Pediatric Clinical Trials: Open, Ongoing and Recruiting Studies

Cardiology Clinical Trials

• Percutaneous Intervention Versus Observational Trial of Arterial Ductus in Low Weight Infants (PIVOTAL)
  Study ID: CAR25008 (PIVOTAL)
  PI: Daniel Duarte-Caceres. View Study Details

• PREEMIE: Study for Treatment of PDA in Premature Infants (PREEMIE)
  Study ID: CAR25032 (PDA-P3-23-01, PREEMIE)
  PI: Shyam Sathanandam. View Study Details

• A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). (CEDAR-HCM)
  Study ID: CAR25047 (20241212)
  PI: Ronald Kanter. View Study Details

Dermatology Clinical Trials

• A Study to Evaluate the Pharmacokinetics, Safety, and Effectiveness of Certolizumab Pegol in Children With Moderate to Severe Chronic Plaque Psoriasis (CIMcare)
  Study ID: DER99037 (PS0007)
  PI: Ana-Margarita Duarte. View Study Details

• A Study to Evaluate the Safety and Efficacy of Ruxolitinib Cream in Pediatric Participants With Nonsegmental Vitiligo
  Study ID: DER24083 (INCB 18424-312)
  PI: Ana-Margarita Duarte. View Study Details

Endocrinology Clinical Trials

• Universal Screening for Type 1 Diabetes in the Primary Clinical Setting
  Study ID: END25055(RDC92)
  PI: Adriana Carrillo. View Study Details

• A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
  Study ID: END24024 (BMN 111-210)
  PI: Adriana Carrillo. View Study Details

• Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
  Study ID: END25024 (LUM-201-10)
  PI: Alejandro Diaz. View Study Details

Gastroenterology Clinical Trials

• Microbiome and Genetic Profile [MGP] of Very Early-Onset Pediatric Inflammatory Bowel Disease and Pediatric Inflammatory Bowel Disease
  Study ID: GAS23122
  PI: Lina Maria Felipez. View Study Details

• Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis
  Study ID: GAS99042 (OVATION)
  PI: Lina Felipez. View Study Details

• Treatment and Acceptability of Specific Carbohydrate Diet for Children with Active Crohn's Disease
  Study ID: GAS99043
  PI: Lina Maria Felipez. View Study Details

• A prospective study evaluating serial intestinal ultrasound (IUS) use in pediatric Inflammatory Bowel Disease (IBD)
  Study ID: GAS24086
  PI: Lina Maria Felipez. View Study Details

• A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis (MIGHTY)
  Study ID: GAS24065 (M19-751)
  PI: Lina Maria Felipez. View Study Details

Hematology and Oncology Clinical Trials

• Triptorelin for the Prevention of Ovarian Damage in Adolescents and Young Adults With Cancer
  Study ID: ONC25053 (ALTE2131)
  PI: Maggie Fader. View Study Details

• A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
  Study ID: ONC99274 (AREN1921)
  PI: Maggie Fader. View Study Details

• A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)
  Study ID: ONC25036 (AREN2231)
  PI: Maggie Fader. View Study Details

• Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma
  Study ID: ONC25026 (ARAR2331)
  PI: Maggie Fader. View Study Details

• Tipifarnib and Naxitamab for Relapsed/​Refractory Neuroblastoma
  Study ID: ONC24067 (BCC022)
  PI: Guillermo De Angulo. View Study Details

• DFMO as Maintenance Therapy for Molecular High/​Very High Risk and Relapsed Medulloblastoma
  Study ID: ONC24008 (BCC016)
  PI: Ossama Maher. View Study Details

• Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors
  Study ID: ONC24057 (BCC021)
  PI: Maggie Fader. View Study Details

• Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)
  Study ID: ONC99078 (AGCT1532)
  PI: Maggie Fader. View Study Details

• A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
  Study ID: ONC23006 (ANHL2121)
  PI: Maggie Fader. View Study Details

• The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study
  Study ID: ONC99101 (APAL2020SC)
  PI: Maggie Fader. View Study Details

• A Study of the Drug Letermovir as Prevention of Cytomegalovirus Infection After Stem Cell Transplant in Pediatric Patients
  Study ID: ONC24035 (ACCL1932)
  PI: Ossama Maher. View Study Details

• Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling
  Study ID: ONC99139 (Ex Vivo 3)
  PI: Maggie Fader. View Study Details

• Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)
  Study ID: ONC99113 (NMTRC012)
  PI: Guillermo De Angulo. View Study Details

• A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab
  Study ID: ONC99271 (AHOD2131)
  PI: Maggie Fader. View Study Details

• Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
  Study ID: ONC99276 (ARST2032)
  PI: Maggie Fader. View Study Details

• A Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma
  Study ID: ONC25006 (ACNS2321)
  PI: Ziad Khatib. View Study Details

• Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma
  Study ID: ONC23118 (BCC018)
  PI: Maggie Fader. View Study Details

• The EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults With B-ALL
  Study ID: ONC99116 (PTCTC ONC1701)
  PI: Jorge Galvez-Silva. View Study Details

• A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss
  Study ID: ONC99270 (ACNS2031)
  PI: Ziad Khatib. View Study Details

• Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
  Study ID: ONC24030 (ANBL2131)
  PI: Maggie Fader. View Study Details

• Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
  Study ID: ONC99077 (AGCT1531)
  PI: Maggie Fader. View Study Details

• Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
  Study ID: ONC99085 (ANBL1531)
  PI: Maggie Fader. View Study Details

• Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy
  Study ID: ONC99062 (AALL1732)
  PI: Maggie Fader. View Study Details

• A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/​or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations
  Study ID: ONC99096 (AAML1831)
  PI: Maggie Fader. View Study Details

• A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
  Study ID: ONC99074 (ACNS1833)
  PI: Ziad Khatib. View Study Details

• Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
  Study ID: ONC99100 (AOST2031)
  PI: Ziad Khatib. View Study Details

• A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT
  Study ID: ONC99099 (ACNS2021)
  PI: Ziad Khatib. View Study Details

• A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)
  Study ID: ONC24032 (ARAR2221)
  PI: Maggie Fader. View Study Details

• Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia
  Study ID: ONC99060 (AALL1621)
  PI: Maggie Fader. View Study Details

• A Study to Test the Addition of the Drug Cabozantinib to Chemotherapy in Patients With Newly Diagnosed Osteosarcoma
  Study ID: ONC99273 (AOST2032)
  PI: Maggie Fader. View Study Details

• Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
  Study ID: ONC24047(BCC020)
  PI: Guillermo De Angulo. View Study Details

Nephrology Clinical Trials

• A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARB (ACTION3)
  Study ID: NEP24046 (DMX-200-301)
  PI: Ana Paredes. View Study Details

• Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases (EPPIK)
  Study ID: NEP99184 (RTRX-RE021-201)
  PI: Ana Paredes. View Study Details

• Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN (APPARENT)
  Study ID: NEP23144 (CLNP023B12302)
  PI: Ana Paredes. View Study Details

• Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G)
  Study ID: NEP23121 (CLNP023B12301)
  PI: Ana Paredes. View Study Details

Neurology Clinical Trials

• REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
  Study ID: NEU24071 (ION582-CS2)
  PI: Paula Schleifer. View Study Details

• Focal Cerebral Arteriopathy Steroid Trial (FOCAS)
  Study ID: NEU23170 (FOCAS)
  PI: Anuj Jayakar. View Study Details

• A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
  Study ID: NEU24088 (LP352-302)
  PI: Matt Lallas. View Study Details

• A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
  Study ID: NEU24061 (LP352-301)
  PI: Matt Lallas. View Study Details

• A Study to Test the Long-term Safety and Tolerability of Brivaracetam in Study Participants With Childhood Absence Epilepsy or Juvenile Absence Epilepsy
  Study ID: NEU24007 (EP0224)
  PI: Anuj Jayakar. View Study Details

• Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder
  Study ID: NEU24078 (20242004)
  PI: Matt Lallas. View Study Details

• A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult Subjects
  Study ID: NEU99205 (BT005)
  PI: John Ragheb. View Study Details

• A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
  Study ID: NEU24062 (NGN RTT-200)
  PI: Paula Schleifer. View Study Details

Orthopedics Clinical Trials

• Distal Radius Interventions for Fracture Treatment (DRIFT)
  Study ID: ORT23139 (2021-4883_DRIFT)
  PI: Monica Payares-Lizano. View Study Details

• Cast or Operation for Medial Epicondyle Fracture Treatment in Children (COMET)
  Study ID: ORT23136 (2022-4916_COMET)
  PI: Monica Payares-Lizano. View Study Details

• Developmental Dysplasia of the Hip: Observation Vs. Bracing
  Study ID: ORT23125 (Brace v. Observational)
  PI: Kevin Horowitz. View Study Details

Primary Care Centers Clinical Trials

• Food for Salud Medically Tailored Grocery Intervention
  Study ID: PCC25017 (Food for Salud)
  PI: Joanna Perdomo. View Study Details

Psychology Clinical Trials

• Wise Girls Workshop Study
  Study ID: PSC24069 (Wise Girls)
  PI: Marina Capo. View Study Details

• The Pediatric Precision Sleep Network
  Study ID: PSC24011 (PPSN)
  PI: Sara Rivero-Conil. View Study Details