Tolerability and Acceptance of Two Oral Hydrocortisone Compounding Formulation for Pediatrics

Adrenal Insufficiency Clinical Trial

Official title:

Tolerability and Acceptance of Two Oral Hydrocortisone Compounding Formulation for Pediatrics

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06435481
• Other study ID #: HIDROGUM21
• Secondary ID: 2021-001069-20
• Status: Not yet recruiting
• Phase: Phase 4
• Start date: September 2024
• Est. completion date: September 2026

Study information

• Verified date: April 2024
• Source: Hospital Universitari Vall d'Hebron Research Institute
• Contact: Carlos Javier Parramón Teixidó
• Phone: +3492746017
• Email: carlosjavier.parramon[@]vallhebron.cat
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The study aims to evaluate the tolerability and acceptance of two compounded formulations of hydrocortisone prepared in the Vall d'Hebron University Hospital (VHUH) Pharmacy Service: one, an oral suspension and the other, chewable tablets prepared using a volume dosing device (M3DIMAKER 3D printer). The main goal is to enhance patient care and adherence among pediatric patients.

This prospective, experimental study employs a randomized, crossover design and will take place solely at VHUH. Approximately 25-30 eligible patients diagnosed with adrenal hyperplasia, isolated primary adrenal insufficiency, or panhypopituitarism will be recruited. Each patient will receive each hydrocortisone formulation for a period of 3 months, totaling 6 months of treatment per patient. All patients will receive the medication at their usual dose and both formulations to assess tolerability and acceptance.

Description:

Adrenal insufficiency arises from inadequate synthesis of adrenal hormones, with primary, secondary, or tertiary forms depending on the defect's location-adrenal, pituitary, or hypothalamic. Hydrocortisone (17-Hydroxycorticosterone) is the preferred cortisol replacement.

Pediatric hydrocortisone formulations are not commercially available in any presentation. Consequently, compounded formulations are prepared as standard practice. Vall d'Hebron University Hospital's Pharmacy Service currently provides a liquid hydrocortisone formulation for these patients. While liquid formulations are often preferred due to their better dose adjustment and improved acceptability by pediatric patients, they have limitations such as shorter shelf life, possible special storage conditions, and taste problems due to bitter active ingredients. In some cases, particularly for chronic treatments with established and constant doses, other options such as capsules may be considered. Capsules offer longer stability, longer expiration dates, and do not require special storage conditions.

In this context, this study aims to determine the tolerability and acceptance of a new solid magistral formula in the form of chewable hydrocortisone tablets, prepared using a volume dosing device (M3DIMAKERTM 3D printer), compared to the usually preferred liquid formulation. Each of the formulations will be administered for three months. Evaluating the results will allow us to improve assistance to pediatric patients by offering the formulation with better tolerability and acceptance. The study will take place at Vall d'Hebron University Hospital, led by the Pharmacy Service in collaboration with the Pediatric Endocrinology Service.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 35
• Est. completion date: September 2026
• Est. primary completion date: September 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

Inclusion Criteria:

• Outpatients of both sexes, = 6 years old without swallowing problems and up to 17 years old, at the time of signing the informed consent document by parent(s) or guardian(s) and/or patients.

• Diagnosis of adrenal hyperplasia or isolated primary adrenal insufficiency or panhypopituitarism (secondary or tertiary adrenal insufficiency).

Exclusion Criteria:

• Known hypersensitivity to any of the excipients in the formulation of hydrocortisone.

• Any disorder or situation (decompensation) that, in the opinion of the investigating physician, poses a risk of non-compliance with the treatment.

Related Conditions & MeSH terms

• Adrenal Insufficiency

Intervention

Drug:
• 3D printed chewable formulation of hydrocortisone
Administration of a novel hydrocortisone formulation provided as chewable tablets. Each tablet, manufactured with a volume dosing device (3D printer), contains a precise dosage of hydrocortisone.
• Oral suspension of hydrocortisone
Administration of a standard hydrocortisone oral suspension. The solution, based on simple syrup, contains 1mg of hydrocortisone per milliliter.

Locations

Country	Name	City	State
Spain	Hospital Universitari Vall d'Hebron	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
Hospital Universitari Vall d'Hebron Research Institute

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Tolerability and acceptance of the two compounded oral hydrocortisone formulations	Tolerability will be assessed using a five-point questionnaire, while acceptability will be measured using the hedonic facial scale.	6 months
Secondary	Efficacy, compliance and safety of each hydrocortisone formulation	Efficacy will be assessed by the variation in hydrocortisone dosage as a percentage compared to the previous visit, the presence of intercurrent issues, and emergency room visits resulting from the underlying condition.; Treatment adherence will be estimated through monitoring medication dispensations from the Pharmacy Service and counting returned medication.; Safety will be evaluated by reporting potential adverse effects of treatment, the presence of intercurrent issues, and emergency room visits during follow-up.	6 months