Clincosm LogoSign in Get a demo

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

Sponsor
Xue-chun Lu (Other)
Overall Status
Recruiting
CT.gov ID
NCT05920512
Collaborator
(none)
10
Enrollment
1
Location
1
Arm
36
Anticipated Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

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 B in participants with severe haemophilia B. The main questions it aims to answer are the possibility of adding a combination regimen to primary treatment for severe haemophilia B. Patients will receive oral sodium valproate extended-release tablets 0.5g/day, sirolimus tablets 1mg/day and osteopontin capsules 0.25μg/day.

Condition or Disease Intervention/Treatment Phase
  • Drug: Sodium valproate extended-release tablets
 Phase 1/Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
10 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
PLA General Hospital
Actual Study Start Date :
Apr 1, 2022
Anticipated Primary Completion Date :
Apr 1, 2025
Anticipated Study Completion Date :
Apr 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Patients with severe haemophilia B

confirmed as hemophilia B and 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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. FIX Activity [through study completion, an average of 1 month]

      FIX activity in peripheral blood

    2. FIX inhibitor concentration [through study completion, an average of 1 month]

      FIX inhibitor concentration in peripheral blood

    Secondary Outcome Measures

    1. frequency of joint bleeding [through study completion, an average of 1 month]

      Record the number of joint bleeds each month

    2. Activated Partial Thromboplastin Time [through study completion, an average of 1 month]

      activated partial thromboplastin time in peripheral blood

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    14 Years to 85 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Patients with clinically confirmed severe haemophilia B;

    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 PLA General Hospital Beijing China 100853

    Sponsors and Collaborators

    • Xue-chun Lu

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    Xue-chun Lu, Director of Hematology Department of the Second Medical Center of PLA General Hospital, Chinese PLA General Hospital
    ClinicalTrials.gov Identifier:
    NCT05920512
    Other Study ID Numbers:
    • S2023-261-02
    First Posted:
    Jun 27, 2023
    Last Update Posted:
    Jun 27, 2023
    Last Verified:
    Jun 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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
    Keywords provided by Xue-chun Lu, Director of Hematology Department of the Second Medical Center of PLA General Hospital, Chinese PLA General Hospital
    Hemophilia B
    Sodium valproate
    Sirolimus
    Calcitriol
    Additional relevant MeSH terms:
    Hemophilia A
    Hemophilia B
    Calcitriol
    Sirolimus
    Valproic Acid

    Study Results

    No Results Posted as of Jun 27, 2023