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

NCT02338440 | Unknown Phase 2 DMC

• 1 other identifier: SNUCH-HSCT-lipoPGE1
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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).

Conditions

Veno-occlusive Disease; Child

Outcome Measures

Primary Outcomes (1)

• pharmacokinetics of lipo-PGE1 (Lipo-PGE1 level (B, P1, P2, O1, O2))

  Lipo-PGE1 drug level of time points (B, P1, P2, O1, O2)

  baseline (pre-dose), 1 to 72 hours from starting continuous infusion of lipo-PGE1

Secondary Outcomes (1)

• Incidence of VOD after transplantation

  until 3 months after transplantation

Study Arms (1)

lipoprostaglandin E1 treatment arm

EXPERIMENTAL

* 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)

Drug: lipoprostaglandin E1

Interventions

lipoprostaglandin E1 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)

Also known as: Alostin

lipoprostaglandin E1 treatment arm

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Patients (or one of parents if patients age \< 19) should sign informed consent.

You may not qualify if:

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

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Hospital

Seoul, Chongno-gu, South Korea

Location

Study Officials

• Hyoung Jin Kang, MD, PhD

  Seoul National University College of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 30, 2014
• First Posted: January 14, 2015
• Study Start: January 1, 2015
• Primary Completion: December 1, 2019
• Study Completion: December 1, 2021
• Last Updated: March 30, 2021

Record last verified: 2021-03

Locations

KR (1)