Exercise Program for Sickle Cell Disease

(SuCCESs Trial)

Recruiting at 1 trial location

Overseen By Victoria G Marchese, PhD

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: University of Maryland, Baltimore

Disqualifiers: Neurological disorder, Lower extremity injury

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 8

Duration: 6 weeks

20 Participants Needed

Trial Summary

What is the purpose of this trial?

The Sickle Cell Children's Exercise Study (SuCCESs) will explore the feasibility and effects of a moderate intensity strengthening, balance, speed, and agility intervention program in children with sickle cell disease.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications.

Is the exercise program for sickle cell disease safe for humans?

The research articles reviewed do not provide specific safety data for the exercise program or the treatments mentioned under different names like SuCCESs, CASGEVY, exagamglogene autotemcel, or exa-cel. However, they do discuss the safety of other treatments for sickle cell disease, such as plerixafor, which was generally well-tolerated in patients. ^{1 2 3 4 5}

How does the exercise program treatment for sickle cell disease differ from other treatments?

The exercise program for sickle cell disease is unique because it focuses on improving physical functioning and cardiopulmonary fitness through moderate endurance exercise, which is not a standard treatment for this condition. Unlike traditional medical treatments, this program involves regular exercise sessions, such as cycling, tailored to be safe and beneficial for individuals with sickle cell disease. ^{6 7 8 9 10}

Eligibility Criteria

The SuCCESs trial is for children aged 6-17 who have been diagnosed with sickle cell disease. It's designed to see if a special exercise program can help them without causing harm.

Inclusion Criteria

I am between 6 and 17 years old and have sickle cell disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Assessment

Participants perform baseline assessments including knee extension strength, locomotor efficiency, exercise tolerance, and rate of muscle activation

1 week

1 visit (in-person)

Treatment

Participants undergo a moderate intensity strengthening, balance, speed, and agility intervention program

6 weeks

6 visits (in-person), 12 home sessions

Post-intervention Assessment

Participants perform post-intervention assessments to measure outcomes such as knee extension strength, locomotor efficiency, exercise tolerance, and rate of muscle activation

1 week

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• SuCCESs

Trial Overview This study tests a moderate intensity exercise program focused on strengthening, balance, speed, and agility in young patients with sickle cell disease to determine its safety and effectiveness.

Participant Groups

Group I: Physical Therapy Exercise Experimental Treatment 1 Intervention

There will be no control group and only one intervention delivered

Who Is Running the Clinical Trial?

University of Maryland, Baltimore

Lead Sponsor

Trials

729

Recruited

540,000+

Findings from Research

A Phase I study involving 28 patients with sickle cell disease found that ICA-17043 was well tolerated with no dose-limiting adverse events, indicating a good safety profile for this medication.

The pharmacokinetics showed that the total systemic exposure to ICA-17043 increased with higher doses, and the drug has a long half-life of 12.8 days, suggesting that once-daily dosing could effectively maintain therapeutic levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dose-escalation study of ICA-17043 in patients with sickle cell disease. Ataga, KI., Orringer, EP., Styles, L., et al.[2022]

In a phase I study involving 15 patients with sickle cell disease, the mobilizing agent plerixafor was found to be well tolerated, with a low rate of serious adverse events, although it did not consistently achieve the desired mobilization of CD34+ hematopoietic progenitor cells.

The study suggested that prior treatment with hydroxyurea may have limited the effectiveness of plerixafor in mobilizing CD34+ cells, indicating that adjustments in hydroxyurea administration may be necessary for future trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of plerixafor dose escalation for the mobilization of CD34+ hematopoietic progenitor cells in patients with sickle cell disease: interim results. Boulad, F., Shore, T., van Besien, K., et al.[2021]

Plerixafor is safe and well-tolerated in adult patients with sickle cell disease (SCD), effectively mobilizing hematopoietic stem cells (HSCs) for potential gene therapy, as demonstrated in a study involving 6 patients.

Using a standard dose of plerixafor (240 µg/kg) and an optimized apheresis protocol, researchers achieved high yields of CD34+ cells, which are crucial for HSC therapies, with counts reaching up to 24.5 × 10^6 cells/kg.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Esrick, EB., Manis, JP., Daley, H., et al.[2021]

Sickle cell disease affects millions globally, with complex complications arising from a single DNA mutation, leading to severe health issues and a reduced lifespan for patients.

While only two drugs have been approved for sickle cell disease treatment so far, recent advancements in research have led to the development of new therapeutic strategies aimed at preventing complications and improving patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Emerging disease-modifying therapies for sickle cell disease. Carden, MA., Little, J.[2020]

Sickle cell disease (SCD) affects around 100,000 Americans and millions globally, with current treatments including red blood cell transfusions and hydroxyurea, while hematopoietic stem cell transplants offer a potential cure but face significant barriers.

L-glutamine has been FDA-approved for patients aged 5 and older to help prevent acute pain episodes by reducing red blood cell oxidant injury, showcasing a novel therapeutic approach in the management of SCD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advances in the Treatment of Sickle Cell Disease. Kapoor, S., Little, JA., Pecker, LH.[2019]

A 12-week home-based exercise program for 13 adolescents with sickle cell anemia showed high feasibility and safety, with 77% of participants completing 89% of the sessions without any exercise-related adverse events.

Although adherence to the exercise duration and target heart rate decreased in the second half of the study, the results suggest that regular exercise could be beneficial for children with sickle cell anemia, warranting further research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Feasibility and safety of home exercise training in children with sickle cell anemia. Liem, RI., Akinosun, M., Muntz, DS., et al.[2017]

A multicenter study involving 40 patients with sickle-cell disease showed that a moderate-intensity endurance exercise program was safe and led to a significant improvement in functional capacity, with trained patients increasing their power output during exercise tests by 7.2 W compared to a decrease in the control group.

No serious adverse events occurred in the training group, while the control group experienced five adverse events, including vaso-occlusive crises, suggesting that exercise may reduce the risk of complications in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Moderate-intensity endurance-exercise training in patients with sickle-cell disease without severe chronic complications (EXDRE): an open-label randomised controlled trial. Gellen, B., Messonnier, LA., Galactéros, F., et al.[2018]

The SPRINTS study is a multicenter trial designed to evaluate the safety and effects of different exercise intensities on inflammation and airway dynamics in children and young adults with sickle cell anemia, involving 70 participants with SCA and 70 healthy controls.

By assessing biomarkers of inflammation, endothelial dysfunction, and tissue oxygenation during exercise, SPRINTS aims to provide crucial insights that could lead to evidence-based exercise guidelines for individuals with sickle cell anemia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Sickle Cell Pro-Inflammatory Response to Interval Testing Study (SPRINTS) in children and young adults with sickle cell anemia - Study design and methodological strategies. Liem, RI., Baynard, T., Hsu, LL., et al.[2021]

A study of 46 children and adolescents with sickle cell disease (SCD) revealed that their lung functional capacity for exercise, measured by the six-minute walk test (6MWT), was lower than expected for their age, indicating potential limitations in physical activity.

Patients with the HbSS/Sβ0-thalassemia genotype walked a shorter distance during the 6MWT compared to those with HbSC/Sβ(+)-thalassemia, suggesting that different SCD genotypes may impact exercise performance and respiratory parameters like respiratory rate and oxygen saturation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of functional capacity for exercise in children and adolescents with sickle-cell disease through the six-minute walk test. Hostyn, SV., Carvalho, WB., Johnston, C., et al.[2022]

Adults with sickle cell disease (SCD) showed significantly lower levels of cardiorespiratory endurance and muscle strength compared to healthy controls, indicating potential fitness challenges associated with the condition.

A positive correlation was found between muscular strength and cardiorespiratory endurance in adults with SCD, suggesting that improving muscle strength may benefit overall cardiopulmonary health in these individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of cardiopulmonary endurance and muscular strength in adults living with sickle cell disease. Ogunsile, FJ., Stewart, KJ., Kanter, J., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Dose-escalation study of ICA-17043 in patients with sickle cell disease. [2022]

2. Italy pubmed.ncbi.nlm.nih.gov

Safety and efficacy of plerixafor dose escalation for the mobilization of CD34+ hematopoietic progenitor cells in patients with sickle cell disease: interim results. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. [2021]

4. Italy pubmed.ncbi.nlm.nih.gov

Emerging disease-modifying therapies for sickle cell disease. [2020]

5. England pubmed.ncbi.nlm.nih.gov

Advances in the Treatment of Sickle Cell Disease. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Feasibility and safety of home exercise training in children with sickle cell anemia. [2017]

7. England pubmed.ncbi.nlm.nih.gov

Moderate-intensity endurance-exercise training in patients with sickle-cell disease without severe chronic complications (EXDRE): an open-label randomised controlled trial. [2018]

8. Netherlands pubmed.ncbi.nlm.nih.gov

The Sickle Cell Pro-Inflammatory Response to Interval Testing Study (SPRINTS) in children and young adults with sickle cell anemia - Study design and methodological strategies. [2021]

9. Brazil pubmed.ncbi.nlm.nih.gov

Evaluation of functional capacity for exercise in children and adolescents with sickle-cell disease through the six-minute walk test. [2022]

10. England pubmed.ncbi.nlm.nih.gov

An evaluation of cardiopulmonary endurance and muscular strength in adults living with sickle cell disease. [2022]