Clinical trials: Syndrome, Metabolic
Sly Syndrome, MPS VII
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans…
- Conditions
- Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
- Trial name
- NCT02418455
- Testing
- UX003
- Where
- 5 sites in United States, Portugal and 1 more
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Celiac Disease
Study of ALV003 in Healthy Adult Volunteers and Subjects With Well-Controlled Celiac Disease Following a Gluten-Containing Meal
ALV003-0812 is a study of the safety and tolerability of a study drug (ALV003) in healthy adult volunteers and in patients with well-controlled celiac disease, following a meal that contains gluten.
- Trial name
- FED (NCT00669825)
- Testing
- ALV003
- Where
- 2 sites in United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Alvine Pharmaceuticals Inc.
Type2 Diabetes Mellitus
Investigate the Efficacy and Safety of Low-Glu in Patients Newly Diagnosed With Type II Diabetes Mellitus
Ficus deltoidea leaves, Cinnamomum cassia and Black seed powdered extract have long been used for the treatment of type 2 diabetes mellitus.
- Trial name
- NCT05343767
- Testing
- Natural Wellness Low-Glu low dose, Natural Wellness Low-Glu high dose, Metformin
- Where
- Egypt
- Volunteers
- 198 people
- Sponsor
- Natural Wellness Egypt
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.
Insulin Resistance - Type A, Insulin Resistance - Type B
A Extension Study of Empagliflozin in Patients With Refractory Diabetes Mellitus With Insulin Resistance ( EMPIRE-02 )
A multicenter, open-label, single-arm, extension study with regard to the safety and efficacy of empagliflozin in patients with refractory diabetes mellitus with insulin resistance.
- Conditions
- Insulin Resistance - Type A, Insulin Resistance - Type B, Lipoatrophic Diabetes Mellitus, Insulin Resistance Syndrome
- Trial name
- EMPIRE-02 (NCT04221152)
- Testing
- Empagliflozin Tablets
- Where
- 5 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Kobe University
Syndrome of Inappropriate Antidiuretic Hormone Secretion (SIADH)
A PK Study of 3 Dosages of Tolvaptan in Patients With Syndrome of Inappropriate Antidiuretic Hormone Secretion (SIADH)
This is a study to evaluate how the body handles and metabolizes (PK) the various doses of the drug Tolvaptan, and what the effect (PD) of the various doses of Tolvaptan are on the content of "salt" in blood and urine
- Trial name
- NCT02009878
- Testing
- tolvaptan
- Where
- 14 sites in Czechia, Denmark and 5 more
- Volunteers
- 30 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Metabolic Syndrome, Insulin-resistance
Metabolic and Cardiovascular Effects of Armolipid Plus in Subjects With Metabolic Syndrome
Aim of the study is to evaluate the effects of Armolipid Plus on insulin sensitivity in patients with MetS and increased LV mass.
- Conditions
- Metabolic Syndrome, Insulin-resistance, Left Ventricular Hypertrophy
- Trial name
- ARMP-11 (NCT02295176)
- Testing
- Armolipid Plus
- Where
- 3 sites in Italy
- Volunteers
- 159 people
- Sponsor
- Rottapharm
Diabetes Mellitus
Observational Study to Assess Glycosylated Hemoglobin Changes After 6 Months of Treatment With Pioglitazone.
The purpose of this study is to asses changes in glycosylated hemoglobin, fasting blood lipids and genetic polymorphism's in peroxisomal proliferator activated receptors--gamma receptor after 6 months of pioglitazone, once daily…
- Trial name
- NCT00449553
- Testing
- Pioglitazone and sulphonylurea, Pioglitazone and metformin
- Where
- 4 sites in Denmark, Iceland and 2 more
- Volunteers
- 326 people
- Sponsor
- Takeda
Crigler-Najjar Syndrome
Clinical Assessment Study in Crigler-Najjar Syndrome
This is a Pre-Phase 1 prospective, non-interventional clinical assessment study to evaluate Crigler-Najjar syndrome subjects requiring daily phototherapy, aged 1 year and older.
- Trial name
- LUSTRO (NCT03078881)
- Where
- 4 sites in United States, Israel and 1 more
- Volunteers
- 8 people
- Sponsor
- Audentes Therapeutics
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
High Blood Pressure, Metabolic Syndrome
Comparison of Aliskiren and Amlodipine on Insulin Resistance and Endothelial Dysfunction in Patients With Hypertension and Metabolic Syndrome
The purpose of this study was to determine the effects of Aliskiren on insulin resistance (IR) and endothelial dysfunction (ED) in patients with high blood pressure and metabolic syndrome.
- Conditions
- High Blood Pressure, Metabolic Syndrome, Insulin Resistance, Endothelial Dysfunction
- Trial name
- NCT00417170
- Testing
- Aliskiren, Amlodipine
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Novartis Pharmaceuticals
Essential Hypertension, Metabolic Syndrome
A Clinical Trial to Evaluate the Blood Pressure Control of Telmisartan or Losartan in Essential Hypertensive Patients With Metabolic Syndrome
This is a multi-center, randomized, open-label, active comparator-controlled, phase 4 clinical trial to evaluate the blood pressure control of Telmisartan or Losartan in essential hypertensive patients with metabolic syndrome
- Trial name
- PRISTINE (NCT05843162)
- Testing
- Telmisartan, Losartan
- Volunteers
- 116 people
- Sponsor
- Chong Kun Dang Pharmaceutical
Genetic Disorder, Prader-Willi Syndrome
Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome
This study is conducted in Europe. The aim of this observational study is to collect data from children with Prader-Willi Syndrome, who have been treated off-label with Norditropin® for more than 12 months to seek approval for…
- Trial name
- NCT00705172
- Testing
- somatropin
- Where
- 3 sites in Denmark, Germany and 1 more
- Volunteers
- 41 people
- Sponsor
- Novo Nordisk A/S
Iron Deficiency Anemia
A Study to Evaluate the Safety and Preliminary Efficacy of a Response-guided Dose Titration of KER-047 in the Treatment of Functional IDA (Iron Deficiency Anemia).
This study aims to explore the safety and preliminary efficacy of a response-guided dose titration of KER-047 in the treatment of functional IDA (Iron deficiency anemia) in MDS (Myelodysplastic syndrome), MF(Myelofibrosis), and…
- Trial name
- NCT05927012
- Testing
- KER-047
- Where
- 5 sites in Australia, Israel
- Sponsor
- Keros Therapeutics, Inc.
Patients With Metabolic Syndrome
A Randomized, Double-Blind Trial Comparing the Efficacy and Safety of Fenofibrate, Metformin, Their Combination and Placebo in Patients With Metabolic Syndrome.
The purpose of this study was to study the effect of different combinations of fenofibrate and metformin on the cluster of metabolic syndrome (MetS) biochemical abnormalities, and to determine the dose combination allowing…
- Trial name
- NCT00703755
- Testing
- Fenofibrate /Metformin
- Where
- 101 sites in Canada, Finland and 7 more
- Volunteers
- 2,288 people
- Sponsor
- Solvay Pharmaceuticals
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Neutropenia
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are…
- Trial name
- NCT06056297
- Testing
- Mavorixafor
- Where
- 114 sites in United States, Argentina and 23 more
- Volunteers
- 176 people
- Sponsor
- X4 Pharmaceuticals
Congenital Adrenal Hyperplasia
Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasia
This study is a parallel arm, randomised, open-label study, including dose titration and admissions for four overnight stays for 24-hour endocrine profiles.
- Trial name
- NCT02716818
- Testing
- Chronocort®, standard glucocorticoid therapy
- Where
- United States
- Volunteers
- 122 people
- Sponsor
- Neurocrine UK Limited
Celiac Disease
A Double-blind Placebo-controlled Study to Evaluate Larazotide Acetate for the Treatment of Celiac Disease
A randomized, double-blind, placebo-controlled Phase 2B study to evaluate the efficacy and safety of Larazotide Acetate in the treatment of patients with celiac disease who have persistent symptoms despite being on a gluten-free…
- Trial name
- NCT01396213
- Testing
- Larazotide Acetate
- Where
- 62 sites in United States, Canada
- Volunteers
- 342 people
- Sponsor
- 9 Meters Biopharma, Inc.
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics