Top Recruiting Trials
20 actively recruiting studies
Purified Cortrophin® Gel Efficacy and Safety Study of 2 Dose Levels in Patients With Acute Gout Flares
Massachusetts General Hospital
n=150
Mass General Hospital
Boston, Massachusetts
Intervention DRUG: Purified Cortophin Gel, 40 U — This will be the 40 U dose of the purified Cortophin Gel
Eligibility Ages 18 Years – 90 Years
Hybrid Florbetaben PET/MRI for Imaging of Cardiac Amyloidosis
University Hospital, Essen
n=25
Department of Nuclear medicine, University Hospital Essen
Essen, North Rhine-Westphalia
Eligibility Ages 40 Years
Cognitive and Neurological Pathologies in Pompe Disease
Duke University
n=45
Duke University Medical Center
Durham, North Carolina
Eligibility Ages 0 Years – 18 Years
The Effect of a Plant-based, Protein-dense Sauce on Essential Amino Acid Concentrations and Gut Fullness in Older Adults
University of Exeter
n=20
University of Exeter
Exeter, Devon
Intervention DIETARY_SUPPLEMENT: ProSauce — 10g additional protein from ProSauce
Eligibility Ages 60 Years
Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
The First Affiliated Hospital of Bengbu Medical University
n=15
The First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui
Intervention GENETIC: NGGT002 Injection — Dose escalation will proceed sequentially from Dose Level 1 to Dose Level 5 in a single-dose administration of NGGT002.
Eligibility Ages 18 Years
Fabry Disease Registry & Pregnancy Sub-registry
Genzyme, a Sanofi Company
n=9000
University of Alabama Birmingham- Nephrology- Site Number : 840018
Birmingham, Alabama
Accelerated Approval
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Genetix Biotherapeutics Inc.
n=120
Boston Children's Hospital
Boston, Massachusetts
Intervention OTHER: No Intervention — This is non-interventional study.
NatiOnal Referral cenTEr Study of Transthyretin Amyloid Cardiomyopathy(ATTR) Patients on Tafamidis
Paolo Milani
n=200
Fondazione IRCCS Policlinico San Matteo
Pavia
Eligibility Ages 18 Years – 99 Years
Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives
University Hospital, Strasbourg, France
n=30
Service de pédiatrie 1 - CHU de Strasbourg - France
Strasbourg
Eligibility Ages 1 Month – 21 Years
Natural History of Wilson Disease
Yale University
n=300
Yale University
New Haven, Connecticut
Pompe Disease Registry Protocol
Genzyme, a Sanofi Company
n=2000
University of Alabama at Birmingham- Site Number : 840106
Birmingham, Alabama
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Khondrion BV
n=220
Massachusetts General Hospital
Boston, Massachusetts
Intervention DRUG: Sonlicromanol — Administration of 90 mg sonlicromanol (100 mg sonlicromanol.HCl) twice daily during 52 weeks
Eligibility Ages 18 Years
Assessing the Safety and Tolerability of NMN in DHDDS-CDG
Eva Morava-Kozicz
n=8
Icahn School of Medicine at Mount Sinai
New York, New York
Intervention DRUG: Nicotinamide Mononucleotide (NMN) Nucleosidase — Name: nicotinamide mononucleotide (NMN)
Form: measured powder
Dose: 250 mg/day
Frequency: Daily
Route of administrat...
Eligibility Ages 4 Years
Functional Impairment in Albinism
Fondation Ophtalmologique Adolphe de Rothschild
n=50
Hôpital Fondation A. de Rothschild
Paris
Intervention OTHER: questionnaire — On-line self-questionnaire, in the form of an 18-question form, the link to which is sent by e-mail to patients with alb...
Eligibility Ages 18 Years
Prospective Research Rare Kidney Stones (ProRKS)
Mayo Clinic
n=220
University of Alabama @ Birmingham
Birmingham, Alabama
Development of Health-related Quality of Life Instrument for Patients With Cystinosis
Cystinose Stiftung
n=300
University Medical Center Hamburg-Eppendorf | Center for Psychosocial Medicine | Department of Medical Psychology
Hamburg
Eligibility Ages 8 Years – 26 Years
Spanish Wilson Disease Registry
Asociación Española para el Estudio del Hígado
n=600
Hospital Clinic
Barcelona
Eligibility Ages 18 Years
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Azafaros B.V.
n=75
UCSF Children's Hospital and Research Center at Oakland
Oakland, California
Intervention DRUG: AZ-3102 — Nizubaglustat
Eligibility Ages 4 Years
Exploratory Study on the Treatment of Gout With Potassium Citrate Sustained-release Tablets
Huashan Hospital
n=312
Huashan hospital, Fudan university
Shanghai
Intervention DRUG: Sodium Bicarbonate Oral Capsule — On the basis of the standard uric acid-lowering treatment plan, sodium bicarbonate should be taken three times a day, 1....
Eligibility Ages 18 Years – 70 Years
Neonatal Screening of Biotinidase Deficiency: Genotype-phenotype Correlation and Clinical Follow-up
IRCCS Azienda Ospedaliero-Universitaria di Bologna
n=180
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Bologna, Bologna
Eligibility Ages 36 Months