Clinical trials: Genetic Conditions
Joint Damage
Effect of Febuxostat on Joint Damage in Hyperuricemic Subjects With Early Gout
This purpose of this study is to assess the effect of febuxostat, once daily (QD), on joint damage in patients with elevated serum urate levels and gout.
- Trial name
- NCT01078389
- Testing
- Febuxostat
- Where
- 56 sites in United States
- Volunteers
- 314 people
- Sponsor
- Takeda
Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1
Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.
- Conditions
- Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome
- Trial name
- NCT01522976
- Testing
- Azacitidine, Laboratory Biomarker Analysis, Lenalidomide
- Where
- 294 sites in United States, Canada
- Volunteers
- 282 people
- Sponsor
- National Cancer Institute (NCI)
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
An Extension Study to Determine the Efficacy and Safety of STI571 in Participants With Chronic Myeloid Leukemia Who Are Refractory to or Intolerant of Interferon-Alpha
During the Core Phase of the study, participants received STI571 at a dose of 400 milligrams (mg) daily for up to 12 months. Participants completing 12 months of therapy were eligible to continue treatment in the Extension Phase…
- Trial name
- NCT00171223
- Testing
- STI571
- Where
- 28 sites in United States, France and 4 more
- Volunteers
- 532 people
- Sponsor
- Novartis Pharmaceuticals
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
Pharmacokinetics and Safety of MM36 Topical Ointment in Pediatric Subjects With Atopic Dermatitis
The purpose of this study is to assess the pharmacokinetic parameters and safety of topical MM36 (OPA-15406) ointment in pediatric subjects with atopic dermatitis under maximal use conditions.
- Trial name
- NCT02945657
- Testing
- MM36 topical ointment, 1%
- Where
- 12 sites in United States, Honduras and 1 more
- Volunteers
- 32 people
- Sponsor
- Medimetriks Pharmaceuticals, Inc
Other Acute Pain
An Efficacy and Safety Trial of Intranasal Ketorolac in Emergency Department Patients for the Treatment of Acute Pain
The purpose of this study is to determine the effectiveness, the safety, and the tolerability of intranasal ketorolac (Sprix) in relieving acute pain in adults ages 18-65 who come to the ED seeking care.
- Trial name
- Sprix (NCT01471639)
- Testing
- intranasal ketorolac
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Down Syndrome, Amyloid Beta-protein
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
To determine the sensitivity of the visual assessment of BAY94-9172 PET images in detecting cerebral β-amyloid in individuals with Down Syndrome (DS) and specificity in individuals without DS.
- Trial name
- NCT00928304
- Testing
- Florbetaben (BAY94-9172)
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Lantheus Biosciences Ltd.
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Norditropin NordiFlex® Device Compared to the Device Previously Used by Patients or Parents
This study is conducted in Europe. The aim of this study is to compare the easiness of use of Norditropin NordiFlex® device to the device previously used by patients or parents.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Foetal Growth Problem, Small for Gestational Age
- Trial name
- NCT01245374
- Testing
- Norditropin NordiFlex®
- Where
- France
- Volunteers
- 103 people
- Sponsor
- Novo Nordisk A/S
Emphysema, Alpha 1-antitrypsin Deficiency (AATD)
Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency
This is a study to assess the safety and pharmacokinetics of weekly infusions of 120 mg/kg of Prolastin-C (alpha1-proteinase inhibitor [alpha1-PI] [Human]), compared to weekly infusions of 60 mg/kg of Prolastin-C in patients with…
- Trial name
- SPARK (NCT01213043)
- Testing
- Prolastin-C, 60 mg/kg, Prolastin-C, 120 mg/kg
- Where
- 5 sites in United States
- Volunteers
- 30 people
- Sponsor
- Grifols Therapeutics LLC
CDK Gene Mutation, Metastatic Malignant Neoplasm in the Brain
Genetic Testing in Guiding Treatment for Patients With Brain Metastases
This phase II trial studies how well genetic testing works in guiding treatment for patients with solid tumors that have spread to the brain.
- Conditions
- CDK Gene Mutation, Metastatic Malignant Neoplasm in the Brain, Metastatic Malignant Solid Neoplasm, NTRK Family Gene Mutation
- Trial name
- NCT03994796
- Testing
- Abemaciclib, PI3K Inhibitor paxalisib, Entrectinib
- Where
- 435 sites in United States
- Volunteers
- 50 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Sepsis
Algorithm for Predicting the Unfavorable Course of Sepsis in Children
A comprehensive strategy will be used to investigate the relationship and correlation between 4 diagnostically significant markers relevant for early diagnosis and prediction of complications and death in the development of…
- Trial name
- NCT05908162
- Testing
- Bood leukocyte subsets
- Where
- Belarus
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- The Republican Research and Practical Center for Epidemiology and Microbiology
Sickle Cell Disease
Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers
The investigators are conducting a Phase II prospective and placebo controlled study of a topical cream containing sodium nitrite compared to the current standard of care.
- Trial name
- NCT02863068
- Testing
- Topical Sodium Nitrite
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- caterina P minniti
Sickle Cell Disease
Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease
The goal of this pilot study is to improve emergency department (ED) pain management for adults with sickle cell disease. Sickle cell disease (SCD) is the most common genetic disorder in the United States, and occurs primarily…
- Trial name
- NCT02222246
- Testing
- Hydromorphone (Standardized, weight-based dosing), Morphine Sulfate (Standardized, weight-based dosing), Hydromorphone (Patient Specific dosing)
- Where
- 2 sites in United States
- Volunteers
- 106 people
- Sponsor
- Duke University
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.
Chronic Myeloid Leukemia
Safety and Efficacy of Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia Patients
This study will further investigate the safety and efficacy of nilotinib in newly diagnosed chronic myeloid leukemia patients in the chronic phase
- Trial name
- ENESTxtnd (NCT01254188)
- Testing
- Nilotinib
- Where
- 100 sites in Algeria, Argentina and 17 more
- Volunteers
- 421 people
- Sponsor
- Novartis Pharmaceuticals
Huntington's Disease, Chorea
Real World Effectiveness and Safety of Deutetrabenazine in Adult Chinese Participants With Huntington's Disease (HD) Chorea in China
The Primary Objective: To evaluate the real-world effectiveness of deutetrabenazine in adult participants with chorea associated with Huntington's disease in China.
- Trial name
- NCT07601516
- Testing
- Deutetrabenazine
- Where
- 3 sites in China
- Volunteers
- 50 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D LLC
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Cystic Fibrosis, Prediabetes
Prevention of Cystic Fibrosis Diabetes
Acute systemic hyperglycemia causes oxidative stress and a pro-inflammatory response. The pro-inflammatory cytokines induced by hyperglycemia are toxic to islet insulin producing cells, and thus worsen glucose intolerance.
- Trial name
- Prevent (NCT00967798)
- Testing
- Sitagliptin
- Where
- 3 sites in United States
- Volunteers
- 33 people
- Sponsor
- Emory University
Hereditary Tyrosinemia, Type I
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to compare the bioavailability of the Test Product, Nitisinone 10 mg Tablet, under fasting and fed conditions (food-effect).
- Trial name
- NCT02750332
- Testing
- Nitisinone
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.