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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| L-Glutamine + Standard Care | Experimental | Participants in this arm will receive oral L-glutamine at a dose of 0.1 g/kg/day for 60 days, in addition to their routine standard care for non-transfusion-dependent thalassemia |
|
| Standard Care Alone | No Intervention | Standard care may include iron chelation therapy and/or hydroxyurea as prescribed by the treating physician. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| L-glutamine | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Pulmonary Artery Pressure (PAP) | 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. | Baseline to 60 days |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Response Rate | Proportion of participants achieving either an absolute reduction in PAP ≥10 mmHg or a relative reduction ≥20% from baseline. | 60 days |
| Safety and Tolerability of L-Glutamine |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Bu'Ali Sina Hospital | Sari | Mazandaran | 4815738477 | Iran |
Individual participant data will not be shared because of the small sample size, the single-center design, and the need to protect patient privacy. Only aggregated results will be reported in publications or presentations.
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| ID | Term |
|---|---|
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D005973 | Glutamine |
| ID | Term |
|---|---|
| D024361 | Amino Acids, Basic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000599 | Amino Acids, Diamino |
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|
Monitoring of adherence, adverse events, and treatment discontinuations related to L-glutamine supplementation.
| Baseline to 60 days |
| Iron Load | Association between change in pulmonary artery pressure (ΔPAP) and iron overload indices (Cardiac T2*, Liver T2*, and Liver Iron Concentration). | Baseline to 60 days |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D021542 | Amino Acids, Neutral |