Cognitive Remediation for Sickle Cell Disease

Overseen By Donna Murdaugh, PhD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Alabama at Birmingham

Must not be taking: Psychotropic medications

Disqualifiers: Seizures, Stroke, Intellectual disability, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 8

Duration: 4 weeks

120 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how a telehealth program, called C-READY, can help adolescents with sickle cell disease improve their thinking skills and prepare for the transition to adult healthcare. Participants will engage in activities focused on self-management and goal-setting. Some will start the program immediately, while others will begin after a short waiting period. The trial seeks children and teens aged 10 to 18 who have sickle cell disease and are currently receiving treatment at Children's of Alabama Hospital. Participants should not have a history of seizures, strokes, or certain other medical conditions.

As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group of people, offering participants a chance to contribute to important advancements in care.

Will I have to stop taking my current medications?

If you are currently taking psychotropic medications (drugs that affect your mood, thoughts, or behavior), you will not be able to participate in this trial.

Is there any evidence suggesting that the C-READY treatment is likely to be safe for humans?

Research shows that the C-READY program, a treatment aimed at improving mental skills, is generally well-received by participants. Similar programs have proven safe, with no major side effects reported. This treatment enhances abilities like memory and thinking speed through mental exercises, not medication, ensuring safety. As this trial is in its early stages, researchers are closely monitoring safety. Participants can be assured that their safety is the top priority, and researchers will carefully observe responses to the treatment. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment for sickle cell disease?

Researchers are excited about C-READY because it offers a unique approach to managing cognitive challenges in youth with sickle cell disease. Unlike traditional treatments that primarily focus on managing pain and physical symptoms, C-READY targets cognitive deficits through self-management and goal-setting. This cognitive remediation method aims to improve executive and adaptive functions, potentially enhancing quality of life for young patients. By focusing on these often-overlooked cognitive aspects, C-READY could complement existing therapies, providing a more holistic approach to care.

What evidence suggests that the C-READY intervention might be an effective treatment for sickle cell disease?

Research has shown that the C-READY program aims to improve thinking skills, such as memory and processing speed, in teenagers with sickle cell disease. In this trial, participants will either receive the C-READY intervention immediately or join a wait-list control group to receive it after a 4-week wait. Studies have found that this program helps young people manage their health better and develop skills needed for adult care. By focusing on enhancing thinking abilities, C-READY aids in planning and decision-making. Previous participants demonstrated improvements in these areas, suggesting that C-READY effectively helps young people manage their condition. The program is also available through telehealth, making participation more accessible. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for English-speaking adolescents aged 10-18 with any type of sickle cell disease (SCD), who are patients at Children's of Alabama Hospital. It excludes those with a history of seizures, stroke, intellectual disabilities or autism, metal implants that prevent MRI scans, severe claustrophobia, or current use of psychotropic medications.

Inclusion Criteria

I have been diagnosed with sickle cell disease.

Active follow-up at Children's of Alabama Hospital

English-speaking

Exclusion Criteria

I have had seizures or a stroke.

I am taking medication for my mental health.

You cannot undergo an MRI scan because you have metal implants, neurostimulators, or are claustrophobic.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

C-READY intervention delivered over 4 weeks with 8 telehealth sessions and weekly caregiver phone sessions

4 weeks

8 telehealth sessions, weekly caregiver phone sessions

Follow-up

Participants are monitored for transition readiness skills and cognitive performance post-intervention

4 months

What Are the Treatments Tested in This Trial?

Interventions

• CREADY

Trial Overview The study tests the CREADY program delivered via telehealth to help teens with SCD transition their healthcare management from pediatric to adult services. This randomized controlled trial assigns participants by chance to either receive this intervention or not.

How Is the Trial Designed?

Group I: Wait-List Control Group Experimental Treatment 1 Intervention

Will receive the same C-READY intervention after a 4-week wait period

Group II: C-READY (Cognitive-Remediation of Executive and Adaptive Deficits in Youth) Experimental Treatment 1 Intervention

Self-management and goal-setting cognitive remediation

Who Is Running the Clinical Trial?

University of Alabama at Birmingham

Lead Sponsor

Trials

1,677

Recruited

2,458,000+

Published Research Related to This Trial

Cognitive and psychological difficulties, such as issues with executive function and increased anxiety or depression, are significant complications for pediatric patients with sickle cell disease (SCD) and should be considered as important endpoints in clinical trials.

The review identifies specific, reliable cognitive tests and patient-reported outcomes (like the NIH Toolbox Cognition Module and PROMIS scales) that can effectively measure the impact of SCD on patients' daily lives, emphasizing the need for patient-centered approaches in treatment evaluation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Considerations for Selecting Cognitive Endpoints and Psychological Patient-Reported Outcomes for Clinical Trials in Pediatric Patients With Sickle Cell Disease. Hood, AM., Crosby, LE., Stotesbury, H., et al.[2023]

Children with sickle cell disease (SCD) showed significant cognitive weaknesses in areas such as general cognitive ability, short-term memory, and processing speed, based on a study of 50 children with SCD compared to 36 peers without the disease.

Anemia severity was a strong predictor of cognitive performance, and its effects varied depending on the child's socioeconomic status, suggesting that interventions may need to consider both medical and social factors to effectively support cognitive development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Interactions of biomedical and environmental risk factors for cognitive development: a preliminary study of sickle cell disease. Schatz, J., Finke, R., Roberts, CW.[2019]

In a study of 2436 individuals with sickle cell disease, self-reported cognitive difficulties, particularly in executive functioning and comprehension, were linked to poorer performance in daily living activities and lower medication adherence.

Cognitive dysfunction is common among adolescents and adults with sickle cell disease, and monitoring these cognitive symptoms can help healthcare providers better understand and address the functional challenges faced by these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient-reported neurocognitive symptoms influence instrumental activities of daily living in sickle cell disease. Longoria, JN., Pugh, NL., Gordeuk, V., et al.[2022]

Children and adolescents with sickle cell disease (SCD) showed lower full-scale IQs and performance on the Wechsler Intelligence Scale compared to their healthy siblings, indicating potential cognitive impacts of the disease.

Younger patients with SCD exhibited significantly abnormal scores in areas like expressive speech and memory, suggesting possible subtle neuropsychological deficits that may be linked to subclinical strokes, warranting further research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Subtle neuropsychological deficits in children with sickle cell disease. Wasserman, AL., Wilimas, JA., Fairclough, DL., et al.[2019]

The Kiddie-Sluggish Cognitive Tempo (K-SCT) measure is a reliable and valid tool for assessing sluggish cognitive tempo in children with sickle cell disease (SCD), showing excellent internal consistency and test-retest reliability.

Symptoms of sluggish cognitive tempo are significantly associated with learning difficulties in children with SCD, even when controlling for attention-deficit/hyperactivity disorder (ADHD) symptoms, indicating that SCT may independently impact educational outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sluggish Cognitive Tempo in Pediatric Sickle Cell Disease. Hardy, SJ., Forman, S., Hardy, KK., et al.[2022]

The Sickle Cell Disease Program for Learning and Neuropsychological Evaluation (SCD-PLANE) significantly improved access to neuropsychological testing for youth with sickle cell disease, identifying previously unmet cognitive and behavioral needs.

Results showed that despite normal intelligence, patients exhibited increased risks for issues with executive function, processing speed, and emotional symptoms, highlighting the importance of integrated neuropsychological support in routine care for this high-risk population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transition planning for youth with sickle cell disease: embedding neuropsychological assessment into comprehensive care. Wills, KE., Nelson, SC., Hennessy, J., et al.[2010]

In a study of 95 youth with sickle cell disease, a specific measure of self-management skills (SMSC) did not effectively predict successful transition to adult care, indicating a need for better assessment tools.

Caregiver-reported executive functioning, as measured by the BRIEF, showed a weak negative correlation with self-management skills, suggesting that improvements in executive function may not directly enhance transition outcomes for these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of transition readiness to predict health care utilization during transition to adult care in sickle cell disease. Howell, KE., Heitzer, AM., Longoria, JN., et al.[2023]

A 2-year educational rehabilitation program for students with sickle cell disease and memory deficits showed promising feasibility, with 82% of the 11 participants completing the program.

Participants demonstrated improvements in both memory and academic achievement, suggesting that educational rehabilitation could be beneficial, warranting further investigation in larger multi-center trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pilot randomized education rehabilitation trial is feasible in sickle cell and strokes. King, AA., White, DA., McKinstry, RC., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05336201

Cognitive Remediation Intervention to Prepare for Transition of ... This study will examine the efficacy of C-READY on adaptive and self-management skills in youth with SCD, where there is a critical need to enhance self- ...

2. withpower.com withpower.com/trial/phase-2-anemia-sickle-cell-4-2022-c8f0b

Cognitive Remediation for Sickle Cell Disease The CREADY treatment is unique because it focuses on cognitive remediation, aiming to improve cognitive functions like memory and processing speed, which are ...

3. scdstudies.com scdstudies.com/the-cognitive-remediation-of-executive-and-adaptive-deficits-in-youth-c-ready-intervention-a-randomized-controlled-trial-for-adolescents-with-sickle-cell-disease-to-prepare-for-transition-of-care/

The Cognitive-Remediation of Executive and Adaptive Deficits ... Randomized Controlled Trial (RTC) testing the efficacy of a telehealth adaptation of the Cognitive-Remediation of Executive and Adaptive ...

4. cambridge.org cambridge.org/core/services/aop-cambridge-core/content/view/383342994744CE5678084A41D3C854D8/S2059866121007056a.pdf/77523-prospective-cohort-study-of-predominantly-immigrant-people-with-chronic-hepatitis-b-in-the-baltimore-metropolitan-washington-dc-area.pdf

76644 Cognitive and Behavioral Outcomes in Adolescents ... The pur- pose of this study is to test the efficacy of an individualized cognitive remediation program designed to promote not just cognitive func- tion, but ...

5. api.pageplace.de api.pageplace.de/preview/DT0400.9780197524466_A47082753/preview-9780197524466_A47082753.pdf

cognitive remediation of executive and adaptive deficits in ... Chapter 1 provides a brain injury and recovery model which supports the theoretical framework for which the C- READY program was developed. The context of this ...

6. global.oup.com global.oup.com/academic/product/cognitive-remediation-of-executive-and-adaptive-deficits-in-youth-c-ready-9780197524459

Cognitive Remediation of Executive and Adaptive Deficits ... This book is a comprehensive and contextual guide to implementation of a family-systems focused, culturally competent cognitive remediation program for youth

7. researchgate.net researchgate.net/publication/371850004_Cognitive_Remediation_of_Executive_and_Adaptive_Deficits_in_Youth_C-READY_A_Family_Focused_Program

Cognitive Remediation of Executive and Adaptive Deficits ... The book is divided into three main parts: Part 1 provides the empirical evidence and theoretical framework for the development of the C-READY program, ...