Non-transfusion Dependent Thalassemia, Pulmonary Artery Pressure, Thalassemia
Conditions
Keywords
L-glutamine, Non-transfusion Dependent Thalassemia, pulmonary artery pressure
Brief summary
The goal of this clinical trial is to learn whether L-glutamine can help lower pulmonary artery pressure in adults with non-transfusion-dependent thalassemia (NTDT). The main questions it aims to answer are: Does L-glutamine reduce pulmonary artery pressure after 60 days of treatment? Is the effect of L-glutamine different from standard care alone? Researchers will compare two groups: Intervention group: Participants receive oral L-glutamine in addition to their standard treatment. Control group: Participants continue with standard treatment only. Participants will: Take either L-glutamine (by mouth) or standard care for 60 days. Undergo echocardiography at the beginning and end of the study to measure pulmonary artery pressure. Attend follow-up visits to monitor safety, adherence, and possible side effects.
Interventions
DRUGL-glutamine
Oral L-glutamine powder, administered at a dose of 0.1 g/kg/day for 60 days in adult patients with non-transfusion-dependent thalassemia (NTDT). The supplement is given in addition to each participant's standard care regimen (e.g., hydroxyurea or iron chelation therapy, as clinically indicated).
Sponsors
Mazandaran University of Medical Sciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adults (≥18 years), any sex. * Diagnosis of non-transfusion-dependent β-thalassemia (NTDT). * Pulmonary artery pressure (PAP) \> 35 mmHg estimated by Doppler echocardiography at screening. * Able and willing to provide written informed consent. * On a stable standard-of-care regimen (e.g., chelation and/or hydroxyurea) per treating physician judgment.
Exclusion criteria
* Age \<18 years. * Refusal or inability to provide informed consent. * Hepatic dysfunction: ALT \>3× upper limit of normal. * Renal dysfunction: serum creatinine \>2× upper limit of normal. * Known hypersensitivity to L-glutamine. * Pregnancy or breastfeeding. * Use of amino-acid/protein supplements within the past 3 months. * History of other cardiac diseases associated with pulmonary hypertension (per investigator assessment).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Pulmonary Artery Pressure (PAP) | Baseline to 60 days | Pulmonary artery pressure (PAP) will be measured by Doppler echocardiography at baseline and at 60 days. The primary endpoint is the change in PAP (ΔPAP = Follow-up PAP - Baseline PAP) to assess whether L-glutamine reduces pulmonary artery pressure compared with standard care alone. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Response Rate | 60 days | Proportion of participants achieving either an absolute reduction in PAP ≥10 mmHg or a relative reduction ≥20% from baseline. |
| Safety and Tolerability of L-Glutamine | Baseline to 60 days | Monitoring of adherence, adverse events, and treatment discontinuations related to L-glutamine supplementation. |
| Iron Load | Baseline to 60 days | Association between change in pulmonary artery pressure (ΔPAP) and iron overload indices (Cardiac T2\*, Liver T2\*, and Liver Iron Concentration). |
Countries
Iran
Outcome results
None listed