None listed
Conditions
Interventions
RHIGF-I/RHIGFBP-3 Type of group
Day one: Single administration of 0.5 mg / kg per day
Day two: Single administration of 0.5 mg / kg per day
Day three: Single administration of 1.0 mg / kg per day
Day four: Single administration of 1.0 mg / kg per day
Sponsors
INSMED INCORPORATED,
Eligibility
Inclusion criteria
Inclusion criteria: • Diagnosis of a GHIS, such as Laron syndrome, la. If it is necessary to confirm the diagnosis (the main investigators can request it), a test of generation of IGF-I will be completed and the insufficiency of the GH will be demonstrated to invoke the IGF-L • 2-18 years old, • Size less than or equal to-3DE for age, • Baseline level of IGF-I less than or equal to -2DE for age, • Maximum level of> 29.2 mu / L (> 13.3 pg / L) of growth hormone using a GH challenge test, • Prepubertal, defined as the mammary stage of Tanner 1 or testicular volume <4 mL, • Baseline level of IGFBP-3 less than or equal to -IDE for the normative age, • Evaluation of the documented speed of the previous period of 12 months, and • With capacity and willingness to give informed consent or assent.
Exclusion criteria
Exclusion criteria: • Children at puberty, with a bone age of> 12 years for girls and> 14 years for boys, • Diagnosis of malignancy, • Diabetes mellitus diagnosis, • Treatment with rhIGF-I interrupted in the 3 years prior to patient selection for this study, • Treatment with growth hormone interrupted less than 6 months before the patient´s recruitment, • Treatment with an experimental drug within 30 days prior to selection, • Neuropathy, nephropathy, retinopathy or other clinically significant microvascular or macrovascular disease, • AST or ALT greater than or equal to 2 times the normal reference interval, • serum creatinine above 150 mmol / L (1.70 mg / dl), • Previous treatment with GnRH analogues or chronic systemic use of corticosteroids, and • Any other condition or treatment that, in the opinion of the investigator, may pose a risk to the patient or interfere with the patient´s ability to comply with this protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Outcome name:Size velocity will be assessed using the 1990 UK standard growth chart and will be calculated by Insmed Inc. For consistency, the height of each patients height should be evaluated from measurements of standing height taken at each visit of the study by the same observer and at the same time of day. Measure:Change in the speed of height (VT) with respect to the basal value Timepoints:During treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Outcome name:Standing and standing stature, weight and cranial perimeter will be measured. Measure:Change with respect to the baseline value in Auxology (height in standing / sitting and cranial perimeter) Timepoints:During treatment ; Outcome name:As part of the physical examination, the pubertal stage should be recorded Measure:Change with respect to the baseline value in Pubertal Stage Timepoints:During treatment ; Outcome name:Bone age will be determined using the TW-RUS method (X-ray of the left hand and wrist). Measure:Change with respect to the baseline value in Bone Age Timepoints:During treatment ; Outcome name:Insmed Inc will calculate the BMI based on the size and weight of patients Measure:Change with respect to the baseline value in BMI Timepoints:During the treatment | — |
Outcome results
None listed