Clinical trials: Malformation
Fabry Disease
A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00283933
- Testing
- migalastat HCl
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Amicus Therapeutics
Cystic Fibrosis
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).
- Trial name
- NCT04043806
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 97 sites in United States, Australia and 9 more
- Volunteers
- 458 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Dermatitis, Atopic
A Dose-Finding Study of GSK2894512 Cream in Subjects With Atopic Dermatitis (AD)
This study will evaluate the efficacy and safety of two concentrations (0.5 percent [%] and 1%) and two application frequencies (once a day and twice a day) of GSK2894512 cream for the topical treatment in adolescent and adult…
- Trial name
- NCT02564055
- Testing
- GSK2894512 1% Cream, GSK2894512 0.5% Cream, Vehicle cream
- Where
- 60 sites in United States, Canada and 1 more
- Volunteers
- 247 people
- Sponsor
- GlaxoSmithKline
Anemia, Sickle Cell
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the…
- Trial name
- NCT01054768
- Testing
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Patent Ductus Arteriosus (PDA)
Closure Of Patent Ductus Arteriosus With the AMPLATZER Duct Occluder the AMPLATZER® Duct Occluder
AGA-004 - The objective of the study is to determine safety, effectiveness and clinical utility of the AMPLATZER Duct Occluder in patients with patent ductus arteriosus.
- Trial name
- NCT00583596
- Testing
- Device closure with AMPLATZER Duct Occluder, Objective Performance Criteria
- Where
- 24 sites in United States
- Volunteers
- 436 people
- Sponsor
- Abbott Medical Devices
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Atopic Dermatitis Eczema, Atopic Dermatitis
Trends in the Epidemiology and Treatment of Atopic Dermatitis in the United Kingdom (UK)
Atopic dermatitis (AD), commonly called eczema, is one of the most frequently occurring skin conditions. It is estimated to affect around one fifth of children in developed countries and is also becoming increasingly common in…
- Conditions
- Atopic Dermatitis Eczema, Atopic Dermatitis, Eczema
- Trial name
- NCT03823794
- Testing
- No intervention
- Where
- United Kingdom
- Volunteers
- 148,166 people, healthy volunteers welcome
- Sponsor
- Momentum Data
Cystic Fibrosis
OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis
The purpose of this trial is to compare the effects of treatment with tobramycin solution for inhalation (TIS) with and without azithromycin in people with cystic fibrosis (CF) age 6 months to 18 years who have early isolation of…
- Trial name
- OPTIMIZE (NCT02054156)
- Testing
- azithromycin, Tobramycin solution for inhalation
- Where
- 45 sites in United States
- Volunteers
- 221 people
- Sponsor
- Bonnie Ramsey
Aplastic Anemia, Pure Red Cell Aplasia
A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
Participants in this study are suffering from rare and serious blood disorders. In aplastic anemia, the bone marrow stops producing red blood cells, platelets, and white blood cells.
- Conditions
- Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
- Trial name
- NCT00001962
- Testing
- Daclizumab
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Anemia, Sickle Cell
Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA.
- Trial name
- NCT00890396
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 163 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Autosomal Dominant Polycystic Kidney Disease
8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of this study is to compare the short-term effects of two tolvaptan formulations in patients with ADPKD.
- Trial name
- NOCTURNE (NCT01451827)
- Testing
- Tolvaptan MR, Tolvaptan IR
- Where
- 35 sites in United States
- Volunteers
- 178 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Respiratory Tract Diseases, Abnormal Mucus Secretions
A Clinical Trial to Compare the Efficacy and Safety of 1-week Treatment of Intravenous N-acetylcysteine (NAC) 600 mg Twice Daily, Ambroxol Hydrochloride 30 mg Twice Daily and Placebo as Expectorant Therapies in Adult Chinese Patients With Respiratory Tract Diseases and Abnormal Mucus Secretions
This is a phase 3, multicenter, randomized, rater- and patient-blind, placebo- and active-controlled, 3-arm parallel group clinical trial.
- Trial name
- NCT03843541
- Testing
- N-acetylcysteine (NAC) 600 mg, Ambroxol hydrochloride 30 mg
- Where
- 32 sites in China
- Volunteers
- 333 people
- Sponsor
- Zambon SpA
Atopic Dermatitis
A Study to Evaluate Upadacitinib in Adolescents and Adults With Moderate to Severe Atopic Dermatitis (Measure Up 2)
The objective of this study is to assess the efficacy and safety of upadacitinib for the treatment of adolescent and adult participants with moderate to severe atopic dermatitis (AD) who are candidates for systemic therapy.
- Trial name
- NCT03607422
- Testing
- Upadacitinib
- Where
- 196 sites in United States, Australia and 21 more
- Volunteers
- 912 people
- Sponsor
- AbbVie
Prostate Cancer
Use of β-hydroxy-β-methylbutyrate to Counteract Muscle Loss in Men With Prostate Cancer on Androgen Ablation
This is a study of HMB plus amino acids in older men with prostate cancer starting androgen deprivation therapy (ADT). The investigators hypothesize that the use of this nutritional supplementation will decrease the loss of…
- Trial name
- NCT01607879
- Testing
- Standard of care ADT + (HMB + arginine + glutamine), Standard of care ADT
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical College of Wisconsin
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Nonsynostotic Plagiocephaly
Brain Characteristics Noted Prior to and Following Treatment
This study will enable investigators to find out if brain structure and characteristics are affected by the shape of the infant's head, and if changes in the brain occur with helmet therapy.
- Trial name
- NCT02392195
- Testing
- Non-interventional
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Duchenne Muscular Dystrophy
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD…
- Trial name
- NCT02760277
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
Cystic Fibrosis
Study of Ataluren (PTC124™) in Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
- Trial name
- NCT00803205
- Testing
- Ataluren
- Where
- 38 sites in United States, Belgium and 9 more
- Volunteers
- 238 people
- Sponsor
- PTC Therapeutics
Familial Partial Lipodystrophy, Nonalcoholic Steatohepatitis
CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy
The study investigators' aim is to determine the long term safety and efficacy of Metreleptin (Myalept,) in promoting amelioration of metabolic abnormalities in patients with all forms of partial lipodystrophy.
- Conditions
- Familial Partial Lipodystrophy, Nonalcoholic Steatohepatitis, NAFLD
- Trial name
- NCT02654977
- Testing
- Metreleptin
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Michigan
Urea Cycle Disorder
Manipulating the Gut Microbiome Study
The objective is to determine if acetohydroxamic acid (AHA) can prevent hydrolysis of urea by inhibiting the bacterial urease of gut flora of both healthy control adults as well as adults with urea cycle disorders
- Trial name
- NCT03181828
- Testing
- Acetohydroxamic Acid Oral Tablet, No treatment
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Nicholas Ah Mew
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