Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension

• ClinicalTrials.gov Identifier: NCT00872170
• Recruitment Status: Completed
• First Posted: March 31, 2009
• Results First Posted: February 21, 2014
• Last Update Posted: February 21, 2014
• Sponsor: HealthCore-NERI
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by (Responsible Party): HealthCore-NERI

• Study Type: Interventional
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Thalassemia; Hypertension, Pulmonary
• Intervention: Drug: Sildenafil
• Enrollment: 27

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Sildenafil	Control
Arm/Group Description	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg	Participants with thalassemia who do not have pulmonary hypertension were part of a control group and were only undergoing screening/baseline assessments.
Period Title: Overall Study
Started	14	13
Completed	13 [1]	13
Not Completed	1	0
Reason Not Completed
Adverse Event	1	0
[1] One patient withdrew from study at week 2.

Baseline Characteristics

Arm/Group Title		Sildenafil	Control	Total
Arm/Group Description		Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg	Participants with thalassemia who do not have pulmonary hypertension were part of a control group and were only undergoing screening/baseline assessments.	Total of all reporting groups
Overall Number of Baseline Participants		14	13	27
Baseline Analysis Population Description		All 27 patients (14 in the Sildenafil group and 13 in the control group) who completed the baseline visit were used.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	14 participants	13 participants	27 participants
		35.6 (11.3)	33.7 (12.6)	34.7 (11.7)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	14 participants	13 participants	27 participants
	Female	2 14.3%	6 46.2%	8 29.6%
	Male	12 85.7%	7 53.8%	19 70.4%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants	Number Analyzed	14 participants	13 participants	27 participants
United States		4	6	10
Canada		2	2	4
United Kingdom		6	5	11
Lebanon		2	0	2

Outcome Measures

1. Primary Outcome

Title	Change in Six-minute Walk Test (6MWT) Distance From Baseline to Week 12 Among Sildenafil Group
Description	Change in six-minute walk test (6MWT) distance was calculated as 6MWT at week 12 minus 6MWT at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and 2 patients who had no 6MWT at 12 weeks were excluded. Patients in control group were only assessed at baseline. Therefore, 8 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	8
Mean (Standard Error); Unit of Measure: meters
	-1.38 (16.01)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	This study had 80% power at level alpha=0.05 to detect a 60 m change in 6MWT among N=10 participants, assuming a 60 m standard deviation for 12-week change.
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.97
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

2. Secondary Outcome

Title	Change in Tricuspid Regurgitant Jet Velocity (TRV) From Baseline to Week 12 Among Sildenafil Group
Description	Change in tricuspid regurgitant jet velocity (TRV) was calculated as TRV at week 12 minus TRV at baseline. The TRV provides an estimate of pulmonary artery pressure.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: m/s
	-0.45 (0.18)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.04
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

3. Secondary Outcome

Title	Change in Echo Left Ventricular End Systolic Volume (LVESV) From Baseline to Week 12 Among Sildenafil Group
Description	Change in echo left ventricular end systolic volume (LVESV) was calculated as LVESV at week 12 minus LVESV at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: ml
	-3.82 (0.98)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.005
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

4. Secondary Outcome

Title	Change in Echo Left Ventricular End Diastolic Volume (LVEDV) From Baseline to Week 12 Among Sildenafil Group
Description	Change in echo left ventricular end diastolic volume (LVEDV) was calculated as LVEDV at week 12 minus LVEDV at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: ml
	-7.89 (2.71)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.02
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

5. Secondary Outcome

Title	Change in Plasma Arginine From Baseline to Week 12 Among Sildenafil Group
Description	Change in Plasma Arginine was calculated as Plasma Arginine at week 12 minus Plasma Arginine at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: µM
	31.77 (15.81)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.05
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

6. Secondary Outcome

Title	Change in Red Blood Cell (RBC) Arginine From Baseline to Week 12 Among Sildenafil Group
Description	Change in Red Blood Cell (RBC) Arginine was calculated as Red Blood Cell (RBC) Arginine at week 12 minus Red Blood Cell (RBC) Arginine at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: µM
	3.67 (1.45)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.02
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

7. Secondary Outcome

Title	Change in Soluble Platelet Selectin (sP-SELECTIN) From Baseline to Week 12 Among Sildenafil Group
Description	Change in Soluble platelet selectin (sP-SELECTIN) was calculated as sP-SELECTIN at week 12 minus sP-SELECTIN at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: ng/ml
	121.13 (57.56)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.04
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

8. Secondary Outcome

Title	Change in Lactate Dehydrogenase (LDH) From Baseline to Week 12 Among Sildenafil Group
Description	Change in Lactate dehydrogenase (LDH) was calculated as LDH at week 12 minus LDH at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and 2 patients who had no LDH at 12 weeks were excluded. Patients in control group were only assessed at baseline. Therefore, 8 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	8
Mean (Standard Error); Unit of Measure: U/L
	23.37 (35.98)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.33
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

9. Secondary Outcome

Title	Change in Cell Free Hemoglobin From Baseline to Week 12 Among Sildenafil Group
Description	Change in Cell Free Hemoglobin was calculated as Cell Free Hemoglobin at week 12 minus Cell Free Hemoglobin at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: ug/ml
	-67.60 (48.03)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.18
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

10. Secondary Outcome

Title	Change in Arginase Concentration From Baseline to Week 12 Among Sildenafil Group
Description	Change in Arginase concentration was calculated as Arginase concentration at week 12 minus Arginase concentration at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: ng/ml
	-17.06 (16.25)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.97
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

11. Secondary Outcome

Title	Change in Arginase Activity From Baseline to Week 12 Among Sildenafil Group
Description	Change in Arginase activity was calculated as Arginase activity at week 12 minus Arginase activity at baseline.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

Of 14 patients in the Sildenafil arm, 4 patients with discrepant Tricuspid Regurgitant Jet Velocity (TRV) measurements between the local site, core lab and NHLBI readings and one patient who withdrew from the study were excluded. Patients in control group were only assessed at baseline. Therefore, 9 Sildenafil and 0 control patients were used.

Arm/Group Title	Sildenafil
Arm/Group Description:	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg
Overall Number of Participants Analyzed	9
Mean (Standard Error); Unit of Measure: U/L
	0.33 (2.88)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Sildenafil
	Comments	[Not Specified]
	Type of Statistical Test	Superiority or Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.96
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	Linear mixed models with participant-specific intercepts and slopes controlled for time effects were used.

Adverse Events

Time Frame	up to Week 12
Adverse Event Reporting Description	At each follow-up visit, Sildenafil patients were interviewed for medical conditions, medical changes, and symptoms that have occurred since the last visit. An Adverse Event (AE) form was completed if any AE was reported. A Serious Adverse Event form was completed for all AEs rated as serious. No AEs were collected for the control group.
Arm/Group Title	Sildenafil		Control
Arm/Group Description	Participants with thalassemia who have pulmonary hypertension received sildenafil for 12 weeks. Sildenafil : Participants received sildenafil for 12 weeks with the following therapy: 50 mg of oral sildenafil three times a day (TID) increased to 100 mg TID as tolerated in adults and children greater than 50 kg; 1 mg/kg sildenafil TID without dose escalation in children less than 50 kg		Participants with thalassemia who do not have pulmonary hypertension were part of a control group and were only undergoing screening/baseline assessments.
All-Cause Mortality
	Sildenafil		Control
	Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--
Serious Adverse Events
	Sildenafil		Control
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/14 (14.29%)		0/0
Eye disorders
Vision/color disturbance	1/14 (7.14%)	1	0/0	0
Respiratory, thoracic and mediastinal disorders
Dyspnea	1/14 (7.14%)	1	0/0	0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Sildenafil		Control
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	11/14 (78.57%)		0/0
Blood and lymphatic system disorders
Hemoglobin decreased	1/14 (7.14%)	1	0/0	0
Eye disorders
Photophobia	1/14 (7.14%)	1	0/0	0
Vision/color disturbance	2/14 (14.29%)	3	0/0	0
Vision blurred	1/14 (7.14%)	1	0/0	0
Ocular/visual - other (shining outline on objects)	1/14 (7.14%)	1	0/0	0
General disorders
Fatigue	1/14 (7.14%)	1	0/0	0
Edema limbs	2/14 (14.29%)	2	0/0	0
Dizziness	1/14 (7.14%)	1	0/0	0
Hepatobiliary disorders
INR increased	1/14 (7.14%)	1	0/0	0
Injury, poisoning and procedural complications
Phlebitis	1/14 (7.14%)	1	0/0	0
Musculoskeletal and connective tissue disorders
Back pain	1/14 (7.14%)	1	0/0	0
Pain-other: sciatic pain of left leg with associated back pain	1/14 (7.14%)	1	0/0	0
Nervous system disorders
Headache	5/14 (35.71%)	6	0/0	0
Reproductive system and breast disorders
Prolonged erection (priapism)	1/14 (7.14%)	2	0/0	0
Respiratory, thoracic and mediastinal disorders
Dyspnea	2/14 (14.29%)	2	0/0	0

Limitations and Caveats

Of 14 patients in the Sildenafil arm, 4 patients with discrepant TRVs between the local site, core lab and NHLBI readings were excluded for the main analyses.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Nancy Gee
• Organization: New England Research Institutes
• Phone: 617-972-3295
• EMail: ngee@neriscience.com

Publications of Results:

Morris CR, Kim HY, Wood J, Porter JB, Klings ES, Trachtenberg FL, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Singer ST, Taher A, Neufeld EJ, Thompson AA, Sachdev V, Larkin S, Suh JH, Kuypers FA, Vichinsky EP; Thalassemia Clinical Research Network. Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension. Haematologica. 2013 Sep;98(9):1359-67. doi: 10.3324/haematol.2012.082065. Epub 2013 Apr 12.

• Responsible Party: HealthCore-NERI
• ClinicalTrials.gov Identifier: NCT00872170
• Other Study ID Numbers: 638; U01HL065238 ( U.S. NIH Grant/Contract )
• First Submitted: March 30, 2009
• First Posted: March 31, 2009
• Results First Submitted: April 24, 2013
• Results First Posted: February 21, 2014
• Last Update Posted: February 21, 2014