Clinical trials: Fetal Developmental Abnormality
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with LGMD2B and FSHD.
- Trial name
- FSHD (NCT02579239)
- Testing
- ATYR1940
- Where
- 6 sites in United States, Denmark and 1 more
- Volunteers
- 18 people
- Sponsor
- aTyr Pharma, Inc.
Opiate Addiction, Pregnancy
Pharmacologically-based Strategies for Opioid Substitution Therapy During Pregnancy
This study is a pharmacodynamic study in pregnant women evaluating the relationship between buprenorphine concentration and outcome such as opioid withdrawal symptoms , NAS scores, neurodevelopmental and neuroanatomic outcomes.
- Trial name
- NCT03831113
- Testing
- Magnitude Group, Frequency Group
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Steve N. Caritis, MD
Cystic Fibrosis
Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
- Trial name
- APPLAUD (NCT03265288)
- Testing
- LAU-7b
- Where
- 40 sites in United States, Australia and 1 more
- Volunteers
- 166 people
- Sponsor
- Laurent Pharmaceuticals Inc.
Erythropoietic Protoporphyria
Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients
The purpose of this study is to evaluate the concentration of afamelanotide in serum after the administration of afamelanotide in adolescent and adult EPP patients.
- Trial name
- NCT06388642
- Testing
- Afamelanotide 16mg implant
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 28 people
- Sponsor
- Clinuvel Europe Limited
IVH- Intraventricular Hemorrhage, Extreme Prematurity
Delayed Cord Clamping With Oxygen In Extremely Low Gestation Infants
This study is being conducted to compare the incidence of preterm infants (up to 28+6 weeks GA) who achieve a peripheral oxygen saturation of 80 percent by 5 minutes of life (MOL) given mask CPAP/PPV with an FiO2 of 1.0 during…
- Conditions
- IVH- Intraventricular Hemorrhage, Extreme Prematurity, Hypoxia Neonatal, Hyperoxia
- Trial name
- DOXIE (NCT04413097)
- Testing
- Delayed Cord Clamping with Low Oxygen concentration, Delayed Cord Clamping with High Oxygen concentration
- Where
- 3 sites in United States
- Volunteers
- 140 people
- Sponsor
- Sharp HealthCare
Mucopolysaccharidosis VI
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk…
- Trial name
- NCT00067470
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- BioMarin Pharmaceutical
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- NCT02586805
- Testing
- DX-2930 - 300mg/2wk, DX-2930 - 300mg/4wk, DX-2930 - 150mg/4wk
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 125 people
- Sponsor
- Shire
Tourette Syndrome
Developing New Treatments for Tourette Syndrome: Therapeutic Trials With Modulators of Glutamatergic Neurotransmission
A joint NIH -Tourette Syndrome Association Conference has emphasized the critical need for the testing and development of new pharmacotherapy for tic suppression in Tourette syndrome (TS).
- Trial name
- NCT01018056
- Testing
- D-serine, Riluzole
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Johns Hopkins University
Atopic Dermatitis, Psoriasis
Evaluating Treatment Resistant Dermatitis TaroIIR
Psoriasis and atopic dermatitis are chronic inflammatory disease that account for a significant amount of patients in most dermatological practices.
- Trial name
- NCT03050294
- Testing
- Phone calls, Desoximetasone 0.25% spray
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Wake Forest University Health Sciences
Von Hippel-Lindau Disease
Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. These tumors are more aggressive in some people than in others.
- Trial name
- NCT00062166
- Where
- United States
- Volunteers
- 340 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with…
- Trial name
- NCT00445978
- Testing
- Sapropterin Dihydrochloride
- Where
- 12 sites in United States
- Volunteers
- 32 people
- Sponsor
- BioMarin Pharmaceutical
Congenital Bleeding Disorder, Haemophilia A
Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
This trial is conducted in China. The aim of this trial is to evaluate the clinical efficacy of turoctocog alfa in treatment of bleeding episodes in Chinese patients with severe haemophilia A (FVIII≤1%).
- Trial name
- guardian TM 7 (NCT02938585)
- Testing
- turoctocog alfa
- Where
- 11 sites in China
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Intracranial Arteriovenous Malformations (AVM)
Protocol for Staged Stereotactic Radiosurgery for Large Arteriovenous Malformations
Stereotactic radiosurgery is a well established treatment option for arteriovenous malformations (AVMs). The potential complications related to radiosurgery are well documented and are predominately related to radiation effects…
- Trial name
- NCT02576535
- Testing
- Fractionated stereotactic radiosurgery, Leskell gamma unit
- Volunteers
- 18 people
- Sponsor
- NYU Langone Health
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant
RATIONALE: Rasburicase may be an effective treatment for graft-versus-host disease caused by a donor stem cell transplant. PURPOSE: This clinical trial is studying how well rasburicase works in preventing graft-versus-host…
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00513474
- Testing
- busulfan, cyclophosphamide, cyclosporin-A
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Massachusetts General Hospital
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Cystic Fibrosis
CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)
This is a Phase 2 study with primary objective of looking whether YPT-01 phage therapy reduces sputum bacterial load in cystic fibrosis subjects with Pseudomonas aeruginosa.
- Trial name
- NCT04684641
- Testing
- Standard Dose YPT-01
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
Cystic Fibrosis
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
This study compares the safety of the tobramycin solution for inhalation with the tobramycin dry powder formulation, used with a simple inhaler
- Trial name
- EAGER (NCT00388505)
- Testing
- Tobramycin Inhalation Powder, Tobramycin Solution for Inhalation
- Where
- 72 sites in United States, Australia and 12 more
- Volunteers
- 517 people
- Sponsor
- Novartis Pharmaceuticals
Long QT Syndrome
Long-term Efficacy Study of Sodium Channel Blocker in LQT3 Patients
The purpose of this study is to determine whether late sodium channel blockade might be effective in shortening the QTc interval in various LQT3 mutations and be considered as a safe therapeutic option for LQT3 patients.
- Trial name
- NCT01648205
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Rochester
Cystic Fibrosis
A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1
The primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic…
- Trial name
- NCT04059094
- Testing
- BI 1265162
- Where
- 26 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- Boehringer Ingelheim
Vaso-occlusive Crisis, Sickle Cell Anemia Crisis
Hyperbaric Oxygen Therapy in Sickle Cell Pain
Sickle cell anemia is a genetic blood disorder where red blood cells are abnormally shaped, crescent- or sickle-shaped, instead of the normal, round shape.
- Trial name
- HAVOC (NCT03412045)
- Testing
- hyperbaric oxygen therapy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.