Back to resultsCombination Regimen With Sodium Valproate for Severe Hemophilia B: a Single-arm, Phase 1, Pilot Trial.
Primary Purpose
Hemophilia B
Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Sodium valproate extended-release tablets
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia B focused on measuring Hemophilia B, Sodium valproate, Sirolimus, Calcitriol
Eligibility Criteria
Inclusion Criteria: Patients with clinically confirmed severe haemophilia B; Expected survival of ≥ 24 weeks with an ECOG score of 0-2; Not having participated in another clinical trial within four weeks; Informed consent signed by the patient or an immediate family member. Exclusion Criteria: Those with other types of blood disorders diagnosed at the morphological or molecular level of the bone marrow; Significantly abnormal cardiopulmonary function; Hepatic or renal insufficiency; Pregnancy or lactation, or inability to use contraception during the trial and for three months before the test and one year after administration Persons who are allergic to the drugs likely to be used or where there is a contraindication to their use; Those with severe uncontrollable infectious diseases or uncontrolled hypertension, malignancy, etc.; Inability to cooperate with a regular follow-up due to psychological, social, family and other geographical circumstances; Any other condition that, in the investigator's opinion, makes participation in this trial inappropriate.
Sites / Locations
- PLA General HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Patients with severe haemophilia B
Arm Description
confirmed as hemophilia B and FIX activity <1%
Outcomes
Primary Outcome Measures
FIX Activity
FIX activity in peripheral blood
FIX inhibitor concentration
FIX inhibitor concentration in peripheral blood
Secondary Outcome Measures
frequency of joint bleeding
Record the number of joint bleeds each month
Activated Partial Thromboplastin Time
activated partial thromboplastin time in peripheral blood
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05920512
Brief Title
Combination Regimen With Sodium Valproate for Severe Hemophilia B: a Single-arm, Phase 1, Pilot Trial.
Official Title
PLA General Hospital
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 1, 2022 (Actual)
Primary Completion Date
April 1, 2025 (Anticipated)
Study Completion Date
April 1, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Xue-chun Lu
4. Oversight
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
Data Monitoring Committee
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia B
Keywords
Hemophilia B, Sodium valproate, Sirolimus, Calcitriol
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Patients with severe haemophilia B
Arm Type
Experimental
Arm Description
confirmed as hemophilia B and FIX activity <1%
Intervention Type
Drug
Intervention Name(s)
Sodium valproate extended-release tablets
Other Intervention Name(s)
sirolimus tablets, calcitriol capsules
Intervention Description
Sodium valproate extended-release tablets 0.5g/day; sirolimus tablets 1mg/day and calcitriol capsules 0.25μg/day.
Primary Outcome Measure Information:
Title
FIX Activity
Description
FIX activity in peripheral blood
Time Frame
through study completion, an average of 1 month
Title
FIX inhibitor concentration
Description
FIX inhibitor concentration in peripheral blood
Time Frame
through study completion, an average of 1 month
Secondary Outcome Measure Information:
Title
frequency of joint bleeding
Description
Record the number of joint bleeds each month
Time Frame
through study completion, an average of 1 month
Title
Activated Partial Thromboplastin Time
Description
activated partial thromboplastin time in peripheral blood
Time Frame
through study completion, an average of 1 month
10. Eligibility
Sex
All
Minimum Age & Unit of Time
14 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with clinically confirmed severe haemophilia B;
Expected survival of ≥ 24 weeks with an ECOG score of 0-2;
Not having participated in another clinical trial within four weeks;
Informed consent signed by the patient or an immediate family member.
Exclusion Criteria:
Those with other types of blood disorders diagnosed at the morphological or molecular level of the bone marrow;
Significantly abnormal cardiopulmonary function;
Hepatic or renal insufficiency;
Pregnancy or lactation, or inability to use contraception during the trial and for three months before the test and one year after administration
Persons who are allergic to the drugs likely to be used or where there is a contraindication to their use;
Those with severe uncontrollable infectious diseases or uncontrolled hypertension, malignancy, etc.;
Inability to cooperate with a regular follow-up due to psychological, social, family and other geographical circumstances;
Any other condition that, in the investigator's opinion, makes participation in this trial inappropriate.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xuechun Lu, M.D.
Phone
13241892863
Email
luxuechun@126.com
First Name & Middle Initial & Last Name or Official Title & Degree
Jundong ZHANG
Phone
15536032300
Email
drzjd123@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xuechun Lu, M.D.
Organizational Affiliation
Department of Hematology, the Second Medical Center of PLA General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
PLA General Hospital
City
Beijing
ZIP/Postal Code
100853
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jundong ZHANG
Phone
15536032300
Email
drzjd123@163.com
12. IPD Sharing Statement
Plan to Share IPD
No
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Combination Regimen With Sodium Valproate for Severe Hemophilia B: a Single-arm, Phase 1, Pilot Trial.
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