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Sandostatine® LP and Hyperinsulinism

Primary Purpose

Congenital Hyperinsulinism

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Sandostatine LP
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Hyperinsulinism focused on measuring Congenital Hyperinsulinism, Hypoglycemia,, Sandostatine subcutaneous in 3 daily injections, Intramuscular injection of Sandostatine® LP per month

Eligibility Criteria

6 Months - 16 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • congenital hyperinsulinism patients
  • age of patients : 6 months to 16 years
  • normoglycemia under sandostatine subcutaneous
  • contraception efficiency
  • signed informed consent

Exclusion Criteria:

  • refusal from parents
  • vesicular lithiasis
  • absence of social security
  • hypersensitivity to octreotide or excipients
  • pregnancy or nursing mother

Sites / Locations

  • Necker Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sandostatine LP

Arm Description

Outcomes

Primary Outcome Measures

Glycemia > or equal to 3 mmol/L, before and after 4 meals +Midnight + 4 am

Secondary Outcome Measures

Abdominal ultra echography, before and after 6 month treatment
Life quality

Full Information

First Posted
September 29, 2009
Last Updated
December 18, 2013
Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Novartis
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1. Study Identification

Unique Protocol Identification Number
NCT00987168
Brief Title
Sandostatine® LP and Hyperinsulinism
Official Title
Replace Sandostatine® in Three Daily Subcutaneous Injections by a Single Intramuscular Injection of Sandostatine® LP Per Month in Patients With a Diffuse Form of Hyperinsulinism
Study Type
Interventional

2. Study Status

Record Verification Date
October 2010
Overall Recruitment Status
Completed
Study Start Date
May 2009 (undefined)
Primary Completion Date
April 2011 (Actual)
Study Completion Date
June 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
Novartis

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
To replace Sandostatine® in three daily subcutaneous injections by a single intramuscular injection of Sandostatine® LP per month in patients with a diffuse form of hyperinsulinism.
Detailed Description
Persistent hyperinsulinemic hypoglycemias of infancy (HI) are characterized by an inappropriate secretion of insulin responsible for profound hypoglycemias which require aggressive treatment to prevent severe and irreversible brain damage. Thanks to the complementarity and to the synergy between paediatricians, paediatric surgeons, radiologists, pathologists and geneticists, an important stage was reached: the recognition of two clinically similar forms of HI but requiring a radically different treatment: a diffuse form and a focal form in the pancreas. The medical treatment is based on proglycem, or diazoxide, then octreotide (Sandostatine ®, Novartis) with a dose of 10 to 50 µg/Kg/jour divided to three subcutaneous injections. Most neonates are resistant to diazoxide and side effects are observed (important edema and hypertrichosis). The Sandostatine® is a much more effective treatment, unfortunately with a short half-life and painful injections. In the cases of resistance to the medical treatment, the distinction of the two forms is essential to guide the surgical treatment : partial pancreatectomy in the focal forms, curing definitively hypoglycemia; subtotal pancreatectomy in the diffuse forms resisting to the medical treatment, leading to a diabetes and a pancreatic exocrine insufficiency. Also, the medical treatment is essential in the case of the diffuse forms to avoid a subtotal pancreatectomy. Mutations in two genes encoding the potassium channels, SUR1 and KIR6.2, are responsible for hyperinsulinism resistant to diazoxide. The Sandostatine® marketed by Novartis exists in two forms, a "rapid" form and a "retarded liberation form". These two molecules have been approved in the treatment of adults in the following indications: Treatment of the clinical symptoms of digestive endocrine tumours Treatment of acromegaly Treatment of primitive thyrotrope adenomata Treatment of unfunctional adenoma Treatment of corticotrope adenoma during (Nelson syndrome) and of functional gonadotrope adenomata After pancreatic surgery Emergency treatment of bleeding secondary to cirrhosis. Sandostatine® has neither approval for hyperinsulinism, nor in children even though many international publications reported efficacy of treatment by Sandostatine® in hyperinsulinemic children since 1983. Also, by consensus most international teams taking care of hyperinsulinism in infancy propose this treatment to their patients. Ten children who have a diffuse form of hyperinsulinism have been treated in our department by Sandostatine® given in three subcutaneous injections for several years, in order to avoid a sub-total pancreatectomy. The only possible adverse effect is the appearance of vesicular lithiasis which can be treated by ursodesoxycholic acid . We changed the Sandostatine® treatment of one of our patients by the Sandostatine® LP (retarded liberation form) after written consent of his two parents. Thus we could stop the three injections per day of Sandostatine®. Sandostatine® LP proved to be as efficient on hypoglycemias as the subcutaneous multi-daily injections (SC). The glycemia values were strictly normal, and no hypoglycaemia was observed. Following this observation, we propose to try to substitute the treatment of Sandostatine® given in several subcutaneous injections by one injection of Sandostatine® LP in 10 children followed in the department of Metabolism for hyperinsulinism. The awaited result of this study is to demonstrate efficacy of Sandostatine® LP and thus replace Sandostatine® in three daily subcutaneous injections by a single intramuscular injection of Sandostatine® LP per month. This study will contribute to an undeniable improvement of the quality of life for the patients and their families.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Hyperinsulinism
Keywords
Congenital Hyperinsulinism, Hypoglycemia,, Sandostatine subcutaneous in 3 daily injections, Intramuscular injection of Sandostatine® LP per month

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Sandostatine LP
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Sandostatine LP
Intervention Description
Intramuscular injection of Sandostatine LP, once per month Dosage : 10 mg, 20 mg, 30 mg
Primary Outcome Measure Information:
Title
Glycemia > or equal to 3 mmol/L, before and after 4 meals +Midnight + 4 am
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Abdominal ultra echography, before and after 6 month treatment
Time Frame
6 months
Title
Life quality
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: congenital hyperinsulinism patients age of patients : 6 months to 16 years normoglycemia under sandostatine subcutaneous contraception efficiency signed informed consent Exclusion Criteria: refusal from parents vesicular lithiasis absence of social security hypersensitivity to octreotide or excipients pregnancy or nursing mother
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pascale De Lonlay, PUPH
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Necker Hospital
City
Paris
ZIP/Postal Code
75015
Country
France

12. IPD Sharing Statement

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Sandostatine® LP and Hyperinsulinism

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