EVOLUTION OF GROWTH RATE IN CHILDRE N WITH GROWTH RETARDATION RELATED TO LONG-TERM GLUCOCORTICOSTEROID THERAPY AND TREATED BY GENOTONORM®

• Conditions: Growth retardation related to long-term glucocorticoid therapy; MedDRA version: 18.0Level: PTClassification code 10053759Term: Growth retardationSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disorders; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: EUCTR2004-002992-17-Outside-EU/EEA
• Lead Sponsor: Pfizer, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-05-14
• Last Update Posted: 10 July 2015

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 98

Inclusion Criteria

•Evidence of a personally signed and dated informed consent document indicating that
the subject (or a legal representative) has been informed of all pertinent aspects of the study.
•Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
•Male and female subjects of childbearing potential must agree to use a highly effective method of contraception throughout the study and for 28 days after the last dose of assigned treatment. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.
•Female subjects who are not of childbearing potential (ie, meet at least one of the following criteria):
•Have undergone hysterectomy or bilateral oophorectomy;
•Have medically confirmed ovarian failure or
•Are medically confirmed to be post menopausal (cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; laboratory confirmation LH and FSH may be indicated if menopause is in doubt.
•Subjects who are clinically pre pubertal
•
•A request for GH treatment may be issued if all the following criteria are fulfilled
1. Bone age < 15 years for a boy and <13 years for a girl.
2. Naïve child: Measured Height < -2 SD.
3. Child currently treated by GH: Annual Growth Rate = 0 SD for CA.
4. Glucocorticosteroid treatment over the last 12 months at least.
5. Glucocorticosteroid treatment is anticipated to be sustained for 1 more year at least.
6. GH treatment request by a physician who can initiate Genotonorm® treatment according to the French SCP. That is to say a physician working in a hospital pediatric department or in a hospital endocrinology and metabolic disease department and, who is a pediatrician and/or a pediatric endocrinologist.
7. The child benefits of the French social security cover.
8. Evidence of a personally signed and dated informed consent document indicating that the patient's parents/guardians and from the patient himself/herself if he/she is able to receive and understand the information have been informed of all pertinent aspects of the trial.
•GH treatment approved by the Treatment monitoring committee

Are the trial subjects under 18? yes
Number of subjects for this age range: 97
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Glucose intolerance on an Oral Glucose Tolerance Test dated less than 3 months or
diabetes mellitus.
2. Syndrome known to be associated with an increased risk of cancer eg, family history
of adenomatous polyposis.
3. Pathological condition or disease for which GH treatment is already approved in France.
4. Participation in any other studies involving investigational or marketed products, concomitantly or within 30 days prior to entry in the study.
5. Unable and/or unlikely to comprehend and/or follow GH treatment and/or the protocol.
6. A previous history of intolerance or hypersensitivity to the study drug, or to drugs with
similar chemical structures.
7. Patients who are known or are suspected allergic to the preservative metacresol
8. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and in the judgment of the investigator, would make the subject inappropriate for entry into this trial. Somatropin must not be used when there is any evidence of activity of a tumour. Intracranial tumours must be inactive and antitumour therapy must be completed prior to starting growth hormone therapy. Treatment should be discontinued if there is evidence of tumour growth.
9. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the subject inappropriate for entry into this trial.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To show an increase in height after 3 years of GH treatment. However subjects will be followed for up to 5 years of treatment. Height in SD for chronological age (CA) after 3 years will be compared to Height in SD for CA before inclusion in the trial.;Secondary Objective: •To estimate the evolution of height and growth rate over 1,2 and 3 years of GH treatment.<br>•To estimate the prognostic factors of total height increase (in SD for CA) and growth rate (SD for CA) after 3 years of GH treatment.<br>•To confirm the good clinical and biological safety of GH treatment in such children.<br>•To confirm the efficacy on height after 5 years of GH treatment.;Primary end point(s): Efficacy: Difference between Height in SD for chronological age (CA) after 3 years and Height in SD for CA at Visit 2.;Timepoint(s) of evaluation of this end point: 3 years after the visit 2.