Hemophilia
Conditions
Keywords
Hemophilia, Sodium valproate, Sirolimus, Calcitriol
Brief summary
The goal of this clinical trial is to determine the clinical efficacy and toxic effects of sodium valproate, sirolimus and calcitriol in the treatment of severe haemophilia in participants with severe haemophilia . The main questions it aims to answer are the possibility of adding a combination regimen to primary treatment for severe haemophilia . Patients will receive oral sodium valproate extended-release tablets 0.5g/day, sirolimus tablets 1mg/day and osteopontin capsules 0.25μg/day.
Interventions
DRUGSodium valproate extended-release tablets
Sodium valproate extended-release tablets 0.5g/day; sirolimus tablets 1mg/day and calcitriol capsules 0.25μg/day.
Sponsors
Xue-chun Lu
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
14 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
1. Patients with clinically confirmed severe haemophilia; 2. Expected survival of ≥ 24 weeks with an ECOG score of 0-2; 3. Not having participated in another clinical trial within four weeks; 4. Informed consent signed by the patient or an immediate family member.
Exclusion criteria
1. Those with other types of blood disorders diagnosed at the morphological or molecular level of the bone marrow; 2. Significantly abnormal cardiopulmonary function; 3. Hepatic or renal insufficiency; 4. Pregnancy or lactation, or inability to use contraception during the trial and for three months before the test and one year after administration 5. Persons who are allergic to the drugs likely to be used or where there is a contraindication to their use; 6. Those with severe uncontrollable infectious diseases or uncontrolled hypertension, malignancy, etc.; 7. Inability to cooperate with a regular follow-up due to psychological, social, family and other geographical circumstances; 8. Any other condition that, in the investigator's opinion, makes participation in this trial inappropriate.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| FVIII/FIX Activity | through study completion, an average of 1 month | FVIII/FIX activity in peripheral blood |
| FVIII/ FIX inhibitor concentration | through study completion, an average of 1 month | FVIII/ FIX inhibitor concentration in peripheral blood |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| frequency of joint bleeding | through study completion, an average of 1 month | Record the number of joint bleeds each month |
| Activated Partial Thromboplastin Time | through study completion, an average of 1 month | activated partial thromboplastin time in peripheral blood |
Countries
China
Contacts
Primary ContactXuechun Lu, M.D.
Backup ContactJundong ZHANG
Outcome results
None listed