Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.

Phase 1

Recruiting

• Conditions: Hemophilia
• Interventions: Drug: Sodium valproate extended-release tablets

• Registration Number: NCT05920512
• Lead Sponsor: Xue-chun Lu

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-04-01
• Study First Submitted: 2023-06-06
• Study First Submitted QC: 2023-06-17
• Study First Posted: 2023-06-27
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-28
• Last Update Posted: 2024-02-29
• Primary Completion: 2025-04-01
• Study Completion: 2025-04-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patients with severe haemophilia	Sodium valproate extended-release tablets	confirmed as hemophilia and FVIII/FIX activity \<1%

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Activated Partial Thromboplastin Time	through study completion, an average of 1 month	activated partial thromboplastin time in peripheral blood
frequency of joint bleeding	through study completion, an average of 1 month	Record the number of joint bleeds each month

Trial Locations

Locations (1)

PLA General Hospital; 🇨🇳 Beijing, China