Growth deficiency due to growth hormone deficiency MedDRA version: 18.1 Level: PT Classification code 10056438 Term: Growth hormone deficiency System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Name: NA
Product Code: NA
Pharmaceutical Form: Lyophilisate and solvent for suspension for injection
INN or Proposed INN: NA
CAS Number: 12629015
Current Sponsor code: CRT076
Other descriptive
Sponsors
Cristália Produtos Químicos Farmacêuticos Ltda.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Children aged 4 years and 13 years (for girls) and 14 years (for boys); Children prepubertal (Tanner stage 1); Bone age less than 11 years (for boys) and 9 years (for girls), documented by radiographs of the hand and wrist (will accept a radiograph performed within 6 months prior to enrollment in the study); Naïve children with growth hormone; Children diagnosed with disturbance in growth due to deficiency of growth hormone documented before initial treatment with r-hGH through: The height z score =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Children aged <4 years; Children with clinical signs of puberty (breast / genitalia and pubic hair Tanner stage 2); Note: Children who enterpuberty during treatment (breast development Tanner 2 / or testicles 4 ml or 2.5 cm in greatest diameter) shall be excluded; Other causes of disorder in children including growth in stature for gestational age (SGA), Turner syndrome, Prader-Willi syndrome, other causes; Children with closed epiphyses bone; Children with physical changes that prevent accurate measurement of height; Children with comorbidities that prevent normal growth. Patients with central hypothyroidism may be included, if well controlled with l-thyroxine, evidenced by normal free T4 dosages; dysmorphic syndrome; Presence of signs of skeletal dysplasia; Evidence of active malignancy or with less than two years of treatment considered curative; uncontrolled growth of benign intracranial tumors; Children with benign intracranial hypertension; Children with clinical evidence of malnutrition considered relevant to the discretion of the investigator; Children with Diabetes mellitus type 1 or 2; severe acute disease, including complications after heart surgery by thoracotomy, abdominal surgery, multiple accidental trauma or acute respiratory failure; concomitant chronic disease which may interfere with the analysis of the study (eg, hyperthyroidism / uncontrolled hypothyroidism, gastrointestinal diseases, cardiorespiratory diseases, liver failure, kidney failure, bone pathologies that can affect the growth, chronic inflammatory diseases and inborn errors of metabolism); Patients with hormone deficiency related diseases by panhypopituitarism may be included if properly controlled; MRI findings that may interfere with the study drug (examples: intracranial hypertension, tumors in the hypothalamic-pituitary region); Children with anti-GH antibodies; Children undergoing treatment for the disorder or attention deficit hyperactivity disorder deficit hyperactivity disorder; Concomitant medication that could influence the secretion of growth (eg, estrogen, androgen, anabolic steroids, steroids, aromatase inhibitors) hormone. Physiological doses of corticosteroids for the treatment of pituitary deficiency are allowed as well as low-dose inhaled and / or nasal steroids for the treatment of asthma or chronic rhinitis (500mcg or less / day beclomethasone or equivalent); or known allergy to the study medications, or any of the excipients or thinner / solvent hypersensitivity; Participation in another clinical study within 3 months before study entry; Statement of legal limitation disability or parent / legal representative
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: This study aims to evaluate the efficacy of recombinant human growth hormone Cristália (r-hGH Cristália) compared to Genotropin® after 12 months of treatment, as measured by growth rate (VC) in prepubertal children with the disorder growth due to deficiency of growth hormone treatment-naive.;Secondary Objective: Z score variation in height (z-score of height after 12 months of treatment - z score high at the start of treatment); Evaluation of immunogenicity of r-hGH periodic quantification of anti- GH antibodies during the study; IGF-1 and IGFBP-3 assessment baseline and periodic serum during the study; glucose and insulin assessment of baseline and periodic serum fasting during the study; Evaluation of the incidence of adverse events during the study;;Primary end point(s): The primary efficacy endpoint is the difference in growth rate (cm / year) between the r-hGH group and Cristália Genotropin® group after 12 months of treatment. The height is measured in millimeters using a stable estadiometer through a standard procedure, done equally in all research centers.;Timepoint(s) of evaluation of this end point: After 12 months of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Difference in height variation, expressed by the variation in z score in height between the r-hGH-Cristália group and the Genotropin® group after 12 months of treatment. The Z score variation in height will be calculated as the difference between the height z score obtained after 12 months of treatment and the z-score of height obtained at the beginning of the treatment period. The Z score of height is the ratio of the actual height minus the average population height for age and sex divided by the standard deviation. The Z score calculation must be performed using the growth curve of the World Health Organization in 2007 to be provided in the study material. The investigator, or person designated by him / her should check the expected height and standard deviation for age and sex of research participants. All this information must be recorded in the source documents of the research participant The height is measured in millimeters using a stable estadiometer through a standard procedure, done equally in all sites.;Timepoint(s) of evaluation of this end point: After 12 months of treatment | — |
Countries
Brazil
Contacts
Public ContactDébora Garcia Rodrigues
Cristália Produtos Químicos Farmacêuticos Ltda.
Outcome results
None listed