Clinical trials

Clinical Trials

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Learn More About our Clinical Trials

It is scientifically proven that children who participate in clinical trials have significantly better outcomes. That’s one of the many reasons research is so vital to us—and why we actively encourage and participate in these trials.

At Cure 4 The Kids Foundation, we believe access to cutting-edge care shouldn’t depend on volume or location. We’ve opened clinical trials for a single patient because we believe every child is worth it. Our patients have access to a wide range of national trials through the Children’s Oncology Group (COG) and NCORP, as well as to our own internally developed, onsite clinical trials tailored to meet the unique needs of the children we serve.

Clinical trials are expensive. They require additional diagnostic testing, ongoing monitoring, and specialized education. But we believe every child deserves access to the best treatment science has to offer—regardless of cost.

That’s why we place a strong emphasis on raising funds to support this important program. We aim to ensure no child is ever denied access to a clinical trial because of financial barriers. When it comes to saving a child’s life, one is never too few.

Children's Oncology Group
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NCORP
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C4K Active Clinical Trials
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Learn More About our Active Clinical Trials

Hematology

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Oncology

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Rare Diseases and Disorders

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Behavioral Health

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Clinical Studies

What is Clinical Research?

Clinical research helps doctors and scientists find new ways to prevent, treat, diagnose, or relieve symptoms of disease in people. Through clinical trials, researchers test the safety and effectiveness of new medications, devices, and other treatments. Unlike standard care, which uses established treatments, clinical research collects data to establish new therapies. Volunteers with specific health conditions participate in clinical research, adding to medical knowledge.

Participation is always voluntary, and safety is closely monitored. Clinical research ultimately drives medical advancements and improves healthcare outcomes. The most common types of clinical trials are interventional studies, observational studies, and compassionate use programs.

What happens if I think I want to participate in clinical research?

If you think you might want to volunteer for a clinical research study, contact our staff to find out more information! Somone from our research team will explain the details of the study to you.

Before you can participate on a clinical study, our doctors and research staff will do a comprehensive review of your medical history to ensure you meet the criteria to participate on a trial. We will ensure that the trial is right for you, that you understand the purpose of the trial, and give you time to ask questions.

No study procedures will be performed without obtaining your informed consent. 

What is Informed Consent?

Informed consent is one of the first most important steps of clinical research.

This is the process by which the patient voluntarily confirms willingness to participate on a clinical study. Before performing informed consent, all aspects of the clinical study are discussed to ensure the patient has a proper understanding of the procedures involved in the study. A through medical review will be done to ensure the patient meets the criteria for a clinical trial. The patient can take the consent form to speak to their families, friends, and other medical professionals. Our doctors and research staff will ensure that all questions are answered before signing.

The informed consent process usually highlights key elements about participation on the trial, such as the purpose of the study, benefits vs risks, confidentiality, compensation and reimbursement, rights and responsibilities, contacts, alternative treatments, and that research is voluntary. The informed consent form is always written in a language that is easy to understand and will be available in multiple languages upon request.

Even after signing the informed consent form, all research participation is voluntary. A participant can withdraw from a clinical trial for any reason and at any time. It will not impact their standard of care treatments with our facility. If a participant chooses to withdraw from a trial, then all withdrawal procedures and alternative treatments will be discussed.

What is Consent and Assent?

Consent and Assent in clinical research refer to the Informed Consent Forms (ICFs) that are required for patients to sign before they can participate in clinical studies. Consents refer to ICFs that are targeted for research participants over the age of 18, or parents of minors. Assents refer to ICFs that are simpler and written in a language that can be understood by pediatric patients aged 7-17.

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Hematology Studies

Title: Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

Protocol: AVA-PED-301

Study Type: Interventional

Status: Closed for Enrollment

Condition/Disease: Immune Thrombocytopenia

Principal/Investigator: Waseem Alhushki, MD

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Title: Prospective, Observational, Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A

Protocol: OBS17523

Study Type: Observational

Status: Open for Enrollment

Condition/Disease: Hemophilia A

Principal/Investigator: Aimee Foord, DO

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Title: A Multicentre Trial Evaluating the Efficacy and Safety of Oral Decitabine Tetrahydrouridine (NDec) in Patients With Sickle Cell Disease

Protocol: NN7533-4470

Study Type: Interventional

Status: Closed for Enrollment

Condition/Disease: Sickle Cell Disease

Principal/Investigator: Aimee Foord, DO

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Title: A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease

Protocol: TAK-577-3001

Study Type: Interventional

Status: Open for Enrollment

Condition/Disease: Von Willebrand Disease (vWD)

Principal/Investigator: Aimee Foord, DO

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Title: ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People with Non-Neoplastic Hematologic Disorders Protocol: ATHN Transcends Study Type: Observational Status: Open for Enrollment Condition/Disease: Non-Neoplastic Hematologic Disorders Principal/Investigator: Aimee Foord, DO Read Study

Oncology Studies

Title: HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Randomization to Either Single Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell Rescue

Protocol: IRB15-00399

Study Type: Interventional

Status: Closed for Enrollment

Condition/Disease: Medulloblastoma, Central Nervous System Embryonal Tumors

Principal/Investigator: Joseph Lasky III, MD

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Title: Single Patient Expanded Access to Nirogacestat for Desmoid Tumors

Protocol: N/A

Study Type: Compassionate Use Program

Status: Open for Enrollment

Condition/Disease: Desmoid Tumor

Principal/Investigator: Waseem Alhushki, MD

Title: Expanded Access Program for Rivoceranib for treatment of Relapsed Osteosarcoma

Protocol: N/A

Study Type: Compassionate Use Program

Status: Open for Enrollment

Condition/Disease: Relapsed Osteosarcoma

Principal/Investigator: Joseph Lasky III, MD

Click below to learn more about COG and NCORP clinical trials that are available at Cure 4 The Kids Foundation!

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Rare Diseases and Disorders Studies

Title: Pompe Disease Registry

Protocol: DIREGC07005

Study Type: Observational

Status: Open for Enrollment

Condition/Disease: Pompe Disease

Principal/Investigator: Sumit Gupta, MD

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Title: Fabry Disease Registry

Protocol: DIREGC07006

Study Type: Observational

Status: Open for Enrollment

Condition/Disease: Fabry Disease

Principal/Investigator: Sumit Gupta, MD

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Title: Mucopolysaccharidosis I (MPS I) Registry

Protocol: DIREGC07008

Study Type: Observational

Status: Open for Enrollment

Condition/Disease: Fabry Disease

Principal/Investigator: Sumit Gupta, MD

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Title: Intrathecal 2-Hydroxypropyl-β-cyclodextrin For Neurological Decline in Patients with Niemann-Pick Disease Type C1

Protocol: N/A

Study Type: Compassionate Use Program

Status: Open for Enrollment

Condition/Disease: Niemann-Pick Disease

Principal/Investigator: Joseph Lasky III, MD

Behavioral Health Studies

Title: Shifting Perspective: Investigative iRest Yoga Nidra Meditation’s Influence on Healthcare Staff Burnout, Well-being, and Self-Care in High-Stake Environments

Protocol: iRest: Shifting Perspectives

Study Type: Internal

Status: Closed for Enrollment

Indication:
Staff Burnout

Principal/Investigator: Danielle Bello, Ph.D