Compassionate Use of SOM230 for Hyperinsulinemic/Hypoglycemia

NCT02835131 | Unknown

• 1 other identifier: 2015-5564
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Congenital hyperinsulinism is a rare condition that can cause life-threatening hypoglycemia. Current treatment for congenital hyperinsulinism is often suboptimal, and such individuals may respond to a new somatostatin analog, pasireotide. This is a compassionate use study of the effects of pasireotide on individuals with suboptimally treated congenital hyperinsulinism.

Conditions

Congenital Hyperinsulinism

Keywords

hyperinsulinemia; hypoglycemia; somatostatin; pasireotide; congenital

Interventions

Pasireotide DRUG

Also known as: Signifor, SOM230

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients aged 18 years or older
• Patients with a confirmed diagnosis of hyperinsulinemic hypoglycemia, if possible by genetic testing
• Patients not controlled by medical therapies (e.g. diazoxide or octreotide) and/or pancreatic surgery or patients not eligible for surgery
• World Health Organization/ Eastern Cooperative Oncology Group Performance Status of 0-2.
• Life expectancy ≥12 weeks
• Adequate end organ function as defined by:
• No evidence of significant liver disease:
• Serum total bilirubin ≤1.5 x upper limit of normal (ULN)
• International Normalized Ratio (INR) \< 1.3
• Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≤ 2 x ULN,
• Alkaline phosphatase ≤ 2.5 x ULN
• Written informed consent obtained prior to treatment to be consistent with local regulatory requirements
• Is suffering from a serious or life-threatening disease or condition
• Does not have access to a comparable or satisfactory alternative treatment (i.e., comparable or satisfactory treatment is not available or does not exist)
• Is not eligible for participation in any of the investigators ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (for example., trial extensions, amendments, etc.), the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient

You may not qualify if:

• Patients with a known hypersensitivity to somatostatin analogs or any component of the pasireotide long acting release (LAR) or subcutaneous. formulations.
• Patients with abnormal coagulation (prothrombin time or activated partial thromboplastin time elevated by 30% above normal limits).
• Patients currently using warfarin / warfarin derivatives
• Patients with symptomatic cholelithiasis.
• Patients who are not biochemically euthyroid. Patients with known history of hypothyroidism are eligible if they are on adequate and stable replacement thyroid hormone therapy for at least 3 months.
• corrected QT interval (QTcF) at screening \> 450 msec in males and QTcF \> 460 msec
• History of syncope or family history of idiopathic sudden death
• Sustained or clinically significant cardiac arrhythmias
• Risk factors for Torsades de Pointes such as hypokalemia, hypomagnesemia, cardiac failure, clinically significant/symptomatic bradycardia, or high-grade atrioventricular block
• Concomitant disease(s) that could prolong QT such as autonomic neuropathy (caused by diabetes, or Parkinson's disease), human immunodeficiency virus (HIV) infection, cirrhosis, uncontrolled hypothyroidism or cardiac failure
• Family history of long QT syndrome
• Concomitant medications known to prolong the QT interval.
• Potassium \< or = 3.5 mmol/L
• Patients who have any severe and/or uncontrolled medical conditions :
• Uncontrolled diabetes as defined by hemoglobinA1c \> 8%,

Sponsors & Collaborators

• Montefiore Medical Center: lead

Study Sites (1)

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

MeSH Terms

Conditions

Congenital Hyperinsulinism; Hyperinsulinism; Hypoglycemia

Interventions

pasireotide

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Vafa Tabatabaie

  Montefiore Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Asst Prof. Department of Medicine (Endocrinology)

Study Record Dates

• First Submitted: July 13, 2016
• First Posted: July 15, 2016
• Last Updated: July 29, 2022

Record last verified: 2022-07

Locations

US (1)