Clinical trial NCT06435481: Tolerability and Acceptance of Two Oral Hydrocortisone Compounding Formulation for Pediatrics
Official title: Tolerability and Acceptance of Two Oral Hydrocortisone Compounding Formulation for Pediatrics
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE4
Conditions: Adrenal Insufficiency
Lead sponsor: Hospital Universitari Vall d'Hebron Research Institute
Sponsor class: OTHER
First posted: 2024-05-30
Enrollment: 35
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Tolerability and acceptance of the two compounded oral hydrocortisone formulations
timeFrame: 6 months
Secondary outcomes:
measure: Efficacy, compliance and safety of each hydrocortisone formulation
timeFrame: 6 months
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
Study officials:
affiliation: Hospital Vall d'Hebron
name: Hospital Universitari Vall d'Hebron
role: STUDY_CHAIR
References: