Clinical trials: Syndrome, Metabolic
Lactose Intolerance
Evaluation of GIMate Handheld Hydrogen Breath Monitor for Diagnosis of Lactose Malabsorption
The goal of this study is to assess the performance of the Vivante Health GIMate Breathalyzer device in diagnosing lactose malabsorption.
- Trial name
- NCT04754724
- Testing
- GIMate, H2 Check
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Vivante Health
Congenital Adrenal Hyperplasia
A Study of the Efficacy, Safety and Tolerability of Chronocort in Treating CAH
Subjects completing study DIUR-005 and those who have already completed study DIUR-003 will be offered the opportunity either to continue Chronocort® therapy or to switch from their current glucocorticoid therapy to Chronocort®…
- Trial name
- NCT03062280
- Testing
- Hydrocortisone
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Neurocrine UK Limited
MELAS
Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
- Trial name
- NCT04475549
- Testing
- IW-6463 Tablets
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Tisento Therapeutics
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
The purpose of this study is to evaluate the efficacy, safety, PK, and PD of atumelnant in adults with classic CAH due to 21-OHD.
- Trial name
- NCT07144163
- Testing
- Atumelnant
- Where
- 81 sites in United States, Argentina and 13 more
- Volunteers
- 150 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Familial Chylomicronemia
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT03544060
- Testing
- Volanesorsen
- Sponsor
- Akcea Therapeutics
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.
- Trial name
- NCT00299000
- Testing
- Naglazyme
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 4 people
- Sponsor
- BioMarin Pharmaceutical
Gaucher Disease, Cerebroside Lipidosis Syndrome
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status.
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00358943
- Where
- 317 sites in United States, Argentina and 50 more
- Volunteers
- 12,000 people
- Sponsor
- Genzyme, a Sanofi Company
Lesch-Nyhan Disease
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam…
- Trial name
- NCT01065558
- Testing
- Ecopipam
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Psyadon Pharma
Menkes Disease, Occipital Horn Syndrome
Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency
Menkes disease and occipital horn syndrome are two forms of copper deficiency that must be diagnosed and treated very early in life to prevent serious developmental problems.
- Conditions
- Menkes Disease, Occipital Horn Syndrome, Unexplained Copper Deficiency
- Trial name
- NCT00811785
- Testing
- Copper Histidine
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Cyprium Therapeutics, Inc.
Type 2 Diabetes Mellitus
Pioglitazone Special Drug Use Surveillance "Metabolic Syndrome in Patients With Type 2 Diabetes Mellitus"
The purpose of this survey is to determine the onset of new cerebral and cardiovascular events and changes in metabolic syndrome parameters in patients with type 2 diabetes mellitus on long-term pioglitazone (Actos Tablets)…
- Trial name
- NCT02002975
- Testing
- Pioglitazone
- Volunteers
- 18,223 people
- Sponsor
- Takeda
Diabetes Mellitus, Type 2
Effect Of Rosiglitazone On Carotid Intima Media Thickness In Patients With Insulin Resistance Syndrome And/Or Type 2 Diabetes
The study investigated the effect of rosiglitazone and placebo on carotid intima media thickness in patients with insulin resistance syndrome and/or type 2 diabetes.
- Trial name
- NCT00306644
- Testing
- Rosiglitazone
- Where
- Sweden
- Volunteers
- 556 people
- Sponsor
- GlaxoSmithKline
Obstructive Sleep Apnea
Maridebart Cafraglutide Versus Placebo in Adult Participants With Obstructive Sleep Apnea Not on Positive Airway Pressure (PAP) Therapy
This Phase 3 clinical trial is designed to evaluate the efficacy and safety of maridebart cafraglutide compared to placebo over a 52-week period in adults with obstructive sleep apnea (OSA) who are not on PAP therapy and are…
- Trial name
- MARITIME-OSA-2 (NCT07226765)
- Testing
- Maridebart cafraglutide
- Where
- 57 sites in United States, Australia and 9 more
- Volunteers
- 250 people
- Sponsor
- Amgen
Hunter Syndrome
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II) Patients
- Trial name
- NCT02044692
- Where
- South Korea
- Volunteers
- 34 people
- Sponsor
- Green Cross Corporation
Nutritional and Metabolic Diseases, Inflammation
A Study of Strawberries and Disease Risk
The purpose of this study is to test whether compounds found in strawberries (polyphenolics which are typically found in berry products, tea, coffee, red wine, and chocolate) will help reduce insulin resistance and inflammation…
- Conditions
- Nutritional and Metabolic Diseases, Inflammation, Physiological Responses
- Trial name
- STRB (NCT01199848)
- Testing
- 10G, 20G, 40G
- Where
- United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- Clinical Nutrition Research Center, Illinois Institute of Technology
Thymidine Kinase 2 Deficiency
A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)
This is a Phase 3b, prospective, single-arm, multicenter, open-label treatment study of the efficacy and safety of MT1621 in pediatric and adolescent patients with thymidine kinase 2 deficiency (TK2d).
- Trial name
- NCT04581733
- Testing
- MT1621
- Sponsor
- Zogenix MDS, Inc.
Colorectal Adenocarcinoma
A Study to Evaluate the Efficacy of VVD-133214 in Combination With Pembrolizumab Compared With Pembrolizumab Alone in Participants With Advanced Cancer of the Colon or Rectum
Researchers are looking for a better way to treat people with advanced colorectal cancer. Colorectal cancer (cancer of the colon or rectum) is one of the most common cancers worldwide.
- Trial name
- NCT07811193
- Testing
- VVD-133214, Pembrolizumab
- Where
- 122 sites in United States, Argentina and 20 more
- Volunteers
- 140 people
- Sponsor
- Vividion Therapeutics, Inc.
Lactose Intolerance
Incidence of Lactose Intolerance Among Self-reported Lactose Intolerant People
This is a 3-sites, double-blinded, randomized, 2X2 cross-over study aiming to compare effects of milk containing only A2 type beta casein versus milk containing both A1 and A2 beta casein proteins on the gastrointestinal symptoms…
- Trial name
- NCT02878876
- Testing
- Oral consumption of milk with sequence A1-A2, Oral consumption of milk with sequence A2-A1
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- a2 Milk Company Ltd.
Metabolic Syndrome
A Clinical Trial of YMC017 in Hypertensive and Hypercholesterolemic Patients With Metabolic Syndrome
This is a Randomized, open-label, 2 groups, parallel design.
- Trial name
- NCT02968160
- Testing
- Telmisartan 40mg + Rosuvastatin 20mg, Telmisartan 80mg + Rosuvastatin 20mg, Telmisartan 40mg, 80 mg
- Where
- South Korea
- Volunteers
- 55 people
- Sponsor
- Yuhan Corporation
Celiac Disease
Study of the Safety, Tolerability and Pharmacokinetics of TIMP-GLIA in Subjects With Celiac Disease
This study is to characterize the safety and tolerability of an investigational drug called TIMP-GLIA when either one or two intravenous doses are given to subjects with celiac disease.
- Trial name
- NCT03486990
- Testing
- TIMP-GLIA
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Takeda
Mucopolysaccharidosis Type IIIA
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance.
- Trial name
- AAVance (NCT03612869)
- Testing
- LYS-SAF302
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- LYSOGENE