Growth Hormone DeficiencyPhase 2OraGrowtH212NCT04806854
PK and PD Study of LUM-201 in Children With Idiopathic Growth Hormone Deficiency: (OraGrowtH212)
A Phase 2 study testing LUM-201 in people with Growth Hormone Deficiency, run by Lumos Pharma at 1 site in Chile.
Trial summary
The goals of this single site trial are to study the pharmacokinetics (PK) and pharmacodynamics of LUM-201 and effects of LUM-201 administration on growth hormone release over time in children with idiopathic pediatric growth hormone deficiency (PGHD). Read more about this study
- Who is it for?
- Ages 4 to 9 years
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- LUM-201
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteChile
- Study dates
- 14 Jul 2021to Jul 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 4 to 9 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Have a chronological age ≥ 4.0 years and ≤ 10.0 years for girls and ≤ 12.0 years for boys.
- Have a minimum body weight of 16 kg at the time of screening.
- Have a bone age examination at screening or within the 6 months prior to screening that is delayed with respect to chronological age. Bone age should be based on the apparent BA of the proximal and distal phalanges, as opposed to the metacarpals, carpals and distal forearm. A central BA reader will make the final determination on BA eligibility.
- Have HT-SDS ≤ -2.0 or a HT-SDS ≥ 2 SD below mean parental HT-SDS. If the other data (slow HV, low IGF-1, peak GH < 10, and 6-month BA delay) are all consistent with idiopathic GHD, the subject may be enrolled with a height SDS > -2.0 after consultation with the MMs.
- Have an accurate baseline height velocity ≤ 5.5 cm/year based on at least 6-months of growth, if available
- Within the past 6 months have a maximal GH response > 3 and < 10 ng/mL from at least one prior GH stimulation test within the past 6 months
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- Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume < 4.0 mL in boys.
- Have an arm span to height ratio > 96.5%
- In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
- Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 30 days prior to the Baseline visit.
Who cannot join
- Any medical or genetic condition which, in the opinion of the PI or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment (e.g., diabetes, , idiopathic short stature (ISS), SHOX-deficiency, skeletal dysplasia, constitutional growth delay, SGA, other).
- A medical or genetic condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201 (e.g., scoliosis).
- Use of any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors (e.g., glucocorticoids).
- Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
- Prior treatment with growth factors including, but not limited to, GH, IGF-1, and GH secretagogues. (These may be used for limited times as a diagnostic test.)
- Suspicion of absence of pituitary function as evidenced by a maximal stimulated GH ≤ 3 ng/mL or pituitary hormone deficiencies beyond GH and thyroid function or a diagnosis of Organic PGHD.
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- At birth, gestational age < 36.0 weeks.
- Participation in any therapeutic trial of investigational drug(s) within the prior 6 months.
- History of spinal, cranial or total body irradiation.
- Recent commencement of non-stimulant therapy to treat attention deficit hyperactivity disorder (ADHD).
Trial locations and contacts
1 site in 1 country.
Chile1 site
Institute of Maternal and Child Research, University of Chile
Santiago
About this study
Official title: A Single-Center, Randomized, Open-Label, Parallel Arm Study of Daily Oral LUM-201 in Naive-to-Treatment, Prepubertal Children With Idiopathic Pediatric Growth Hormone Deficiency (PGHD)
The goals of this single site trial are to study the pharmacokinetics (PK) and pharmacodynamics of LUM-201 and effects of LUM-201 administration on growth hormone release over time in children with idiopathic pediatric growth hormone deficiency (PGHD).
Read the detailed description
This trial will have a single screening visit to assess if subjects are eligible to start study therapy. Once subjects have completed screening, and if they are determined to be eligible, they will return to the clinic for a 12-hour pulsatility study. On Day Two of the trial, subjects will be randomized to receive one of two oral daily doses of LUM-201 and PK samples will be collected. All subjects will have an equal chance of being placed in either of the two groups.
The trial consists of treatment until subjects achieve near adult height. After screening, subjects will return to the clinic monthly for the first 6 visits and every three months thereafter. At most clinic visits, subjects will have a physical exam, blood and urine collections. At the Month 6 visit subjects will repeat the 12-hour pulsatility and PK studies.
- Conditions
- Growth Hormone Deficiency
- Being tested
- LUM-201
- Keywords
- GHD, PGHD, LUM-201, growth hormone secretagogue, height, catch-up growth, pulsatility, pharmacokinetics, pharmacodynamics, PK, PD, idiopathic
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUM-201Drug
Administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| LUM-201 (1.6 mg/kg/day) | Experimental | Not described Receives: Drug: LUM-201 |
| LUM-201 (3.2 mg/kg/day) | Experimental | Not described Receives: Drug: LUM-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of Augmented Growth Hormone (GH) Pulsatility
12-hour mean GH concentration
Measured: Day 1 to Month 6
Pharmacokinetics of LUM-201 and M8
Plasma concentrations (Cmax/Steady State)
Measured: Day 1 to Month 6
Secondary outcomes (2)
Incidence of adverse events in children with idiopathic GHD
Number of events
Measured: up to 8 years
Height standard deviation score (SDS)
Change in HT-SDS from Baseline to Month 6 and Month 12
Measured: Day 1 to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2021
- Study start14 Jul 2021
- Primary completionApr 2030 (estimated)
- Study completionJul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lumos Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04806854
- Sponsor's protocol ID
- LUM-201-03
- Acronym
- OraGrowtH212
- Status last verified
- Oct 2023
- First submitted
- 17 Mar 2021
- Last updated
- 4 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Center, Randomized, Open-Label, Parallel Arm Study of Daily Oral LUM-201 in Naive-to-Treatment, Prepubertal Children With Idiopathic Pediatric Growth Hormone Deficiency (PGHD)
- ClinicalTrials.gov ID
- NCT04806854
- Acronym / sponsor ID
- OraGrowtH212 / LUM-201-03
- Conditions
- Growth Hormone Deficiency
- Keywords
- GHD, PGHD, LUM-201, growth hormone secretagogue, height, catch-up growth, pulsatility, pharmacokinetics, pharmacodynamics, PK, PD, idiopathic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Lumos PharmaIndustry (pharma, biotech, devices)
- Study start
- 14 Jul 2021 (actual)
- Primary completion
- Apr 2030 (estimated)
- Study completion
- Jul 2030 (estimated)
- First posted
- 19 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goals of this single site trial are to study the pharmacokinetics (PK) and pharmacodynamics of LUM-201 and effects of LUM-201 administration on growth hormone release over time in children with idiopathic pediatric growth hormone deficiency (PGHD).
Detailed description
This trial will have a single screening visit to assess if subjects are eligible to start study therapy. Once subjects have completed screening, and if they are determined to be eligible, they will return to the clinic for a 12-hour pulsatility study. On Day Two of the trial, subjects will be randomized to receive one of two oral daily doses of LUM-201 and PK samples will be collected. All subjects will have an equal chance of being placed in either of the two groups.
The trial consists of treatment until subjects achieve near adult height. After screening, subjects will return to the clinic monthly for the first 6 visits and every three months thereafter. At most clinic visits, subjects will have a physical exam, blood and urine collections. At the Month 6 visit subjects will repeat the 12-hour pulsatility and PK studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LUM-201 (1.6 mg/kg/day) | Experimental | Drug: LUM-201 |
| LUM-201 (3.2 mg/kg/day) | Experimental | Drug: LUM-201 |
Who may qualify
- Ages
- 4 to 9 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Have a chronological age ≥ 4.0 years and ≤ 10.0 years for girls and ≤ 12.0 years for boys.
- Have a minimum body weight of 16 kg at the time of screening.
- Have a bone age examination at screening or within the 6 months prior to screening that is delayed with respect to chronological age. Bone age should be based on the apparent BA of the proximal and distal phalanges, as opposed to the metacarpals, carpals and distal forearm. A central BA reader will make the final determination on BA eligibility.
- Have HT-SDS ≤ -2.0 or a HT-SDS ≥ 2 SD below mean parental HT-SDS. If the other data (slow HV, low IGF-1, peak GH < 10, and 6-month BA delay) are all consistent with idiopathic GHD, the subject may be enrolled with a height SDS > -2.0 after consultation with the MMs.
- Have an accurate baseline height velocity ≤ 5.5 cm/year based on at least 6-months of growth, if available
Key exclusion criteria (10 listed)
- Any medical or genetic condition which, in the opinion of the PI or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment (e.g., diabetes, , idiopathic short stature (ISS), SHOX-deficiency, skeletal dysplasia, constitutional growth delay, SGA, other).
- A medical or genetic condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201 (e.g., scoliosis).
- Use of any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors (e.g., glucocorticoids).
- Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
- Prior treatment with growth factors including, but not limited to, GH, IGF-1, and GH secretagogues. (These may be used for limited times as a diagnostic test.)
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 |
|---|---|---|
| LUM-201Administered orally once daily | Drug | LUM-201 (1.6 mg/kg/day), LUM-201 (3.2 mg/kg/day) |
Primary outcome
Evaluation of Augmented Growth Hormone (GH) Pulsatility
Time frame: Day 1 to Month 6
12-hour mean GH concentration
Pharmacokinetics of LUM-201 and M8
Time frame: Day 1 to Month 6
Plasma concentrations (Cmax/Steady State)
2 secondary outcomes
Incidence of adverse events in children with idiopathic GHD
Time frame: up to 8 years
Height standard deviation score (SDS)
Time frame: Day 1 to Month 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2023.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
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 LUM-201 in people with Growth Hormone Deficiency, run by Lumos Pharma at 1 site in Chile.
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 10 inclusion and 10 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 4 Oct 2023. 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 Chile. 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?
This study is still under way. Results are not available in the source record yet.
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?
Evaluation of Augmented Growth Hormone (GH) Pulsatility (time frame: Day 1 to Month 6). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Oct 2023.