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Conditions: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Mucopolysaccharidosis Type II (MPS II)
Interventions: RGX-121; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 5 Years to 17 Years · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Oakland, California

Conditions: Lumbar Spinal Stenosis
Interventions: Superion™ IDS device; Device
Lead sponsor: Boston Scientific Corporation; Industry
Eligibility: 45 Years and older
Enrollment: 166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2041
U.S. locations: 23
States / cities: Carlsbad, California • Larkspur, California • Rancho Mirage, California + 20 more

Conditions: Ovarian Neoplasms
Interventions: Niraparib, Carboplatin, Paclitaxel; Drug
Lead sponsor: Tesaro, Inc.; Industry
Eligibility: 18 Years and older · Female only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 11
States / cities: San Francisco, California • Miami, Florida • Tampa, Florida + 7 more

Conditions: Hunter Syndrome
Interventions: Idursulfase-IT, Elaprase; Drug
Lead sponsor: Takeda; Industry
Eligibility: 3 Years to 18 Years · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2024
U.S. locations: 7
States / cities: Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more

Conditions: Adult Glioblastoma, Depression, Recurrent Adult Brain Tumor
Interventions: vorinostat, temozolomide, magnetic resonance spectroscopic imaging, survey administration; Drug · Procedure · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2012
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Interventions: Not listed
Lead sponsor: University of Minnesota; Other
Eligibility: 6 Years and older
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2019
U.S. locations: 3
States / cities: Oakland, California • Minneapolis, Minnesota • New York, New York

Conditions: Mucopolysaccharidosis II, MPS II
Interventions: SB-913; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 5 Years to 65 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 5
States / cities: Oakland, California • Chicago, Illinois • New York, New York + 2 more

Conditions: Rhinitis, Sinusitis
Interventions: Quinine Sulfate, Placebo; Drug
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Lumbar Spinal Stenosis
Interventions: Superion® Indirect Decompression System (IDS); Device
Lead sponsor: Boston Scientific Corporation; Industry
Eligibility: 45 Years and older
Enrollment: 1,674 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 66
States / cities: Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 60 more

Conditions: Mucopolysaccharidosis II
Interventions: Observational; Other
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 1 Month to 8 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

Conditions: Cocaine Use Disorder
Interventions: Cariprazine Oral Capsule [Vraylar], Placebo oral capsule; Drug
Lead sponsor: Anna Rose Childress, Ph.D.; Other
Eligibility: 18 Years to 65 Years
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Hunter Syndrome
Interventions: Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg); Other · Drug
Lead sponsor: Shire; Industry
Eligibility: 3 Years to 18 Years · Male only
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Healthy Adults
Interventions: Dextroamphetamine, Placebo, [18F]Fallypride, [18F]-FE-PE2I; Drug · Diagnostic Test
Lead sponsor: Vanderbilt University; Other
Eligibility: 20 Years to 65 Years
Enrollment: 65 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Opioid Use Disorder
Interventions: Semaglutide, Placebo; Drug · Other
Lead sponsor: Brigham and Women's Hospital; Other
Eligibility: 18 Years and older
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Urinary Retention Postoperative
Interventions: Tamsulosin, Placebo; Drug
Lead sponsor: Allegheny Singer Research Institute (also known as Allegheny Health Network Research Institute); Other
Eligibility: 18 Years and older · Female only
Enrollment: 154 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2026
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Hunter Syndrome
Interventions: idursulfase-IT, No IT treatment; Biological · Other
Lead sponsor: Shire; Industry
Eligibility: Up to 18 Years · Male only
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina

Conditions: Spinal Deformity, Spinal Degenerative Disorder, Spinal Fusion Failure, Spinal Trauma, Spinal Tumor Case
Interventions: Follow-up schedule: pre-operative baseline up to 24-months post-procedure; Device
Lead sponsor: Medtronic Spinal and Biologics; Industry
Eligibility: 18 Years and older
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2031
U.S. locations: 23
States / cities: Tucson, Arizona • Little Rock, Arkansas • Sacramento, California + 19 more

Conditions: Tobacco Use Disorder
Interventions: Ramelteon, Placebo; Drug
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years to 65 Years
Enrollment: 140 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Pancreatitis
Interventions: Indomethacin, Placebo suspension; Drug
Lead sponsor: Yale University; Other
Eligibility: 18 Years and older
Enrollment: 117 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2012
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Chronic Rhinosinusitis (Diagnosis)
Interventions: Lactobacillus sakei proBio65, Isotonic Saline Solution; Drug · Other
Lead sponsor: University of Illinois at Chicago; Other
Eligibility: 18 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions: SB-318, SB-913, SB-FIX; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 5
States / cities: Oakland, California • Chicago, Illinois • New York, New York + 2 more

Conditions: Mucopolysaccharidosis II
Interventions: iduronate-2-sulfatase enzyme replacement therapy, Placebo; Biological
Lead sponsor: Shire; Industry
Eligibility: 5 Years to 25 Years · Male only
Enrollment: 96 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2005
U.S. locations: 4
States / cities: Oakland, California • St Louis, Missouri • Chapel Hill, North Carolina + 1 more

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PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

A 5-year Superion™ IDS Clinical Outcomes Post-Approval Evaluation (SCOPE)

A Study Comparing Niraparib Versus Platinum-Taxane Doublet Chemotherapy as Neoadjuvant Treatment in Participants With Homologous Recombination-Deficient Stage III/IV Ovarian Cancer (COHORT-C)

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Magnetic Resonance Spectroscopy Imaging in Predicting Response to Vorinostat and Temozolomide in Patients With Recurrent or Progressive Glioblastoma

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Topical Irrigation Therapy for CRS

Postmarket Registry for Evaluation of the Superion® Spacer

Mucopolysaccharidosis Type II Observational

Cariprazine Effects on Brain and Behavior in Cocaine Use Disorder

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Decision Making Study in Young and Middle-Aged Adults: Part II

Semaglutide for Helping Opioid Recovery

Impact of Tamsulosin on Post-Operative Urinary Retention Following Pelvic Reconstructive Surgery

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

The Ailliance Post-Market Clinical Study

Effect of Ramelteon on Smoking Abstinence

A Double-blind Study to Determine if Intraduodenal Indomethacin Can Decrease the Incidence of Post-ERCP Pancreatitis

Topical Probiotic Sinus Irrigations for Treating Chronic Sinusitis

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)