Trial Outcomes & Findings for Growth Hormone in a Patient With a Dominant-Negative GHR Mutation (NCT NCT05382637)

NCT ID: NCT05382637

Last Updated: 2025-07-02

Results Overview

Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 1 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2025-07-02

Participant Flow

Participant milestones

Measure	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. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Growth Hormone n=1 Participants The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	9.75 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants
Baseline Height standard deviation score	-3.18 Standard deviation score from the CDC n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean

Outcome measures

Measure	Growth Hormone n=1 Participants The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Growth Hormone Dose	0.250 mg/kg/day

SECONDARY outcome

Timeframe: 1 year

Growth velocity during 1st year of growth hormone treatment

Outcome measures

Measure	Growth Hormone n=1 Participants The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Growth Velocity	8.7 cm/year

SECONDARY outcome

Timeframe: Baseline to 1 Year

Change in age and sex adjusted height standard deviation score based on the US CDC growth charts over the course of the 1st year of treatment with growth hormone. Height standard deviation score based on the US CDC growth chart data. The CDC provides tables of both height percentiles and standard deviations of the distribution of heights by age and sex. The CDC has a SAS program that allows for calculation of an individual's height standard deviation score based on the population data. This is the standard way that height is recorded in many growth studies.

Outcome measures

Measure	Growth Hormone n=1 Participants The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. Somatropin: Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
Height Standard Deviation Score Using Center for Disease Control (CDC) Growth Chart	0.81 Standard deviations

Adverse Events

Growth Hormone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Andrew Dauber, MD

Children's National Hospital

Phone: 202-476-2121

Email: adauber@childrensnational.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place