Clinical trials: Fetal Developmental Abnormality
Hemophilia A, Prophylaxis of Bleeding
Prophylaxis Regimen for Hemophilia A Patients
Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII).
- Trial name
- PREDICT (NCT05036278)
- Testing
- Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
- Where
- 12 sites in United States
- Volunteers
- 21 people
- Sponsor
- Bayer
Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin.
- Conditions
- Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome
- Trial name
- NCT02960997
- Testing
- Sirolimus, 2%, Vehicle
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Stanford University
Sickle Cell Disease
Families Taking Control (FTC): Family-based Problem-solving Intervention for Children With Sickle Cell Disease
This study aims to develop an effective, brief, family-based intervention targeting quality of life and school functioning for youth with sickle cell disease.
- Trial name
- FTC (NCT02273310)
- Testing
- Problem-Solving Skills Training for Disease Management
- Volunteers
- 83 people
- Sponsor
- University of Pennsylvania
Phenylketonuria
Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain reduced blood Phe concentrations in PKU subjects.
- Trial name
- NCT00924703
- Testing
- rAvPAL-PEG
- Where
- 14 sites in United States
- Volunteers
- 68 people
- Sponsor
- BioMarin Pharmaceutical
Epistaxis
NOrth American Study for the Treatment of Recurrent epIstaxis With DoxycycLine: The NOSTRIL Trial
The purpose of this research study is to determine whether Doxycycline can be used to control nosebleeds (epistaxis) for patients with Hereditary Hemorrhagic Telangiectasia (HHT).
- Trial name
- NOSTRIL (NCT04167085)
- Testing
- Doxycycline
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of California, Los Angeles
Elective Colonoscopy
Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep®
The purpose of this dose finding/comparative efficacy study is to first single out the most appropriate dose of mannitol for bowel preparation (phase II) and, subsequently, demonstrate the non-inferiority of the efficacy of…
- Trial name
- SATISFACTION (NCT04759885)
- Testing
- Phase II: NTC015 low dose, Phase II: NTC015 medium dose, Phase II: NTC015 high dose
- Where
- 35 sites in France, Germany and 2 more
- Volunteers
- 886 people
- Sponsor
- NTC srl
Mucopolysaccharidosis I, Hurlers Syndrome
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
- Conditions
- Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT00912925
- Testing
- rhIDU (recombinant human-Alpha-L-Iduronidase)
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company
Retinitis Pigmentosa, Visual Impairment
Environmental Localization Mapping and Guidance for Visual Prosthesis Users
This study is driven by the hypothesis that independent navigation by blind users of visual prosthetic devices can be greatly aided by use of an autonomous navigational aid that provides information about the environment and…
- Conditions
- Retinitis Pigmentosa, Visual Impairment, Visual Prosthesis
- Trial name
- SLAM (NCT04359108)
- Testing
- Navigation system mode: Argus Vision, Navigation system mode: Depth Vision, Navigation system mode: Depth Vision with Haptic / Audio
- Where
- 2 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis
Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration…
- Trial name
- SIMPLICITY (NCT02734810)
- Testing
- Liprotamase Powder for Oral Solution
- Where
- 21 sites in United States
- Volunteers
- 15 people
- Sponsor
- Anthera Pharmaceuticals
Autosomal Dominant Polycystic Kidney Disease
The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
This is a Phase 2, open-label, parallel-group, multiple dose study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and tolerability of multiple doses of lixivaptan in Autosomal Dominant Polycystic Kidney…
- Trial name
- NCT03487913
- Testing
- Lixivaptan
- Where
- 13 sites in United States
- Volunteers
- 31 people
- Sponsor
- Palladio Biosciences
Metastatic Urothelial Carcinoma, Unresectable Urothelial Carcinoma
Pemigatinib + Pembrolizumab vs Pemigatinib Alone vs Standard of Care for Urothelial Carcinoma (FIGHT-205)
The purpose of this study is to evaluate the safety and efficacy of pemigatinib plus pembrolizumab or pemigatinib alone versus the standard of care for participants with metastatic or unresectable urothelial carcinoma who are not…
- Trial name
- NCT04003610
- Testing
- Pemigatinib, Pembrolizumab, Gemcitabine
- Where
- 79 sites in United States, Austria and 14 more
- Volunteers
- 7 people
- Sponsor
- Incyte Corporation
Cystic Fibrosis, Healthy
Utilizing Exhaled Breathe Condensate Collection to Study Ion Regulation in Cystic Fibrosis
Our aims were to determine if exhaled breath condensate (EBC) could detect differences in ion regulation between cystic fibrosis (CF) and healthy and measure the effect of the albuterol on EBC ions in these populations.
- Trial name
- NCT01880723
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- University of Arizona
Duchenne Muscular Dystrophy
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age.
- Trial name
- NCT02167217
- Testing
- Prednisolone
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- Washington University School of Medicine
Duchenne Muscular Dystrophy
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
- Trial name
- NCT04956289
- Testing
- Viltolarsen
- Where
- 8 sites in United States, China and 4 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Sickle Cell Disease
Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet assessments to identify type, intensity, frequency, and phase of pain among SCD adolescents is lacking.
- Trial name
- NCT03291613
- Testing
- Pinpoint App
- Where
- 2 sites in United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Fetal Growth Retardation
Placental Lesions in Fetal Growth Restrictions
The purpose of this research study is to develop imaging methods to diagnose placental injury in pregnancies diagnosed with fetal growth restriction (FGR).
- Trial name
- NCT04839185
- Testing
- Ferumoxytol infusion, MRI scan
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Wisconsin, Madison
Sickle Cell Anemia
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or…
- Trial name
- NCT03020615
- Testing
- Hydroxyurea
- Where
- 4 sites in United States
- Volunteers
- 58 people
- Sponsor
- St. Jude Children's Research Hospital
Cystic Fibrosis
Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
The purpose of this study was to evaluate the safety and efficacy of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
- Trial name
- AIR-CF2 (NCT00104520)
- Testing
- AZLI 75 mg two times a day (BID)/three times a day (TID)
- Where
- 56 sites in United States
- Volunteers
- 211 people
- Sponsor
- Gilead Sciences
Birt-Hogg-Dube Syndrome
Assessment of Safety of Air Travel in Patients With Birt-Hogg-Dube Syndrome
The aim of this study is to conduct survey-based assessments for the safety of air travel in patients with Birt-Hogg-Dube syndrome (BHD).
- Trial name
- NCT03040115
- Testing
- Non-Interventional Study
- Where
- United States
- Volunteers
- 104 people
- Sponsor
- University of Cincinnati
Diabetes Mellitus, Type 1
A Research Trial of Aralast in New Onset Diabetes (RETAIN)
The drug Alpha-1 Antitrypsin (AAT, Aralast NP) is being tested in this study as an anti-inflammatory drug (a medication that decreases inflammation, which is part of the body's normal ability to fight infection and respond to…
- Trial name
- RETAIN (NCT01183468)
- Testing
- Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
- Where
- 15 sites in United States
- Volunteers
- 17 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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