Clinical trials: Syndrome, Metabolic
Multiple System Atrophy
A Biomarker Study of ATH434 in Participants With MSA
This study will assess the safety and efficacy of ATH434 in participants with a clinical diagnosis of Multiple System Atrophy
- Trial name
- NCT05864365
- Testing
- ATH434
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Alterity Therapeutics
Type 2 Diabetes, Cortisol Excess
Trial Against INtractable Type 2 Diabetes (CAPTAIN-T2D)
CAPTAIN-T2D will take place in two parts. Part 1 (Screening) will evaluate patients with type 2 diabetes and elevated cortisol risk factors for trial eligibility and the presence of elevated cortisol.
- Trial name
- CAPTAIN-T2D (NCT07296484)
- Testing
- clofutriben
- Where
- 61 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- Sparrow Pharmaceuticals
Sanfilippo Syndrome Type A
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including…
- Trial name
- NCT01047306
- Testing
- assessment
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Shire
Anemia, Iron-Deficiency, Kidney Failure, Chronic
Effect of Ferrlecit® Versus Oral Iron on Iron Deficient Chronic Kidney Disease (CKD) Patients
This study compares the effect of Ferrlecit® (a form of intravenous iron) to ferrous sulfate (a form of oral iron) in treating anemia and iron deficiency in chronic kidney disease patients who are not receiving erythropoietic…
- Trial name
- NCT00224055
- Testing
- Sodium Ferric Gluconate Complex in Sucrose Injection, Ferrous sulfate tablets
- Where
- 24 sites in United States, Puerto Rico
- Volunteers
- 89 people
- Sponsor
- Watson Pharmaceuticals
Familial Chylomicronemia Syndrome (FCS)
CS-121 APOC3 Base Editing in FCS
This is an open-label, single-arm, dose-escalation Phase I clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid…
- Trial name
- NCT07176923
- Testing
- CS-121
- Where
- China
- Volunteers
- 15 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
HIV Infections, Hepatitis B
ProAlgaZyme Novel Algae Infusion: Applications in Immunodeficiency
The purpose of this study is to determine the safety and effects in HIV patients of supplementation (4-20 fl. oz. daily) with ProAlgaZyme, a novel fermentation product of a freshwater algae ecosystem, on markers of immune status…
- Conditions
- HIV Infections, Hepatitis B, Dyslipidemia
- Trial name
- NCT00493506
- Testing
- ProAlgaZyme
- Where
- Cameroon
- Volunteers
- 59 people
- Sponsor
- Health Enhancement Products, Inc.
MPS III B, Mucopolysaccharidosis Type IIIB
A Treatment Study of Mucopolysaccharidosis Type IIIB
The study's primary objectives are to evaluate the safety and tolerability of AX 250 administered to subjects with MPS IIIB via an ICV reservoir and catheter and to evaluate the impact of AX 250 on cognitive function in patients…
- Trial name
- MPS IIIB (NCT02754076)
- Testing
- AX 250
- Where
- 7 sites in United States, Colombia and 5 more
- Volunteers
- 23 people
- Sponsor
- Allievex Corporation
MPS IV A
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed…
- Trial name
- NCT00884949
- Testing
- BMN 110
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Leigh Syndrome
A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)
The goal of this clinical trial is to investigate oral TTI-0102 to evaluate its pharmacokinetics, safety, and preliminary efficacy in patients with Leigh syndrome spectrum, in three sequences.
- Trial name
- NCT06990984
- Testing
- TTI-0102: cysteamine-pantetheine disulfide, D-Mannitol
- Volunteers
- 18 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
Lowe Syndrome
An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
The primary objective of this trial is to: 1. Evaluate the efficacy of piclidenoson to increase renal uptake of 99mTc-labeled DMSA, in comparison to baseline, after 6 months (26 weeks) of treatment as a measure the reabsorption…
- Trial name
- NCT07410455
- Testing
- Piclidenoson
- Where
- Italy
- Volunteers
- 5 people
- Sponsor
- Can-Fite BioPharma
Nephropathic Cystinosis
DFT383 in Pediatric Participants With Nephropathic Cystinosis
An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase.
- Trial name
- CYStem (NCT06910813)
- Testing
- DFT383
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Hunter Syndrome
Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome
As the HGT-HIT-046 (NCT01506141) and SHP609-302 (NCT02412787) studies are completed and closed, this post-trial access (PTA) program provides TAK-609 to participants in these studies for whom the benefit:risk ratio of continued…
- Trial name
- NCT05795361
- Testing
- Idursulfase-IT
- Where
- 22 sites in United States, Australia and 3 more
- Sponsor
- Takeda
HIV-Associated Lipodystrophy Syndrome
Study of Reyataz in HIV-infected Patients With Lipodystrophy Syndrome
The purpose of this clinical research study is to learn if human immunodeficiency virus (HIV)-infected subjects with abdominal fat accumulation on their highly active antiretroviral treatment (HAART) regimen have better changes…
- Trial name
- NCT00135356
- Testing
- Atazanavir (ATV) + ritonavir (RTV), continuation of backbone 2 nucleoside reverse transcriptase inhibitor (NRTIs), continuation of current HAART (boosted protease inhibitor [PI] combination + 2 NRTIs)
- Where
- 30 sites in United States, Canada and 7 more
- Volunteers
- 219 people
- Sponsor
- Bristol-Myers Squibb
Prader-Willi Syndrome
Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.
- Trial name
- NCT03714373
- Testing
- DCCR
- Where
- 28 sites in United States, United Kingdom
- Volunteers
- 115 people
- Sponsor
- Soleno Therapeutics, Inc.
Cardiovascular Diseases
A Study of AMR101 to Evaluate Its Ability to Reduce Cardiovascular Events in High-Risk Patients With Hypertriglyceridemia and on Statin
AMR101 (icosapent ethyl [ethyl-EPA]) is a highly purified ethyl ester of eicosapentaenoic acid (EPA) developed by Amarin Pharma Inc.
- Trial name
- REDUCE-IT (NCT01492361)
- Testing
- AMR101, Statin therapy
- Where
- 410 sites in United States, Australia and 9 more
- Volunteers
- 8,179 people
- Sponsor
- Amarin Pharma Inc.
Fanconi Anemia Complementation Group A, Fanconi Anemia
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial.
- Trial name
- NCT04437771
- Testing
- Safety and efficacy assessments
- Where
- Spain
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Metabolic Syndrome
Effect of Crestor (Rosuvastatin) on Lipid Levels in Patients With Metabolic Syndrome
The primary objective is to evaluate the efficacy of rosuvastatin therapy on plasma lipid profile (Low Density Lipoprotein (LDL), High-Density Lipoprotein (HDL), total cholesterol, triglyceride) in patients with metabolic…
- Trial name
- EFFORT (NCT00815659)
- Testing
- rosuvastatin
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 97 people
- Sponsor
- AstraZeneca
Diabetes Mellitus
Efficacy of Pioglitazone and Fortamet Combination Therapy in Subjects With Type 2 Diabetes
The purpose of this study is to determine the efficacy of pioglitazone and metformin combination therapy, once daily (QD), on glycosylated hemoglobin in adults with type 2 diabetes.
- Trial name
- NCT00754403
- Testing
- Pioglitazone and metformin, Metformin
- Volunteers
- 312 people
- Sponsor
- Takeda
Metabolic Syndrome, Pre-diabetes
Effect of Pasteurized Akkermansia Muciniphila on Insulin Resistance in Otherwise Healthy Subjects With Dysglycaemia
The purpose of this study is to demonstrate the efficacy of pasteurized Akkermansia muciniphila (pAKK) in improving insulin sensitivity in hyperglycaemic, but otherwise healthy persons with metabolic syndrome.
- Conditions
- Metabolic Syndrome, Pre-diabetes, Overweight and Obesity
- Trial name
- NCT05114018
- Testing
- Pasteurized A. muciniphila
- Where
- 2 sites in Germany, Ireland
- Volunteers
- 143 people, healthy volunteers welcome
- Sponsor
- A-Mansia Biotech S.A.