A Multi-center, Double-Blind, Randomized, Two-Arm, Parallel-Group, Placebo Controlled Study to Assess the Efficacy and Safety of ELGN-2112 on Intestinal Malabsorption in Preterm Infants

PI: Eilon Shany

A Phase 3, Multi-Center, Randomized, Double- Blind, Placebo-Controlled Study to Assess the Efficancy and Safety of APREMILAST (CC-10004) in Pediatric Subjects from 6 through 17 Years of age with Moderate to Severe Plaque Psoriasis

PI: Amir Horev. Funded: Celgene Corporation.

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Outpatient Study Evaluating the Pharmacokinetics, Efficacy, and Safety of Baricitinib in Pediatric Patients with Moderate to Severe Atopic Dermatitis. BREEZE-AD-PEDS. Protocol I4V-MC-JAIP. Genetic Sub Study.

PI: Amir Horev. Funded: Eli Lilly

"A randomized, double-blind, placebo- and active controlled multicenter trial to demonstrate efficacy of subcutaneous secukinumab compared to placebo and etanercept (in a single-blinded arm) after twelve weeks of treatment, and to assess the safety, tolerability, and long-term efficacy in subjects from 6 to less than 18 years of age with severe chronic plaque psoriasis"

PI: Amir Horev. Funded: Novartis

A Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Combination with Topical Corticosteroids in Adolescent and Adult Subjects with Moderate to Severe Atopic Dermatitis

PI: Amir Horev. Funded: AbbVie

OBS15333 PEDISTAD

PI: Amir Horev. Funded: IQVIA

A Phase 3, Multicenter, Randomized, Double-blind, Placebo- controlled, Parallel-group, Outpatient Study Evaluating the Pharmacokinetics, Efficacy, and Safety of Baricitinib in Pediatric Patients with Moderate to Severe Atopic Dermatitis. BREEZE-AD-PEDS. Protocol I4V-MC-JAIP.

PI: Amir Horev. Funded: Eli Lilly

A Phase 3b, Multi Center, Open-label, Long-term Extension Study of Apremilast (CC-10004) in Pediatric Subjects from 6 Through 17 Years of Age with Moderate to Severe Plaque Psoriasis

PI: Amir Horev. Funded: Celgene Corporation

A multicenter study to evaluate long-term safety and tolerability of open label sacubitril/valsartan in pediatric patients with heart failure due to systemic left ventricle systolic dysfunction who have completed study CLCZ696B2319

PI: Aviva Levitas. Funded: Novartis

An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in newborns and infants up to the age of 12 months with neonatal and infantile onset of urea cycle disorders (UCD) using a 15N ammonium chloride tracer compared to newborns and infants without UCD

PI: Orna Staretz. Funded: GCP Academy

Compassionate treatment study for the administration of intravenous Trappsol® Cyclo ™ (HPBCD) in patients with Niemann-Pick disease type C (NPC-1) that have finished participation in CTD-TCNPC-201 research protocol

PI: Orna Staretz. Funded: CTD Holdings

A Phase 3, Double blind, Randomized, Placebo-controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl β cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1

PI: Orna Staretz. Funded: CTD Holdings

An open-label single-arm Phase IIb study of F901318 as treatment of invasive fungal infections due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., and other resistant fungi in patients lacking suitable alternative treatment options.

PI: Lior Nesher. Funded: IQVIA

EMAS

PI: Iris Neuman

A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) - RESTORE

PI: Eylon Shani. Funded: Novartis

A Randomized, Double-Blind, Phase 3 Study to Evaluate the Efficacy and Safety of Vedolizumab Intravenous as Maintenance Therapy in Pediatric Subjects with Moderately to Severely Active Ulcerative Colitis Who Achieved Clinical Response Following Open–Label Vedolizumab Intravenous Therapy

PI: Baruch Yerushalmi. Funded: PPD

A Randomized, Double-Blind, Phase 3 Study to Evaluate the Efficacy and Safety of Vedolizumab Intravenous as Maintenance Therapy in Pediatric Subjects with Moderately to Severely Active Crohn’s Disease Who Achieved Clinical Response Following Open-Label Vedolizumab Intravenous Therapy

PI: Boris. Funded: PPD

A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study, with an Optional Open-Label Extension to Assess the Safety, Tolerability, and Pharmacokinetics of Obeticholic Acid in Pediatric Subjects with Biliary Atresia

PI: Baruch Yerushalmi

Immunologic and environmental characteristics of patients with immunodeficiency and inflammatory bowel disease

PI: Baruch Yerushalmi

A 6-month multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and PK/PD of an age-and body weight-adjusted oral finerenone regimen, in addition to an ACEI or ARB, for the treatment of children, 6 months to <18 years of age, with chronic kidney disease and proteinuria (FIONA)

PI: Ruth Shreiber. Funded: Bayer

A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF CARISBAMATE (YKP509) AS ADJUNCTIVE TREATMENT FOR SEIZURES ASSOCIATED WITH LENNOX-GASTAUT SYNDROME IN CHILDREN AND ADULTS, WITH OPTIONAL OPEN-LABEL EXTENSION

PI: Iris Neuman. Funded: PPD

A Phase 3b Extension Study to Evaluate the Long-term Safety of Vedolizumab Intravenous in Pediatric Patients With Ulcerative Colitis or Crohn’s Disease

PI: Baruch Yerushalmi

Real-World Use of Novel Treatments in Patients with Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States

PI: Iris Neuman. Funded: IQVIA

A Randomized, Double-Blind, Sparing-effect Placebo Controlled, with Cross-over Study to Evaluate the Safety, Tolerability and Efficacy of SCI-210 in Children with Autism Spectrum Disorder (ASD)

PI: Gal Meiri. Funded: TCA