Glibentek in Patients With Neonatal Diabetes Secondary to Mutations in K+-ATP Channels

NCT02375828 | Completed Phase 3 DMC

• 1 other identifier: 2014-003436-39
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The understanding of the molecular mechanisms of neonatal diabetes has deeply changed the therapy of patients carrying mutations in the K-ATP channel. Indeed, those patients are not treated anymore by insulin injections but by glibenclamide an oral anti-diabetic drug widely used in type 2 diabetes. Anyway, its galenic form (pills of 5 mg) is not suitable for children and difficult to administrate to infants or young children. The purpose of this study is to determine if a new galenic form of this durg is more suitable and as efficient as pills in children with neonatal diabetes.

Conditions

Neonatal Diabetes Secondary to Mutation in the Potassium Channel

Keywords

Acceptability; Phase 3; Neonatal diabetes

Outcome Measures

Primary Outcomes (2)

• Acceptability of an oral solution of glibenclamide (Hedonic visual scale)

  Hedonic visual scale

  2 months after the change from pills to oral solution.

• Acceptability of an oral solution of glibenclamide (Hedonic visual scale)

  Hedonic visual scale

  3 months after the change from pills to oral solution.

Secondary Outcomes (7)

• Tolerance of an oral solution of glibenclamide (Self administrated questionnaries)

  2 months after the change from pills to oral solution.

• Tolerance of an oral solution of glibenclamide (Self administrated questionnaries)

  3 months after the change from pills to oral solution.

• Recording pharmaceutical data on pills and oral solution of glibenclamide (Blood drug dosage)

  At inclusion

• Recording pharmaceutical data on pills and oral solution of glibenclamide (Blood drug dosage)

  2 months after the switch from pills to oral solution

• No alteration in metabolic control of the disease

  During the first month of administration

Study Arms (1)

Patients with neonatal diabetes

EXPERIMENTAL

Patients with neonatal diabetes

Drug: Glibenclamide

Interventions

Glibenclamide DRUG

Glibenclamide pills will be administrated during one month at the previously used dosage. During the first month of the study we wwil record pharmacokinetic data, number of hypoglycaemia and the administration problems associated to this galenic form. At the end of the first month of enrolment, patients will be given oral solution of glibenclamide for the 4 remaining months. Pharmacocinetic data, number of hypoglycaemia and parents and children feeling about pratictability of administration will be then recorded.

Patients with neonatal diabetes

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age below 18 years
• Neonatal diabetes secondary to documented mutation in one of the 2 sub units of potassium channels
• Patients already treated by glibenclamide pills
• Signed consent

You may not qualify if:

• Families unable to fill in the questionnaries
• Patients unable to answer to the visual hedonic squale
• Patients unable to take the oral solution
• Known allergy to sulfonylureas or to other antidiabetic or antibiotic sulfamides
• Insulin therapy associated to glibenclamide
• Miconazole therapy
• Porphyria
• Breast feeding
• Severe renal failure (creatinine clearance below 30 ml/mn)
• Liver failure (prothombine time below 70)
• Not affiliated to the health care system

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Hôpital Universitaire Necker Enfants Malades

Paris, 7501, France

Location

Related Publications (4)

• Beltrand J, Baptiste A, Busiah K, Bouazza N, Godot C, Boucheron A, Djerada Z, Gozalo C, Berdugo M, Treluyer JM, Elie C, Polak M; GLID-KIR study group. Glibenclamide oral suspension: Suitable and effective in patients with neonatal diabetes. Pediatr Diabetes. 2019 May;20(3):246-254. doi: 10.1111/pedi.12823. Epub 2019 Feb 21.

  PMID: 30684309 BACKGROUND

• Busiah K, Drunat S, Vaivre-Douret L, Bonnefond A, Simon A, Flechtner I, Gerard B, Pouvreau N, Elie C, Nimri R, De Vries L, Tubiana-Rufi N, Metz C, Bertrand AM, Nivot-Adamiak S, de Kerdanet M, Stuckens C, Jennane F, Souchon PF, Le Tallec C, Desiree C, Pereira S, Dechaume A, Robert JJ, Phillip M, Scharfmann R, Czernichow P, Froguel P, Vaxillaire M, Polak M, Cave H; French NDM study group. Neuropsychological dysfunction and developmental defects associated with genetic changes in infants with neonatal diabetes mellitus: a prospective cohort study [corrected]. Lancet Diabetes Endocrinol. 2013 Nov;1(3):199-207. doi: 10.1016/S2213-8587(13)70059-7. Epub 2013 Sep 6.

  PMID: 24622368 BACKGROUND

• Pearson ER, Flechtner I, Njolstad PR, Malecki MT, Flanagan SE, Larkin B, Ashcroft FM, Klimes I, Codner E, Iotova V, Slingerland AS, Shield J, Robert JJ, Holst JJ, Clark PM, Ellard S, Sovik O, Polak M, Hattersley AT; Neonatal Diabetes International Collaborative Group. Switching from insulin to oral sulfonylureas in patients with diabetes due to Kir6.2 mutations. N Engl J Med. 2006 Aug 3;355(5):467-77. doi: 10.1056/NEJMoa061759.

  PMID: 16885550 BACKGROUND

• Babenko AP, Polak M, Cave H, Busiah K, Czernichow P, Scharfmann R, Bryan J, Aguilar-Bryan L, Vaxillaire M, Froguel P. Activating mutations in the ABCC8 gene in neonatal diabetes mellitus. N Engl J Med. 2006 Aug 3;355(5):456-66. doi: 10.1056/NEJMoa055068.

  PMID: 16885549 BACKGROUND

MeSH Terms

Interventions

Glyburide

Intervention Hierarchy (Ancestors)

Sulfonylurea Compounds; Urea; Amides; Organic Chemicals; Sulfones; Sulfur Compounds

Study Officials

• Michel Polak, MD, PhD

  Hopital Universitaire Necker Enfants Malades, Assistance publique - hôpitaux de Paris, Faculté de medicine Paris Descartes, Université Sorbonne Paris cité

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 24, 2015
• First Posted: March 3, 2015
• Study Start: March 20, 2015
• Primary Completion: March 4, 2016
• Study Completion: July 22, 2019
• Last Updated: September 8, 2025

Record last verified: 2025-09

Locations

FR (1)