Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With β-TTD

Official title

Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With Transfusion-dependent β-thalassemia

Overview

The goal of this clinical trial is to compare physical ability of patients with transfusion-dependent β-thalassemia to control subjects. The main question it aims to answer is: is there a difference in power output at first lactate threshold between patients with transfusion-dependent β-thalassemia and control subjects during maximal incremental exercise test. Participants will have to realise differents exams including measurement of body compposition, maximal incremental exercise test, vertical jump, vasoreactivity test, blood sampling, quality of life questionnaires and neuromuscular assessments.

Interventions

• Other Maximal incremental exercise test
  Participants will perform a symptom-limited maximal incremental exercise test on a cycle ergometer.

Primary outcome measures

• Power at the first lactate threshold (PSL1) collected during the maximal incremental exercise test, compared between the two groups [Time frame: 7 days after a transfusion for patient]

Secondary outcome measures (12)

• Comparison of power values at lactate threshold 2 between βTTD patients and control subjects. [Time frame: 7 days after a transfusion for patients]
• Comparison between βTTD patients and control subjects of oxygen consumption efficiency slope values (OUES for Oxygen Uptake Efficiency Slope) [Time frame: 7 days after a transfusion for patients]
• Comparison between βTTD patients and control subjects of cardiac output values combining heart rate and systolic ejection volume values during maximal exercise. [Time frame: 7 days after a transfusion for patients]
• Comparison of muscle mass values between βTTD patients and control subjects [Time frame: 7 days after a transfusion for patients]
• Comparison between βTTD patients and control subjects of first-second expiratory flow values (FEV1, L) [Time frame: 7 days after a transfusion for patients]
• Comparison between βTTD patients and control subjects of maximum grip strength, jump height and reactive strength index values [Time frame: 7 days after a transfusion for patients]
• Compare neuromuscular function between βTTD patients and control subjects [Time frame: 7 days after a transfusion for patients]
• Compare the level of physical activity between βTTD patients and control subjects [Time frame: During inclusion visit, at day 1]
• Compare anxiety levels between βTTD patients and control subjects. [Time frame: During second visit, occuring during a transfusion visit and no longer than 3 months after the inclusion.]
• Comparison between βTTD patients and control subjects of muscle reoxygenation slope values upon removal of arterial occlusion [Time frame: 7 days after a transfusion for patients]
• Correlation within the βTTD group between physical ability and cardiac iron load, hepatic iron load and EPO [Time frame: 7 days after a transfusion for patients]
• Correlation within the βTTD group between testosterone (men) or oestrogen (women) levels and muscle mass in kg [Time frame: 7 days after a transfusion for patients]

Eligibility criteria

Inclusion criteria

• Patients aged 18 years or older

• Patients with transfusion-dependent β-thalassaemia (B0 or B+)
• Patients who have undergone blood transfusions at intervals of between 1 and 8 weeks for at least 5 years.
• Have given their free and informed written consent after being informed of the purpose of the study, its conduct and its risks.
• Be affiliated with a social security scheme.
• Not participating in any other interventional studies during the duration of this study

Control subjects must meet all of the following criteria to participate in the study:

• Subject aged 18 years or older
• Matched in age and gender to a βTTD patient included in the study.
• Declaring to be free of known acute or chronic pathologies.
• Have given their free written consent after being informed of the purpose of the study, its conduct and its risks.
• Be affiliated with a social security scheme.
• Not participate in any other interventional study during the duration of this study.
• Have 'low' or 'moderate' IPAQ results (inactive or slightly active).

Exclusion criteria

• \- Having received a bone marrow transplant.
• History of thromboembolic disease.
• Hospitalised for cardiac decompensation in the last 12 months.
• Lack of use of limbs (amputee, paraplegic, quadriplegic)
• Unable to comply with protocol requirements for social, family or other reasons, as determined by the investigator.
• Known concomitant medical condition that could affect compliance with the protocol
• benefiting of enhanced protection, namely minors, persons deprived of their liberty by judicial or administrative decision, adults under legal protection and, finally, patients in emergency situations.
• Unable to give consent.
• Pregnant or breastfeeding women.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Allocation

Non-randomized

Model

Parallel assignment

Masking

Single blind

Primary purpose

Basic science

Study locations

France · 1 center

• CH Métropole Savoie — Chambéry

Publications

• Noguer M, Berthon P, Makowski C, Messonnier LA. Impaired physical ability in patients with transfusion-dependent beta-thalassaemia: Can regular physical activity be a countermeasure? Br J Haematol. 2025 Jan;206(1):86-93. doi: 10.1111/bjh.19847. Epub 2024 Nov 19. PMID 39562756

Identifiers

NCT: NCT07207577 · CHMS25002 · 2025-A00051-48

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗