Phase 1 Haemophilia Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You must be willing and able to sign a consent form agreeing to take part in the study.
• You can be any age, including infants, as long as you weigh at least 3 kg (about 6.6 lbs).
• In the investigator's opinion, you are able to follow the study's requirements.
• You have been diagnosed with a severe form of von Willebrand disease (VWD) — a blood clotting disorder — confirmed by past medical records at the study site, based on specific blood test results or a confirmed genetic mutation.
• Alternatively, you have both VWD and a related condition called hemophilia A, confirmed by past medical records and specific blood test results.
• You are willing and able to take the study drug (emicizumab) as a preventive (prophylactic) treatment throughout the study.
• Your doctor has recommended preventive treatment because of how much bleeding you experience.
• If you are already on a preventive treatment for VWD, you may still join — provided it is not emicizumab, it has been more than 18 months since you last took emicizumab (if ever), and you are willing to stop your current preventive treatment.
• If you are someone who has menstrual periods and could become pregnant, you must agree to either abstain from heterosexual intercourse or use a highly reliable form of contraception (less than 1% failure rate per year, such as hormonal contraceptives, an IUD, or surgical sterilisation) throughout the study. Note: methods like the calendar or withdrawal method are not acceptable.

Who may not be able to join:

• Patients or infants who weigh less than 3 kg (about 6.6 lbs).
• People with mild or low VWD who do not meet the specific diagnostic criteria described above.
• People who have another bleeding or clotting disorder in addition to VWD, including clotting problems caused by liver cirrhosis (liver scarring).
• People who are currently taking emicizumab, or who have taken it within the past 18 months.
• People who have been treated for a blood clot condition (such as deep vein thrombosis or pulmonary embolism) in the past 12 months, are currently being treated for one, or currently show signs of a blood clot — with a limited exception for past clots related to a medical catheter that are no longer being treated.
• People with certain other health conditions — such as lupus, inflammatory bowel disease, or antiphospholipid syndrome — that may increase the risk of bleeding or clotting.
• People who, in the investigator's opinion, are at high risk of a serious condition affecting small blood vessels called thrombotic microangiopathy (TMA), including those with a personal or family history of TMA (confirm with trial site).
• People who would refuse a blood transfusion or blood products if medically necessary.
• People with any serious medical condition or significant abnormal lab results that, in the investigator's opinion, would make it unsafe for them to participate.
• People who have recently taken certain other experimental (investigational) drugs — specifically, any experimental drug for a bleeding disorder within a certain number of days before the study starts, or any other experimental drug within 30 days before the study starts (confirm exact timeframes with trial site).
• People who have had a significant allergic reaction to a monoclonal antibody therapy (a type of drug similar to emicizumab) or to any ingredient in the emicizumab injection.
• People who are pregnant, breastfeeding, or planning to become pregnant during the study.
• Women who could become pregnant must have a negative pregnancy blood test within 7 days before the study begins.
• People who, in the investigator's opinion, have had a problem with illegal drug use or heavy alcohol use within the past 12 months.
• People who have had a serious infection requiring antibiotic treatment (by mouth or through a drip) within 30 days before the screening visit.

Important: Always verify eligibility with the trial site directly before applying.