Clinical Research Directory

Cardiac Dysfunction in Childhood Cancer Survivors

This multicenter, prospective cohort study evaluates early cardiac dysfunction in adult survivors of childhood cancer. The hypothesis of this study is that cardiac dysfunction can be detected earlier when using speckle tracking echocardiography as novel echocardiographic technique compared to conventional echocardiography.

The Impact of Fatigue on Quality of Life and Activities of Daily Living in Pediatric Oncology Patients

This study aims to examine the impact of fatigue on the quality of life and daily living activities of children undergoing cancer treatment.In this cross-sectional study of 89 children aged 2-18 years, fatigue, quality of life, and functional independence were assessed using various scales.The basic hypothesis of the study is that as fatigue increases, children's daily activities and quality of life will be negatively affected.

Male Fertility Preservation Using Cryopreservation of Testicular Tissue Before Highly Gonadotoxic Cancer Treatment

Background: Due to the remarkable improvement in treatments these last decades, long term survival can be expected in more than 80% of childhood cancer patients. Unfortunately, cancer treatments can be harmful to the gonads and can affect reproductive and endocrine functions. While loss of fertility is a major concern for most patients, sperm cryopreservation should be offered to all pubertal male patients. For prepubertal boys, only the experimental option of testicular biopsy in order to cryopreserve testicular stem cells can be proposed. Primary aims \- To cryopreserve testicular tissue of prepubertal patient receiving highly gonadotoxic oncological treatment. Secondary aims * To cryopreserve testicular tissue after failure of sperm cryopreservation in pubertal patient with high risk of infertility * To create a database in order to record clinical and biological follow-up data * To create a research biobank for future research projects Multicentric study: HUG, CHUV, UKBB

Female Fertility Preservation Using Ovarian Tissue Cryopreservation Before Highly Gonadotoxic Cancer Treatment

Background: Due to the remarkable improvement in treatments these last decades, long term survival can be expected in more than 80% of childhood cancer patients. Unfortunately, cancer treatments can be harmful to the gonads and can affect reproductive and endocrine functions. While loss of fertility is a major concern for most patients, only the experimental option of ovarian tissue cryopreservation can be proposed to prepubertal girls with a high risk of infertility. For pubertal patient, cryopreservation of mature oocytes after ovarian stimulation can be offered if oncological treatment debut can be delayed. As it is often not possible, ovarian tissue cryopreservation can also be offered. Primary aims \- To cryopreserve ovarian tissue of pre or peripubertal patient who will be receiving highly gonadotoxic oncological treatment. Secondary aims * To create a database in order to record clinical and biological follow-up data * To pool resources with Fertisave Registry * To create a research biobank for future research projects Multicentric study: HUG, CHUV

Heuristic Tool To Improve Symptom Self-Management in Adolescents and Young Adults With Cancer

Determine the effects of the Computerized Symptom Assessment Tool (C-SCAT) versus usual care on the primary outcomes of self-efficacy for symptom management and symptom self-management behaviors

Imaging Skeletal Muscle Mitochondrial OXPHOS Activity In Acute Lymphoblastic Leukemia Survivors

The participants are being asked to take part in this trial, because the participant is a survivor of childhood cancer or agreed to be part of a volunteer group to understand the relation between cancer and cancer treatment and muscle weakness in survivors of Acute Lymphoblastic Leukemia (ALL). ALL is cancer of the blood and bone marrow. Primary Objective • To compare muscle mtOXPHOS activity and satellite cell content among ALL survivors and controls. Secondary Objective * To evaluate the association between muscle mtOXPHOS, muscle satellite cell content and physical performance in ALL survivors. * To evaluate the association of muscle morphology and epigenetics with muscle mtOXPHOS in ALL survivors.

ASIA Down Syndrome Acute Lymphoblastic Leukemia 2016

To evaluate the outcome of a prednisolone and low dose methotrexate based protocol in Down syndrome children with ALL (DS-ALL) in an Asia-wide study. The treatment protocol was modified based upon backbone of Taiwan Pediatric Oncology Group (TPOG)-ALL protocol in which risk classification will be guided by level of flow minimal residual disease (MRD) instead.

Fertility in Young Adults Who Did (Not) Store Testicular Tissue Before a Treatment Leading to Fertility Problems

The goal of this prospective comparative interventional cohort study is to assess the fertility status of young adult men (≥18 years) who received gonadotoxic treatment during childhood for the treatment of cancer or hematological disorders. These treatment protocols are highly gonadotoxic (i.e. they may cause later fertility problems) and therefore these patients have been proposed to store some testicular tissue during childhood as an option to preserve their fertility. The main questions this study aims to answer are (1) the impact of the received gonadotoxic treatment on the later fertility status and (2) the additional impact of a testicular biopsy procedure (performed at a young age to harvest testicular tissue for storage) on the future fertility. Participants will be asked to undergo a physical examination by a fertility specialist, to undergo a scrotal ultrasound, to give a blood sample, and to provide a semen sample. Researchers will compare the patients fertility status between the different received gonadotoxic treatment protocols, between patients who underwent a testicular biopsy procedure at a young age and those who did not, and compare the patients fertility status with the reproductive health of spontaneously conceived young adults.

Protocol For Collecting Data On Patients With Childhood Cancer

Progress in the development of curative therapy for pediatric malignancies has resulted in increasing numbers of long-term childhood cancer survivors. This protocol is a means to provide continuing review of outcome and late toxicity for all patients actively being treated and previously treated for childhood cancer at St. Jude Children's Research Hospital.

Remote Exercise Video Adaptations to Maximize Physical Activity in Childhood Cancer

The goal of this observational study is to leverage childhood cancer survivor input to adapt video content of a digital video disc-(DVD) delivered evidence-based PA intervention, originally designed for community-dwelling older adults. Primary Objective: \- To leverage childhood cancer survivor input to adapt video content for an evidence-based remote exercise intervention.

Patient- Generated Health Data to Predict Childhood Cancer Survivorship Outcomes

Some childhood cancer survivors have health problems as the result of previous cancer treatment. This study is being done to determine if we can better predict the risk a childhood cancer survivor might have for developing future health issues. The goal of this study is to enable regular monitoring of patient-generated health data (PGHD), including symptoms, physical activity, energy expenditure, sleep behavior and heart rate variability, and utilize these data in predicting survivor-specific risk of late effects to improve survivorship care and outcomes.

An Open-Label Intervention Trial to Reduce Senescence and Improve Frailty in Adult Survivors of Childhood Cancer

This is a first-in survivor pilot study with the goal of establishing preliminary evidence of efficacy, safety, and tolerability of two senolytic regimens to reduce markers of cellular senescence (primary outcome: p16\^INK4a) and improve frailty (primary outcome: walking speed) in adult survivors of childhood cancer. If successful, this pilot would provide the preliminary evidence needed for a phase 2, randomized, placebo-controlled trial to establish efficacy. Primary Objective * The primary aim of this proposal is to test the efficacy of two, short duration senolytic regimens: 1) combination of Dasatinib plus Quercetin and 2) Fisetin alone, to improve walking speed and decrease senescent cell abundance in blood (p16\^INKA): * Primary endpoints of this trial will be change in walking speed and senescent cell abundance in blood (p16\^INK4A) determined at baseline and again at 60 days, within an individual arm. Extended follow up at 150 days will assess the permanence of change after completion of the trial. Secondary endpoints of this trial will be effect of intervention on additional measures of frailty (beyond walking speed; Fried criteria) and on other cell senescence markers, markers of inflammation, insulin resistance, bone resorption, and cognitive function. Secondary Objectives The secondary aim is to test the safety and tolerability of two different senolytic therapies. Exploratory Objectives * To compare the efficacy of the two senolytic regimens in improving walking speed and decreasing senescent cell abundance * To evaluate the longitudinal pattern in measures of frailty.

An Evaluation of Concordance of Smartwatch ECG and One Clinical ECG and Comparison of The Two ECGS in Terms of Predictive Risks

The participant is being asked to take part in this trial, because the participant is a survivor of childhood cancer. Primary Objective To evaluate remote cardiomyopathy prediction via smartwatch and one clinical ECG and assess the concordance of the two ECGs in terms of predicted risk. Secondary Objective To build a novel predictive model solely on smartwatch ECG to predict risk for cardiomyopathy.

Social Experiences of Adolescents and Young Adults With Cancer

Participants are being asked to take part in this clinical trial, a type of research study, because investigators want to learn more about the social experiences adolescents and young adults who are being treated or have been treated for cancer. Primary Objectives * Describe differences in social experience variables (peer connectedness, perceived social competence, parent versus peer attachment) based on treatment status: on versus off therapy. * In on-therapy patients, describe differences in social experience variables (peer connectedness, perceived social competence, parent versus peer attachment) based on developmental stage: high school versus post-high school. Secondary Objectives * Assess the social support and peer interaction needs of AYA with cancer as a means of determining stakeholder interest and need for psychosocial interventions targeting social experiences. * Using qualitative interviews, explore patient perceptions of the impact of cancer on social experiences among AYA, particularly with regards to changes in friendships as a result of the cancer diagnosis and the role of the hospital in helping or hindering friendship maintenance/development. Exploratory Objectives * Explore differences in social experience by demographic, disease and treatment factors, including: gender, diagnostic category (brain tumor, leukemia/lymphoma, solid tumor), late effects/symptom burden, and treatment (e.g., treatment intensity, serious medical events). * Explore associations between perceived impact of cancer and social experience in AYA. * Explore associations between use of social media and social experience. * Explore associations between social experiences and overall functioning (quality of life, distress, coping). * Explore the possibility of subclasses of AYA by using person-centered analyses to empirically derive profiles of social experience.

Assessment of Remote Approaches for Identification of Autonomic Dysfunction Among Survivors of Leukemia and Lymphoma

This study seeks to determine if diagnosing cardiac autonomic dysfunction (AD) can be done remotely with the same accuracy as in-person testing. If so, the identification of AD could happen sooner, facilitating remote studies of the condition and potentially reducing the risk of illness. Childhood cancer survivors, particularly survivors of acute lymphoblastic leukemia (ALL) and Hodgkins's lymphoma (HL), appear to be at increased risk for AD. Primary Objectives: * To determine the sensitivity and specificity of heart rate variability (HRV), measured remotely with biosensor technology (Actigraph LEAP), compared to in-person assessment using the Ewing battery as the reference standard to identify cardiac autonomic dysfunction (AD) among survivors of leukemia and lymphoma. * To determine the sensitivity and specificity of the Composite Autonomic Symptom Scale 31 (COMPASS31) compared to the Ewing battery to identify AD among leukemia and lymphoma survivors.

Proof-of-Concept Testing of the Cardiovascular Health Equity Through Food (CHEF) Intervention in Childhood Cancer Survivors

The goal of the Cardiovascular Health Equity Through Food (CHEF) program is to make it easier for participants and families to cook and eat healthy foods during and following childhood cancer treatment with the long-term goal of supporting heart health.

Early Diagnosis and Cardiovascular Risk Stratification in Children Exposed to Cancer Therapies

The goal of this prospective interventional study is to improve the detection of subclinical chronic Cancer Therapy-Related Cardiovascular Toxicity (CTRCT) and evaluate the added value of advanced cardioechography, ergospirometry, and specific biomarkers in pediatric cancer survivors (aged 2 to 25) who received potentially cardiotoxic treatments (chemotherapy/thoracic radiotherapy). The main questions it aims to answer are: * Can advanced echocardiography (including strain and myocardial work), ECG, and ergospirometry effectively diagnose earlier subclinical cardiac impairment in this population? * What is the prevalence of cardiovascular risk factors (including physical activity levels and biological markers like proBNP/troponins) * Can new genetic or biological markers be identified to help optimizing the detection of CTRCT? At time of follow-up, if they agree, participants will: * Complete validated questionnaires regarding quality of life, physical activity, and sedentary behavior. * Undergo a cardiopulmonary exercise test (ergospirometry) for those aged over 8 years. * Wear an accelerometer (ActiGraph GT3X) for 7 consecutive days to monitor physical activity. * Provide an additional blood sample during routine follow-up for the creation of a biobank dedicated to analyzing markers of senescence, fibrosis, apoptosis, and genetic polymorphisms.

Mindfulness Interventions to Improve Health Activation, Coping, and Stress Among Childhood Cancer Survivors

This study will explore feasibility and acceptability of using a daily mobile mindfulness app to cope with stress. In addition, feasibility of digital health data collection procedures including wrist-worn devices and ecological momentary assessments will be examined. PRIMARY OBJECTIVES * Determine the feasibility and acceptability of completing the 30-day mobile mindfulness program * Evaluate the feasibility of digital health data collection procedures to detect autonomic nervous system (ANS) activation associated with stress (e.g., electrodermal activity, pulse rate variability, skin temperature) via wearable devices and self-reported ecological momentary assessments (EMA) among adult survivors of childhood cancer in the SJLIFE cohort * Generate preliminary data to estimate the effect size and power needed for an extramurally funded randomized controlled trials (RCT) examining the impact of daily mindfulness mediation on biomarkers of ANS activation, coping, health behaviors, and cardiometabolic outcomes in a cohort of survivors of childhood cancer.

Biomarkers of Brain Injury in Children With Brain Tumors

The goal of this observational study is to identify biomarkers of treatment-induced brain injury in children treated for primary brain tumors. The main question it aims to answer is: Can investigators identify sensitive plasma biomarker(s) of treatment-induced brain injury in children with primary brain tumors? Researchers will compare results between different treatment modalities (surgery, chemotherapy, radiation versus radiation-naive) and a healthy age- and sex-matched control population, to identify treatment-specific biomarkers. Participants will provide plasma samples at the following time points: before surgery, 1-2 weeks after surgery, as well as at 3-, 6-, 12-, 18-, 24-, and 36 months after surgery. Participants who receive radiation treatment will also provide plasma samples before and during treatment (approximately every 2 weeks). Where possible, plasma samples are also collected before the start of any new treatment (e.g., chemotherapy). Healthy controls will provide samples once.

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

A companion platform trial to test novel targeted agents based on the patient's tumor profile.

DENTOFACIAL-PBT Stage 2 - Feasibility Study

What is the health problem? Proton beam therapy (PBT), a type of radiotherapy treatment, can be especially beneficial to children and teenagers with cancer. PBT works by damaging cancer cells, but it can also damage healthy parts of the body nearby. This can include the teeth and jaws if the cancer is in the head and neck region. Unfortunately, the investigators do not yet fully understand the potential side effects of PBT on the teeth or jaws. What is the aim? The DENTOFACIAL-PBT research project aims to learn about the side effects to the teeth and jaws in children treated with PBT. This stage of the research aims to determine how frequently issues ('toxicities') in the development of the teeth and growth of the jaws have occurred in previously treated patients. DENTOFACIAL-PBT is funded by the National Institute for Health and Care Research. How is the researched being approached? The investigators will ask head and neck childhood cancer survivors treated with PBT overseas or in Manchester to support this research. Surveys will be sent initially, asking questions about their teeth and jaws after treatment. If interested, these survivors can be involved in the next part of this stage, which is a mixed-methods feasibility study. This will involve online interviews and dental assessments with some and the collection of dental information from a participant's dentist. Radiation dose to the teeth and the jaw bones will then be linked to any side effects seen. What will be the outcome? The information gained in this study will allow the research team to look at how age, location and amount of dose given affects the toxicities seen. Information learnt will help develop the final stage of the DENTOFACIAL-PBT study. In this, a new toxicity reporting tool will be developed which aims to improve the communication between dentists, doctors, and cancer patients.

Examination of the Relationship Between Quality of Life and Caregiving Burden Among Caregivers of Pediatric Oncology Patients

Primary Aim The primary aim of this study is to determine the quality of life and caregiving burden levels of caregivers of pediatric oncology patients and to examine the relationship between these two variables. Secondary Aims To evaluate the differences between caregivers' sociodemographic characteristics (age, gender, education level, marital status, income level, etc.) and their quality of life. To examine the differences between caregivers' sociodemographic characteristics and their caregiving burden levels. To determine whether caregivers' quality of life and caregiving burden differ according to the diagnosis and treatment characteristics of pediatric oncology patients (duration of diagnosis, type of treatment, frequency of hospitalization, etc.). Significance of the Study Childhood cancer is a process that deeply affects not only the patient but also the family, particularly the caregivers. Family members who provide care for children often face intense physical, psychological, social, and economic burdens. These challenges reduce caregivers' quality of life and increase their caregiving burden. Identifying the relationship between the quality of life and caregiving burden among caregivers of pediatric oncology patients is highly important in planning support services for families during the care process and in strengthening family-centered care practices by healthcare professionals. The findings of this study will contribute to the development of family-centered approaches in nursing care, the design of psychosocial support programs, and a better understanding of caregivers' needs.

Patients At Risk for Sensory Screening (HPARSS) to Enhance Sensory Deficit Screening in Childhood Cancer Survivors

The overall goal of this study is to attempt to overcome the organizational barriers that impede prompt screening for at-risk sensory deficits in childhood cancer survivors (CCS). Using a cross sectional design study, collaborators in the Informatics Research branch of the Institute of Informatics at the Washington University School of Medicine will identify CCS at risk for sensory deficits based upon their therapy exposure to generate the highlighting patients at risk for sensory screening (HPARSS) document. The investigators will utilize the HPARSS that will link therapy related risks for sensory deficits to specific screening procedures prompting the primary oncology provider to implement screening, diagnostic testing, and therapy.

Cryopreservation of Prepubertal Testicular Tissue for Preservation of Fertility in Young Boys With Cancer

Prepubertal boys treated for cancer are at risk of infertility as a result of their treatment. At present there are no options to preserve fertility in these boys. With informed consent, the investigators aim to cryopreserve (freeze) testis tissue biopsies taken from boys undergoing cancer therapy with a high risk of infertility. A portion of cryopreserved tissue will be securely stored, which in the future may be used to restore fertility in the patients. A second portion of the tissue will be used for laboratory work aimed at understanding the conditions required for development of the germ cells. Patients will be recruited from the Oncology Department at the Royal Hospital for Children and Young People (RHCYP) in Edinburgh. Tissue will be obtained from prepubertal patients with cancer who are about to undergo treatment with a high risk (\>80%) of infertility. Informed consent will be obtained from the patients legal guardian (and patient if applicable) for the procedure and subsequent use of tissue. A testis biopsy from one testis will be performed and if possible will coincide with a planned routine procedure requiring general anaesthetic in order to avoid the need for additional anaesthesia. The procedure will take place at RHCYP during the initial assessment and prior to the commencement of cancer treatment and will not result in delay of treatment. Tissue will be assessed for the presence of sperm for storage using existing methods. In addition, tissue will be cryopreserved by Tissue and Cells Directorate, SNBTS in Edinburgh. The tissue will be stored for up to 55 years in accordance with current legislation (HFEA Act as amended 2005). Research will be carried out at the MRC Centre for Reproductive Health in accordance with Human Tissue (Scotland) Act 2006.