Growth Hormone in a Patient With a Dominant-Negative GHR Mutation

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-26

Study Completion Date

2028-09-01

Brief Summary

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This is a prospective interventional study designed for a single patient with a dominant-negative mutation in the growth hormone receptor gene (GHR) which results in increased levels of growth hormone binding protein (GHBP). The patient will receive escalating doses of growth hormone titrated to achieve an insulin like growth factor-1 level above the mean and then growth response to therapy will be monitored.

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Detailed Description

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Conditions

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Growth Disorders

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Growth hormone

The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations.

Group Type EXPERIMENTAL

Somatropin

Intervention Type DRUG

Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating

Interventions

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Somatropin

Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Provision of signed and dated informed consent form
2. Stated willingness to comply with all study procedures and availability for the duration of the study
3. Be the specific subject with the a specific mutation in GHR leading to high GHBP.

Minimum Eligible Age

9 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No