Clinical Trial on PREnatal Dexamethasone In Congenital adrenal hyperplasia Therapy - PREDICT -

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Philipps-Universitaet Marburg

Participant type

Pediatric, Patients

Age range

In Utero, 0-17 years, 18-64 years

Gender

Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Hormonal diseases [C19]

Trial duration

5 Aug 2025 → ongoing

Decision date (initial)

2024-11-19

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

German Federal Ministry of Education and Research (Bundesministerium für Bildung und Forschung)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety

To investigate if a reduced dexamethasone dose is effective and non-inferior to the currently experimentally used high (“standard”) dose regimen in preventing prenatal virilization in females with classic congenital adrenal hyperplasia (CAH) and to investigate the effect on maternal weight gain during gestation (GWG)

Secondary objectives 1

1. To investigate safety of treated mothers and children (stillbirths, miscarriages, maternal side effects, maternal quality of life, maternal mental health and sleep, child´s birth weight and height at birth, week of gestation at birth, infant nervous system development, malformations in the child)

Conditions and MedDRA coding

Congenital adrenal hyperplasia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Adult pregnant females with risk of classic CAH in their fetuses (genetic mutations in both parents)
2. Written informed consent of the pregnant mother and the legal guardians of the unborn child
3. GW <8+0 post menstruationem

Exclusion criteria 8

1. Co-morbid condition requiring daily administration of a medication (or use of any medications/supplements) that interferes with the metabolism of glucocorticoids
2. Clinical or biochemical evidence of hepatic or renal disease. Creatinine over twice the upper limit of normal (ULN) or elevated liver function tests (ALT or AST >2 times ULN])
3. Subjects on regular daily inhaled, topical, nasal or oral steroids for any indication other than therapy of CAH in the fetus or in the CAH affected mother herself
4. History of malignancy (other than basal cell carcinoma successfully treated)
5. Participation in a clinical trial of an investigational or licensed drug or device within the 3 months prior to inclusion in this study.
6. Body weight <50 kg or >95 kg before pregnancy
7. Subjects unable to comply with the requirements of the protocol
8. Pregnancy with twins or multiples

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Closure of urogenital sinus (Prader stage)
2. Gestational weight gain (GWG) of mother

Secondary endpoints 2

1. Parameters of treatment consequences/safety in treated mothers and children
2. safety parameters: stillbirths, miscarriages, maternal side effects and adverse events, serious adverse events (AEs, SAEs) in treated mothers and children, maternal quality of life, maternal mental health and sleep, child´s birth weight and height at birth, week of gestation at birth, infant nervous system development, malformations in the child)

Investigational products

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

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Philipps-Universitaet Marburg

Sponsor organisation

Philipps-Universitaet Marburg

Address

Karl-Von-Frisch-Strasse 4

City

Marburg

Postcode

35043

Country

Germany

Scientific contact point

Organisation

Philipps-Universitaet Marburg

Contact name

KKS Marburg

Public contact point

Organisation

Philipps-Universitaet Marburg

Contact name

KKS Marburg

Third parties 4

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 12 files

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

Application history

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