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

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-016096-29-GB

Enrollment

Registered

2010-03-17

Start date

2010-03-05

Completion date

Unknown

Last updated

2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pulmonary Hypertension in Thalassaemia patients

Interventions

Trade Name: Viagra Product Name: Viagra Pharmaceutical Form: Tablet INN or Proposed INN: Sildenafil citrate CAS Number: 171599-83-0 Other descriptive name: Viagra, Revatio Concentration unit: mg milli

Eligibility

Sex/Gender

All

Inclusion criteria

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: 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate whether 12 weeks of treatment with oral sildenafil increases Six Minute Walk Time (6MWT) distance in thalassaemia patients with Pulmonary Hypertension (PHT). Hypothesis: Mean twelve-week change in 6MWT distance will be greater than +60m in thalassaemia patients with PHT treated with oral sildenafil.;Secondary Objective: 1) To evaluate whether 12 weeks of treatment with oral sildenail improves hemodynamics, pulmonary function and structure, hemolysis, arginine-nitric oxide dysregulation, hypercoagulability, inflammation, and cardiac load in thalassaemia patients with PHT. 2)To characterize PHT in thalassaemia with respect to exercise capacity, hemodynamics, pulmonary function and structure, hemolysis, arginine-nitric oxide dysregulation, hypercoagulability, inflammation, and cardiac load.;Primary end point(s): Changes in 6 Minute Walk Time (6MWT) distances will be calculated from each participant's baseline and will be analyzed in a linear model including time since treatment initiation and baseline 6MWT distance as fixed covariates and participant-specific intercepts as random terms. The primary outcome variable, 12-week change in 6MWT distance, will be estimated at the mean baseline 6MWT distance.	—

Measure

Time frame

Main Objective: To evaluate whether 12 weeks of treatment with oral sildenafil increases Six Minute Walk Time (6MWT) distance in thalassaemia patients with Pulmonary Hypertension (PHT). Hypothesis: Mean twelve-week change in 6MWT distance will be greater than +60m in thalassaemia patients with PHT treated with oral sildenafil.;Secondary Objective: 1) To evaluate whether 12 weeks of treatment with oral sildenail improves hemodynamics, pulmonary function and structure, hemolysis, arginine-nitric oxide dysregulation, hypercoagulability, inflammation, and cardiac load in thalassaemia patients with PHT. 2)To characterize PHT in thalassaemia with respect to exercise capacity, hemodynamics, pulmonary function and structure, hemolysis, arginine-nitric oxide dysregulation, hypercoagulability, inflammation, and cardiac load.;Primary end point(s): Changes in 6 Minute Walk Time (6MWT) distances will be calculated from each participant's baseline and will be analyzed in a linear model including time since treatment initiation and baseline 6MWT distance as fixed covariates and participant-specific intercepts as random terms. The primary outcome variable, 12-week change in 6MWT distance, will be estimated at the mean baseline 6MWT distance.

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Countries

United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026