Selected Clinical Studies from Cleveland Clinic Children’s

To learn more about these and other active Cleveland Clinic Children’s studies, please send a message to pedsresearch@ccf.org.

Title: A Phase 4, Open-Label, Non-randomized, Multicenter Study to Evaluate Safety and Efficacy of Intravenous Administration of OPTISON™ for Contrast- Enhanced Echocardiography in Pediatric Patients

Sponsor: GE Healthcare

Ages Eligible: 9 to 17 years

Primary Investigator: Francine Erenberg, MD

For more information, visit clinicaltrials.gov.

Title: A Phase 3, Randomized, Double-Blind, Multinational, Placebo-Controlled Study to Evaluate Efficacy and Safety of Teplizumab (PRV-031), a Humanized, FcR Non-Binding, anti-CD3 Monoclonal Antibody, in Children and Adolescents with Newly Diagnosed Type 1 Diabetes (T1D) (PROTECT)

Sponsor: Provention Bio

Ages Eligible: 8 to 17 years

Primary Investigator: Anzar Haider, MD

For more information, visit clinicaltrials.gov.

Title: A Phase 3 Multicenter, Double-Blind, Placebo-Controlled Trial of Viralym-M (ALVR-105) for the Treatment of Patients with Virus-Associated Hemorrhagic Cystitis After Allogeneic Hematopoietic Cell Transplant (Protocol AVM-003-HC)

Sponsor: AlloVir

Ages Eligible: Any age

Primary Investigator: Rabi Hanna, MD

For more information, visit clinicaltrials.gov.

Title: A phase 3 randomized trial for patients with de novo AML comparing standard therapy including gemtuzumab ozogamicin (GO) to CPX-351 with GO, and the addition of the FLT3 inhibitor gilteritinib for patients with FLT3 mutations

Ages Eligible: up to 22 years

Primary Investigator: Rabi Hanna, MD

Sponsor: Children’s Oncology Group

For more information, visit clinicaltrials.gov.

Title: A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects with Severe Sickle Cell Disease

Sponsor: Editas Medicine

Ages Eligible: 18 to 50 years

Primary Investigator: Rabi Hanna, MD

For more information, visit clinicaltrials.gov.

Title: A Phase II Pilot Study of Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults with High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant: A Multicenter Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) Study (EXCEL Trial)

Sponsor: Nationwide Children’s Hospital

Ages Eligible: Less than or equal to 25 years

Primary Investigator: Rabi Hanna, MD

For more information, visit clinicaltrials.gov.

Title: Phase 1/2, Double-Blind, Placebo-Controlled, Dose Escalation and Expansion Study of ALVR106 in Addition to Standard of Care for the Treatment of High-Risk Patients with Respiratory Viral Infections After Hematopoietic Cell Transplant

Sponsor: AlloVir

Ages Eligible: 17 to 75 years

Primary Investigator: Rabi Hanna, MD

For more information, visit clinicaltrials.gov.

Title: Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients with Chronic Acid Sphingomyelinase Deficiency (ASMD)

Sponsor: Sanofi

Ages Eligible: 3 years and older

Primary Investigator: Rabi Hanna, MD

For more information, visit clinicaltrials.gov.

Title: A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to < 18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)

Sponsor: Alexion Pharmaceuticals

Ages Eligible: 1 month to 17 years

Primary Investigator: Seth Rotz, MD

For more information, visit clinicaltrials.gov.

Title: Evolutionary Inspired Therapy For Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma

Sponsor: National Pediatric Cancer Foundation

Ages Eligible: Any age

Primary Investigator: Matteo Trucco, MD

For more information, visit clinicaltrials.gov.

Title: A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination with Azacitidine in Patients with Recurrent, Resectable Osteosarcoma

Sponsor: National Pediatric Cancer Foundation

Ages Eligible: up to 39 years

Primary Investigator: Matteo Trucco, MD

For more information, visit clinicaltrials.gov.

Title: A Phase I/II Open-Label Trial of CPI-613® (devimistat) plus Hydroxychloroquine to Evaluate Maximally Tolerated Dose (MTD), Safety and Efficacy in Patients with Relapsed or Refractory Clear Cell Sarcoma (CCS)

Sponsor: Rafael Pharmaceuticals

Ages Eligible: 11 years to 75 years

Primary Investigator: Matteo Trucco, MD

For more information, visit clinicaltrials.gov.

Title: Precision Medicine Proof of Concept for TNF Inhibition in FSGS and Treatment Resistant Minimal Change Disease

Sponsor: University of Michigan

Ages Eligible: 6 years to 70 years

Primary Investigator: John Sedor, MD (Katherine Dell, MD, pediatric co-investigator)

For more information, visit clinicaltrials.gov.

Title: An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome

Sponsor: Acadia Pharmaceuticals

Ages Eligible: 5 to 22 years

Primary Investigator: Sumit Parikh, MD

For more information, visit clinicaltrials.gov.

Title: A Phase 3 Study of Adjunctive XEN496 in Pediatric Subjects With KCNQ2 Developmental and Epileptic Encephalopathy

Sponsor: Xenon Pharmaceuticals

Ages Eligible: 1 month to 6 years

Primary Investigator: Elia Pestana-Knight, MD

For more information, visit clinicaltrials.gov.

Title: A Master Protocol to Test the Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (SIMPLIFY)

Sponsor: Cystic Fibrosis Foundation

Ages Eligible: 12 years and older

Primary Investigator: Eliot Dasenbrook, MD (Nathan Kraynack, MD, pediatric co-investigator)

For more information, visit clinicaltrials.gov.