PHASE II / III, OPEN AND MULTICENTRIC CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFECTIVENESS OF FACTOR I SIMILAR GROWTH TO INSULIN / PROTEIN 3 OF UNION TO FACTOR I OF GROWTH SIMILAR TO INSULIN (RHIGF-I / RHIGFBP-3 ) ADMINISTERED FOR 12 MONTHS TO CHILDREN AND ADOLESCENTS WITH SYNDROME OF INSENSIBILITY TO GROWTH HORMONE (GHIS) LIKE LARON SYNDROME

Not Applicable

• Conditions: -M892; M892

• Registration Number: PER-061-04
• Lead Sponsor: INSMED INCORPORATED,

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2004-09-29
• Last Update Posted: 4 September 2023

Recruitment & Eligibility

• Status: Complete
• Sex: Not specified
• Target Recruitment: 0

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

• 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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified