Growth deficiency due to growth hormone deficiency
Conditions
Interventions
Experimental group (r-hGH Cristáli): 47 participants with failure to thrive will use the product investigational recombinant growth hormone. The product should be administered every day, with 0,33ug p
Drug
Biological/vaccine
D06.472.699.631.525.425
Sponsors
ICF - Instituto de Ciências Farmaceuticas de Estudos e Pesquisas LTDA
Cristalia Produtos Químicos Farmacêuticos Ltda
Eligibility
Age
4 Years to 14 Years
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 <-2.0 SD for age and sex; A response to a test stimulus GH release with peak blood 7 ng / ml in the presence of abnormalities morphostructural hypothalamic-pituitary region shown by Nuclear Magnetic Resonance (NMR) or; response to stimulus two tests of GH release with peak 7 ng / ml in the presence of a normal NMR (stimulating agents are acceptable insulin, clonidine, glucagon, arginine and L-dopa). Historical values of GH in blood up to 12 months prior to enrollment in the study will be acceptable. An MRI performed up to 2 years before inclusion in the study is acceptable; History of growth velocity below the mean for the normal population for at least 6 months prior to inclusion in the study, according to the table of VC Tanner (must be historical height data with a minimum of 6 months and maximum of 18 months. Researcher must ensure that the measurements were performed in a standardized way in standard stadiometer (rigid deck and rigid ruler); Baseline IGF-I -0.5 SD for age and sex (results provided by the central laboratory).
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 enter puberty 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, cardio-respiratory 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 |
|---|---|
| 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 research centers. | — |
Countries
Brazil
Contacts
Public ContactRicardo ;Vanessa Baradelli;Assao
Cristalia Produtos Químicos Farmacêuticos Ltda;Cristalia Produtos Químicos Farmacêuticos Ltda
Outcome results
None listed