Oral Revatio in Pediatric PAH- Long term follow on study to A1481131

• Register: EUCTR
• Last refreshed on: 17 June 2013
• Main ID: EUCTR2005-000963-25-SE
• Date of registration: 06/04/2005
• Prospective Registration: Yes
• Primary sponsor: Pfizer Limited, Ramsgate Road, Sandwich, Kent, UK
• Public title: Oral Revatio in Pediatric PAH- Long term follow on study to A1481131
• Scientific title: A Multicenter, Long-Term Extension Study to Assess Safety of Oral Sildenafil in the Treatment of Subjects Who Have Completed Study A1481131 - N/A
• Date of first enrolment: 30/05/2005
• Target sample size: 200
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2005-000963-25
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Low, medium and high sildenafil dose groups Number of treatment arms in the trial: 3

Countries of recruitment

Brazil, Chile, Colombia, Costa Rica, Finland, Guatemala, Hungary, India, Italy, Malaysia, Mexico, Poland, Russian Federation, Sweden, Taiwan, Thailand, United Kingdom

Contacts

• Name: Clinical Trials gov Call cneter
• Affiliation: Pfizer Inc

• Name: Clinical Trials gov Call cneter
• Affiliation: Pfizer Inc

Key inclusion & exclusion criteria

Inclusion criteria:
· Pediatric subjects, who enrolled in and completed Study A1481131;
· Females of childbearing potential who are sexually active must be practicing a suitable method of birth control so that in the opinion of the investigator, they will not become pregnant during the study; and
· The investigator must obtain written informed consent and assent where applicable before the subject is considered for enrollment in Study A1481156. The inclusion of a subject more than once in the same clinical trial is not permissible.
All inclusion criteria must be adhered to.



Are the trial subjects under 18? yes
Number of subjects for this age range: 217
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
• Pediatric subjects, who did not complete Study A1481131.
• Subjects with known hereditary degenerative retinal disorders (such as retinitis
pigmentosa) or history of non-arteritic anterior ischemic optic neuropathy (NAION).

The exclusion criteria must be adhered to.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Circulatory and Respiratory Physiological Phenomena [G09]

Pulmonary Arterial Hypertension
MedDRA version: 14.1 Level: PT Classification code 10064911 Term: Pulmonary arterial hypertension System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders

Intervention(s)

Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: sildenafil (as citrate)
CAS Number: N/A
Other descriptive name: UK-92,480
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-

Trade Name: Revatio
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: sildenafil (as citrate)
CAS Number: N/A
Other descriptive name: UK-92,480
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Primary end point(s): Safety Parameters:
· Standard safety data;
· Ocular safety measures: Change from A1481156 baseline in ocular safety tests;
· Need for downtitration in dose due to intolerability;
· Need for discontinuation due to intolerability; and
· Growth and development.

Other Outcome Parameters:
To assess 1-year efficacy data, the following endpoints will be described;
Percent change from A1481131 baseline to Week 36 of the extension Study A1481156 in the following exercise endpoints in subjects who are developmentally able to perform the CPX test:
· Peak VO2 normalized to body weight using an exercise tolerance test (bicycle ergometry);
· Respiratory Exchange Ratio (RER);
· Time to maximum VO2;
· Total ventilation (VE);
Change from A1481131 baseline to Week 36 of the extension Study A1481156 in:
· CHQ-PF28 as assessed by the physical and psychosocial scales;
· WHO PH functional class;
· End tidal O2 and CO2;
· Anaerobic threshold (AT);
· Percent predicted peak VO2 in subjects who are developmentally able to perform the CPX test;
· Subject (Parent)/Physician global assessment;
· Background therapy.

To assess sustainability of effect after A1481131, the following endpoints will be described;
From A1481156 baseline to assessment times specified in study schedule:
· Percent change in peak VO2 normalized to body weight using an exercise tolerance test (bicycle ergometry) in subjects developmentally able to perform the exercise;
· Change in CHQ PF28 scales;
· Change in WHO PH functional class.

Main Objective: The primary objective is to assess the safety and tolerability of oral sildenafil in the chronic treatment of pediatric subjects with PAH.

Timepoint(s) of evaluation of this end point: Weeks 12,24,36, 48 every 12 weeks

Secondary Objective: The secondary objectives are;
- To describe long-term (=1 year) efficacy of oral sildenafil in these subjects
- To assess survival status of subjects who have discontinued study drug

Secondary Outcome(s)

Secondary end point(s): Long-term Efficacy:'Other Outcome Parameters: To assess 1-year efficacy data, the following endpoints will be described; Percent change from A1481131 baseline to Week 36 of the extension Study A1481156 in the following exercise endpoints in subjects who are developmentally able to perform the CPX test: •Peak VO2 normalized to body weight using an exercise tolerance test (bicycle ergometry); •Respiratory Exchange Ratio (RER); •Time to maximum VO2; •Total ventilation (VE); Change from A1481131 baseline to Week 36 of the extension Study A1481156 in: •CHQ-PF28 as assessed by the physical and psychosocial scales; •WHO PH functional class; •End tidal O2 and CO2; •Anaerobic threshold (AT); •Percent predicted peak VO2 in subjects who are developmentally able to perform the CPX test; •Subject (Parent)/Physician global assessment; •Background therapy. To assess sustainability of effect after A1481131, the following endpoints will be described; From A1481156 baseline to assessment times specified in study schedule: •Percent change in peak VO2 normalized to body weight using an exercise tolerance test (bicycle ergometry) in subjects developmentally able to perform the exercise

Timepoint(s) of evaluation of this end point: Weeks 12,24,36, 48 every 12 weeks

Secondary ID(s)

A1481156

Source(s) of Monetary Support

Pfizer Limited, Ramsgate Road, Sandwich. Kent UK