Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia

Mazandaran University of Medical Sciences8 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Pulmonary Artery Pressure Thalassemia Non-transfusion Dependent Thalassemia

Interventions

L-glutamineDRUG

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).

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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).

Outcomes

Primary Outcomes

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.

Time frame: Baseline to 60 days

Secondary Outcomes

Clinical Response Rate

Proportion of participants achieving either an absolute reduction in PAP ≥10 mmHg or a relative reduction ≥20% from baseline.

Time frame: 60 days

Safety and Tolerability of L-Glutamine

Monitoring of adherence, adverse events, and treatment discontinuations related to L-glutamine supplementation.

Time frame: 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).

Time frame: Baseline to 60 days

Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes