Pilot of oral Sildenafil for the treatment of Pulmonary Hypertension in Thalassaemia with comparison to controls. - Pulmonary Hypertension (PHT)Study

• Conditions: Pulmonary Hypertension in Thalassaemia patients

• Registration Number: EUCTR2009-016096-29-GB
• Lead Sponsor: niversity College London

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-03-17
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

INCLUSION CRITERIA

General:
1) Alpha, beta or E-beta thalassaemia confirmed by Hb-electrophoresis or molecular diagnosis
2) Age at entry at least 16 years

Intervention:
1) Pulmonary hypertension defined as TRjet velocity by Doppler echocardiography greater than 2.5 m/s

Control:
1) Lack of pulmonary hypertension defined as TRjet velocity by Doppler echocardiography less than 2.5 m/s

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

Exclusion Criteria

EXCLUSION CRITERIA
1) Pregnancy or lactation at baseline
2) Hypersensitivity to arginine or sildenafil based on prior use.

Any of the following medical conditions:
- Severe renal insufficiency defined as use of hemodialysis or serum creatinine greater than 2.5mg/dl at the time of screening.
- Cardiac disease with adjustment of cardiac medications within the last 60 days
- Symptomatic coronary artery disease as indicated by a history of chest pain, angina, claudication or surgery to treat coronary artery disease within the last year
- Stroke defined as a new focal neurological deficit lasting more than 24 hours within the last 45 days.
- New diagnosis of pulmonary embolism by ventilation-perfusion scan, angiography or any other technique within the last 90 days
- History of retinal detachment or retinal hemorrhage in the last 180 days
- Use of nitrate-based vasodilators, prostacyclin (inhaled, subcutaneous or intravenous), endothelin antagonists, or any other medication for pulmonary hypertension.
- Acute asthma exacerbation requiring use of prednisone within the last 60 days.
- Initiation or dosage increase of calcium channel blockers within the last 30 days.
- Initiation of any other cardiac or pulmonary medication within the last 90 days.
- Patients with a TRjet velocity of exactly 2.5 are not eligible for either group.
- Presence of any other condition, which in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient's compliance with the protocol, including but not limited to alcohol or drug abuse.
- No measurable TRjet on Doppler echocardiography (i.e. presence of PHT cannot be confirmed or ruled out).
- Inability or refusal to provide informed consent.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified