Glucagon-Like Peptide 1 Receptor Agonist in Diabetes Mellitus Management in Children and Adolescents With Transfusion-Dependent Thalassemia

NCT07370922 | Completed Phase 2 DMC

• 1 other identifier: FMASU MS 794/2024
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

Blood transfusion and iron-chelation therapy have prolonged and improved the quality of life in patients with β-thalassemia. The improvement was mainly due to the decrease in mortality from heart failure Such a treatment, however, leads to chronic iron overload and frequently to endocrine complications, especially the development of diabetes. The prevalence of diabetes mellitus (DM) in β-thalassemia varies from 9.7% to 29% and the overall prevalence of impaired fasting glucose (IFG) and impaired glucose tolerance (IGT) is 17.2% and 12.4% respectively in transfusion dependent thalassemia (TDT) patients. GLP-1 is a proglucagon derived peptide that is released from gut endocrine cells in response to nutrient intake. This molecule is rapidly inactivated by the action of dipeptidyl peptidase IV (DPP-4) which limits its use as therapeutic agent. Recent guidelines by the American Diabetes Association and the European Association for the Study of Diabetes recommend that for patients with type 2 diabetes, GLP-1 receptor agonists (GLP-1RAs) are preferable to insulin as the initial injection therapy and are also the preferred choice for addition to basal insulin for combination injection therapy. An increasing number of clinical trials of agents in youth-onset T2D resulted in the availability of more efficacy data and regulatory approval for two Glucagon-like peptide-1 (GLP-1) receptor agonists (Liraglutide and Exenatide) in Pediatrics. The Efficacy of the daily GLP-1 agonist, Liraglutide, in youth-onset T2D wasstudied in the Ellipse trial, which demonstrated placebo-subtracted. HbA1c lowering of 1% and 1.5% at 26 and 52 weeks, respectively. This glycemic reduction was accompanied by a small decrease in BMI z-score. Liraglutide (Victoza 0.6-1.8 mg a day) subsequently received approval by the FDA for use in youth 12-17 years of age. Recently, extended release exenatide (Bydureon BCise 2 mg) was approved as a once-weekly injection for youth 10-17 years of age based on data from the BCB114 study showing superiority to placebo in lowering HbA1c with a between-group difference of 0.85 percentage points. Hence, the aim of this study is to assess the efficacy and safety of GLP-1 receptor agonist versus conventional insulin therapy in the management of diabetes mellitus in children with transfusion -dependent Thalassemia.

Conditions

Transfusion Dependent Beta Thalassemia

Keywords

GLP-1 receptor agonist; Dulaglutide; Diabetes; Transfusion Dependent Thalassemia

Outcome Measures

Primary Outcomes (1)

• Change in glycemic variability using continuous glucose monitoring after 6 months.

  Change in glycemic variability using continuous glucose monitoring after 6 months.

  6 months

Study Arms (2)

Dulaglutide group

EXPERIMENTAL

Drug: Dulaglutide 0.75Mg/0.5Ml Inj Pen

Control group

ACTIVE COMPARATOR

Control group

Drug: control group (insulin)

Interventions

Dulaglutide 0.75Mg/0.5Ml Inj Pen DRUG

Dulaglutide subcutaneous injection in a dose of 0.75 mg once weekly for 6 months.

Dulaglutide group

control group (insulin) DRUG

Insulin basal blocs according to guidelines

Control group

Eligibility Criteria

• Age: 10 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 10-18 years old.
• Children with TDT according to the Thalassemia International Federation (TIF) guidelines (Farmakis et al., 2022).
• Children with diabetes mellitus according to the International Society for Pediatric and Adolescent Diabetes (ISPAD) 2022 guidelines (Libman et al., 2022).

You may not qualify if:

• Other hemoglobinopathies as alpha thalassemia or sickle thalassemia patients.
• Other disorders that may affect glucose homeostasis rather than β-TM.
• Autoimmune disease, collagen diseases, hypo- or hyper-thyroidism, infections, tumors, hematological diseases other than β-TM.
• Personal or family history of medullary thyroid carcinoma (MTC) or multiple endocrine neoplasia type 2 (MEN 2).
• Intake of any vitamins or food supplements one month before study and participation in a previous investigational drug study within the three months preceding screening.

Sponsors & Collaborators

• Ain Shams University: lead

Study Sites (1)

Faculty of medicine, Ain Shams University

Cairo, Egypt

Location

MeSH Terms

Conditions

Diabetes Mellitus

Interventions

dulaglutide; Control Groups; Insulin

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods; Proinsulin; Insulins; Pancreatic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: assistant professor

Study Record Dates

• First Submitted: January 9, 2026
• First Posted: January 27, 2026
• Study Start: November 22, 2024
• Primary Completion: November 22, 2025
• Study Completion: January 5, 2026
• Last Updated: January 27, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

EG (1)