Trial · NCT01927861

Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome

NCT01927861, Phase 3, Completed, Novo Nordisk A/S; results first posted 2019-10-14

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome. Trials, record NCT01927861. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01927861

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
NCT number: NCT01927861
Status: Completed
Phases: Phase 3
Enrollment: 51 participants, actual
Lead sponsor: Novo Nordisk A/S
Conditions: Genetic Disorder, Noonan Syndrome
Interventions: somatropin
First posted: 2013-08-23
Last updated: 2020-08-10
Results first posted: 2019-10-14

Source ClinicalTrials.gov

Registered outcomes67

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Height SDS (Japanese National Reference Data)	Baseline, week 104	Height SDS was calculated using the formula: SDS = (height - mean)/SD, where height was the height variable measured, mean and SD of height by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height and negative SDS indicated lesser height than the mean of the reference population. The change from baseline (week 0) in the height SDS after 104 weeks of treatment was analysed using an analysis of covariance (ANCOVA) model with treatment as a fixed effect and baseline height SDS as a covariate. Positive value in change from baseline indicated that SDS was better than baseline SDS. Missing values were imputed using the last observation carried forward (LOCF) method.
Secondary	Height Velocity SDS	Baseline to week 52	Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 52) and height at baseline (week 0) divided by time between those measurement time points and multiplied by 365 days. Height velocity SDS was calculated using the formula: SDS = (height velocity - mean)/SD, where height velocity was the height velocity variable measured, mean and SD of height velocity by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height velocity and negative SDS indicated lesser height velocity than the mean of the reference population.
Secondary	Height Velocity SDS	Week 52 to week 104	Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 104) and height at week 52 divided by time between those measurement time points and multiplied by 365 days. Height velocity SDS was calculated using the formula: SDS = (height velocity - mean)/SD, where height velocity was the height velocity variable measured, mean and SD of height velocity by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height velocity and negative SDS indicated lesser height velocity than the mean of the reference population.
Secondary	Height Velocity	Baseline to week 52	Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 52) and height at baseline (week 0) divided by time between those measurement time points and multiplied by 365 days.
Secondary	Height Velocity	Week 52 to week 104	Height velocity is change in height per year. The height velocity was calculated as the difference between current height (week 104) and height at week 52 divided by time between those measurement time points and multiplied by 365 days.
Secondary	Incidence of Treatment Emergent Adverse Events	During 104 weeks of treatment	A treatment emergent adverse event (TEAE; for the pivotal phase) was defined as an event that had onset date on or after the date of visit 2 (week 0; start of treatment) and no later than the date of visit 12 (104 weeks; end of pivotal phase). For withdrawal participants (if any), an adverse event with onset date no later than 7 days after the last day of NN-220 treatment was included.
Secondary	Change in IGF-I (Insulin-like Growth Factor-I)	Baseline, week 104	Change from baseline (within 4 weeks prior to week 0) in IGF-I was evaluated after 104 weeks of treatment. Missing values were imputed using the LOCF method.
Secondary	Change in HbA1c (Glycosylated Haemoglobin)	Baseline, week 104	Change from baseline (within 4 weeks prior to week 0) in HbA1c was evaluated after 104 weeks of treatment.
Secondary	Change in Clinical Laboratory Tests (Haematology: Erythrocytes)	Baseline, week 104	Change from baseline (within 4 weeks prior to week 0) in haematological parameter - erythrocytes. Missing values were imputed using the LOCF method.
Secondary	Change in Clinical Laboratory Tests (Haematology: Leukocytes and Thrombocytes)	Baseline, week 104	Change from baseline (within 4 weeks prior to week 0) in haematological parameter - leukocytes and thrombocytes. Missing values were imputed using the LOCF method.

1 to 10 of 67Source ClinicalTrials.gov

See all 67 registered outcomes

Outcomes67

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Height SDS (Japanese National Reference Data)	Baseline, week 104	FAS.	Standard deviation score	LEAST_SQUARES_MEAN
SECONDARY	Height Velocity SDS	Baseline to week 52	FAS.	Standard deviation score	MEAN
SECONDARY	Height Velocity SDS	Week 52 to week 104	FAS.	Standard deviation score	MEAN
SECONDARY	Height Velocity	Baseline to week 52	FAS.	Centimeters per year (cm/year)	MEAN
SECONDARY	Height Velocity	Week 52 to week 104	FAS.	cm/year	MEAN
SECONDARY	Incidence of Treatment Emergent Adverse Events	During 104 weeks of treatment	Safety analysis set (SAS) which included all participants who received at least one dose of trial product (NN-220, 0.033 mg/kg/day or NN-220, 0.066 mg/kg/day).	Events	NUMBER
SECONDARY	Change in IGF-I (Insulin-like Growth Factor-I)	Baseline, week 104	SAS.	Nanograms per milliliter (ng/mL)	MEAN
SECONDARY	Change in HbA1c (Glycosylated Haemoglobin)	Baseline, week 104	SAS.	Percentage of HbA1c	MEAN
SECONDARY	Change in Clinical Laboratory Tests (Haematology: Erythrocytes)	Baseline, week 104	SAS.	10^12 cells per liter (10^12 cells/L)	MEAN
SECONDARY	Change in Clinical Laboratory Tests (Haematology: Leukocytes and Thrombocytes)	Baseline, week 104	SAS. Number analyzed = participants with available data.	10^9 cells per liter (10^9 cells/L)	MEAN

1 to 10 of 67Source ClinicalTrials.gov

See all 67 outcomes

Results by group322

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change in Height SDS (Japanese National Reference Data)	NN-220 0.033 mg/kg/Day	0.84	Standard Error 0.09	Standard deviation score
Change in Height SDS (Japanese National Reference Data)	NN-220 0.066 mg/kg/Day	1.47	Standard Error 0.09	Standard deviation score
Height Velocity SDS	NN-220 0.033 mg/kg/Day	2.80	Standard Deviation 1.13	Standard deviation score
Height Velocity SDS	NN-220 0.066 mg/kg/Day	5.01	Standard Deviation 1.92	Standard deviation score
Height Velocity SDS	NN-220 0.033 mg/kg/Day	0.58	Standard Deviation 1.59	Standard deviation score
Height Velocity SDS	NN-220 0.066 mg/kg/Day	2.65	Standard Deviation 1.76	Standard deviation score
Height Velocity	NN-220 0.033 mg/kg/Day	7.88	Standard Deviation 0.87	Centimeters per year (cm/year)
Height Velocity	NN-220 0.066 mg/kg/Day	9.90	Standard Deviation 1.49	Centimeters per year (cm/year)
Height Velocity	NN-220 0.033 mg/kg/Day	6.20	Standard Deviation 0.95	cm/year
Height Velocity	NN-220 0.066 mg/kg/Day	7.99	Standard Deviation 1.20	cm/year

1 to 10 of 322Source ClinicalTrials.gov

See all 322 results by group

Participant flow18

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Pivotal Phase (104 Weeks)	STARTED	NN-220 0.033 mg/kg/Day	25
Pivotal Phase (104 Weeks)	STARTED	NN-220 0.066 mg/kg/Day	26
Pivotal Phase (104 Weeks)	COMPLETED	NN-220 0.033 mg/kg/Day	25
Pivotal Phase (104 Weeks)	COMPLETED	NN-220 0.066 mg/kg/Day	26
Pivotal Phase (104 Weeks)	NOT COMPLETED	NN-220 0.033 mg/kg/Day	0
Pivotal Phase (104 Weeks)	NOT COMPLETED	NN-220 0.066 mg/kg/Day	0
Extension Phase (104 Weeks)	STARTED	NN-220 0.033 mg/kg/Day	25
Extension Phase (104 Weeks)	STARTED	NN-220 0.066 mg/kg/Day	26
Extension Phase (104 Weeks)	COMPLETED	NN-220 0.033 mg/kg/Day	25
Extension Phase (104 Weeks)	COMPLETED	NN-220 0.066 mg/kg/Day	23

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events148

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Abdominal pain	Gastrointestinal disorders	0.033 mg/kg/Day	2	25	2
other	Abdominal pain	Gastrointestinal disorders	0.066 mg/kg/Day	2	26	10
other	Arthralgia	Musculoskeletal and connective tissue disorders	0.033 mg/kg/Day	2	25	2
other	Arthralgia	Musculoskeletal and connective tissue disorders	0.066 mg/kg/Day	2	26	2
other	Arthropod bite	Injury, poisoning and procedural complications	0.033 mg/kg/Day	2	25	2
other	Arthropod bite	Injury, poisoning and procedural complications	0.066 mg/kg/Day	3	26	8
other	Arthropod sting	Injury, poisoning and procedural complications	0.033 mg/kg/Day	0	25	0
other	Arthropod sting	Injury, poisoning and procedural complications	0.066 mg/kg/Day	2	26	3
other	Asthma	Respiratory, thoracic and mediastinal disorders	0.033 mg/kg/Day	3	25	4
other	Asthma	Respiratory, thoracic and mediastinal disorders	0.066 mg/kg/Day	2	26	3

1 to 10 of 148Source ClinicalTrials.gov

See all 148 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	0.033 mg/kg/Day	0	25
Total, all-cause mortality	0.066 mg/kg/Day	0	26
Total, other adverse events	0.033 mg/kg/Day	24	25
Total, other adverse events	0.066 mg/kg/Day	26	26
Total, serious adverse events	0.033 mg/kg/Day	9	25
Total, serious adverse events	0.066 mg/kg/Day	10	26

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
29109363	RESULT	PubMed
32269181	RESULT	PubMed
41285480	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
0.033 mg/kg/Day vs 0.066 mg/kg/Day: deaths from any cause	Trial comparisons
0.033 mg/kg/Day vs 0.066 mg/kg/Day: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01927861

GET https://conductscience.org/api/trials/v1/NCT01927861/registered?page=2

API reference For AI assistants