First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study

Merck KGaA (EMD Serono)

Status and phase: Completed; Phase 4

Conditions: Turner Syndrome; Idiopathic Growth Hormone Deficiency

Treatments: Other: Blood sampling

Study type: Interventional

Funder types: Industry

Identifiers

NCT01419249

EMR 200104_010

Details and patient eligibility

About

PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner syndrome (TS) will respond to a treatment with recombinant human growth hormone (r-hGH).

Full description

This study is an open-label, interventional, retrospective, multicenter, international study, single-arm, non-randomized, and non-controlled study. The subject's trial participation includes a single visit. During the visit, subjects who give consent to participate in the trial will undergo blood sampling for genetic markers testing and retrospective data will be collected relative to the first year of the subject's r-hGH treatment.

The r-hGH treatment followed by the subject is indicated the pediatric population, therefore most of the subjects included into the trial will be below 18 years old.

This study is a non-investigational medicinal product (IMP) trial therefore no drug product data is provided.

Enrollment: 458 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Pre-established diagnosis of IGHD or TS based on classical criteria with at least 1 year of r-hGH therapy and with Tanner stage 1 at treatment start
• Retrospective availability of a complete set of clinical, auxological and biological parameters necessary for building the predictive model
• Other protocol defined inclusion criteria could apply

Exclusion criteria

• Acquired growth hormone deficiency (GHD)
• Any drug or disease that could affect growth during the first year of r-hGH treatment
• Other protocol defined exclusion criteria could apply

Trial design

458 participants in 1 patient group

Retrospective cohort

Other group

Treatment:

Other: Blood sampling