Clinical trial NCT05920512: Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.
Official title: PLA General Hospital
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Hemophilia
Keywords: Hemophilia
Sodium valproate
Sirolimus
Calcitriol
Lead sponsor: Chinese PLA General Hospital
Sponsor class: OTHER
First posted: 2023-06-27
Enrollment: 10
Sex: ALL
Minimum age (years): 14
Maximum age (years): 85
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: FVIII/FIX Activity
timeFrame: through study completion, an average of 1 month
measure: FVIII/ FIX inhibitor concentration
timeFrame: through study completion, an average of 1 month
Secondary outcomes:
measure: frequency of joint bleeding
timeFrame: through study completion, an average of 1 month
measure: Activated Partial Thromboplastin Time
timeFrame: through study completion, an average of 1 month
Other outcomes:
Locations:
city: Beijing
country: China
facility: PLA General Hospital
Study officials:
affiliation: Department of Hematology, the Second Medical Center of PLA General Hospital
name: Xuechun Lu, M.D.
role: PRINCIPAL_INVESTIGATOR
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