Clinical trials: Syndrome, Metabolic
Mucopolysaccharidosis IVA (Morquio A Syndrome)
BMN 110 Phase 3B in Australian Patients
There is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment…
- Trial name
- MOR-AUS (NCT01966029)
- Testing
- BMN 110
- Where
- 5 sites in Australia
- Volunteers
- 13 people
- Sponsor
- BioMarin Pharmaceutical
Hypertension, Heart Failure
OneShot Renal Denervation Registry
This is a multi-center, prospective registry designed to monitor the outcomes of renal denervation with the OneShot Device in a real-world patient population.
- Conditions
- Hypertension, Heart Failure, Diabetes Mellitus, Sleep Apnea
- Trial name
- NCT01844037
- Testing
- OneShot Ablation System
- Where
- Italy
- Volunteers
- 51 people
- Sponsor
- Medtronic Endovascular
Congenital Adrenal Hyperplasia
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal…
- Trial name
- CONnECT (NCT05063994)
- Testing
- Chronocort, Cortef
- Where
- 21 sites in United States, France and 1 more
- Volunteers
- 55 people
- Sponsor
- Neurocrine UK Limited
Diabetes Mellitus
Efficacy of Pioglitazone Compared to Glyburide in Treating Subjects With Type 2 Diabetes Mellitus and Mild Cardiac Disease
The purpose of this study is to evaluate the cardiovascular effects of pioglitazone, once daily (QD), versus glyburide when administered to patients with type 2 diabetes mellitus and mild cardiac disease.
- Trial name
- NCT00521742
- Testing
- Pioglitazone, Glyburide
- Volunteers
- 300 people
- Sponsor
- Takeda
Overweight, Metabolic Syndrome X
Effects of NUTRIOSE®FB Dietary Fiber Supplementation on Satiety, Body Fat, and Metabolic Syndrome in Overweight Adult Men
The purpose of this study was to determine the effects of NUTRIOSE® supplementation on body composition, satiety, and determinants of metabolic syndrome in overweight men.
- Trial name
- NCT01044680
- Testing
- NUTRIOSE
- Where
- China
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Roquette Freres
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03811028
- Testing
- SOBI003
- Where
- 3 sites in United States, Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Ketosis, Epilepsy
Market Research - Acceptability Study for a Range of MCT Products
This is a market research, observational study to evaluate the tolerability and acceptability of MCT supplements for young children and young people with intractable epilepsy, GLUT-1 or PDHD from 3 years to adulthood.
- Conditions
- Ketosis, Epilepsy, Ketogenic Diet, Fatty Acid Oxidation Disorder
- Trial name
- NCT04309149
- Testing
- MCT fats
- Where
- 2 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Dr. Schär AG / SPA
Mucopolysaccharidosis I
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
- Trial name
- NCT02371226
- Testing
- AGT-181 (HIRMAb-IDUA)
- Where
- 6 sites in United States
- Volunteers
- 3 people
- Sponsor
- ArmaGen, Inc
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
Mucopolysaccharidosis IIIB
Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103 in participants with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with evaluable signs or symptoms of developmental delay.
- Trial name
- NCT02324049
- Testing
- SBC-103
- Where
- 4 sites in United States, Spain and 1 more
- Volunteers
- 11 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Obese, Sleep Apnea, Obstructive
Eval EarlySense Bet Lowenstein Sleeplab
The study objective is to collect heart rate, respiration rate and motion data of patients admitted to sleep lab and to determine the accuracy level of the EarlySense system vs.
- Conditions
- Obese, Sleep Apnea, Obstructive, Central Apnea, Sleep Disorders
- Trial name
- NCT01978340
- Where
- Israel
- Volunteers
- healthy volunteers welcome
- Sponsor
- EarlySense Ltd.
HIV Infections
ROCKET I - Randomized Open Label Switch for Cholesterol Elevation on Kivexa Evaluation Trial
This study aims to investigate whether patients switching their backbone from Kivexa to Truvada, who already have raised total cholesterol prior to switching, have an improvement in their total cholesterol after 12 weeks of…
- Trial name
- ROCKET I (NCT00615810)
- Testing
- Atripla, Kivexa plus Sustiva
- Where
- United Kingdom
- Volunteers
- 159 people
- Sponsor
- Gilead Sciences
Diabetes Mellitus
Efficacy and Safety of TAK-559 Combined With Glyburide in Treating Subjects With Type 2 Diabetes Mellitus.
The purpose of this study is to determine the safety and efficacy of TAK-559, once daily (QD), combined with glyburide in treating Type 2 Diabetes.
- Trial name
- ORIGAMI (NCT00759720)
- Testing
- TAK-559 and glyburide, Glyburide
- Volunteers
- 447 people
- Sponsor
- Takeda
Hypertension, Hyperhomocysteinemia
CSPPT- Chronic Kidney Diseases Study
The purpose of this trial is to confirm that enalapril maleate and folic acid tablets is more effective in preventing renal function decline among the patients with primary hypertension when compared to enalapril maleate.
- Trial name
- CSPPT-CKD (NCT01871740)
- Testing
- Enalapril maleate and folic acid tablets, Enalapril maleate
- Where
- 2 sites in China
- Sponsor
- Shenzhen Ausa Pharmed Co.,Ltd
Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
This extension study will allow participants to continue receiving treatment with HGT-1410 and to initiate treatment in patients who received no-treatment in Study HGT-SAN-093, and will evaluate the long-term safety and efficacy…
- Trial name
- NCT02350816
- Testing
- HGT-1410
- Where
- 8 sites in United States, France and 5 more
- Volunteers
- 17 people
- Sponsor
- Shire
Hyperlipidemia Combined
A Controlled-Study of Fenofibrate 145 mg and Ezetimibe 10 mg in Type IIb Dyslipidemic Patients With Features of the Metabolic Syndrome
Type IIb dyslipidemia is characterized by elevated triglycerides (TG ³ 150 mg/dL), and elevated LDL-C levels (³160 mg/dL). When in addition, HDL-C is low, the presence of the lipid triad, elevated TG and LDL-C and low levels of…
- Trial name
- NCT00349284
- Testing
- Combination of 145 mg fenofibrate and 10 mg ezetimibe
- Where
- 43 sites in Belgium, France and 1 more
- Volunteers
- 181 people
- Sponsor
- Solvay Pharmaceuticals
Hemophilia B, Mucopolysaccharidosis I
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol.
- Conditions
- Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
- Trial name
- LTFU (NCT04628871)
- Testing
- SB-318, SB-913, SB-FIX
- Where
- 5 sites in United States
- Volunteers
- 13 people
- Sponsor
- Sangamo Therapeutics
Obesity, Metabolic Syndrome
Association of Genes to Resistance to Weight Loss in Obese Patients
The overall goal of this study is to determine whether variations in the perilipin, and several other, genes would be a useful tool for physicians who are caring for morbidly obese patients to guide therapy.
- Conditions
- Obesity, Metabolic Syndrome, Dyslipidemia
- Trial name
- NCT00858221
- Where
- United States
- Volunteers
- 824 people
- Sponsor
- Interleukin Genetics, Inc.
Genetic Disorder, Prader-Willi Syndrome
Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers
This trial is conducted in United States of America (USA). The aim of this trial is to examine the bioequivalence of Norditropin® versus Genotropin® in healthy adult volunteers.
- Conditions
- Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature
- Trial name
- NCT01401244
- Testing
- somatropin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
HIV Infections
A Comparison of Three Anti-HIV Drug Combinations in HIV-Infected Patients
The purpose of this study is to look at the effects of different anti-HIV drug combinations on body fat changes in HIV-positive patients who have never received anti-HIV therapy before.
- Trial name
- NCT00005106
- Testing
- Lamivudine/Zidovudine, Abacavir sulfate, Nelfinavir mesylate
- Where
- 39 sites in United States
- Volunteers
- 230 people
- Sponsor
- Glaxo Wellcome