Treatment Plan for an Individual Patient With Pasireotide for Hyperinsulinemic Hypoglycemia

NCT03103009 | Completed Phase 1 FDA Drug

• 1 other identifier: 16-0393
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

Somatostatin analogues are a last resort for medical intervention in hyperinsulinemic hypoglycemia (HH). The hypoglycemia is very debilitating and can be even life threatening. There is limited experience with pasireotide in hyperinsulinemic hypoglycemia (only one publication); there is more experience with octreotide, both in adults and children successful interventions with octreotide in hyperinsulinemic hypoglycemia have been published. Pasireotide via its different somatostatin receptor binding profile has clear effects on insulin, glucagon and incretin secretion and can ultimately lead to hyperglycemia. This mode of action (especially the effects on insulin and incretin secretion) could be very useful in the setting of hyperinsulinemic hypoglycemia.

Conditions

Hyperinsulinemic Hypoglycemia; Post Gastrointestinal Tract Surgery Hypoglycaemia

Outcome Measures

Primary Outcomes (1)

• Recording of Hypoglycemic events

  The primary outcome is the number of hypoglycemic events occurring.

  Up to 2 years

Study Arms (1)

pasireotide

EXPERIMENTAL

Interventions - One patient will be given a drug, pasireotide (Signifor), on a compassionate use basis for treatment of post-gastric bypass hypoglycemia. The minimal dose will be 0.3 mg subcutaneous daily and the maximal dose will be 0.6 mg subcutaneous twice daily. The patient may continue treatment with pasireotide indefinitely unless the patient experiences unacceptable toxicity, disease progression and/or treatment is discontinued at the discretion of the treating physician or Novartis or withdrawal of consent.

Drug: Pasireotide

Interventions

Pasireotide DRUG

Somatostatin analogues are a last resort for medical intervention in hyperinsulinemic hypoglycemia (HH). The hypoglycemia is very debilitating and can be even life threatening. There is limited experience with pasireotide in hyperinsulinemic hypoglycemia (only one publication); there is more experience with octreotide, both in adults and children and successful interventions with octreotide in hyperinsulinemic hypoglycemia have been published. Pasireotide via its different somatostatin receptor binding profile has clear effects on insulin, glucagon and incretin secretion and can ultimately lead to hyperglycemia. This mode of action (especially the effects on insulin and incretin secretion) could be very useful in the setting of hyperinsulinemic hypoglycemia.

Also known as: Signifor

pasireotide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• 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
• WHO/ ECOG 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 ULN
• INR \< 1.3
• ALT and 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 IMP's ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (e.g., 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 eligible for this Treatment Plan must not meet any of the following criteria:
• Patients with a known hypersensitivity to somatostatin analogs or any component of the pasireotide LAR or s.c. formulations.
• Patients with abnormal coagulation (PT or aPTT 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.
• 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 AV block
• Concomitant disease(s) that could prolong QT such as autonomic neuropathy (caused by diabetes, or Parkinson's disease), HIV, 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 :

Sponsors & Collaborators

• University of Colorado, Denver: lead
• Novartis: collaborator

Study Sites (1)

University of Colorado Denver

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Congenital Hyperinsulinism

Interventions

pasireotide

Condition Hierarchy (Ancestors)

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

Study Officials

• Helen Lawler, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

• Mike McDermott, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Treatment Plan for an individual patient with pasireotide for Hyperinsulinemic Hypoglycemia (Compassionate Use Protocol for Pasireotide)

Study Record Dates

• First Submitted: March 31, 2017
• First Posted: April 6, 2017
• Study Start: March 22, 2017
• Primary Completion: December 22, 2017
• Study Completion: December 22, 2017
• Last Updated: September 29, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will share

Locations

US (1)