Pediatric Clinical Trials: Open, Ongoing and Recruiting Studies

To inquire about current studies please contact us at:

NCRI.recruitment@nicklaushealth.org

Cardiology Clinical Trials

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

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

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

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

  • Phase I/​II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)
    Study ID: BMT23108 (ORCA-Q)
    PI: Jorge Galvez-Silva. 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

  • PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma
    Study ID: ONC23174 (CONNECT1906)
    PI: Ziad Khatib. 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

  • MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
    Study ID: ONC23127 (CONNECT2108)
    PI: Ziad Khatib. View Study Details

  • ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
    Study ID: ONC23173 (CONNECT1905)
    PI: Ziad Khatib. 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

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

  • Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma
    Study ID: ONC23150 (ACCL1931)
    PI: Maggie Fader. 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 Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) (TransIT)
    Study ID: BMT23109 (TransIT)
    PI: Jorge Galvez-Silva. 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

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

  • Randomized Study in Children and Adolescents With Migraine: Acute Treatment
    Study ID: NEU23013 (C4951002-BHV3000-311)
    PI: Wilson Heredia Nunez. View Study Details

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

  • Humanitarian Device Exemption (Dystonia IRB)
    Study ID: NEU99202 (H020007)
    PI: Toba Niazi. View Study Details

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

  • A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
    Study ID: NEU24048 (STK-001-DS-301)
    PI: Matt Lallas. View Study Details

  • Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric Diffuse Intrinsic Pontine Gliomas (DIPG)
    Study ID: NEU23134 (BT016)
    PI: Toba Niazi. View Study Details

  • A Study to Evaluate the Efficacy, Safety, and Tolerability of Brivaracetam as Monotherapy in Patients 2 to 25 Years of Age With Childhood Absence Epilepsy or Juvenile Absence Epilepsy (EXPAND)
    Study ID: NEU23003 (N01269)
    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

  • 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