Trial Outcomes & Findings for Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213) (NCT NCT05250063)
NCT ID: NCT05250063
Last Updated: 2026-05-15
Results Overview
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
COMPLETED
PHASE2
15 participants
Day 1 to Month 12
2026-05-15
Participant Flow
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).
Participant milestones
| Measure |
LUM-201 (3.2 mg/kg/Day)
LUM-201: Administered orally once daily
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)
Baseline characteristics by cohort
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=15 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=11 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=11 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=11 Participants
|
|
Age, Continuous
|
102 months
STANDARD_DEVIATION 25.5 • n=11 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=11 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=11 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=11 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=11 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=11 Participants
|
|
Region of Enrollment
Poland
|
6 participants
n=11 Participants
|
|
Baseline Height-Standard Deviation Score (SDS)
|
-1.32492 Height-SDS
STANDARD_DEVIATION 0.37944 • n=11 Participants
|
|
Baseline Insulin-like growth factor 1 (IGF-1) SDS
|
0.9343 SDS
STANDARD_DEVIATION 1.1716 • n=11 Participants
|
PRIMARY outcome
Timeframe: Day 1 to Month 12Population: Per Protocol Population Analyzed
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
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=9 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer
|
0.098 cm/year
Standard Deviation 0.775
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: Summary of Treatment Emergent Adverse Events by System Organ Class and Preferred Term Intent to Treat Population
Number of events
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=15 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD)
|
42 occurences
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: Blood samples were unable to be collected/analyzed for various subjects/timepoints. Per Protocol Population Analyzed
Mean GH Values change from Day 1 to month 12 (Pre-Dose and 1 hour Post-Dose)
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Growth Hormone (GH) Values
Month 12, Pre-Dose
|
-0.05 ng/ml
Standard Deviation 0.68
|
|
Growth Hormone (GH) Values
Month 12, 1 hour Post-Dose
|
1.17 ng/ml
Standard Deviation 2.81
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: Per Protocol Population Analyzed
Mean change from Day 1 to month 12 (IGF-1 Values Pre-Dose)
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Insulin-Like Growth Factor 1 (IGF-1) Values
|
-8.75 nmol/L
Standard Deviation 14.59
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: Per Protocol Population Analyzed
Mean change from day 1 to month 12 (IGFBP-3 Values Pre-Dose)
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values
|
-58.033 nmol/L
Standard Deviation 33.092
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: 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
Mean change from day 1 to month 12 in HT-SDS
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Height (HT) Standard Deviation Score (SDS)
|
0.08229 Standard Deviation Score
Standard Deviation 0.20574
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: Per Protocol Population Analyzed
Mean change in Weight from day 1 to month 12
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Change in Weight
|
4.19 kg
Standard Deviation 1.74
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: 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
Mean change in Weight-SDS from day 1 to month 12
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Change in Weight - Standard Deviation Score (SDS)
|
0.62279 Standard Deviation Score
Standard Deviation 0.30934
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: Per Protocol Population Analyzed
Mean change from day 1 to month 12 in BMI
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Change in Body Mass Index (BMI)
|
0.969 kg/m^2
Standard Deviation 0.695
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: 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
Mean change from Day 1 to month 12 in BMI-SDS
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Change in Body Mass Index (BMI) - Standard Deviation Score (SDS)
|
0.32057 Standard Deviation Score
Standard Deviation 0.30768
|
SECONDARY outcome
Timeframe: Month 6Population: Per Protocol Population Analyzed
Mean change from day 1 to month 6 in bone age, measured by X-ray of left hand and wrist using Greulich \& Pyle atlas
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Change in Bone Age
|
2.01 year
Standard Deviation 0.81
|
SECONDARY outcome
Timeframe: Day 1 to Month 12Population: 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
Mean Change from day 1 to month 12 for IGF-1 - SDS Pre-dose Values
Outcome measures
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=10 Participants
LUM-201: Administered orally once daily
|
|---|---|
|
Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS)
|
-1.1083 Standard Deviation Score
Standard Deviation 1.1529
|
Adverse Events
LUM-201 (3.2 mg/kg/Day)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
LUM-201 (3.2 mg/kg/Day)
n=15 participants at risk
LUM-201: Administered orally once daily
|
|---|---|
|
Infections and infestations
Respiratory Infection
|
6.7%
1/15 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Number of events 3 • 1 year
|
|
Immune system disorders
Drug hypersensitivity
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Bronchitis
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Ear infections
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Hordeolum
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Influenza
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
Infections and infestations
Pharyngitis
|
13.3%
2/15 • Number of events 4 • 1 year
|
|
Infections and infestations
Pharyngitis streptococcal
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Tooth abscess
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Infections and infestations
Upper respiratory tract infections
|
20.0%
3/15 • Number of events 4 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
Radius fracture
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Investigations
Body temperature increased
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Increased appetite
|
13.3%
2/15 • Number of events 3 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.7%
1/15 • Number of events 2 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Joint noise
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Nervous system disorders
Headache
|
6.7%
1/15 • Number of events 2 • 1 year
|
|
Psychiatric disorders
Irritability
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
13.3%
2/15 • Number of events 2 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Lichen planus
|
6.7%
1/15 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.3%
2/15 • Number of events 3 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60