Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia

Effect of Aerobic Exercise Paired With Strength Conditioning on Bone Health in Patients With Thalassemia

The goal of this clinical trial is to determine if a weight bearing exercise intervention can improve body composition and bone health in adolescents and adults with Thalassemia.

The main questions it aims to answer are:

• Does participation in a 12-week weight bearing exercise intervention change total body lean mass and percentage body fat (as assessed by DXA) in adolescents and adults with Thalassemia?
• Does participation in a 12-week weight bearing exercise intervention change muscle function (assessed by hand grip strength, sit to stand and vertical jump) and endurance (assessed by the 6 minute walk test) in adolescents and adults with Thalassemia?
• Does participation in a 36 week weight bearing exercise intervention (30 min/day; 5x/week) change bone mineral density as assessed by DXA in adolescents and adults with Thalassemia?

Researchers will compare participants' change in body composition, muscle mass, and muscle function during a "Usual Activity" period (12 weeks) with an exercise intervention (Period 1: 12 weeks) to see if exercise can improve body composition and muscle function.

The intervention will then be extended an additional 24 weeks for a total of 36 weeks of exercise (Period 2) to explore the change in bone mineral density between between "Usual Activity" and "Exercise Intervention" (Period 2) in individuals with Thalassemia.

During the intervention period, participants will engage in a self-directed exercise regime of either weight bearing aerobic exercise or strength training exercises (30 min/day; 5x/week).

Study Overview

• Status: Recruiting
• Conditions: Thalassemia
• Intervention / Treatment: Behavioral: Exercise

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Ellen Fung, PhD; Phone Number: 4939 510-428-3885; Email: ellen.fung@ucsf.edu
• Study Contact Backup: Name: Raquel Manzo, BS; Phone Number: 510-428-3429; Email: raquel.manzo@ucsf.edu

Study Locations

• United States
  • California
    • Oakland, California, United States, 94609
      • Recruiting
      • UCSF Benioff Children's Hospital Oakland
      • Contact: Ellen Fung, PhD; Phone Number: 4939 510-428-3885; Email: ellen.fung@ucsf.edu
      • Contact: Raquel Manzo; Email: raquel.manzo@ucsf.edu
    • Oakland, California, United States, 94609
      • Recruiting
      • University of California, San Francisco

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age: 14 - 40 years
• BMD Z-score at any skeletal site < -1.0
• Diagnosed with thalassemia (any genotype, regardless of transfusion dependency)
• Vitamin D (25-Hydroxy) drawn within the previous 12 months >20 ng/mL
• English speaking, able to consent

Exclusion Criteria:

• Patients who self-identify as 'exercisers' e.g. routinely exercise for minimum of 45 min/day, 5x/week
• Pregnant (unable to conduct bone density measurements in pregnant females)
• Hypogonadal, must be on replacement sex hormone therapy for min of 6 months
• Cardiac T2* by Magnetic Resonance Imaging of <20 ms (e.g. evidence of cardiac iron overload)
• Recent long bone or vertebral fracture (within the last 6 months)
• Cognitive impairment limiting ability to understand instructions during orientation
• Other conditions known to influence bone health or body composition as determined by the investigator
• Patients at risk for cardiovascular disease yet have not received a routine cardiology assessment within the previous 12 months
• Bone medication (e.g. Zometa, Prolia, Forteo) use in previous 2 years

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: Usual Activity; Subjects will participate in usual daily activities during this period

Behavioral: Exercise; Exercise for 30 min a day 5x a week with a choice between Aerobic or strength training. Subjects will choose an aerobic activity to perform 3x/week (e.g. brisk walking, hiking, jogging). They will start at 15-20 min/day depending on fitness level and build up by 5 min intervals/week as tolerated to 50 min/day or 150 min/week by 12 weeks. Subjects will choose strength exercises to perform 2x/week, on days separate from aerobic activity. They will start with 1-2 exercises (e.g. squats, lunges, planks with and without resistance) and gradually add exercises to include all muscle groups (arms, legs, chest, back, abdomen). Start with 2 sets of 10 reps, and work up to 3 sets of 15 reps of given exercises.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total Body Lean Mass (grams)	Total body lean mass assessed by Dual Energy X-Ray Absorptiometry (DXA)	12 weeks (Period 1) and 36 weeks (Period 2)
Percentage Body Fat	Percentage body fat assessed by Dual Energy X-Ray Absorptiometry (DXA)	12 weeks (Period 1) and 36 weeks (Period 2)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
6 minute walk test (6MWT)	Distanced walked during a 6MWT	12 weeks (Period 1) and 36 weeks (Period 2)
Vertical Jump	Vertical jump from a standing height (cm)	12 weeks (Period 1) and 36 weeks (Period 2)
Bone Mineral Density (BMD)	Bone mineral density at the spine, left hip, left distal radius and whole body as assessed by Dual Energy X-Ray Absorptiometry (DXA)	36 weeks (Period 2)

Collaborators and Investigators

• Sponsor: University of California, San Francisco
• Investigators: Principal Investigator: Ellen Fung, PhD, University of California, San Francisco

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: July 7, 2025
• First Submitted That Met QC Criteria: July 7, 2025
• First Posted (Actual): July 15, 2025

Study Record Updates

• Last Update Posted (Actual): July 15, 2025
• Last Update Submitted That Met QC Criteria: July 7, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: Exercise; Body composition; Bone; Muscle function
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia
• Other Study ID Numbers: Thal Exercise

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No