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Displaying 1 - 20 of 100

Open Actively Recruiting

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.

This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).

Phase: Phase 2
Primary Purpose: Prevention
Gender: All
Contact: Natalie Solis
Open Actively Recruiting

A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder

This is a multicenter, double-blind, parallel-group, placebo controlled, 2-part study to evaluate the efficacy and safety of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

Phase: Phase III
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Angela Martinez
Open Actively Recruiting

A Study to Compare Early Use of Vinorelbine and Maintenance Therapy for Patients With High Risk Rhabdomyosarcoma

This phase III trial compares the safety and effect of adding vinorelbine to vincristine, dactinomycin, and cyclophosphamide (VAC) for the treatment of patients with high risk rhabdomyosarcoma (RMS). High risk refers to cancer that is likely to recur (come back) after treatment or spread to other parts of the body. This study will also examine if adding maintenance therapy after VAC therapy, with or without vinorelbine, will help get rid of the cancer and/or lower the chance that the cancer comes back. Vinorelbine and vincristine are in a class of medications called vinca alkaloids. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy. Cyclophosphamide is in a class of medications called alkylating agents. Vinorelbine, vincristine, dactinomycin and cyclophosphamide are chemotherapy medications that work by slowing or stopping the growth of cancer cells in the body. This trial may have the potential to eliminate rhabdomyosarcoma for a long time or for the rest of patient's life.

Phase: Phase III
Primary Purpose: Treatment
Gender: All
Age Group: Children
Contact: Mikayla Marvis Henderson
Open Actively Recruiting

Stopping Tyrosine Kinase Inhibitors in Affecting Treatment-Free Remission in Patients With Chronic Phase Chronic Myeloid Leukemia

This phase II trial studies how stopping tyrosine kinase inhibitors will affect treatment-free remission in patients with chronic myeloid leukemia in chronic phase. When the level of disease is very low, it's called molecular remission. TKIs are a type of medication that help keep this level low. However, after being in molecular remission for a specific amount of time, it may not be necessary to take tyrosine kinase inhibitors. It is not yet known whether stopping tyrosine kinase inhibitors will help patients with chronic myeloid leukemia in chronic phase continue or re-achieve molecular remission.

Phase: Phase 2
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Mikayla Marvis Henderson
Open Actively Recruiting

Mobile Health and Social Media Physical Activity Intervention Among Adolescent and Young Adult Childhood Cancer Survivors, the StepByStep Study

This phase III trial compares a multi-component mobile health and social media physical activity intervention versus wearing a physical activity tracker alone among adolescent and young adult childhood cancer survivors. Regular physical activity helps maintain healthy weight, energy levels, and health. Adolescents and young adults who complete treatment for cancer are often less active. They may gain weight and have more health problems compared to people the same age who have not had treatment for cancer. Comparing the 2 programs will help researchers learn how to increase physical activity levels over time and also how changes in physical activity levels affect health and quality of life over time.

Phase: N/A
Primary Purpose: Supportive Care
Gender: All
Age Group: Children
Contact: Mikayla Marvis Henderson
Open Actively Recruiting

Endophenotypes of Persistent Post Concussive Symptoms

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

Contact: Yena Kim
Open Actively Recruiting

secretin levels in refeeding-induced polyuria (cross section)

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

Age Group: All
Contact: Katherine Perry
Open Actively Recruiting

Blood and tissue banking from sarcoma patients receiving immunotherapy

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

Phase: N/A
Age Group: All
Contact: THEODORE NOWICKI
Open Actively Recruiting

Long-Term Clinical, Immunologic, and Virologic Profiles of Children who Received Early Treatment for HIV

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

Contact: Michele Carter
Open Actively Recruiting

A Prospective Prevalence Study in Adolescent and Adult Patients Dependent on Parenteral Nutrition to Assess the Incidence of Intestinal Failure-Associated Liver Disease

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

Age Group: All
Contact: Susan Feist
Open Actively Recruiting

Basimglurant in Children and Adolescents With TSC

The study intends to show that basimglurant provides effective seizure control in children and adolescents with Tuberous Sclerosis Complex (TSC).

Phase: Phase II
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Angela Martinez
Open Actively Recruiting

HM15136 Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)

This study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15136 when used as add-on therapy in subjects with CHI with persistent hypoglycemia while on standard of care treatment (SoC). HM15136 will be administered once weekly in multiple doses to subjects in multiple age including pediatric to find appropriate exposure-response data.

Phase: Phase II
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Angela Martinez
Investigator: Erin R. Okawa, MD
Open Actively Recruiting

Pharmacokinetic Properties of Antiretroviral and Anti-Tuberculosis Drugs During Pregnancy and Postpartum

The purpose of this study is to evaluate the pharmacokinetic (PK) properties of antiretroviral (ARV) and anti-tuberculosis (TB) drugs administered during pregnancy and postpartum.

Phase: Phase 4
Primary Purpose: Treatment
Gender: Female
Contact: Michele Carter
Open Actively Recruiting

A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery

This phase II trial studies the side effects and how well nirogacestat works in treating patients patients less than 18 years of age with desmoid tumors that has grown after at least one form of treatment by mouth or in the vein that cannot be removed by surgery. Nirogacestat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Phase: N/A
Primary Purpose: Treatment
Gender: All
Age Group: Children
Contact: Mikayla Marvis Henderson
Open Actively Recruiting

A Mobile Phone Intervention to Promote Adherence to Survivorship Care Among Adolescent and Young Adult Cancer Survivors

Adolescents and young adults (AYA) survivors of childhood cancer receive inadequate surveillance for treatment-related late effects. This study evaluates the acceptability and feasibility of a mobile phone messaging intervention targeting behavior changes related to receiving survivorship care among AYA survivors.

Content from an existing text-messaging survivorship education intervention will be adapted to a novel text-messaging platform, Chorus. Ten AYA survivors will be recruited to participate in the expanded 12-week text messaging intervention. In-depth interviews will be conducted with each participant during the study, and a focus group with all participants will be conducted at the end of the study. Qualitative analysis will determine areas for improvement in the text messaging intervention in order to ensure acceptability and feasibility in AYA survivors. The text messaging intervention on Chorus will be adapted to better suit the needs of AYA survivors based on the results of the qualitative analysis. Mobile phone interventions offer a cost-effective, age-appropriate approach to AYA survivorship care education. If effective, the text-messaging intervention will be evaluated in a randomized, controlled pilot trial to improve rates of survivorship care and clinical outcomes for AYA survivors.

Phase: Pilot/Feasibility
Primary Purpose: Health Services Research
Gender: All
Age Group: Adults
Contact: Mark Garcia
Investigator:
Mark Garcia
Open Actively Recruiting

A Study to Evaluate EDP 938 Regimens in Children With RSV

A 2-part study to evaluate the safety, pharmacokinetics and efficacy of EDP-938 in children with RSV infection.

Phase: Phase II
Primary Purpose: Treatment
Gender: All
Age Group: Children
Open Actively Recruiting

A Study of ZN-c3 in Combination With Gemcitabine in Subjects With Osteosarcoma

This is a phase 1/2 study of ZN-c3 in combination with gemcitabine in adult and pediatric subjects with relapsed or refractory osteosarcoma.

Phase: Phase I/II
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Rubi Arias
Open Actively Recruiting

Surveillance and Treatment to Prevent Fetal Atrioventricular Block Likely to Occur Quickly (STOP BLOQ)

Fetal complete (i.e., third degree, 3°) atrioventricular block (AVB), identified in the 2nd trimester of pregnancy in an otherwise normally developing heart, is almost universally associated with maternal anti-Ro autoantibodies and results in death in a fifth of cases. To date treatment of 3° AVB has been ineffective in restoring normal rhythm (NR) which may be because current surveillance is limited to once- weekly fetal echocardiograms. It is hypothesized that there may be a vital transition period of several hours in which incomplete block (2° AVB) may be successfully treated avoiding fully advanced irreversible 3° AVB. To optimize the likelihood of timely detection of the transition period this study comprises three steps: 1) to risk stratify for high titer anti-Ro antibodies, which are necessary but not sufficient to develop fetal AVB; 2) to empower mothers to identify 2° AVB by using fetal heart rate and rhythm monitoring (FHRM) at home, and 3) to rapidly treat mothers who detect an abnormality by monitoring with an urgent echocardiogram that confirms 2° AVB with the hope of reversing 2° AVB before it becomes permanent (3° AVB). In addition, it will be determined if FHRM reduces the need for weekly echoes. Although mothers with low titer anti-Ro will not be continued in Step 2 and therefore not followed by FHRM, birth ECGs will be collected to confirm that low titer antibodies do not confer risk. It is anticipated that this study will provide an evidenced based surveillance strategy for those mothers at high risk of having a child with 3° AVB.

Phase: Phase 3
Primary Purpose: Treatment
Gender: Female
Contact: Kimberly Schweigman
Investigator: Gary M. Satou, MD
Open Actively Recruiting

Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum

This is a prospective Phase II multi-center study with an upfront 16-week, randomized, double-blind, placebo-controlled period, and extension periods, to assess the efficacy, safety and pharmacokinetics of alpelisib in pediatric and adult participants with PIK3CA-related overgrowth spectrum (PROS).

Phase: Phase II
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Mikayla Marvis Henderson
Open Actively Recruiting

A Study to Compare Blinatumomab Alone to Blinatumomab With Nivolumab in Patients Diagnosed With First Relapse B-Cell Acute Lymphoblastic Leukemia (B-ALL)

This phase II trial studies the effect of nivolumab in combination with blinatumomab compared to blinatumomab alone in treating patients with B-cell acute lymphoblastic leukemia (B-ALL) that has come back (relapsed). Down syndrome patients with relapsed B-ALL are included in this study. Blinatumomab is an antibody, which is a protein that identifies and targets specific molecules in the body. Blinatumomab searches for and attaches itself to the cancer cell. Once attached, an immune response occurs which may kill the cancer cell. Nivolumab is a medicine that may boost a patient's immune system. Giving nivolumab in combination with blinatumomab may cause the cancer to stop growing for a period of time, and for some patients, it may lessen the symptoms, such as pain, that are caused by the cancer.

Phase: Phase II
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Mikayla Marvis Henderson