Clinical Research Directory

Engaging Parents in Neuropsychological Rehabilitation for Childhood Cancer Survivors: The ImPACT Program - A Pilot Study

The aim of the present pilot study is to evaluate the feasibility, acceptability, and preliminary efficacy of a novel, context-sensitive neuropsychological rehabilitation intervention for childhood cancer survivors and their families.

Biology and Benefits of Music Play and Stories for Kids/Parents During ALL Treatment

Music therapy has become a standard palliative care service in many pediatric and adult hospitals; however, a majority of music therapy research has focused on the use of music to improve psychosocial dimensions of health, without considering biological dimensions. This study builds on prior work examining the psychosocial mechanisms of action underlying an Active Music Engagement (AME) intervention, designed to help manage emotional distress and improve positive health outcomes in young children with cancer and parents, by examining its effects on biomarkers of stress and immune function. The purposes of this two group, randomized controlled trial are to examine biological mechanisms of effect and dose-response relationships of AME on child/parent stress during the consolidation phase of Acute Lymphoblastic Leukemia (ALL) treatment. Specific aims are to: Aim 1. Establish whether AME lowers child and parent cortisol during ALL treatment. Aim 2. Examine cortisol as a mediator of AME effects on child and parent outcomes during ALL treatment. Aim 3 (exploratory). Examine the dose-response relationship of AME on child and parent cortisol during ALL treatment. Findings will provide a more holistic understanding about how active music interventions work to mitigate cancer-related stress and its potential to improve immune function, with direct implications for the evidence-based use of music to improve health.

Illuminate: A Clinical Study Evaluating CAR T Immune Cell Therapy (BCB-276) for Patients With Diffuse Intrinsic Pontine Glioma (DIPG).

This study will evaluate BCB-276, an investigational B7-H3-targeted Chimeric Antigen Receptor (CAR) T cell therapy, in children and young adults with diffuse intrinsic pontine glioma (DIPG). DIPG is a rare and aggressive brain tumor with limited treatment options. CAR T cell therapy uses a patient's own immune cells that are changed in a laboratory to recognize and attack cancer cells. The purpose of this study is to determine whether BCB-276, when given after completion of standard radiation therapy, is safe and can improve survival for patients with DIPG. To participate, individuals must be between 1 and 26 years of age when they join the study, have a diagnosis of DIPG, and enroll for treatment within 6 weeks of completing initial radiation therapy. Participants must not have received prior anti-cancer therapy beyond radiation with or without temozolomide prior to joining this study. BCB-276 is administered intraventricularly (into the fluid around the brain), which requires placement of a catheter for treatment. BCB-276 is given every 2 weeks at a research center over a period of several months (approximately 7-8 months). Participation includes travel to a study site, procedures to support treatment administration, sample collection, and ongoing monitoring for safety and effectiveness, with follow-up visits lasting up to about 2 years.

A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia

The goal of this clinical trial is to learn if sonrotoclax (BGB-11417) is safe and may help treat children and adolescents with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has come back after treatment or has not responded to treatment. The study will also learn how the body processes sonrotoclax when it is given with other medicines. The main questions it aims to answer are: * Is sonrotoclax safe and well tolerated when given with other anti-cancer medicines? * How does the body absorb, process, and remove sonrotoclax? * Does treatment with sonrotoclax, in combination with other medicines, help reduce or eliminate leukemia? Researchers will give sonrotoclax together with other anti-cancer medicines to participants with relapsed or refractory AML or ALL. Participants will: * Take sonrotoclax in combination with other anti-cancer medicines * Have regular clinic visits for physical exams, blood tests, heart monitoring, and other safety assessments. * Provide blood samples to measure how the body processes sonrotoclax. * Have tests to evaluate how their leukemia responds to treatment. * Continue treatment as long as it is helping and side effects remain manageable, according to the study plan.

Improving Communication Between AYA Oncology Patients and Clinicians: A Patient-Centered Intervention

The overarching goal is to develop and demonstrate proof-of-concept of ReSPECT (Reproductive and Sexual Health Patient Education and Communication Tool), a multi-modal communication intervention to improve adolescent and young adult (AYA)- clinician sexual and reproductive health (SRH) communication in the outpatient oncology clinic setting.

Larotrectinib to Enhance RAI Avidity in Differentiated Thyroid Cancer

Papillary thyroid cancer (PTC) is the most common form of differentiated thyroid cancer (DTC). The traditional first line treatment for patients with advanced DTC after surgical resection is radioactive iodine (RAI) therapy. However, less than a quarter of patients with lung metastases will achieve a complete response to RAI therapy, and this therapy carries the risk of pulmonary fibrosis and an increasingly recognized risk of secondary malignancies.

Longitudinal Identity Study of Childhood Cancer Survivors

The study investigators plan to conduct a longitudinal questionnaire study in adolescents and emerging adults (14-25 years of age at the start of the study) who survived childhood cancer to chart their identity development and broader social functioning. Additionally, the functioning of these survivors will be related to the functioning of their parents and siblings. The investigators shall focus especially on the current experience and impact of the earlier cancer experience. They will investigate to what extent the experience of a life-threatening disease has an effect on the daily life of survivors and over time and how the survivors develop through the course of adolescence and emerging adulthood on the psychosocial level. The formation of an adult identity is a very challenging task during adolescence and the way to adulthood and the fact that these youth had cancer during their childhood may especially complicate this process of identity formation. Furthermore, both parental and sibling functioning will be taken into account, which will allow us to examine inter-generational mechanisms (thus parental functioning that possibly impacts youth functioning and vice versa) and sibling functioning in these families. To investigate the latter, at each timepoint of the longitudinal study a sibling between 14 and 25 years of age at the start of the study will be included (if there is more than one sibling in a family, ideally the sibling who is closest in age will be the one who participates in the study). Moreover, a community sample that is matched on age and sex with the survivor of pediatric cancer will be assessed. This will allow the investigators to make well-founded comparisons regarding identity development and broader psychosocial functioning.

Psychological Benefits of a Normalized Camping Experience for Children With Cancer

Background: * Cancer has an enormous impact on the psychological and social well-being of the family unit. The life-threatening connotations of cancer single out the ill child from his peer/family group as one who is different, and often unable to maintain a normal lifestyle. Physical sequelae of cancer and its treatment accentuate the differences between these children and their normal peers/siblings. * It is important that children with cancer be prepared to function outside of protected situations and begin to develop skills of separation and independence. For healthy children, some of these latter skills are acquired by a camping experience. Such an experience for the patient with cancer is frequently precluded by their dependence on medical facilities and the physical limitations of their activities. * The goal of this study will be to assess the short and long term benefits of the "normalized" camping experience, provided in conjunction with Special Love, Inc., on the patients and staff. In particular, we will seek to determine whether such a comprehensive experience is capable of influencing the attitudes and life experiences of patients and staff in a positive manner. Objectives: -To evaluate the impact of an enriched normalized camping experience on the quality of life of the pediatric cancer patient. In particular, attempts will be made to measure the manner in which this experience influences the child's sense of well-being and self-esteem as well as his or her relationship with parents, family, and peers. Eligibility: * Children 7-17 years of age who are currently being treated for cancer or are up to 3 years post therapy OR Young adults with cancer (YACers) 18-25 years of age who are acting as counselors at Camp Fantastic * All children/young adults will be selected for camp after careful screening by a multidisciplinary committee consisting of medical and program directors. * At the discretion of the multidisciplinary committee consisting of medical and program directors, special exceptions may be made for children with extenuating circumstances. Design: * Assessment of benefit may include interviews with children and families before, during and following camp. Observational data on the child's performance at camp will be noted. * Medical and nursing personnel will consist of staff from the Pediatric Branch at the NCI, other units within the NIH, and participating institutions. * Special Love members, the Program Director at the 4-H Center camp (site of the camp) and Pediatric Branch staff at the NCI will coordinate the camp program, taking into account the medical needs of each camper. * Every attempt will be made to provide a full agenda of age appropriate activities for the patients. * The length of the camping experience for children with cancer will be for 7 days beginning on a Sunday and extending through the following Saturday morning. Patients will be transported to the camp from the NIH Clinical Center and the Virginia hospitals by bus.

Music Therapy for Oral Mucositis Pain in Pediatric Patients

This mixed-methods pilot study will examine the effect of music therapy on oral mucositis pain in pediatric oncology patients. Oral mucositis, characterized by painful ulcerative lesions in the mouth, is a common side effect of high-dose chemotherapy and hematopoietic stem cell or bone marrow transplantation. Despite its high prevalence and impact on quality of life, effective pain management strategies for pediatric oral mucositis remain limited and often rely heavily on medications that may cause significant side effects or provide insufficient relief. Music therapy may offer a promising non-pharmacologic adjunct for reducing pain, yet no studies to date have specifically evaluated its use for oral mucositis pain in pediatric patients.

Digital Art Therapy for Young Cancer Survivors

This is a single-site, single-arm, interventional study assessing the feasibility of the ARTCan Therapy Application (App) and whether it is an acceptable means of administering art therapy to young adult cancer survivors. The ARTCan Therapy App guides participants through a 6-week digital art therapy program. Subjects will participate in weekly art therapy prompts guided by the app and will complete weekly mental health quality of life (MHQoL) surveys during the intervention. In addition, baseline and end-of-intervention patient-reported outcome measures (PROMIS-DSF8a) and an acceptability survey will be administered. The hypothesis is that digital art therapy is feasible for young adult cancer survivors with self-reported mood issues and is an acceptable means of administering art therapy in the patient population.

The Comparative Effectiveness Evaluation of the Impact of Digital Education

This study is being conducted to determine how best to educate caregivers about cancer and its treatment. When caregivers are well-informed, they are more confident in supporting their child through treatment, which can improve treatment adherence.

Murottal as Adjunctive Intervention in Pediatric

This study aims to evaluate whether listening to the recitation of Surah Ar-Rahman (Murottal) before bedtime can help improve anxiety and sleep quality in hospitalized children with blood cancer. Twenty children aged 8-12 years will be divided into two groups: one group will receive standard care plus the Murottal intervention for 7 days, while the other group will receive standard care only. Anxiety and sleep quality will be measured using validated questionnaires (SCARED and SDSC) before and after the intervention. We hope this study will provide evidence that Murottal can be a safe, non-invasive, and culturally appropriate supportive therapy for pediatric cancer patients.

Umbilical Cord Blood Infusion to Promote Hematopoietic and Immune Reconstitution in Children With Relapsed or Refractory Malignant Solid Tumors

Pediatric malignant solid tumors (including neuroblastoma, Wilms tumor, hepatoblastoma, rhabdomyosarcoma, osteosarcoma, Ewing sarcoma, and germ cell tumors) account for approximately 60% of childhood malignancies. Despite multimodal therapy, children with relapsed or refractory disease have poor outcomes. Repeated lines of intensive salvage chemotherapy cause cumulative damage to the hematopoietic system, resulting in profound and prolonged myelosuppression (absolute neutrophil count \<0.5×10\^9/L or platelet count \<20×10\^9/L) that often fails to recover despite standard supportive care with short-acting recombinant human granulocyte colony-stimulating factor (rhG-CSF) and recombinant human thrombopoietin (rhTPO). This leads to chemotherapy delays that compromise treatment continuity and intensity. Umbilical cord blood (UCB) contains hematopoietic progenitor cells, NK cells, T lymphocytes, and various hematopoietic and immunomodulatory factors. UCB infusion has been shown to accelerate hematopoietic recovery after chemotherapy and may enhance anti-tumor immune function. This is a single-center, single-arm, prospective exploratory study. Approximately 10 children (aged 1-18 years) with relapsed or refractory malignant solid tumors who develop severe post-chemotherapy myelosuppression with delayed hematopoietic recovery (persistent ANC \<1.0×10\^9/L and platelets \<50×10\^9/L despite ≥7 days of rhG-CSF/rhTPO support, resulting in chemotherapy delay) will receive UCB infusion in addition to continuing background anti-tumor therapy and standardized hematopoietic supportive care. Each patient may receive up to 3 UCB infusions. The follow-up period is 12 weeks from the first infusion. The study aims to: (1) evaluate the effect of UCB infusion on promoting hematopoietic recovery and on-schedule chemotherapy completion (compared with institutional historical controls); (2) evaluate immune reconstitution after UCB infusion, including dynamics of T-lymphocyte subsets, NK cells, B cells, and serum cytokines; (3) assess the safety of UCB infusion; and (4) explore differences in hematopoietic recovery and immune reconstitution across tumor types and treatment backgrounds.

Pediatric Oncology Nutrition Intervention Trial

Nearly 60% of pediatric patients diagnosed with cancer develop malnutrition caused by a combination of disease burden, side effects of chemotherapy, and the intensity of cancer treatment. These patients are known to have an increased risk of infection, treatment-related toxicity, inferior clinical outcomes, and increased risk of mortality. Malnutrition may progress to cancer cachexia, characterized by anorexia, increased inflammation, decreased fat, and decreased muscle mass with subsequent weight loss, which is associated with decreased overall survival. The goal of the proposed research is to determine changes in body composition, weight status, and nutritional status between common nutrition interventions including oral nutrition supplements (ONS), appetite stimulants, and enteral nutrition (EN) among pediatric cancer patients. A secondary goal of this research is to utilize the findings to develop clinical nutrition guidelines for this patient population. The specific objective of the research proposed is to solve the lack of evidence to adequately treat nutritional deficits in the pediatric oncology population. Without this data, there is a lack of clinical consistency in the initiation and selection of appropriate nutrition interventions to provide a more definitive pathway of care. This study can help formulate a clinical guideline for this patient population before, during, and after treatment.

Vomiting Prevention in Children With Cancer

The goal of this single arm trial is to learn if a machine learning (ML) model predicting the risk of vomiting within the next 96 hours will impact vomiting outcomes in inpatient cancer pediatric patients. The main questions it aims to answer are whether an ML model predicting the risk of vomiting within the next 96 hours will: Primary 1\. Reduce the proportion with any vomiting within the 96-hour window Secondary 1. Reduce the number of vomiting episodes 2. Increase the proportion receiving care pathway-consistent care 3. Impact on specific antiemetic medication administrations Exploratory: To determine whether deployment of the ML model reduces the proportion of patients with any nausea within 96 hours and to characterize references to the algorithm in clinical notes following deployment, and to determine the number of times the prediction report dashboard was opened. Newly admitted participants will have a ML model predict the risk of vomiting within the next 96 hours according to their medical admission information. The prediction will be made at 8:30 AM following admission. Pharmacists will be charged with bringing information about patients' vomiting risk to the attention of the medical team and implementing interventions.

Barriers and Facilitators in Access to OT/PT in Pediatric Cancer Survivors

This study aims to identify the modifiable facilitators and barriers to occupational and physical therapy care for children during and after cancer treatment that may be contributing to disparities in health outcomes. The investigators will identify these facilitators and barriers by surveying and interviewing healthcare administrators at MD Anderson Cancer center. The investigators will also survey and interview physicians, advanced practice providers, and occupational and physical therapists to obtain their feedback on these issues.

Integration of Palliative Care in Pediatric Onco-hematology

PALONCOPED is a multicentre, interregional, observational and qualitative study aiming to better understand the early integration of palliative care in children with cancer. This study compares the practices, representations and temporalities of integration according to the type of cancer, in particular between hematological malignancies and solid tumors. The study seeks to identify the obstacles and levers encountered by professionals in pediatric onco-haematology and pediatric palliative care resource teams. It will be conducted in the hospitals of Nantes, Angers and Tours, based on about 45 semi-structured individual interviews with healthcare professionals. The data will be analyzed according to a reflexive thematic analysis, supplemented if relevant by a lexical analysis. The project aims to identify ways to improve for organizational and relational improvement, to strengthen collaboration between teams, and to promote earlier, coordinated care pathways adapted to children and their families.

PediRISE Feasibility

The goal of this research study is to learn whether investigators can successfully give the PediRISE program to families-in other words, whether most families are interested in participating in a study about the PediRISE program, including a 50-50 chance of receiving standard usual care, and a 50-50 chance of receiving the PediRISE support program. The names of the study groups in this research study are: * PediRISE Program Group * Usual Care Group

CAYA Cancer Prospective Cohort Study

Cancer is a leading cause of illness and death among children, adolescents, and young adults(CAYAs), especially in low- and middle-income countries(LMICs), where access to timely diagnosis and treatment is often limited. As a result, patients in these settings may experience higher rates of treatment complications, interruptions, and poorer outcomes compared with those in high-income countries (HICs). This is a prospective, multicenter observational study that will follow children, adolescents, and young adults(CAYAs) with cancer who are receiving routine care at participating hospitals in low - and middle - income countries(LMICs). The study does not involve experimental treatments or changes to standard medical care. Information will be collected from medical records and from questionnaires that address access to care and social factors affecting treatment. By describing treatment outcomes and the challenges patients and families face during cancer care, this study aims to provide data that can help inform future efforts to improve access to care and cancer outcomes in resource-limited settings.

Durvalumab and Tremelimumab for Pediatric Malignancies

The purpose of the study is to determine the recommended dose of durvalumab and tremelimumab (immunotherapy drugs) in pediatric patients with advanced solid and hematological cancers and expand in a second phase to test the efficacy of these drugs once this dose is determined.

Pharmacogenomic Testing in Pediatric Hematology/Oncology Patients

Pharmacogenomic (PGx) testing involves analyzing variants of genes associated with drug metabolism, transport and medication targets. PGx testing uses an individual's genetic factors, such as single nucleotide polymorphisms (SNPs), to personalize therapy or dose a selection of medications. PGx testing has traditionally been used to test single genes, but there are now platforms allowing a panel of genes to be tested at once. To date there has not been a comprehensive screening of pediatric oncology patients to determine the prevalence of genetic variants that may affect anticancer therapy and supportive care medications. This study would allow us to summarize the frequency of clinically relevant gene-drug interactions and actionable genetic polymorphisms in pediatric oncology patients.

Generating Novel Translational and Therapeutic Strategies Based on a Multicenter, Pediatric and AYA Evolutionary Tumor Board (pedsETB)

To evaluate the ability of a multidisciplinary and multisite group, the Pediatric Evolutionary Tumor Board (pedsETB), to develop additional therapeutic strategies in patients without curative options or with suboptimal outcomes.

Biomarkers of Resiliency In Childhood Cancer Surgery

This observational study is to better understand how children and their families recover after the stress of major surgery for cancer so that investigators can create ways to improve resilience during recovery. The main questions it aims to answer are: 1. Can information obtained from patients and their caregivers wearing smartwatches and answering questionnaires be used to measure how patients are recovering from surgery? 2. Are there specific patterns in patients' circulating proteins and metabolites that are associated with stress after surgery? Participants, including pediatric patients undergoing surgery for cancer and their primary caregiver, will be asked to: * wear a smartwatch * complete questionnaires * allow for extra blood to be drawn for this research study when they are having their regular blood draws for clinical purposes These actions will occur at baseline prior to patients' surgery and then afterwards for up to one year. There are no changes to participants' clinical care or surgical care as a result of the study. Investigators will also collect participants' clinical information and cancer-specific outcomes. Participants will be remunerated for their time.

Central Venous Catheter Care Training for Pediatric Oncology Nurses

The knowledge level of nurses regarding care techniques plays a crucial role in the prevention of central venous catheter-related infections. This study was designed to evaluate the effect of training on central venous catheter care (CVCC), provided to nurses caring for pediatric oncology patients, on their knowledge level and infection prevention behaviors. The study aims to determine the effectiveness of an educational program intended to enhance nurses' knowledge and practical skills in preventing CVC-related infections, thereby contributing to evidence-based practices that improve patient safety.