Trial Outcomes & Findings for Augmenting Growth Hormone to Ameliorate Nonalcoholic Fatty Liver Disease in Adolescents (NCT NCT02726542)

NCT ID: NCT02726542

Last Updated: 2022-09-07

Results Overview

change in hepatic fat fraction between baseline and 24 weeks as measured by hydrogen magnetic resonance spectroscopy

• Recruitment status: COMPLETED
• Study phase: EARLY_PHASE1
• Target enrollment: 24 participants
• Primary outcome timeframe: 24 weeks
• Results posted on: 2022-09-07

Participant Flow

Participant milestones

Measure	Growth Hormone Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment (no study treatment - observation only)
Overall Study STARTED	13	11
Overall Study COMPLETED	10	10
Overall Study NOT COMPLETED	3	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Augmenting Growth Hormone to Ameliorate Nonalcoholic Fatty Liver Disease in Adolescents

Baseline characteristics by cohort

Measure	Growth Hormone n=13 Participants Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=11 Participants (no study treatment - observation only)	Total n=24 Participants Total of all reporting groups
Age, Continuous	25 years STANDARD_DEVIATION 3 • n=5 Participants	23 years STANDARD_DEVIATION 4 • n=7 Participants	24 years STANDARD_DEVIATION 4 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	6 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaskan Native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized White	9 Participants n=5 Participants	6 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Other or more than 1 race	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic (independent of race)	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Region of Enrollment United States	13 participants n=5 Participants	11 participants n=7 Participants	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Population: all available data used

change in hepatic fat fraction between baseline and 24 weeks as measured by hydrogen magnetic resonance spectroscopy

Outcome measures

Measure	Growth Hormone n=9 Participants Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=9 Participants (no study treatment - observation only)
Change in Hepatic Fat Fraction	-3.0 Percent (Hepatic fat fraction, percent) Standard Deviation 5.2	0.3 Percent (Hepatic fat fraction, percent) Standard Deviation 3.5

SECONDARY outcome

Timeframe: 24 weeks

Population: all available data used

change in AST between baseline and 24 weeks

Outcome measures

Measure	Growth Hormone n=10 Participants Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=10 Participants (no study treatment - observation only)
Change in Aspartate Aminotransferase (AST)	-3 units per liter Standard Deviation 9	-1 units per liter Standard Deviation 4

SECONDARY outcome

Timeframe: 24 weeks

Population: all available data used

change in ALT between baseline and 24 weeks

Outcome measures

Measure	Growth Hormone n=10 Participants Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=10 Participants (no study treatment - observation only)
Change in Alanine Aminotransferase (ALT)	-2 units per liter Standard Deviation 9	1 units per liter Standard Deviation 8

SECONDARY outcome

Timeframe: 24 weeks

Population: all available data used

change in GGT between baseline and 24 weeks

Outcome measures

Measure	Growth Hormone n=10 Participants Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=10 Participants (no study treatment - observation only)
Change in Gamma Glutamyl Transferase (GGT)	-9 units per liter Standard Deviation 30	5 units per liter Standard Deviation 9

SECONDARY outcome

Timeframe: 24 weeks

Population: all available data used

Change in visceral adipose tissue cross-sectional area at the 4th lumbar vertebra as measured by magnetic resonance imaging between baseline and 24 weeks

Outcome measures

Measure	Growth Hormone n=9 Participants Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=9 Participants (no study treatment - observation only)
Change in Visceral Adipose Tissue	-6 square centimeters Standard Deviation 50	6 square centimeters Standard Deviation 24

Adverse Events

Growth Hormone

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

No Treatment

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure	Growth Hormone n=13 participants at risk Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=11 participants at risk (no study treatment - observation only)
Surgical and medical procedures Hospitalization	7.7% 1/13 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.

Other adverse events

Measure	Growth Hormone n=13 participants at risk Somatropin given by daily subcutaneous injection. Dose will begin at 1mg and be titrated based on insulin-like growth factor 1 (IGF-1) levels. somatropin: Norditropin (growth hormone) given by injection using a pen-device	No Treatment n=11 participants at risk (no study treatment - observation only)
Nervous system disorders Headache transient	30.8% 4/13 • Number of events 5 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	9.1% 1/11 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
General disorders Injection Site Bruising	30.8% 4/13 • Number of events 6 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
Infections and infestations Upper Respiratory Tract Infection	15.4% 2/13 • Number of events 2 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
Skin and subcutaneous tissue disorders Acne	7.7% 1/13 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
Gastrointestinal disorders Gingival hyperplasia	7.7% 1/13 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
Immune system disorders Acute allergic reaction	7.7% 1/13 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
Musculoskeletal and connective tissue disorders Tightness in Jaw	7.7% 1/13 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.
Injury, poisoning and procedural complications Motor Vehicle Accident	7.7% 1/13 • Number of events 1 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.	0.00% 0/11 • Adverse events were collected from participants while they were enrolled in the 6 month study period. Participants were asked about adverse events at every study visit. Of note, participants receiving GH were seen for 4 additional visits compared to those receiving no treatment in this open-label study, and thus had more visits at which adverse events were assessed.

Additional Information

Takara Stanley, MD

Massachusetts General Hospital

Phone: 617-726-5312

Email: tstanley@mgh.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place