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Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Institutes of Health and JDRF
Summary: This is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo-controlled clinical trial. The study aims assess the efficacy, safety and mode of action of hydroxychloroquine to prevent progression from Stage 1 (normal glucose tolerance) to Stage 2 (abnormal glucose tolerance) or Stage 3 (clinically overt) T1D.
Eligibility: Individuals at Stage 1 T1D (two or more antibodies and normal glucose tolerance), age > 3 years and previously enrolled in TrialNet Pathway to Prevention Study.
Primary Investigator: Sumana Narasimhan, MD
Sponsor: Mirum Pharmaceuticals Inc.
Summary: This phase 3 study will evaluate the efficacy of maralixibat versus placebo on the severity and frequency of pruritus and on total serum bile acid (sBA) levels. The study will also evaluate the safety, tolerability and pharmacokinetics of maralixibat versus placebo.
Eligibility: Individuals > 12 months and < 18 years of age with a diagnosis of PFIC and persistent pruritus, biochemical abnormalities or pathological evidence of progressive liver disease.
Primary Investigator: Vera Hupertz, MD
Sponsor: Turning Point Therapeutics, Inc.
Summary: This is a phase 1/2, open-label, single-arm, multicenter, multi-cohort study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of the ALK/ROS1/NTRK inhibitor, repotrectinib, in pediatric and young adult subjects with advanced or metastatic solid tumors, primary CNS tumors, or anaplastic large cell lymphoma (ALCL) with anaplastic lymphoma kinase (ALK), receptor tyrosine kinase encoded by the gene ROS1 (ROS1), or neurotrophic receptor kinase genes encoding TRK kinase family (NTRK1-3) alterations. The Phase 1 dose escalation part will determine the Pediatric Recommended Phase 2 Dose (RP2D). Phase 2 of the study comprises three cohorts depending on gene alterations.
Eligibility: Pediatric subjects < 12 years old with locally advanced or metastatic solid tumors, including a primary central nervous system (CNS) tumor, or ALCL, with disease progression or who is non-responsive or intolerant to available therapies and for which no standard or available curative therapy exists. Subjects from Phase 1 are eligible for enrollment in Phase 2. Additional subjects 12 years to < 25 years old may be directly enrolled into the Phase 2 part of the study. Phase 2 subjects have advanced solid tumors (including primary CNS tumors) or ALCL with documented genetic ALK, ROS1 or NTRK1-3 alteration.
Primary Investigator: Matteo Trucco, MD
Sponsor: Atara Biotherapeutics, Inc.
Summary: This is a multicenter, open-label, single-arm phase 3 study to assess the efficacy and safety of tabelecleucel for the treatment of Epstein-Barr Virus positive (EBV+) post-transplant lymphoproliferative disorder (PTLD) in the setting of (a) solid organ transplant (SOT) after failure of rituximab or rituximab plus chemotherapy, or (b) hematopoietic cell transplant (HCT) after failure of rituximab.
Eligibility: Males and females of any age must have a diagnosis of locally assessed, biopsy-proven EBV+ PTLD with a prior history of SOT of kidney, heart, lung, pancreas, small bowel or any combination of these (SOT cohort); or prior allogeneic HCT (HCT cohort). Subjects must also have had treatment failure of rituximab or interchangeable biosimilar monotherapy or rituximab plus any concurrent or sequentially administered chemotherapy regimen for treatment of PTLD.
Primary Investigator: Rabi Hanna, MD
Sponsor: Chiesi Farmaceutici S.p.A.
Summary: Neonates will be randomly assigned in a 2:1 ratio to receive either CHF6563 (a sublingual form of buprenorphine solution) or morphine for the treatment of neonatal opioid withdrawal syndrome (NOWS). This study will assess the efficacy, safety, tolerability and pharmacokinetics of CHF6563 in babies with NOWS.
Eligibility: Newborns of gestational age of 36 weeks or greater and up to 7 days old exposed to opioids during the last month before delivery showing signs of NOWS with or without concomitant drug withdrawal syndromes requiring treatment.
Primary Investigator: Jalal Abu-Shaweesh, MD
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Summary: A two-arm, randomized, controlled, prospective, clinical trial to determine whether a multi-component program of behavioral interventions will result in a reduced risk of stone disease recurrence/progression over a 2-year period. Behavioral interventions will target increased fluid intake (sufficient to achieve a urine output > 2.5 L daily or > 30 mL/kg/day for those study participants < 18 years and weighing < 75 kg.
Eligibility: Study participants > 12 years of age with > 1 symptomatic stone event in past 3-5 years.
Primary Investigator: Sri Sivalingam, MD
Sponsor: Cystic Fibrosis Foundation, Inc. (CFF)
Summary: The prospective, multicenter observational study aims to collect physiological and clinical data plus biospecimens from patients with cystic fibrosis (CF) patients who are prescribed new cystic fibrosis transmembrane conductance regulator (CFTR) modulator triple combination therapy (TCT) (elexacaftor, tezacaftor and ivacaftor) before and after treatment with that modulator.
Eligibility: Subjects > 12 years old with a diagnosis of CF.
Primary Investigator: Elliott Dasenbrook, MD
Sponsor: West-Ward Pharmaceutical Corp.
Summary: The objective of this study is to evaluate the dose effect of Phenylephrine Hydrochloride Injection, USP on the treatment of clinically relevant decreased blood pressure in the pediatric population, > 12- to 16-year-old patients undergoing general and neuroaxial anesthesia. The study also seeks to describe changes in blood pressure and heart rate, the time to onset and to maximal response, and the duration of response; to assess the safety of the product in this population, and to characterize the pharmacokinetics of phenylephrine.
Eligibility: Subjects > 12 to 16 years old scheduled for a procedure that requires general or neuroaxial anesthesia.
Primary Investigator: Surendrasingh Chhabada, MD
Summary: Dynamic Compression Braces have shown to be an effective alternative treatment to surgery for pectus carinatum. This study investigates patients’ overall satisfaction with the treatment methods and results.
Eligibility: Patients > 14 years of age treated for pectus carinatum from the inception of the bracing program at the Cleveland Clinic in 2016 to present.
Primary Investigator: Anthony DeRoss, MD
Sponsor: ACADIA Pharmaceuticals Inc.
Summary: This study investigates the efficacy, safety, tolerability and pharmacokinetics of treatment with oral trofinetide versus placebo in girls and women with Rett syndrome. Efficacy of trofinetide will utilize measures for behavior, communication, quality of life and gross motor skills.
Eligibility: Female subjects 5 to 20 years of age, inclusive, with classic/typical Rett syndrome.
Primary Investigator: Sumit Parikh, MD
Summary: This study will evaluate the efficacy of PF-06651600, a Janus Kinase 3 inhibitor, compared to placebo in adult and adolescent alopecia areata subjects with 50% or greater scalp hair loss on regrowth of lost hair and to characterize the exposure response of PF-06651600 on regrowth of lost hair.
Eligibility: Male and female subjects age 12 years and older must have moderate to severe AA with > 50% hair loss of the scalp without evidence of terminal hair regrowth with the previous 6 months and with the current episode of hair loss < 10 years.
Primary Investigator: Wilma Bergfeld, MD
Summary: This study aims to assess the combined clinical and microbiological efficacy, safety and tolerability of gepotidacin compared to nitrofurantoin in female participants with acute cystitis in the Microbiological Intent-to-Treat nitrofurantoin-Susceptible (micro-ITT NTF-S) Population.
Eligibility: Female subjects > 12 years old with clinical signs and symptoms of acute cystitis and presence of nitrites or pyuria in urine sample.
Primary Investigator: Terence Isakov, MD
Sponsor: F. Hoffman-La Roche Ltd.
Summary: This 2-year, phase 2 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of ocrelizumab in children and adolescents with relapsing-remitting multiple sclerosis (RRMS). The study will also serve to determine the dose of ocrelizumab to be further investigated in the subsequent phase 3 study in children and adolescents.
Eligibility: Children and adolescents ages > 10 to < 18 years with RRMS.
Primary Investigator: Mary Rensel, MD
Sponsor: Cincinnati Children’s Hospital Medical Center
Summary: This multicenter, cross-sectional investigation aims to identify biochemical and drug concentration targets for pediatric-onset Crohn’s Disease (CD) in order to personalize anti-TNF dosing regimens and improve long-term outcomes. The study will collect biospecimens (blood, stool and intestinal biopsies) to support the development of specific pharmacodynamic biomarkers of treatment response to anti-TNF (infliximab, adalimumab or either biosimilar) and the discovery of specific anti-TNF concentrations associated with mucosal healing in children and young adults with CD.
Eligibility: Individuals > 1 year to < 22 years of age diagnosed with Crohn’s Disease and who have received anti-TNF therapy for at least 6 months undergoing a clinically indicated colonoscopy.
Primary Investigator: Jacob Kurowski, MD
Sponsor: National Institutes of Health
Summary: This is an observational study designed to assess the impact of renal risk variants in the APOL1 gene on the functional outcomes of kidneys transplanted from living and deceased donors with recent African ancestry and on the kidney function of living kidney donors with recent African ancestry in a prospective manner.
Eligibility: Individuals who are living kidney donors with self-reported recent African ancestry (defined as African American, Afro-Caribbean, Hispanic black or African) or recipients of a kidney transplant from an eligible living or deceased donor with recent African ancestry including recipients of multi-organ transplants including a kidney plus an additional organ (i.e., liver, heart, lung, pancreas, etc.) or pediatric en bloc and dual kidney transplants.
Principal Investigator: Emilio Poggio, MD