Sandostatine® LP and Hyperinsulinism

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00987168
Recruitment Status : Completed
First Posted : September 30, 2009
Last Update Posted : December 19, 2013
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

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.

Condition or disease Intervention/treatment Phase
Congenital Hyperinsulinism Drug: Sandostatine LP Phase 2

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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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : May 2009
Actual Primary Completion Date : April 2011
Actual Study Completion Date : June 2011

Arm Intervention/treatment
Experimental: Sandostatine LP Drug: Sandostatine LP
Intramuscular injection of Sandostatine LP, once per month Dosage : 10 mg, 20 mg, 30 mg

Primary Outcome Measures :
  1. Glycemia > or equal to 3 mmol/L, before and after 4 meals +Midnight + 4 am [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Abdominal ultra echography, before and after 6 month treatment [ Time Frame: 6 months ]
  2. Life quality [ Time Frame: 6 months ]

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Ages Eligible for Study:   6 Months to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00987168

Necker Hospital
Paris, France, 75015
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Principal Investigator: Pascale De Lonlay, PUPH Assistance Publique - Hôpitaux de Paris

Responsible Party: Assistance Publique - Hôpitaux de Paris Identifier: NCT00987168     History of Changes
Other Study ID Numbers: CRC 07024
First Posted: September 30, 2009    Key Record Dates
Last Update Posted: December 19, 2013
Last Verified: October 2010

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Congenital Hyperinsulinism
Sandostatine subcutaneous in 3 daily injections
Intramuscular injection of Sandostatine® LP per month

Additional relevant MeSH terms:
Congenital Hyperinsulinism
Glucose Metabolism Disorders
Metabolic Diseases
Pancreatic Diseases
Digestive System Diseases
Infant, Newborn, Diseases
Gastrointestinal Agents
Antineoplastic Agents, Hormonal
Antineoplastic Agents