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Showing 169–192 of 1,509 matching trials from the live ClinicalTrials.gov search.
Completed No phase listed Observational Accepts healthy volunteers

Intracardiac Flow Assessment in Cardiac Amyloidosis

NCT05379101
Conditions
Amyloidosis Cardiac, Healthy Adults
Interventions
Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
40 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Healthy
Interventions
Nikkomycin Z
Drug
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Tucson, Arizona
Conditions
GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
Interventions
miglustat, Ketogenic Diet
Drug · Other
Lead sponsor
University of Minnesota
Other
Eligibility
Up to 204 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 6 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Tampa, Florida + 9 more
Completed Phase 3 Interventional Results available

Atorvastatin Three Year Pediatric Study

NCT00827606
Conditions
Familial Hypercholesterolemia
Interventions
atorvastatin
Drug
Lead sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Industry
Eligibility
6 Years to 15 Years
Enrollment
272 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
7
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Gainesville, Florida + 3 more
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years to 60 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
2
States / cities
San Francisco, California • Washington D.C., District of Columbia
Enrolling by invitation No phase listed Observational

Registry of Patients With Hypophosphatasia

NCT02306720
Conditions
Hypophosphatasia (HPP)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
1,571 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2031
U.S. locations
20
States / cities
Centennial, Colorado • Hartford, Connecticut • Tampa, Florida + 16 more
Conditions
Cancers
Interventions
Laparoscopic surgery
Procedure
Lead sponsor
Erin Rowell
Other
Eligibility
Up to 11 Years · Female only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2035
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Conditions
X-linked Hypophosphatemia
Interventions
KRN23
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years to 65 Years
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1992
U.S. locations
11
States / cities
Los Angeles, California • San Francisco, California • Miami, Florida + 7 more
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Conditions
Fabry Disease
Interventions
Replagal
Biological
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Iowa City, Iowa • Fairfax, Virginia
Conditions
Joint Damage
Interventions
Febuxostat, Placebo for Febuxostat
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
314 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
56
States / cities
Mesa, Arizona • Tucson, Arizona • Burbank, California + 53 more
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
New Haven, Connecticut • Miami, Florida • Chicago, Illinois + 2 more
Conditions
Glycogen Storage Disease Type IA
Interventions
DTX401, Placebo, Oral prednisolone, Placebo for oral prednisolone
Genetic · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
8 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Farmington, Connecticut + 6 more
Conditions
Mucopolysaccharidosis I
Interventions
JR-171
Drug
Lead sponsor
JCR Pharmaceuticals Co., Ltd.
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Oakland, California
Conditions
Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
Interventions
Antioxidants, Cholesterol
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Up to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Completed No phase listed Observational Accepts healthy volunteers

Decision Support for Parents Receiving Information About Child's Rare Disease

NCT01875640
Conditions
Disorder of Sex Development, Intersex Conditions, Congenital Adrenal Hyperplasia, Hypospadias
Interventions
Not listed
Lead sponsor
University of Michigan
Other
Eligibility
Not listed
Enrollment
63 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Ann Arbor, Michigan • Philadelphia, Pennsylvania + 1 more
Conditions
Hunter Syndrome
Interventions
No treatment
Other
Lead sponsor
Shire
Industry
Eligibility
Up to 70 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions
BMN 110
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 5 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
2
States / cities
Oakland, California • Manhasset, New York
Conditions
Gaucher Disease Type 1
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
16 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia