Lanreotide Autogel Treatment of Patients With Congenital Hyperinsulinism of Infancy

• Sponsor: Sheba Medical Center (Other)
• Overall Status: Unknown status
• CT.gov ID: NCT01070758
• Collaborator: (none)
• Enrollment: 10
• Location: 1
• Duration (Months): 60
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The purpose of our study is to evaluate the efficacy and safety of Lanreotide Autogel in children with congenital hyperinsulinism already treated with Octreotide by pump.

Congenital hyperinsulinism is a genetic disorder characterized by inappropriate insulin secretion resulting in persistent hypoglycemia (low blood sugars. Patients exposed to recurrent hypoglycemic episodes are at increased risk of developmental disorders, so identification and prompt management of patients are essential. Many patients are treated with the somatostatin analog Octreotide which is administered by continuous infusion using a pump (we use an insulin pump). This treatment may pose a huge burden and be stressful for patients and families as it demands intensive daily care. In an effort to simplify the daily care of our patients and improve their quality of life we will study the efficacy and safety of Lanreotide Autogel - a long-acting somatostatin analog that can be administered by injection once a month

Condition or Disease	Intervention/Treatment	Phase
Congenital Hyperinsulinism	Drug: Lanreotide autogel	Phase 4

Detailed Description

The purpose of our study is to evaluate the efficacy and safety of Lanreotide Autogel in children with CHI already treated with Octreotide by pump.

Patients and methods. Congenital hyperinsulinism (CH) is a genetic disorder characterized by dysregulated insulin secretion resulting in persistent hypoglycemia. Identification and prompt management of patients are essential, as patients exposed to recurrent hypoglycemic episodes are at increased risk of developmental disorders. Many patients are treated with the somatostatin analog Octreotide which is administered by continuous infusion using a pump. This treatment may pose a huge burden and be stressful for patients and families as it demands intensive daily care. In an effort to simplify the daily care of our patients and improve their quality of life we will study the efficacy and safety of Lanreotide Autogel(Lan-ATG)- a long-acting somatostatin analog that can be administered by injection once a month.

This trial will include children with CH, who are treated with Octreotide by pump. We believe that children older than 2 years old will benefit most from this therapy. At this age, some of the parents encounter technical problems with the pump, as the children are prone to play with the pump and take out the needles. It's also very difficult to place the children in day care, because they need continuous follow up.

The dose of Lan-ATG will be calculated according to the surface area of the patient. The dose used in adults is usually 60 mg and we will adapt the patient's dose according to the body surface area and also according to the daily dose of Octreotide used with the pump. The starting dose will be 40 mg/m².

The patients will be gradually weaned from the pump following the first injection of Lan-ATG (10% decrease every 3-4 days for a total of a month).

Every patient will serve as his/her own control.

The following examinations will be done in every child:

1. Continuous blood glucose monitoring during 72 hours with a glucosensor, to exclude asymptomatic hypoglycemia - once in 6 months.

2. Growth velocity every 3 months.

3. Bone age once a year.

4. Routine laboratory tests (biochemistry, CBC and thyroid function tests) every six months.

5. Biliary US once in 6 months. During the follow up we will try to expand the distance between injections, based on our knowledge that most of the patients with CH are known to enter remission after the age of 4-5 years.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Treatment With Lanreotide Autogel (Somatostatin Analogue) in Patients With Congenital Hyperinsulinism of Infancy Already Treated With Somatostatin Analog by Pump
• Study Start Date: Feb 1, 2010
• Anticipated Primary Completion Date: Feb 1, 2015
• Anticipated Study Completion Date: Feb 1, 2015

Outcome Measures

Primary Outcome Measures

1. Euglycemia as recorded by Continuous Glucose Monitoring System (CGMS) [1 year]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 8 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Age 2-8 years,

• Diagnosed with congenital hyperinsulinism,

• Treated by Octreotide continuous infusion (pump).

Exclusion Criteria:

• Family not interested in participating.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pediatric Endocrinology Unit, Sheba Medical Center	Tel-Hashomer, Ramat-Gan		Israel	52653

Sponsors and Collaborators

• Sheba Medical Center

Investigators

• Principal Investigator: Dalit Modan, M.D., Sheba Medical Ceter, Tel-Hashomer, Israel

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Dr. Dalit Modan, Pediatric Endocrinologist, Sheba Medical Center
• ClinicalTrials.gov Identifier: NCT01070758
• Other Study ID Numbers: SHEBA-10-7165-DM-CTIL
• First Posted: Feb 18, 2010
• Last Update Posted: Dec 13, 2012
• Last Verified: Dec 1, 2012

• Keywords provided by Dr. Dalit Modan, Pediatric Endocrinologist, Sheba Medical Center: Congenital hyperinsulinism; Somatostatin analog; Lanreotide autogel; Hypoglycemia
• Additional relevant MeSH terms: Congenital Hyperinsulinism; Nesidioblastosis; Hyperinsulinism; Lanreotide

Study Results

No Results Posted as of Dec 13, 2012