Pharmacokinetic Study of Lipo-PGE1 for Prevention of VOD After HSCT

NCT ID: NCT02338440

Last Updated: 2021-03-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2/PHASE3

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-01-31

Study Completion Date

2021-12-31

Brief Summary

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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

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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.

Conditions

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Veno-occlusive Disease Child

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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lipoprostaglandin E1 treatment arm

* lipoprostaglandin E1 1mcg/kg/day, continuous infusion
* lipoprostaglandin E1 1.5mcg/kg/day, continuous infusion (for patients with elevating total bilirubin, hepatomegaly, right upper quadrant abdominal pain, unexplained weight gain)

Group Type EXPERIMENTAL

lipoprostaglandin E1

Intervention Type DRUG

Blood sampling before administration (baseline), between 1 hour to 72 hours from administration (plateau 1), 72 hours after engraftment (plateau 2), when the clinician determine to do blood sampling because of change of kidney/liver function change (optional 1), 72 hours from the change of lipoprostaglandin E1 concentration change (optional 2)

Interventions

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lipoprostaglandin E1

Blood sampling before administration (baseline), between 1 hour to 72 hours from administration (plateau 1), 72 hours after engraftment (plateau 2), when the clinician determine to do blood sampling because of change of kidney/liver function change (optional 1), 72 hours from the change of lipoprostaglandin E1 concentration change (optional 2)

Intervention Type DRUG

Other Intervention Names

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Alostin

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Seoul National University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Hyoung Jin Kang, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Seoul National University College of Medicine

Locations

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Seoul National University Hospital

Seoul, Chongno-gu, South Korea

Site Status

Countries

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South Korea

Other Identifiers

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SNUCH-HSCT-lipoPGE1

Identifier Type: -

Identifier Source: org_study_id

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