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New treatment option for Growth Hormone Deficiency (GHD)
Official title Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
ClinicalTrials.gov ID: NCT06948214
What this study is testing
What is LUM-201?
LUM-201 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for growth hormone deficiency (ghd).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The OraGrowtH Phase 3 Trial is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate the LUM-201 predictive enrichment marker (LUM-201 PEM) strategy to select subjects likely to respond to therapy with daily oral LUM-201.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 11
You may be able to join if
- people must be naïve to treatment and prepubertal
- people must have a maximal GH response of \< 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
- Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
- Morning or random cortisol level of ≥ 7.0 μg/dL
- ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
You likely can't join if
- Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature...
- Arm span to height ratio \> 2 SDs below the mean for age and sex
- A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201
- Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous...
- Current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the Screening...
- Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency
See the full eligibility criteria
- people must be naïve to treatment and prepubertal
- people must have a maximal GH response of \< 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
- Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
- Morning or random cortisol level of ≥ 7.0 μg/dL
- ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
- Baseline height velocity (HV) based on ≥ 6 months of growth assessments \< 25th percentile for age and sex
- Bone Age delay of ≥ 12 months compared to the chronological age
- 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. people diagnosed with hypothyroidism must have documented successful treatment for at least 3 months prior to Day 1
- Baseline IGF-1 standard deviation score (SDS) ≤ -1.0
- Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment.
- Arm span to height ratio \> 2 SDs below the mean for age and sex
- A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201
- Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors
- Current inflammatory diseases requiring systemic corticosteroid treatment for \> 2 consecutive weeks within the last 3 months prior to the Screening Visit
- Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency
- Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM
- Any people suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit
- Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments
- Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin \> upper limit of normal (ULN)
- Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months
- Body weight ≤ 14.0 kg
- BMI \ +2 SDs for age and sex based on WHO standards
- Birth weight for gestational age \< 3rd percentile based on WHO standards
- Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4
- History of spinal, cranial, or total body irradiation
- Attention deficit hyperactivity disorder (ADHD) diagnosis
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Madera, California, United States
- Orange, California, United States
- Sacramento, California, United States
- San Diego, California, United States
- Centennial, Colorado, United States
- Greenwood Village, Colorado, United States
- Washington D.C., District of Columbia, United States
- Hollywood, Florida, United States
- Miami, Florida, United States
- Orlando, Florida, United States
- Tallahassee, Florida, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- New Orleans, Louisiana, United States
- Minneapolis, Minnesota, United States
- Kansas City, Missouri, United States
- Las Vegas, Nevada, United States
- New Brunswick, New Jersey, United States
- Staten Island, New York, United States
+ 21 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Madera, California, United States; Orange, California, United States; Sacramento, California, United States; San Diego, California, United States; Centennial, Colorado, United States and 35 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.