Clinical trial NCT02338440: Pharmacokinetic Study of Lipo-PGE1 for Prevention of VOD After HSCT
Official title: Pharmacokinetic Study of Lipo-PGE1 for Prevention of VOD After HSCT
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Veno-occlusive Disease
Child
Lead sponsor: Seoul National University Hospital
Sponsor class: OTHER
First posted: 2015-01-14
Enrollment: 30
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Veno-occlusive disease (VOD) after hematopoietic stem cell transplantation (HSCT) remains the major complication. VOD occurs in 11-31% of pediatric HSCT and the mortality reaches up to 50%.

Prostaglandin E1 (PGE1) have been reported to prevent and relieve the severity of VOD by Gluckman et al.. Lipo-PGE1 is a transporter of PGE1, which is superior in concentrating PGE1 and acts for prolonged time. Because of the prolonged effective time, lipo-PGE1 acts comparable effects by 1/4\~1/8 of PGE1 dose. Empirically, pediatric HSCT centers adopt lipo-PGE1 in dose of 1 mcg/kg/day (0.042 mcg/kg/hr), which is 1/7 of the dose recommended by Gluckman, et al. This prospective study will investigate the concentration of lipo-PGE1 with preventive lipo-PGE1 with empirical dose (1 mcg/kg/day).

Detailed description: Primary Objective: To evaluate the pharmacokinetics of lipo-PGE1. Secondary objective: To evaluate the relation between lipo-PGE1 concentration and VOD occurrence, severity.

Inclusion criteria

1. Patients undergoing HSCT with high risk of VOD who cannot use low dose heparin.

   * high risk of VOD : previous radiotherapy, liver function abnormality, children, conditioning regimen including busulfan or total body irradiation
   * contraindication of heparin : low platelet count, bleeding tendency, allergy
2. Patients (or one of parents if patients age \< 19) should sign informed consent.

Exclusion criteria

1. Patient with heart failure.
2. Patient with bleeding (intracranial hemorrhage, gastrointestinal tract bleeding, hemoptysis).
3. History of hypersensitivity reaction as shock to lipo-PGE1.
4. Psychiatric disorder that would preclude compliance.
5. If the clinician decides that there is a condition improper for the clinical study.

Eligibility criteria: Inclusion Criteria:

1. Patients undergoing HSCT with high risk of VOD who cannot use low dose heparin.

   * high risk of VOD : previous radiotherapy, liver function abnormality, children, conditioning regimen including busulfan or total body irradiation
   * contraindication of heparin : low platelet count, bleeding tendency, allergy
2. Patients (or one of parents if patients age \< 19) should sign informed consent.

Exclusion Criteria:

1. Patient with heart failure.
2. Patient with bleeding (intracranial hemorrhage, gastrointestinal tract bleeding, hemoptysis).
3. History of hypersensitivity reaction as shock to lipo-PGE1.
4. Psychiatric disorder that would preclude compliance.
5. If the clinician decides that there is a condition improper for the clinical study.

Primary outcomes:
measure: pharmacokinetics of lipo-PGE1 (Lipo-PGE1 level (B, P1, P2, O1, O2))
timeFrame: baseline (pre-dose), 1 to 72 hours from starting continuous infusion of lipo-PGE1
Secondary outcomes:
measure: Incidence of VOD after transplantation
timeFrame: until 3 months after transplantation
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
state: Chongno-gu
Study officials:
affiliation: Seoul National University College of Medicine
name: Hyoung Jin Kang, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: