Trials / Active Not Recruiting
Active Not RecruitingNCT05382637
Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- Children's National Research Institute · Academic / Other
- Sex
- Male
- Age
- 9 Years
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Somatropin | Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating |
Timeline
- Start date
- 2022-08-26
- Primary completion
- 2023-09-01
- Completion
- 2028-09-01
- First posted
- 2022-05-19
- Last updated
- 2025-07-02
- Results posted
- 2025-07-02
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05382637. Inclusion in this directory is not an endorsement.