Growth DisordersPhase 2NCT05382637
Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
A Phase 2 study testing Somatropin in people with Growth Disorders, run by Children's National Research Institute at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 9 years and older, men only
- Conditions the study is for
- Growth Disorders
- What is being tested?
- Somatropin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Aug 2022to 1 Sep 2028
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Be the specific subject with the a specific mutation in GHR leading to high GHBP.
Who cannot join
- There are no exclusion criteria for this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
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
- Growth Disorders
- Being tested
- Somatropin
- Medical subject headings
- Growth Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SomatropinDrug
Daily growth hormone at a starting dose of 50 mcg/kg/day and escalating
| Study group | Role | What they receive |
|---|---|---|
| Growth hormone | Experimental | The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. Receives: Drug: Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Growth Hormone Dose
Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean
Measured: 1 year
Secondary outcomes (2)
Growth Velocity
Growth velocity during 1st year of growth hormone treatment
Measured: 1 year
Height Standard Deviation Score Using Center for Disease Control (CDC) Growth Chart
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.
Measured: Baseline to 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2022
- Study start26 Aug 2022
- Primary completion1 Sep 2023
- Study completion1 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Growth DisordersStudies of the same condition, open and under way.
- More trials run by Children's National Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Andrew Dauber, Children's National Research Institute
- Investigators
- Andrew Dauber, MD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05382637
- Sponsor's protocol ID
- STUDY00000211
- Status last verified
- Jun 2025
- First submitted
- 16 May 2022
- Last updated
- 2 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Merchant N, Houchin L, Boucher K, Dauber A. A Clinical Trial of High-Dose Growth Hormone in a Patient With a Dominant-Negative Growth Hormone Receptor Mutation. J Clin Endocrinol Metab. 2024 Oct 15;109(11):2937-2942. doi: 10.1210/clinem/dgae244.
PubMed 38597155
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Growth Hormone in a Patient With a Dominant-Negative GHR Mutation
- ClinicalTrials.gov ID
- NCT05382637
- Acronym / sponsor ID
- STUDY00000211
- Conditions
- Growth Disorders
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 26 Aug 2022 (actual)
- Primary completion
- 1 Sep 2023 (actual)
- Study completion
- 1 Sep 2028 (estimated)
- First posted
- 19 May 2022 (actual)
- Results
- Posted 2 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Growth hormone | Experimental | Drug: SomatropinThe participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations. |
Who may qualify
- Ages
- 9 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Be the specific subject with the a specific mutation in GHR leading to high GHBP.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SomatropinDaily growth hormone at a starting dose of 50 mcg/kg/day and escalating | Drug | Growth hormone |
Primary outcome
Growth Hormone Dose
Time frame: 1 year
Dose of growth hormone required in mg/kg/day to achieve an IGF-1 level above the mean
2 secondary outcomes
Growth Velocity
Time frame: 1 year
Height Standard Deviation Score Using Center for Disease Control (CDC) Growth Chart
Time frame: Baseline to 1 Year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38597155Merchant N, Houchin L, Boucher K, Dauber A. A Clinical Trial of High-Dose Growth Hormone in a Patient With a Dominant-Negative Growth Hormone Receptor Mutation. J Clin Endocrinol Metab. 2024 Oct 15;109(11):2937-2942. doi: 10.1210/clinem/dgae244.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew Dauber, MD principal investigator
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Somatropin in people with Growth Disorders, run by Children's National Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 2 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jul 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Growth Hormone Dose (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05382637, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2025.