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This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.
This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable.
The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LUM-201 (3.2 mg/kg/day) | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM-201 | Drug | Administered orally once daily |
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| Measure | Description | Time Frame |
|---|---|---|
| Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer | Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12 | Day 1 to Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD) | Number of events | Day 1 to Month 12 |
| Growth Hormone (GH) Values | Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rady Children's Hospital | San Diego | California | 92123 | United States | ||
| Children's National Hospital |
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Each screened subject will enter the Subject Number previously assigned in the LUM-201-01 trial, at the Screening/Baseline visit (visit 1). Subject Numbers will not be reassigned. All subjects will receive a single oral 3.2 mg/kg dose of LUM-201 at the Screening/Baseline visit (visit 1) which will include measurement of GH concentrations at baseline and 60 ± 10 minutes after dosing. All subjects will commence their dosing at the Screening/Baseline visit (visit 1).
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| ID | Title | Description |
|---|---|---|
| FG000 | LUM-201 (3.2 mg/kg/Day) | LUM-201: Administered orally once daily |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 19, 2022 | Mar 4, 2026 |
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| Day 1 to Month 12 |
| Insulin-Like Growth Factor 1 (IGF-1) Values | Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose) | Day 1 to Month 12 |
| Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values | Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose) | Day 1 to Month 12 |
| Height (HT) Standard Deviation Score (SDS) | Mean change from day 1 to month 12 in HT-SDS | Day 1 to Month 12 |
| Change in Weight | Mean change in Weight from day 1 to month 12 | Day 1 to Month 12 |
| Change in Weight - Standard Deviation Score (SDS) | Mean change in Weight-SDS from day 1 to month 12 | Day 1 to Month 12 |
| Change in Body Mass Index (BMI) | Mean change from day 1 to month 12 in BMI | Day 1 to Month 12 |
| Change in Body Mass Index (BMI) - Standard Deviation Score (SDS) | Mean change from Day 1 to month 12 in BMI-SDS | Day 1 to Month 12 |
| Change in Bone Age | Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas | Month 6 |
| Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS) | Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values | Day 1 to Month 12 |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Indiana University School of Medicine | Indianapolis | Indiana | 46202 | United States |
| M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic | Minneapolis | Minnesota | 55454 | United States |
| The Children's Mercy Hospital | Kansas City | Missouri | 64111 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| University of Virginia Health System | Charlottesville | Virginia | 22908 | United States |
| Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki | Lodz | Poland |
| Sonomed - Centrum Medyczne | Szczecin | Poland |
| Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka | Warsaw | Poland |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | LUM-201 (3.2 mg/kg/Day) | LUM-201: Administered orally once daily |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | months |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Baseline Height-Standard Deviation Score (SDS) | This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. | Mean | Standard Deviation | Height-SDS |
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| Baseline Insulin-like growth factor 1 (IGF-1) SDS | This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. | Mean | Standard Deviation | SDS |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer | Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12 | Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | cm/year | Day 1 to Month 12 |
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| Secondary | Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD) | Number of events | Summary of Treatment Emergent Adverse Events by System Organ Class and Preferred Term Intent to Treat Population | Posted | Number | occurences | Day 1 to Month 12 |
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| Secondary | Growth Hormone (GH) Values | Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose) | Blood samples were unable to be collected/analyzed for various subjects/timepoints. Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | ng/ml | Day 1 to Month 12 |
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| Secondary | Insulin-Like Growth Factor 1 (IGF-1) Values | Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose) | Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | nmol/L | Day 1 to Month 12 |
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| Secondary | Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values | Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose) | Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | nmol/L | Day 1 to Month 12 |
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| Secondary | Height (HT) Standard Deviation Score (SDS) | Mean change from day 1 to month 12 in HT-SDS | This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease. Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | Standard Deviation Score | Day 1 to Month 12 |
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| Secondary | Change in Weight | Mean change in Weight from day 1 to month 12 | Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | kg | Day 1 to Month 12 |
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| Secondary | Change in Weight - Standard Deviation Score (SDS) | Mean change in Weight-SDS from day 1 to month 12 | This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease. Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | Standard Deviation Score | Day 1 to Month 12 |
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| Secondary | Change in Body Mass Index (BMI) | Mean change from day 1 to month 12 in BMI | Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | kg/m^2 | Day 1 to Month 12 |
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| Secondary | Change in Body Mass Index (BMI) - Standard Deviation Score (SDS) | Mean change from Day 1 to month 12 in BMI-SDS | This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease. Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | Standard Deviation Score | Day 1 to Month 12 |
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| Secondary | Change in Bone Age | Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas | Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | year | Month 6 |
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| Secondary | Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS) | Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values | This is not a Z-Score analysis. Standard deviation score of 0 is the population mean for the age/sex. Normal range is typically -2 to +2 SDS. A positive score is an improvement and a negative score a decrease. Per Protocol Population Analyzed | Posted | Mean | Standard Deviation | Standard Deviation Score | Day 1 to Month 12 |
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1 year
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | LUM-201 (3.2 mg/kg/Day) | LUM-201: Administered orally once daily | 0 | 15 | 0 | 15 | 13 | 15 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain upper | Gastrointestinal disorders | Systematic Assessment |
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| Pyrexia | General disorders | Systematic Assessment |
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| Drug hypersensitivity | Immune system disorders | Systematic Assessment |
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| Bronchitis | Infections and infestations | Systematic Assessment |
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| Ear infections | Infections and infestations | Systematic Assessment |
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| Hordeolum | Infections and infestations | Systematic Assessment |
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| Influenza | Infections and infestations | Systematic Assessment |
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| Pharyngitis | Infections and infestations | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | Systematic Assessment |
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| Tooth abscess | Infections and infestations | Systematic Assessment |
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| Upper respiratory tract infections | Infections and infestations | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | Systematic Assessment |
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| Radius fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Body temperature increased | Investigations | Systematic Assessment |
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| Increased appetite | Metabolism and nutrition disorders | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Joint noise | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Irritability | Psychiatric disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Lichen planus | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Respiratory Infection | Infections and infestations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Lumos Pharma LLC | Lumos Pharma LLC | 515-598-2921 | clinical.trials@lumos-pharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 30, 2025 | Mar 4, 2026 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D001849 | Bone Diseases, Endocrine |
| D007018 | Hypopituitarism |
| D010900 | Pituitary Diseases |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D004700 | Endocrine System Diseases |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Month 12, 1 hour Post-Dose |
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