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Conditions: Tics, Tourette Disorder, Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions: Habit Reversal Training; Behavioral
Lead sponsor: University of Utah; Other
Eligibility: 8 Years to 17 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Advanced Pancreatic Carcinoma, Metastatic Pancreatic Carcinoma, Stage II Pancreatic Cancer AJCC v8, Stage III Pancreatic Cancer AJCC v8, Stage IV Pancreatic Cancer AJCC v8
Interventions: Pemigatinib, Computed Tomography (CT), Magnetic Resonance Imaging, Optical Coherence Tomography, Bone Scan, Ophthalmoscopy; Drug · Procedure
Lead sponsor: Sameek Roychowdhury; Other
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 109
States / cities: Birmingham, Alabama • Mobile, Alabama • Montgomery, Alabama + 106 more

Conditions: Congenital Sucrase-isomaltase Deficiency (CSID)
Interventions: Not listed
Lead sponsor: QOL Medical, LLC; Industry
Eligibility: Up to 18 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 19
States / cities: Los Angeles, California • Oakland, California • Aurora, Colorado + 16 more

Conditions: 16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder), ACTL6B, ADNP, AHDC1, ANK2, ANKRD11, ARID1B, ASH1L, BCL11A, CHAMP1, CHD2, CHD8, CSNK2A1, CTBP1, CTNNB1 Gene Mutation, CUL3, DDX3X, DNMT3A, DSCAM, DYRK1A, FOXP1, GRIN2A, GRIN2B, HIVEP2-Related Intellectual Disability, HNRNPH2, KATNAL2, KDM5B, KDM6B, KMT2C Gene Mutation, KMT2E, KMT5B, MBD5, MED13L, PACS1, PPP2R5D-Related Intellectual Disability, PTCHD1, REST, SCN2A Encephalopathy, SETBP1 Gene Mutation, SETD5, SMARCA4 Gene Mutation, SMARCC2, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability, TBR1, ARHGEF9, HNRNPU, PPP3CA, PPP2R1A, SLC6A1, 2p16.3 Deletions, 5q35 Deletions, 5q35 Duplications, 7q11.23 Duplications, 15Q13.3 Deletion Syndrome, 16p11.2 Triplications, 16P12.2 Microdeletion, 16P13.11 Microdeletion Syndrome (Disorder), 17Q12 Microdeletion Syndrome (Disorder), 17Q12 Duplication Syndrome, 17Q21.31 Deletion Syndrome, 17q21.3 Duplications, ACTB, ADSL, AFF2, ALDH5A1, ANK3, ARX, ATRX Gene Mutation, AUTS2 Syndrome, BCKDK, BRSK2, CACNA1C, CAPRIN1, CASK, CASZ1, CHD3, CIC, CNOT3, CREBBP Gene Mutation, CSDE1, CTCF, DEAF1, DHCR7, DLG4, EBF3, EHMT1, EP300 Gene Mutation, GIGYF1, GRIN1, GRIN2D, IQSEC2-Related Syndromic Intellectual Disability, IRF2BPL, KANSL1, KCNB1, KDM3B, NEXMIF, KMT2A, MBOAT7, MEIS2, MYT1L, NAA15, NBEA, NCKAP1, NIPBL, NLGN2, NLGN3, NLGN4X, NR4A2, NRXN1, NRXN2, NSD1 Gene Mutation, PHF21A, PHF3, PHIP, PSMD12, RELN, RERE, RFX3, RIMS1, RORB, SCN1A, SETD2 Gene Mutation, SHANK2, SIN3A, SLC9A6, SON, SOX5, SPAST, SRCAP, TAOK1, TANC2, TCF20, TLK2, TRIO, TRIP12, UPF3B, USP9X, VPS13B, WAC, WDFY3, ZBTB20, ZNF292, ZNF462, 2Q37 Deletion Syndrome, 9q34 Duplications, 15q15 Deletions, 15Q24 Deletion, NR3C2, SYNCRIP, 2q37.3 Deletion, 6q16 Deletion, 15q11.2 BP1-BP2 Deletion, 16p13.3 Deletion, 17Q11.2 Microduplication Syndrome (Disorder), 17p13.3, Xq28 Duplication, CLCN4, CSNK2B, DYNC1H1, EIF3F, GNB1, MED13, MEF2C, RALGAPB, SCN1B, YY1, Xp11.22 Duplication, PACS2, MAOA, MAOB, HNRNPC, HNRNPD, HNRNPK, HNRNPR, HNRNPUL2, 5P Deletion Syndrome, TCF7L2 Gene Mutation, HECW2, PPM1D, RNU4-2, SNAP25, FOXP2, ITSN1
Interventions: Not listed
Lead sponsor: Simons Searchlight; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2050
U.S. locations: 2
States / cities: Boston, Massachusetts • Lewisburg, Pennsylvania

Conditions: Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC, Hemoglobin Beta Thalassemia
Interventions: Decision Aid Tool, Standard Practice; Other
Lead sponsor: Emory University; Other
Eligibility: 8 Years to 80 Years
Enrollment: 134 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Atopic Dermatitis
Interventions: EDP1815, Placebo; Drug
Lead sponsor: Evelo Biosciences, Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 421 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 27
States / cities: Birmingham, Alabama • Fountain Valley, California • Fremont, California + 22 more

Conditions: Congenital Adrenal Hyperplasia
Interventions: Abiraterone acetate, Placebo, Hydrocortisone, Fludrocortisone; Drug
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 2 Years to 9 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 4
States / cities: Los Angeles, California • Bethesda, Maryland • Ann Arbor, Michigan + 1 more

Conditions: Sickle Cell Disease
Interventions: SelG1, Placebo; Drug
Lead sponsor: Reprixys Pharmaceutical Corporation; Industry
Eligibility: 16 Years to 65 Years
Enrollment: 198 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 49
States / cities: Birmingham, Alabama • Mobile, Alabama • Little Rock, Arkansas + 46 more

Conditions: Congenital Myasthenic Syndrome, CMS
Interventions: ARGX-119, Placebo; Biological · Other
Lead sponsor: argenx; Industry
Eligibility: 18 Years and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 2
States / cities: Sacramento, California • Chicago, Illinois

Conditions: Duchenne Muscular Dystrophy
Interventions: RGX-202; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 1 Year and older · Male only
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 19
States / cities: Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more

Conditions: Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions: Not listed
Lead sponsor: Columbia University; Other
Eligibility: 5 Years and older
Enrollment: 106 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2028
U.S. locations: 3
States / cities: Palo Alto, California • Boston, Massachusetts • New York, New York

Conditions: Atopic Dermatitis (AD)
Interventions: CGB-500 Ointment with 1% tofacitinib, Ruxolitinib 1.5% Cream QD; Drug
Lead sponsor: CAGE Bio Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025
U.S. locations: 1
States / cities: Fremont, California

Conditions: Endothelial Dysfunction
Interventions: Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Hackensack, New Jersey

Conditions: Cystic Fibrosis
Interventions: azithromycin 250 mg tablets, placebo tablets; Drug
Lead sponsor: CF Therapeutics Development Network Coordinating Center; Network
Eligibility: 6 Years to 18 Years
Enrollment: 263 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 31
States / cities: Phoenix, Arizona • Hartford, Connecticut • Atlanta, Georgia + 27 more

Conditions: Multiple Endocrine Neoplasia, Pancreatic Neuroendocrine Tumors, Hyperparathyroidism
Interventions: Genome Sequencing, Data Review; Other
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: Not listed
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2023
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Healthy
Interventions: ALXN1840, Placebo, Moxifloxacin; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 57 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Austin, Texas

Conditions: Skin Laxity
Interventions: Dermal Cooling System; Device
Lead sponsor: R2 Dermatology; Industry
Eligibility: 18 Years and older
Enrollment: 28 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2017
U.S. locations: 2
States / cities: Folsom, California • Fremont, California

Conditions: Pre-diabetes, Overweight, Metabolic Syndrome
Interventions: Diabetes Genetic Counseling, No genetic counseling; Behavioral
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 21 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Lynch Syndrome
Interventions: Let's Talk Genetics Providers, Let's Talk Patients; Behavioral
Lead sponsor: UNC Lineberger Comprehensive Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Hereditary Angioedema
Interventions: Icatibant, Placebo; Drug
Lead sponsor: Shire; Industry
Eligibility: 18 Years and older
Enrollment: 98 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 39
States / cities: Alabaster, Alabama • Birmingham, Alabama • Scottsdale, Arizona + 35 more

Conditions: Rothmund-Thomson Syndrome
Interventions: Calcium stable isotope; Other
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 4 Years and older
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2017
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Achondroplasia
Interventions: Not listed
Lead sponsor: Pfizer; Industry
Eligibility: 0 Years to 15 Years
Enrollment: 315 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 14
States / cities: Los Angeles, California • Torrance, California • Wilmington, Delaware + 7 more

Conditions: Healthy Volunteers
Interventions: BMS-986533, Famotidine, Placebo, Dabigatran Etexilate, Rosuvastatin; Drug
Lead sponsor: Bristol-Myers Squibb; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 136 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Lincoln, Nebraska

Conditions: Colorectal Cancer, Lynch Syndrome
Interventions: Omega-3 fatty acid ethyl esters (2 gram); Drug
Lead sponsor: University of Kansas Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 1
States / cities: Kansas City, Kansas

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Investigating Changes in Premonitory Urges During Habit Reversal Training for Tics

A Phase II Nationwide, Fully Decentralized, Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreatic Cancer With FGFR Genetic Alterations

Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS)

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

Decision Aid for Therapeutic Options In Sickle Cell Disease

A Study Investigating the Effect of EDP1815 in the Treatment of Mild, Moderate and Severe Atopic Dermatitis

Androgen Reduction in Congenital Adrenal Hyperplasia

Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

A Study to Assess Efficacy and Safety of CGB-500, 1% Tofacitinib Versus an Active Comparator for Atopic Dermatitis

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa

Variables That Are Correlated to Developing Multiple Endocrine Neoplasia (MEN) and Pancreatic Neuroendocrine Tumors (PNET)

A Study of the Cardiac Effects of ALXN1840 in Healthy Adults

Dermal Cryotherapy in Patients Undergoing Abdominoplasty

Genetic Counseling and Lifestyle Change for Diabetes Prevention

Overcoming Barriers to Uptake of Cascade Screening

A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)

Calcium Absorption in Patients With Rothmund-Thomson Syndrome

Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Single and Multiple Dose Study to Evaluate Safety and Pharmacokinetics of BMS-986533 in Healthy Participants and Assessments of Food and pH Effects on Relative Bioavailability, and Drug-Drug Interaction Potential in Healthy Participants

Omega 3 Fatty Acids in Colorectal Cancer (CRC) Prevention in Patients With Lynch Syndrome (COLYNE)