Tolerability and acceptance of two oral hydrocortisone medications for Pediatrics.

2024-519149-31-00 Protocol HIDROGUM21 Therapeutic use (Phase IV) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 1 sites · Protocol HIDROGUM21

Overview

Sponsor-declared trial summary

Phase Therapeutic use (Phase IV)
Status Authorised, recruitment pending
Participants planned 35
Countries 1
Sites 1

Adrenal hyperplasia or isolated primary adrenal insufficiency or panhypopituitarism (secondary or tertiary adrenal insufficiency).

To evaluate the tolerability and acceptance of the two hydrocortisone compounding formulation prepared in the Hospital Pharmacy Service.

Key facts

Sponsor
Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca
Participant type
Pediatric, Patients
Age range
0-17 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Hormonal diseases [C19]
Decision date (initial)
2024-10-31
Transition trial
Yes
Low-intervention
Yes
Rare-disease indication
No
Vulnerable population
No

External identifiers

EU CT number
2024-519149-31-00
EudraCT number
2021-001069-20

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others

To evaluate the tolerability and acceptance of the two hydrocortisone compounding formulation prepared in the Hospital Pharmacy Service.

Secondary objectives 1

  1. To evaluate adherence to treatment.

Conditions and MedDRA coding

Adrenal hyperplasia or isolated primary adrenal insufficiency or panhypopituitarism (secondary or tertiary adrenal insufficiency).

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

  1. Outpatients of both sexes, ≥ 6 years of age without swallowing problems and up to 17 years, at the time of signing the informed consent document by parent (s) or guardian (s) and / or patients.
  2. Diagnosis of adrenal hyperplasia or isolated primary adrenal insufficiency or panhypopituitarism (secondary or tertiary adrenal insufficiency).

Exclusion criteria 2

  1. Known hypersensitivity to any of the excipients in the hydrocortisone compounding.
  2. Any disorder or situation (decompensation) that, in the opinion of the medical researcher, poses a risk of not complying with the treatment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. The sensory acceptability of the formulation.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Hydrocortisone Ph. Eur.

SUB171070 · Substance

Active substance
Hydrocortisone Ph. Eur.
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
12 mg/m2 milligram(s)/square meter
Max total dose
1080 mg/m2 milligram(s)/square meter
Max treatment duration
3 Month(s)
Authorisation status
Authorised
ATC code
- — -
Marketing authorisation
-
Paediatric formulation
Yes
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Formula magistral

Comparator 1

Hydrocortisone Ph. Eur.

SUB171070 · Substance

Active substance
Hydrocortisone Ph. Eur.
Pharmaceutical form
TABLET
Route of administration
ORAL
Max daily dose
12 mg/m2 milligram(s)/square meter
Max total dose
1080 mg/m2 milligram(s)/square meter
Max treatment duration
3 Month(s)
Authorisation status
Authorised
ATC code
- — -
Marketing authorisation
-
Paediatric formulation
Yes
Orphan designation
No
Modified vs. Marketing Authorisation
Yes
Modification description
Formula magristal

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca

Sponsor organisation
Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca
Address
Passeig De La Vall D'Hebron 119-129
City
Barcelona
Postcode
08035
Country
Spain

Scientific contact point

Organisation
Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca
Contact name
Carlos Javier Parramón-Teixidó

Public contact point

Organisation
Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca
Contact name
Carlos Javier Parramón-Teixidó

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Spain Authorised, recruitment pending 35 1
Rest of world 0

Investigational sites

Spain

1 site · Authorised, recruitment pending
Hospital Universitari Vall D Hebron
Pharmacy service, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 6 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_Protocol EU CT 2024-519149-31-00_for publication 6
Recruitment arrangements (for publication) K1_Recruitment arrangements 1
Subject information and informed consent form (for publication) L1_SIS and ICF_minor patients_ESP 6
Subject information and informed consent form (for publication) L1_SIS and ICF_parents or legal representative_ESP 6
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Hidrocortisona Base_for publication 1
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC_Hidrocortisona Base_for publication 1

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-10-25 Spain Acceptable
2024-10-31
2024-10-31