Clinical trials: The Metabolic Syndrome
Mitochondrial Diseases, Mitochondrial Myopathies
The KHENERGYZE Study
Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins of the various enzyme complexes of the OXPHOS.
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS Syndrome
- Trial name
- NCT04165239
- Testing
- KH176
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 27 people
- Sponsor
- Khondrion BV
Obesity, Obstructive Sleep Apnea
Maridebart Cafraglutide Versus Placebo in Adult Participants With Obstructive Sleep Apnea on Positive Airway Pressure 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 receiving positive airway…
- Trial name
- MARITIME-OSA-1 (NCT07225686)
- Testing
- Maridebart cafraglutide
- Where
- 61 sites in United States, Australia and 9 more
- Volunteers
- 250 people
- Sponsor
- Amgen
Body Weight, Metabolic Syndrome
Evaluation of the Body Slimming Effect of TCI904
To assess the efficacy of Lactobacillus delbrueckii ssp. bulgaricus TCI904 on body slimming
- Trial name
- NCT05805488
- Testing
- TCI904 sachet
- Where
- Taiwan
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- TCI Co., Ltd.
Sanfilippo Syndrome Type A
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device…
- Trial name
- NCT06567769
- Testing
- GC1130A
- Where
- 4 sites in United States, South Korea
- Volunteers
- 9 people
- Sponsor
- GC Biopharma Corp
Fanconi Anemia
A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia
This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and…
- Trial name
- NCT04522375
- Testing
- FP-045
- Sponsor
- Foresee Pharmaceuticals Co., Ltd.
Celiac Disease
Study of the Safety, Tolerability, Pharmacokinetics and Biomarker of DONQ52 in Celiac Disease Patients
This study is to characterize the safety and tolerability of an investigational drug called DONQ52 and consists of a single ascending dose part (Part A) and a multiple ascending dose part (Part B) in well-controlled celiac…
- Trial name
- NCT05425446
- Testing
- DONQ52
- Where
- 21 sites in United States, Australia
- Volunteers
- 56 people
- Sponsor
- Chugai Pharmaceutical
Diabetes Mellitus
Efficacy Study of Pioglitazone and Glimepiride on the Rate of Progression of Atherosclerotic Disease.
The primary purpose of this study is to compare the effects of pioglitazone, once daily (QD), versus glimepiride on the amount of thickening of the carotid artery.
- Trial name
- CHICAGO (NCT00225264)
- Testing
- Pioglitazone, Glimepiride
- Where
- United States
- Volunteers
- 458 people
- Sponsor
- Takeda
Autoimmune Diseases, Ulcerative Colitis
A Biospecimen Collection Study to Identify the Targets of Disease-Reactive T Cells in Patients With Autoimmune Disease
The most clinically meaningful way to discover new targets of T cells in autoimmune diseases is to study the tissues of patients with active autoimmune disease mediated organ inflammation.
- Conditions
- Autoimmune Diseases, Ulcerative Colitis, Multiple Sclerosis, Scleroderma
- Trial name
- NCT06587828
- Testing
- Companion blood samples with procedure
- Where
- 11 sites in United States
- Volunteers
- 238 people
- Sponsor
- TScan Therapeutics, Inc.
Congenital Adrenal Hyperplasia, 21-OHD
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
- Trial name
- NCT05128942
- Testing
- Tildacerfont
- Where
- 13 sites in United States
- Volunteers
- 67 people
- Sponsor
- Spruce Biosciences
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome
RGX-111 Gene Therapy in Patients With MPS I
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system.
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT03580083
- Testing
- RGX-111
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
Mucopolysaccharidosis I, Hurler's Syndrome
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease.
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00146770
- Testing
- Aldurazyme
- Where
- 25 sites in United States, Brazil and 5 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company