

Serious infection requiring oral or IV antibiotics within 30 days prior to screening

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Kansas City?

Yes, this clinical trial (NCT05500807) has an active research site in Kansas City, MO that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Von Willebrand Disease, Type 3 Treatment Options in Kansas City, MO

If you're searching for von willebrand disease, type 3 treatment options in Kansas City, MO, this clinical trial (NCT05500807) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Kansas City research site is actively enrolling participants for this clinical trial. You'll receive care from experienced von willebrand disease, type 3 specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all von willebrand disease, type 3 clinical trials near you to find additional studies recruiting in your area.

Von Willebrand Disease, Type 3 Clinical Trial in Kansas City, MO

Expert Care in Kansas City

IRB Approved

No-Cost Care

Apply for This Kansas City Location

Why Participate?

Simple Process