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

February 28, 2024 updated by: Xue-chun Lu

PLA General Hospital

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
      • Beijing, China, 100853
        • Recruiting
        • PLA General Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Patients with severe haemophilia
confirmed as hemophilia and FVIII/FIX activity <1%
Drug: Sodium valproate extended-release tablets
Sodium valproate extended-release tablets 0.5g/day; sirolimus tablets 1mg/day and calcitriol capsules 0.25μg/day.
Other Names:
  • sirolimus tablets
  • calcitriol capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
FVIII/FIX Activity
Time Frame: through study completion, an average of 1 month
FVIII/FIX activity in peripheral blood
through study completion, an average of 1 month
FVIII/ FIX inhibitor concentration
Time Frame: through study completion, an average of 1 month
FVIII/ FIX inhibitor concentration in peripheral blood
through study completion, an average of 1 month

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Xue-chun Lu

Investigators

  • Principal Investigator: Xuechun Lu, M.D., Department of Hematology, the Second Medical Center of PLA General Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2022

Primary Completion (Estimated)

April 1, 2025

Study Completion (Estimated)

April 1, 2025

Study Registration Dates

First Submitted

June 6, 2023

First Submitted That Met QC Criteria

June 17, 2023

First Posted (Actual)

June 27, 2023

Study Record Updates

Last Update Posted (Estimated)

February 29, 2024

Last Update Submitted That Met QC Criteria

February 28, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • S2023-261-02

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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