Clinical trials: Congenital Deformity
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Cardiovascular Disease
Study of Non-Invasive Regional Oxygen Saturations in Pediatric Patients Undergoing Cardiac Catheterization
The purpose of this study is to validate the Nonin Near-infrared spectroscopy (NIRS) device to measure regional oxygen saturation in a cohort of children with cardiovascular disease undergoing cardiac catheterization.
- Trial name
- NCT00939224
- Testing
- Model 7600 Regional Oximeter System
- Where
- 3 sites in United States
- Volunteers
- 100 people
- Sponsor
- Nonin Medical, Inc
Cystic Fibrosis, Adherence, Medication
Evaluation of the ReX-C System, in Patients Receiving CFTR Modulators for the Treatment of Cystic Fibrosis (CF).
Cystic fibrosis (CF) is a progressive multisystem disorder characterized by abnormalities in the transport of chloride ions in human airway epithelial cells, leading to frequent lung infections, decreased pulmonary function…
- Trial name
- NCT04215796
- Testing
- ReX-C system
- Where
- 2 sites in Israel
- Volunteers
- 10 people
- Sponsor
- Dosentrx Ltd.
Cystic Fibrosis
Safety, Pharmacokinetic and Pharmacodynamic Study of MP-376 in Patients With Cystic Fibrosis
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT00503490
- Testing
- MP-376 (Levofloxacin solution for Inhalation)
- Where
- 14 sites in United States
- Volunteers
- 40 people
- Sponsor
- Amgen
Type II Hyperlipidaemia
Efficacy and Safety of JTT-705 300, 600 And 900mg in Comparison With Placebo in Patients With Type II Hyperlipidaemia
To demonstrate the effect of JTT-705 doses from 300 mg to 900 mg on the elevation of HDL-C and on the inhibition of CETP activity versus placebo, in patients presenting with mild dyslipidaemia.
- Trial name
- NCT00686010
- Testing
- JTT-705 300mg, JTT-705 600mg, JTT-705 900mg
- Where
- Netherlands
- Volunteers
- 198 people
- Sponsor
- Japan Tobacco Inc.
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Neonatal Abstinence Syndrome
Donor Human Milk in Neonatal Abstinence Syndrome
This study is designed to develop pilot data on the acceptability and benefit of donor human milk for infants undergoing pharmacologic treatment for NAS.
- Trial name
- DHM&NAS (NCT02182973)
- Testing
- donor human milk
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Neolac Inc dba Medolac Laboratories
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals
Basal Cell Carcinoma in Basal Cell Nevus Syndrome
Study of ASN-002 to Treat Basal Cell Carcinomas (BCCs) in Individuals With Basal Cell Nevus Syndrome (BCNS)
The primary objective is to confirm the safety of treating multiple BCCs once weekly x 3 weeks in individuals with Basal Cell Nevus Syndrome (BCNS).
- Trial name
- NCT03208296
- Testing
- ASN-002
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Ascend Biopharmaceuticals Ltd
Mucopolysaccharidosis (MPS)
A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil
The main aims of the study are to learn about the percentage of mucopolysaccharidosis type II (MPS II) in adults in Brazil as well as about the diagnosis process. No study medicines will be provided to participants in this study.
- Trial name
- NCT05155488
- Testing
- No Intervention
- Where
- Brazil
- Volunteers
- 250 people
- Sponsor
- Takeda
Hereditary Angioedema
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.
- Trial name
- OPuS-1 (NCT01984788)
- Testing
- BCX4161
- Where
- 6 sites in Germany, United Kingdom
- Volunteers
- 24 people
- Sponsor
- BioCryst Pharmaceuticals
Single Ventricle, Hypoplastic Left Heart Syndrome (HLHS)
Long-term Safety Study Following to JRM-001 Treatment in Pediatric Patients With Congenital Heart Disease
The objective of this observational study is to investigate the long-term safety of autologous cardiac stem cells (JRM-001) administered to patients with single ventricle physiology.
- Trial name
- NCT07884318
- Where
- Japan
- Volunteers
- 40 people
- Sponsor
- Metcela Inc.
Atopic Dermatitis, Eczema
A Trial of a Botanical Drug Containing East Indian Sandalwood Oil (EISO) For Treatment of Atopic Dermatitis
This trial will be a double-blind, randomized, placebo-controlled, safety, tolerability and efficacy trial of SAN007 (5% East Indian sandalwood oil in a cream formulation) treatment regimen when administered daily for up to 28…
- Trial name
- NCT02871479
- Testing
- SAN007 5% cream, SAN007 10% cream
- Where
- 5 sites in United States
- Volunteers
- 71 people
- Sponsor
- Santalis Pharmaceuticals, Inc.
Patent Foramen Ovale
Paradigm I Clinical Trial: Study of the PFX Closure System in Subjects With Cryptogenic Stroke, Transient Ischemia Attack or Paradoxical Embolism
The primary objective of this study is to demonstrate the safety of the PFX Closure System when utilized for patients with patent foramen ovale (PFO) suffering from cryptogenic stroke, transient ischemic attacks or paradoxical…
- Trial name
- NCT00196027
- Testing
- PFX Closure System
- Where
- Germany
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Cierra
Stargardt's Macular Dystrophy
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
- Trial name
- NCT02941991
- Testing
- hESC-RPE
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
- Trial name
- NCT02131623
- Where
- 5 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Atopic Dermatitis
A Safety, Tolerability and Pharmacokinetic Study of LEO 29102 in Subjects With Atopic Dermatitis
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of 7 days treatment with LEO 29102 in adult patients with atopic dermatitis.
- Trial name
- NCT01005823
- Testing
- LEO 29102 cream
- Where
- Netherlands
- Volunteers
- 36 people
- Sponsor
- LEO Pharma
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
- Trial name
- NCT07290257
- Testing
- Livmarli Oral Product
- Where
- 15 sites in Belgium, France and 6 more
- Volunteers
- 230 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Bloating
A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population
The purpose of this study is to investigate the effects of OPTI-BIOME™ (Bacillus subtilis MB40), a probiotic supplement, on bloating, gas and abdominal discomfort symptoms in otherwise healthy adults.
- Trial name
- NCT02950012
- Testing
- OPTI-BIOME™ Bacillus subtilis MB40
- Where
- 3 sites in United States, Canada
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- BIO-CAT Microbials, LLC
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.