Clinical trials: Genetic Conditions
Alpha1-antitrypsin Deficiency
Aralast alpha1-proteinase Inhibitor Surveillance Study
The primary objectives of this Phase 4, open label, prospective U.S. surveillance study are to evaluate the health outcomes of Alpha 1-Antitrypsin (AAT)-deficient subjects who are initiating treatment with ARALAST on…
- Trial name
- NCT00313144
- Testing
- ARALAST Alpha1-Proteinase Inhibitor
- Where
- United States
- Volunteers
- 127 people
- Sponsor
- Baxalta now part of Shire
Bardet-Biedl Syndrome, POMC Deficiency
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.
- Trial name
- NCT05194124
- Testing
- Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg
- Where
- 7 sites in United States, Canada and 4 more
- Volunteers
- 19 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Hunter Syndrome
An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV)…
- Trial name
- NCT01506141
- Testing
- Idursulfase-IT, Elaprase
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- Takeda
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
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
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
Cystic Fibrosis, Chronic Pancreatitis
Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon
Exocrine pancreatic insufficiency (EPI) is a condition that is caused by the inadequate pancreatic enzymes needed for normal digestion and is commonly associated with a wide range of chronic diseases, including cystic fibrosis…
- Trial name
- NCT05069597
- Testing
- CREON
- Where
- 21 sites in United States
- Volunteers
- 30 people
- Sponsor
- AbbVie
Thalassemia Major
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as…
- Trial name
- NCT02049450
- Testing
- ruxolitinib
- Where
- 7 sites in Greece, Italy and 3 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
Efficacy and Safety Study of the Use of Benralizumab for Patients With Moderate to Severe Atopic Dermatitis
The purpose of the study is to compare the efficacy and safety of benralizumab versus placebo and to compare benralizumab dosing regimens during extension period.
- Trial name
- NCT04605094
- Testing
- Benralizumab
- Where
- 51 sites in United States, Australia and 6 more
- Volunteers
- 194 people
- Sponsor
- AstraZeneca
Neoplasms, Breast
Pazopanib Plus Lapatinib Compared To Lapatinib Alone In Subjects With Advanced Or Metastatic Breast Cancer
This study is being conducted to compare the efficacy and safety of pazopanib in combination with lapatinib with that of lapatinib alone in subjects with locally advanced or metastatic breast cancer whose tumors overexpress the…
- Trial name
- NCT00347919
- Testing
- pazopanib (GW786034) 400 mg, lapatinib (GW572016) 1500 mg, lapatinib (GW572016) 1000 mg
- Where
- 84 sites in United States, Canada and 14 more
- Volunteers
- 189 people
- Sponsor
- GlaxoSmithKline
Endometriosis
A Clinical Study To Investigate The Effectiveness And Safety Of Tanezumab In Treating Pain Associated With Endometriosis
The purpose of this study is to determine whether tanezumab is effective and safe in the treatment of pain associated with endometriosis.
- Trial name
- NCT00784693
- Testing
- Tanezumab
- Where
- 30 sites in United States
- Volunteers
- 48 people
- Sponsor
- Pfizer
Familial Adenomatous Polyposis Coli, FAP
Chemoprevention Trial in Familial Adenomatous Polyposis (FAP) Coli Using EPA
This purpose of this study is to investigate whether the number and size of rectal polyps can be reduced in patients with Familial Adenomatous Polyposis (FAP) by using a highly-purified form of a naturally occurring substance…
- Trial name
- NCT00510692
- Testing
- Eicosapentanoic Acid (EPA), Endoscopy, Biopsies taken
- Where
- United Kingdom
- Volunteers
- 58 people
- Sponsor
- S.L.A. Pharma AG
Lower Urinary Tract Symptoms, Bladder Outlet Obstruction
A Study of Mirabegron (YM178) in Men With Lower Urinary Tract Symptoms (LUTS) and Bladder Outlet Obstruction (BOO)
This study examined the safety, tolerability, and efficacy of mirabegron (YM178) compared to placebo.
- Trial name
- NCT00410514
- Testing
- Mirabegron
- Where
- 27 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- Astellas Pharma Inc
Asymptomatic Hyperuricemia
A Study to Assess the Effect of Intensive Uric Acid (UA) Lowering Therapy With RDEA3170, Febuxostat, Dapagliflozin on Urinary Excretion of UA
This is a randomized, placebo controlled, double-blind, 2-way crossover study conducted on asymptomatic hyperuricemic patients. The core study consists of screening period, 2 treatment periods (verinurad + febuxostat +…
- Trial name
- NCT03316131
- Testing
- Verinurad, Febuxostat, Dapagliflozin
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Mucopolysaccharidosis II
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
- Trial name
- NCT00069641
- Testing
- Iduronate-2-sulfatase enzyme replacement therapy
- Where
- 9 sites in United States, Brazil and 2 more
- Volunteers
- 96 people
- Sponsor
- Shire
Tourette's Disorder, Tic Disorder
Safety and Tolerability of Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to determine safety of Once-daily aripiprazole in reducing Total Tic Severity in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01727713
- Testing
- Aripiprazole
- Where
- 33 sites in United States, Canada and 2 more
- Volunteers
- 110 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Arthritis, Osteoarthritis
Tanezumab in Osteoarthritis of the Knee
Test the efficacy and safety of 3 doses in Osteoarthritis of the knee in patients.
- Trial name
- NCT00733902
- Testing
- tanezumab
- Where
- 82 sites in United States
- Volunteers
- 697 people
- Sponsor
- Pfizer
Atopic Dermatitis
Study of TS-022 in Adult Patients With Atopic Dermatitis With Pruritus (POC)
The purpose and (primary) objectives of this study are to evaluate the efficacy, safety, and tolerability of TS-022 in adults with atopic dermatitis who have moderate to very severe pruritus (itching), following a 28-day regimen…
- Trial name
- NCT00914186
- Testing
- TS022, Vehicle
- Where
- 15 sites in United States
- Volunteers
- 122 people
- Sponsor
- Taisho Pharmaceutical R&D Inc.
Alpha1-Antitrypsin Deficiency
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency
This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of Alpha-1 MP (alpha1-proteinase inhibitor (human), modified process) for 8 weeks.
- Trial name
- NCT02870309
- Testing
- Alpha-1 MP
- Where
- 2 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC