hemophilia B
Conditions
Sponsors
Dongguan Taixin Hospital
Eligibility
Sex/Gender
All
Age
7 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Aged >=7 years old, able to understand, agree and sign informed consent form or parent / legal guardian must be able to understand, agree and sign informed consent form; 2. The disease is stable and the expected survival time of the subject is> 6 months; 3. Subjects must have participated in treatment in a professional institution and have been followed up for more than two years, with a comprehensive evaluation of the disease including a mental assessment; detailed medical records including data from at least the past two years for self-control; 4. No treatment with hydroxyurea, 5-azacytidine, cytarabine, etc. within three months before enrollment.
Exclusion criteria
Exclusion criteria: 1. HIV seropositive, hepatitis B surface antigen positive or hepatitis B core antibody positive with HBV DNA positive, hepatitis C virus antibody positive, and syphilis antibody positive; 2. Contraindications for anesthesia and bone marrow; 3. Severe bacterial, fungal, mycoplasma, virus and other infections; 4. history of malignant tumors; 5. Presence or suspected hereditary familial tumor syndrome (including but not limited to hereditary breast and ovarian cancer syndromes, hereditary nonpolyposis colorectal cancer syndromes, familial adenoma polyposis, etc.) in immediate family members; 6. History of mental illness, mental disorder or recent drug or alcohol use; 7. Complex autoimmune diseases may cause difficulty in transfusion; 8. Subjects who are pregnant or breast-feeding or are unable to perform contraception; 9. People with severe organ damage include the following: (1) Severe cerebrovascular disease or cognitive psychological sequelae, including hemiplegia; (2) Subjects with severe liver disease, glutamine aminotransferase (ALT)> 3 x upper limit of normal, or subjects with cirrhosis and liver fibrosis; (3) Heart disease with ejection fraction <25% or cardiac MRI T2 * test <10ms; (4) Severe kidney disease and creatinine clearance <30% of normal value; (5) Pulmonary diseases include pulmonary fibrosis, pulmonary hypertension, or significant decline in lung function (eg, partial oxygen pressure <90mmHg and / or CO diffusion <50% of expected value); (6) Endocrine disorders include insulin-dependent diabetes mellitus, hyperthyroidism or insufficiency, and hyperparathyroidism or insufficiency; 10. Participate in another clinical study within 30 days before the screening period; 11. Bone marrow test found chromosomal abnormalities.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse events / adverse events;Clinical manifestations of bleeding and factor IX activity level; | — |
Countries
China
Contacts
Public ContactChunfu Li ?Jianyun Liao
Dongguan Taixin Hospital
Outcome results
None listed