Combination treatment for severe hemophilia using sodium valproate, sirolimus, and calcitriol
PLA General Hospital
This study is testing if a new combination of three medications can improve treatment for people with severe hemophilia.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 14 Years to 85 Years |
| Sex | All |
| Sponsor | Chinese PLA General Hospital Academic / other |
| Locations | 1 site (Beijing) |
| Trial ID | NCT05920512 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the effectiveness and safety of a combination regimen of sodium valproate, sirolimus, and calcitriol in patients with severe hemophilia. Participants will receive oral sodium valproate extended-release tablets at a dose of 0.5g per day, along with sirolimus tablets and osteopontin capsules. The study seeks to determine if this combination can enhance the primary treatment for severe hemophilia. It is a single-arm, Phase 1 pilot trial, focusing on both clinical efficacy and potential toxic effects.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with clinically confirmed severe hemophilia who are expected to survive for at least 24 weeks.
Not a fit: Patients with other blood disorders, significant organ dysfunction, or those who are pregnant or lactating may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with severe hemophilia, potentially improving their quality of life.
How similar studies have performed: While this approach is novel, similar studies exploring combination therapies for hemophilia have shown promise in enhancing treatment outcomes.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Beijing
- PLA General Hospital — Beijing, China (Recruiting)
Study contacts
- Principal investigator: Xuechun Lu, M.D. — Department of Hematology, the Second Medical Center of PLA General Hospital
- Study coordinator: Xuechun Lu, M.D.
- Email: luxuechun@126.com
- Phone: 13241892863
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.