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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine how a special exercise program can help children with sickle cell disease. The program focuses on building strength, improving balance, and boosting speed and agility. The goal is to assess whether these exercises are feasible and beneficial for children with this condition. Children aged 6-17 with sickle cell disease, without unrelated neurological disorders or recent leg injuries, are suitable candidates for the study. As an unphased trial, this study offers a unique opportunity for children to potentially enhance their quality of life through innovative exercise programs.

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.

What prior data suggests that this exercise program is safe for children with sickle cell disease?

Research has shown that exercise programs, like the one in the SuCCESs study, are generally safe for children with sickle cell disease. Although specific safety data for the SuCCESs program is not yet available, exercise programs are usually well-tolerated and safe for many individuals.

Clinical trials at the "Not Applicable" phase often test non-drug methods like exercise routines, which are less likely to cause serious side effects compared to drug trials. Exercise is a common component of many health plans and is often recommended to improve overall well-being.

In summary, the SuCCESs exercise program is expected to be safe based on knowledge of similar exercise programs. Participants should feel confident about joining, but discussing any concerns with a healthcare provider is always important. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the SuCCESs exercise program for sickle cell disease because it offers a novel approach compared to traditional treatments, which typically focus on medication to manage pain and prevent complications. Unlike standard care options like hydroxyurea or blood transfusions, SuCCESs introduces a structured physical therapy exercise regimen as a way to potentially improve overall health and quality of life for individuals with sickle cell disease. This approach is different because it emphasizes physical activity, which could enhance circulation and reduce pain episodes, offering a complementary strategy alongside medical treatments.

What evidence suggests that this exercise program is effective for sickle cell disease?

Research has shown that exercise benefits people with sickle cell disease. Studies have found that exercise improves blood flow and oxygen delivery, potentially reducing the risk of painful blockages in blood vessels. Low to moderate-intensity exercise programs can enhance physical abilities without causing harm. Additionally, daily exercise can help manage pain and strengthen muscles. This trial will focus on a structured exercise program as the sole intervention, which might improve the quality of life for children with sickle cell disease. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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 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]

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 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]

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8446254/

Effectiveness of a home-based therapeutic exercise program ... Results suggest that daily home-based exercises for a three-month period ameliorate pain and improve disability related to lower back pain and muscle strength.

2. link.springer.com link.springer.com/article/10.1007/s44337-024-00047-1

The role of exercise in sickle cell anemia management Exercise has been shown to improve blood flow, oxygen delivery, and tissue perfusion, reducing the risk of vaso-occlusion and tissue ischemia [ ...

3. apta.confex.com apta.confex.com/apta/csm2025/meetingapp.cgi/Paper/47653

Are Physical Therapy Interventions Safe for Persons with ... The main findings across all the studies were that low to moderate-intensity exercise programs improved the functional capacity of individuals with SCD without ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04705792

Physical Rehabilitation in Sickle Cell Anemia The objective of this project is to evaluate the effects of functional rehabilitation on quality of life in adult patients over 18 years of SCA.

5. sciencedirect.com sciencedirect.com/science/article/pii/S0006497121050369

Quantitative Assessment of Physical Activity in Adults with ... The majority of SCD participants reported sub-optimal levels of activity and currently do not meet the national guidelines of physical activity ...

6. casgevy.com casgevy.com/sickle-cell-disease/study-information

CASGEVY® Clinical Trial and Results Study purpose: The study looked at the safety of CASGEVY and how well CASGEVY worked, like how many people went without a severe VOC* for at least 12 months ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38661449/

Exagamglogene Autotemcel for Severe Sickle Cell Disease The safety of exa-cel was also assessed. Results: A total of 44 patients received exa-cel, and the median follow-up was 19.3 months (range ...

8. fda.gov fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

FDA Approves First Gene Therapies to Treat Patients with ... The safety and effectiveness of Lyfgenia is based on the analysis of data from a single-arm, 24-month multicenter study in patients with sickle ...

9. news.vrtx.com news.vrtx.com/news-releases/news-release-details/vertex-presents-positive-long-term-data-casgevytm-0

Vertex Presents Positive Long-Term Data On CASGEVY ... The longest follow up for both SCD and TDT patients now extends more than 5 years, with a median of 33.2 months and 38.1 months, respectively.

10. ashpublications.org ashpublications.org/ashclinicalnews/news/8012/Full-Results-of-Exa-Cel-Study-Show-Continued

Full Results of Exa-Cel Study Show Continued Safety, Efficacy ... The study met both its primary and secondary endpoints: of the 30 evaluable patients, 97% were free from VOCs for at least 12 consecutive months, and 100% were ...