" An Open Label Pilot Study Evaluating Safety and Evidence of Therapeutic Effect of IV Admin of 2-0, 3-0 Desulfated Heparin, Treatment of Exacerbation of Protein Losing Enteropathy (PLE) Associated With Single Ventricle Palliative Surgery"

NCT01161641 Phase 1 TERMINATED

Protein Losing Enteropathy (PLE) is a serious medical condition that may develop in children and adults with congenital heart disease for which a palliative procedure known as the "Fontan procedure" has been performed. The loss of serum proteins into the gastrointestinal tract that is associated with PLE can cause serious symptoms and life-threatening complications. A number of clinical studies have suggested that heparin administration can have clinical benefit in children with PLE, however the risk of bleeding associated with the administration of heparin is an important concern and commonly limits its administration. ODSH is a desulfated heparin with minimal anticoagulation properties but which, in pre-clinical studies, appears to have the potential to replace heparin and greatly reduce the risk of bleeding. This open label study is to assess the safety and evidence of therapeutic effect of the administration of ODSH as a 4-day continuous intravenous infusion in patients with an exacerbation of their PLE.

Details

Lead sponsor	Jazz Pharmaceuticals
Phase	Phase 1
Status	TERMINATED
Enrolment	5
Start date	2010-07
Completion	2012-02

Conditions

• Protein Losing Enteropathy

Interventions

• ODSH at 0.125 mg/kg/h
• ODSH at 0.375 mg/kg/h
• ODSH at 0.250 mg/kg/h

Primary outcomes

• Clinical improvement of PLE symptoms and signs. — Day 4 after 96 hours of IV continuous infusion of ODSH
  Clinical Improvement of signs \& symptoms of PLE such as diarrhea, abdominal pain, peripheral edema, and ascites. Visual/categorical scales will be used by the study subjects and the investigators to assess diarrhea/abdominal pain intensity as well as The Global Impression of Improvement. The investigator will assess peripheral edema and ascites.
• Fecal alpha 1 antitrypsin (FA1AT) — Day 4 after 96 hours of ODSH IV continuous infusion
  Decrease of Fecal alpha 1 antitrypsin level at Day 4 compared to baseline.
• Serum albumin levels. — Day 4 after 96 hours of continuous IV infusion of ODSH
  Serum albumin levels at Day 4 compared to Baseline. A serum albumin level of 3 or more mg/dL or at least an increase of 25% from baseline at Day 4 or the reduction in the need for additional albumin infusions will be considered as clinically significant.
• ODSH safety — Day 1, 2, 3 and 4
  aPTT as a measure of coagulation effect from ODSH will be measured every day. If the aPTT value is 8 or more seconds higher than the upper limit of normal value of aPTT for the study site then the ODSH infusion rate will be decreased as recommended in the protocol. Coagulation and bleeding abnormalities will be monitored closely by the investigator. Liver enzymes will also be monitored during the study.

Countries

United States