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A Study of Tadalafil in Pediatric Participants With Pulmonary Arterial Hypertension (PAH)

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ClinicalTrials.gov Identifier: NCT01824290
Recruitment Status : Completed
First Posted : April 4, 2013
Results First Posted : March 23, 2020
Last Update Posted : November 5, 2021
Sponsor:
Information provided by (Responsible Party):
Eli Lilly and Company

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Hypertension, Pulmonary
Interventions Drug: Tadalafil
Drug: Placebo
Drug: ERA as specific PAH treatment
Enrollment 35
Recruitment Details  
Pre-assignment Details Per protocol and statistical analysis plan (SAP), the primary and secondary analysis from period 1 were performed to compare all tadalafil participants together versus all placebo participants together.
Arm/Group Title Placebo Tadalafil Placebo/Tadalafil Tadalafil/Tadalafil
Hide Arm/Group Description Period 1: Participants received placebo orally by tablets once a day. Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received placebo during period 1.

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received tadalafil during period 1.

Period Title: Period 1: Double Blind
Started 18 17 0 0
Received at Least One Dose of Study Drug 18 17 0 0
Received 20 mg 0 4 0 0
Received 40 mg 0 13 0 0
Completed 15 15 0 0
Not Completed 3 2 0 0
Reason Not Completed
Entry Criteria Not Met             1             0             0             0
Parent/Caregiver Decision             1             1             0             0
Investigator Reported Clinical Worsening             1             1             0             0
Period Title: Period 2: Open-Label Treatment
Started 0 0 16 [1] 16 [1]
Received 20 mg 0 0 3 3
Received 40 mg 0 0 13 13
Completed 0 0 13 13
Not Completed 0 0 3 3
Reason Not Completed
Adverse Event             0             0             0             1
Parent/caregiver Decision             0             0             1             1
Withdrawal by Subject             0             0             2             1
[1]
As per the protocol, period 2 eligibility were participants who completed period 1 or experienced clinical worsening (CW) in period 1. 1 participant that had CW in period 1 continued to period 2.
Arm/Group Title Placebo Tadalafil Total
Hide Arm/Group Description Period 1: Participants received placebo orally by tablets once a day. Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day. Total of all reporting groups
Overall Number of Baseline Participants 18 17 35
Hide Baseline Analysis Population Description
All participants who received at least one dose of study drug. Per protocol and statistical analysis plan (SAP), the primary and secondary analysis were performed to compare all tadalafil participants together versus all placebo participants together.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 18 participants 17 participants 35 participants
12.8  (3.39) 14.1  (3.49) 13.5  (3.45)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 17 participants 35 participants
Female 9 10 19
Male 9 7 16
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 17 participants 35 participants
Hispanic or Latino 7 8 15
Not Hispanic or Latino 6 4 10
Unknown or Not Reported 5 5 10
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 17 participants 35 participants
American Indian or Alaska Native 1 3 4
Asian 1 1 2
Native Hawaiian or Other Pacific Islander 0 0 0
Black or African American 2 1 3
White 14 12 26
More than one race 0 0 0
Unknown or Not Reported 0 0 0
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 18 participants 17 participants 35 participants
Japan 1 1 2
Turkey 2 1 3
Brazil 9 6 15
Mexico 1 4 5
Israel 2 4 6
France 1 1 2
Germany 1 0 1
Poland 1 0 1
6 Minute Walk Distance  
Mean (Standard Deviation)
Unit of measure:  Meters
Number Analyzed 18 participants 17 participants 35 participants
476.7  (105.11) 485.8  (160.231) 481.1  (132.77)
1.Primary Outcome
Title Period 1: Change From Baseline to Week 24 in a 6 Minute Walk (MW) Distance in Meters
Hide Description 6MWD in meters assessed in a subset of participants who are ≥6 to <18 years of age who are developmentally capable of performing a 6MW test. Change from baseline was derived using mixed model repeated measures (MMRM) with terms for treatment group, visit, baseline 6MWD, and treatment-by-visit interaction.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of study drug who were > = 6 to < 18 years of age and were capable of performing a 6MW test.
Arm/Group Title Placebo Tadalafil
Hide Arm/Group Description:
Period 1: Participants received placebo orally by tablets once a day.
Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.
Overall Number of Participants Analyzed 15 15
Least Squares Mean (Standard Error)
Unit of Measure: Meters
36.60  (20.776) 60.48  (20.410)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Tadalafil
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 23.88
Confidence Interval (2-Sided) 80%
-14.25 to 62.00
Parameter Dispersion
Type: Standard Error of the Mean
Value: 29.114
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Period 1: Time to Adjudicated Clinical Worsening (CW)
Hide Description Clinical worsening was defined as any of the following: death,lung or heart transplantation,atrial septostomy or Potts' shunt,hospitalization for Pulmonary Arterial Hypertension(PAH) progression,new onset syncope,initiation of new PAH therapy(including increase in the dose of existing PAH specific concomitant therapy,such as endothelin receptor agonist or beraprost medication), or increase of 1 or more in World Health Organization(WHO) Functional Class(except for participants already in Class IV;only for participants unable to perform the 6 minute walk(6MW) test;worsening of WHO functional class by 1 or more for participants who can perform a 6 minute walk(6MW) test and who have a decrease of ≥ 20% in the 6 minute walk distance(for those participants who are ≥6 years of age). Criteria for CW(from Period 1) were adjudicated by an independent,blinded study-specific Clinical Endpoint Committee(CEC).This adjudication was used for data analysis, and was not used to guide subject treatment.
Time Frame Baseline through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of study drug.
Arm/Group Title Placebo Tadalafil
Hide Arm/Group Description:
Period 1: Participants received placebo orally by tablets once a day.
Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.
Overall Number of Participants Analyzed 18 17
Median (95% Confidence Interval)
Unit of Measure: Weeks
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
[1]
There was no confirmed adjudicated CW case to report.
3.Secondary Outcome
Title Period 1: Percentage of Participants Who Experience CW
Hide Description Clinical worsening was defined as any of the following: death,lung or heart transplantation,atrial septostomy or Potts' shunt,hospitalization for Pulmonary Arterial Hypertension(PAH) progression,new onset syncope, initiation of new PAH therapy(including increase in the dose of existing PAH specific concomitant therapy,such as endothelin receptor agonist or beraprost medication),or increase of 1 or more in World Health Organization(WHO) Functional Class(except for participants already in Class IV; only for participants unable to perform the 6 minute walk(6MW) test;worsening of WHO functional class by 1 or more for participants who can perform a 6 minute walk(6MW) test and who have a decrease of ≥ 20% in the 6 minute walk distance(for those participants who are ≥6 years of age).Criteria for CW(from Period 1) were adjudicated by an independent,blinded study-specific Clinical Endpoint Committee(CEC).This adjudication was used for data analysis, and was not used to guide subject treatment.
Time Frame Baseline through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of study drug.
Arm/Group Title Placebo Tadalafil
Hide Arm/Group Description:
Period 1: Participants received placebo orally by tablets once a day.
Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered orally by tablets once a day.
Overall Number of Participants Analyzed 18 17
Measure Type: Number
Unit of Measure: percentage of participants
0 0
4.Secondary Outcome
Title Period 1: Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil at Steady-state
Hide Description Period 1: Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil at steady-state
Time Frame Week 2, Week 4, Week 16 and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least one dose of study drug and had evaluable PK data.
Arm/Group Title 20 mg Tadalafil 40 mg Tadalafil
Hide Arm/Group Description:
Period 1: 20 mg tadalafil administered orally by tablets once a day with concomitant endothelin receptor antagonist (ERA).
Period 1: 40 mg tadalafil administered orally by tablets once a day with concomitant ERA.
Overall Number of Participants Analyzed 4 13
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Liter Per Hour (L/hr)
With concomitant bosentan Number Analyzed 3 participants 13 participants
3.63
(38.1%)
4.49
(28.2%)
No bosentan (ERA: Macitentan) Number Analyzed 1 participants 0 participants
NA [1] 
(NA%)
[1]
For n = 1, geometric mean and geometric coefficient of variation could not be calculated. Individual value is 2.68.
5.Secondary Outcome
Title Period 2: Percentage of Participants Who Experience CW
Hide Description Clinical worsening was defined as any of the following: death, lung or heart transplantation, atrial septostomy or Potts' shunt, hospitalization for Pulmonary Arterial Hypertension (PAH) progression, new onset syncope, initiation of new PAH therapy (including increase in the dose of existing PAH specific concomitant therapy, such as endothelin receptor agonist or beraprost medication), or increase of 1 or more in World Health Organization(WHO) Functional Class (except for participants already in Class IV; only for participants unable to perform the 6 minute walk (6MW) test; worsening of WHO functional class by 1 or more for participants who can perform a 6 minute walk (6MW) test and who have a decrease of ≥ 20% in the 6 minute walk distance (for those participants who are ≥6 years of age).
Time Frame Period 2 Baseline through Study Completion (Up to 24 Months)
Hide Outcome Measure Data
Hide Analysis Population Description
Period 2: All participants who received at least one dose of study drug.
Arm/Group Title Placebo/Tadalafil - Open-Label Treatment Tadalafil/Tadalafil - Open-Label Treatment
Hide Arm/Group Description:

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received placebo during period 1.

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received tadalafil during period 1.

Overall Number of Participants Analyzed 16 16
Measure Type: Number
Unit of Measure: percentage of participants
12.5 18.8
6.Secondary Outcome
Title Period 2: Time to First Occurrence of CW
Hide Description Clinical worsening was defined as any of the following: death, lung or heart transplantation, atrial septostomy or Potts' shunt, hospitalization for Pulmonary Arterial Hypertension (PAH) progression, new onset syncope, initiation of new PAH therapy (including increase in the dose of existing PAH specific concomitant therapy, such as endothelin receptor agonist or beraprost medication), or increase of 1 or more in World Health Organization(WHO) Functional Class (except for participants already in Class IV; only for participants unable to perform the 6 minute walk (6MW) test; worsening of WHO functional class by 1 or more for participants who can perform a 6 minute walk (6MW) test and who have a decrease of ≥ 20% in the 6 minute walk distance (for those participants who are ≥6 years of age).
Time Frame Period 2 Baseline through Study Completion (Up to 24 Months)
Hide Outcome Measure Data
Hide Analysis Population Description
Period 2: All participants who received at least one dose of study drug, who entered the open-label treatment Period
Arm/Group Title Placebo/Tadalafil - Open-Label Treatment Tadalafil/Tadalafil - Open-Label Treatment
Hide Arm/Group Description:

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received placebo during period 1.

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received tadalafil during period 1.

Overall Number of Participants Analyzed 16 16
Median (95% Confidence Interval)
Unit of Measure: Months
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
[1]
Median and CI are not estimable due to inadequate events of Clinical Worsening (CW) using Kaplan-Meier survival Estimates.
Time Frame Baseline Up To 24 Months
Adverse Event Reporting Description All participants who received at least one dose of study drug. Gender specific events only occurring in male or female participants have had the number of participants at risk adjusted accordingly.
 
Arm/Group Title Placebo - Double Blind Tadalafil - Double Blind Placebo/Tadalafil - Open-Label Treatment Tadalafil/Tadalafil - Open-Label Treatment
Hide Arm/Group Description Period 1: Participants received placebo orally by tablets once a day. Period 1: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received placebo during period 1.

Period 2: 20 mg middle weight cohort or 40 mg for heavy weight cohort administered tadalafil orally by tablets once a day.

Participants had received tadalafil during period 1.

All-Cause Mortality
Placebo - Double Blind Tadalafil - Double Blind Placebo/Tadalafil - Open-Label Treatment Tadalafil/Tadalafil - Open-Label Treatment
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/18 (0.00%)      0/17 (0.00%)      0/16 (0.00%)      0/16 (0.00%)    
Hide Serious Adverse Events
Placebo - Double Blind Tadalafil - Double Blind Placebo/Tadalafil - Open-Label Treatment Tadalafil/Tadalafil - Open-Label Treatment
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/18 (0.00%)      0/17 (0.00%)      4/16 (25.00%)      1/16 (6.25%)    
Blood and lymphatic system disorders         
Anaemia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Cardiac disorders         
Acute right ventricular failure  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations         
Gastroenteritis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Pneumonia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Haemoptysis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
1
Term from vocabulary, MedDRA 24.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo - Double Blind Tadalafil - Double Blind Placebo/Tadalafil - Open-Label Treatment Tadalafil/Tadalafil - Open-Label Treatment
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/18 (44.44%)      15/17 (88.24%)      11/16 (68.75%)      12/16 (75.00%)    
Blood and lymphatic system disorders         
Anaemia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 2/16 (12.50%)  2 0/16 (0.00%)  0
Eosinophilia  1  1/18 (5.56%)  1 0/17 (0.00%)  0 0/16 (0.00%)  0 0/16 (0.00%)  0
Cardiac disorders         
Palpitations  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Supraventricular tachycardia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Tachycardia  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Ventricular extrasystoles  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Ear and labyrinth disorders         
Ear pain  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Eye disorders         
Asthenopia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Photopsia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Gastrointestinal disorders         
Abdominal pain upper  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 2/16 (12.50%)  2
Dental caries  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Enterocolitis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Gastrooesophageal reflux disease  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Irritable bowel syndrome  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Nausea  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Plicated tongue  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Vomiting  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 3/16 (18.75%)  3
General disorders         
Asthenia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Exercise tolerance decreased  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Pyrexia  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Immune system disorders         
Hypersensitivity  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Seasonal allergy  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  2
Infections and infestations         
Acute sinusitis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 2/16 (12.50%)  2 0/16 (0.00%)  0
Ascariasis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Body tinea  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Bronchitis  1  1/18 (5.56%)  1 0/17 (0.00%)  0 0/16 (0.00%)  0 0/16 (0.00%)  0
Ear infection  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  2
Gastroenteritis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Impetigo  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Influenza  1  0/18 (0.00%)  0 3/17 (17.65%)  3 0/16 (0.00%)  0 1/16 (6.25%)  1
Nasopharyngitis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  2 3/16 (18.75%)  4
Otitis media acute  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Paronychia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Pharyngitis  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 2/16 (12.50%)  4
Rhinitis  1  0/18 (0.00%)  0 1/17 (5.88%)  1 2/16 (12.50%)  2 0/16 (0.00%)  0
Sinusitis  1  1/18 (5.56%)  1 1/17 (5.88%)  1 0/16 (0.00%)  0 1/16 (6.25%)  2
Tonsillitis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Tracheobronchitis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Upper respiratory tract infection  1  1/18 (5.56%)  1 3/17 (17.65%)  3 0/16 (0.00%)  0 2/16 (12.50%)  2
Urinary tract infection  1  1/18 (5.56%)  1 1/17 (5.88%)  1 0/16 (0.00%)  0 1/16 (6.25%)  1
Vaginal infection  1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/8 (0.00%)  0 0/10 (0.00%)  0
Viral infection  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Viral tonsillitis  1  1/18 (5.56%)  1 0/17 (0.00%)  0 0/16 (0.00%)  0 0/16 (0.00%)  0
Viral upper respiratory tract infection  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Injury, poisoning and procedural complications         
Bone contusion  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Penis injury  1  0/9 (0.00%)  0 0/7 (0.00%)  0 1/8 (12.50%)  1 0/6 (0.00%)  0
Investigations         
Blood creatine phosphokinase increased  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Hepatic enzyme increased  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Metabolism and nutrition disorders         
Decreased appetite  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Dyslipidaemia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Vitamin b12 deficiency  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Vitamin d deficiency  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Musculoskeletal and connective tissue disorders         
Arthralgia  1  1/18 (5.56%)  1 2/17 (11.76%)  2 0/16 (0.00%)  0 2/16 (12.50%)  2
Back pain  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Musculoskeletal chest pain  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Pain in extremity  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Patellofemoral pain syndrome  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Pharyngeal neoplasm  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Nervous system disorders         
Dizziness  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 3/16 (18.75%)  3
Headache  1  2/18 (11.11%)  6 5/17 (29.41%)  5 2/16 (12.50%)  2 3/16 (18.75%)  3
Presyncope  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 1/16 (6.25%)  1
Somnolence  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Syncope  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Psychiatric disorders         
Anxiety  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Excessive masturbation  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Insomnia  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Reproductive system and breast disorders         
Menorrhagia  1  0/9 (0.00%)  0 1/10 (10.00%)  1 1/8 (12.50%)  1 1/10 (10.00%)  2
Metrorrhagia  1  0/9 (0.00%)  0 0/10 (0.00%)  0 0/8 (0.00%)  0 1/10 (10.00%)  1
Penis disorder  1  0/9 (0.00%)  0 1/7 (14.29%)  1 0/8 (0.00%)  0 0/6 (0.00%)  0
Polymenorrhoea  1  0/9 (0.00%)  0 0/10 (0.00%)  0 0/8 (0.00%)  0 1/10 (10.00%)  1
Spontaneous penile erection  1  0/9 (0.00%)  0 1/7 (14.29%)  2 1/8 (12.50%)  1 0/6 (0.00%)  0
Uterine haemorrhage  1  0/9 (0.00%)  0 0/10 (0.00%)  0 1/8 (12.50%)  1 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Cough  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Dyspnoea  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Epistaxis  1  1/18 (5.56%)  1 2/17 (11.76%)  2 1/16 (6.25%)  1 1/16 (6.25%)  1
Oropharyngeal pain  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 1/16 (6.25%)  1
Pulmonary arterial hypertension  1  1/18 (5.56%)  1 0/17 (0.00%)  0 0/16 (0.00%)  0 0/16 (0.00%)  0
Respiratory distress  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Skin and subcutaneous tissue disorders         
Dermatitis allergic  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  2
Haemorrhage subcutaneous  1  0/18 (0.00%)  0 1/17 (5.88%)  3 0/16 (0.00%)  0 0/16 (0.00%)  0
Livedo reticularis  1  1/18 (5.56%)  1 0/17 (0.00%)  0 0/16 (0.00%)  0 0/16 (0.00%)  0
Photosensitivity reaction  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Pruritus  1  0/18 (0.00%)  0 0/17 (0.00%)  0 1/16 (6.25%)  1 0/16 (0.00%)  0
Rash  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Swelling face  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Social circumstances         
Menarche  1  1/9 (11.11%)  1 0/10 (0.00%)  0 0/8 (0.00%)  0 0/10 (0.00%)  0
Vascular disorders         
Deep vein thrombosis  1  0/18 (0.00%)  0 0/17 (0.00%)  0 0/16 (0.00%)  0 1/16 (6.25%)  1
Flushing  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
Hypotension  1  0/18 (0.00%)  0 1/17 (5.88%)  1 0/16 (0.00%)  0 0/16 (0.00%)  0
1
Term from vocabulary, MedDRA 24.0
Indicates events were collected by systematic assessment
The study is mainly descriptive in a small number of children with PAH and there were no participants enrolled in the light weight cohort.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: Eli Lilly and Company
Phone: 800-545-5979
EMail: ClinicalTrials.gov@lilly.com
Layout table for additonal information
Responsible Party: Eli Lilly and Company
ClinicalTrials.gov Identifier: NCT01824290    
Other Study ID Numbers: 10609
H6D-MC-LVHV ( Other Identifier: Eli Lilly and Company )
2012-002354-23 ( EudraCT Number )
First Submitted: April 1, 2013
First Posted: April 4, 2013
Results First Submitted: March 6, 2020
Results First Posted: March 23, 2020
Last Update Posted: November 5, 2021