Clinical trials: Abnormalities
Erythropoietic Protoporphyria (EPP)
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.
- Trial name
- NCT03520036
- Testing
- MT-7117 low dose, MT-7117 high dose
- Where
- 9 sites in United States
- Volunteers
- 102 people
- Sponsor
- Tanabe Pharma America, Inc.
Infant, Premature, Diseases, Infant, Small for Gestational Age
Optimizing Individual Nutrition in Preterm Very Low Birth Weight Infants
In preterm infants fed human milk, milk needs to be fortified to meet nutrient recommendations. Fortification can be 1) standard, 2) individualized (adjusted based on daily human milk nutrient analysis and milk volume), or 3)…
- Trial name
- NCT02372136
- Testing
- Individualized Nutrition, Optimized nutrition
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- University of Texas Southwestern Medical Center
Pediatric Glaucoma, Elevated IOP in Pediatric Patients
Study of Travoprost Ophthalmic Solution, 0.004% Compared to Timolol (0.5% or 0.25%) in Pediatric Glaucoma Patients
The purpose of this study is to evaluate the intraocular pressure (IOP)-lowering efficacy of Travoprost 0.004% POLYQUAD (PQ) ophthalmic solution compared to Timolol ophthalmic solution (0.5% or 0.25%) in pediatric glaucoma…
- Conditions
- Pediatric Glaucoma, Elevated IOP in Pediatric Patients, Ocular Hypertension in Pediatric Patients
- Trial name
- NCT01652664
- Testing
- Travoprost 0.004% PQ ophthalmic solution, Timolol, 0.5% or 0.25% ophthalmic solution, Travoprost Vehicle
- Volunteers
- 184 people
- Sponsor
- Alcon Research
Leukemia
Clofarabine and Daunorubicin in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as clofarabine and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00814164
- Testing
- clofarabine, daunorubicin hydrochloride, cytogenetic analysis
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Roswell Park Cancer Institute
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
Immune Tolerance Induction Study
An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction…
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT00701701
- Testing
- Myozyme® (alglucosidase alfa)
- Where
- 5 sites in United States, Israel
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company
Stargardt Disease
Safety and Efficacy of Emixustat in Stargardt Disease
The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease. Funding Source -- FDA OOPD
- Trial name
- SeaSTAR (NCT03772665)
- Testing
- Emixustat
- Where
- 29 sites in United States, Brazil and 9 more
- Volunteers
- 194 people
- Sponsor
- Kubota Vision Inc.
Amyloid Cardiomyopathy
Open-Label Study of AG10 in Patients With Cardiomyopathy
This prospective, multicenter open-label study will evaluate the long-term safety, tolerability, PK and PD of acoramidis administered on a background of stable heart failure therapy.
- Trial name
- NCT03536767
- Testing
- Acoramidis
- Where
- 9 sites in United States
- Volunteers
- 47 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Fabry Disease
A Study of Replagal in Treatment-naïve Adults With Fabry Disease
In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney function and heart structure of…
- Trial name
- NCT04840667
- Testing
- REPLAGAL
- Where
- 28 sites in Canada, Finland and 6 more
- Volunteers
- 17 people
- Sponsor
- Shire
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Biliary Atresia
Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy
This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy).
- Trial name
- NCT06121375
- Testing
- OCA
- Where
- 24 sites in Australia, Canada and 8 more
- Volunteers
- 28 people
- Sponsor
- Intercept Pharmaceuticals
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited
Neurofibromatosis 1
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T
This trial is evaluating the use of oral Everolimus to determine if there is a reduction in the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6 month period.
- Trial name
- DCLNF1 (NCT02332902)
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- The University of Texas Health Science Center, Houston
Huntington's Disease
Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
This study is a 26 week, randomized, parallel group, double blind comparison of PF-02545920 5 mg, PF-02545920 20 mg, and placebo dosed BID in the treatment of motor impairment of subjects with Huntington's Disease.
- Trial name
- NCT02197130
- Testing
- PF-02545920
- Where
- 57 sites in United States, Canada and 3 more
- Volunteers
- 272 people
- Sponsor
- Pfizer
Patent Ductus Arteriosus, Prematurity
Early Versus Late Use of Ibuprofen for Patent Ductus Arteriosus (PDA) Closure
The primary objective is to evaluate the Patent Ductus Arteriosus (PDA) closure rate of early vs. late use of Ibuprofen (Ibu). The investigators believe that early use of Ibu will have a higher PDA closure rate than later use of…
- Trial name
- NCT00833365
- Testing
- Ibuprofen
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Utah
Respiratory Distress Syndrome, Newborn
Randomized Controlled Trial of Surfactant Delivery Via Laryngeal Mask Airway (LMA) Versus Endotracheal Intubation
In this study, newborn babies with respiratory distress syndrome (RDS), receiving oxygen via nasal CPAP, and needing surfactant treatment will be randomized to standard delivery of surfactant via and endotracheal tube…
- Trial name
- NCT01042600
- Testing
- Endotracheal tube insertion, Laryngeal mask airway insertion
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Albany Medical College
Atrial Septal Defect
Closure of Atrial Septal Defects With the AMPLATZER Septal Occluder - Post Approval Study
The purpose of this study is to prospectively evaluate the incidence of hemodynamic compromise and to obtain long-term survival data on patients with the AMPLATZER Septal Occluder
- Trial name
- NCT00650936
- Testing
- AMPLATZER Septal Occluder
- Where
- 51 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Abbott Medical Devices
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia (M7)
Alvocidib, Cytarabine, and Mitoxantrone in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
This randomized phase II trial is studying two different schedules of alvocidib to compare how well they work when given together with cytarabine and mitoxantrone in treating patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT00795002
- Testing
- alvocidib, mitoxantrone hydrochloride, cytarabine
- Where
- 2 sites in United States
- Volunteers
- 78 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease, Sickle Cell Anemia
Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body.
- Trial name
- NCT04476277
- Testing
- Biotin label
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Cystic Fibrosis
A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)
identify and validate biomarkers that might reflect partial restoration of CFTR function and can be used to monitor disease progression, and ii) evaluate the mechanistic effects of CFTR modulators and other relevant therapies in…
- Trial name
- PROSPECT (NCT02477319)
- Testing
- Observational
- Where
- 38 sites in United States
- Volunteers
- 452 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Fabry Disease, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company