Clinical trials: Other Metabolic Diseases
- Condition Other Metabolic Diseases
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Hurler Syndrome, Inherited Metabolic Disorder
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy
- Trial name
- NCT03639844
- Testing
- rivogenlecleucel, rimiducid
- Where
- 2 sites in United States
- Sponsor
- Bellicum Pharmaceuticals
Diabetes, Diabetes Mellitus, Type 2
A Trial Investigating the Efficacy and Safety of Insulin Detemir Versus Insulin NPH in Combination With Metformin and Diet/Exercise in Children and Adolescents With Type 2 Diabetes Insufficiently Controlled on Metformin With or Without Other Oral Antidiabetic Drug(s) With or Without Basal Insulin
This trial is conducted globally. The aim of the trial is to investigate the efficacy and safety of insulin detemir versus insulin Neutral Protamine Hagedorn (NPH) in combination with the maximum tolerated dose of metformin and…
- Trial name
- iDEAt2 (NCT02131272)
- Testing
- Insulin detemir, Insulin NPH, Diet/exercise
- Where
- 82 sites in United States, Argentina and 22 more
- Volunteers
- 42 people
- Sponsor
- Novo Nordisk A/S
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Type 2 Diabetes
Characteristics of Empagliflozin Initiators
Empagliflozin, a sodium glucose cotransporter 2 (SGLT-2) inhibitor, was launched in the United Kingdom (UK) in August 2014. It can be expected that patients initiating empagliflozin may differ in their characteristics from…
- Trial name
- NCT03050619
- Testing
- Empagliflozin, Other SGLT2 inhibitors, Other non-insulin GLDs
- Where
- Germany
- Volunteers
- 31,908 people
- Sponsor
- Boehringer Ingelheim
Iron Overload, Sickle Cell Disease
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron…
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- NCT02443545
- Testing
- Deferiprone
- Where
- 13 sites in United States, Canada and 3 more
- Volunteers
- 134 people
- Sponsor
- ApoPharma
Type 2 Diabetes (Treated With Exenatide or Other Oral Antidiabetic Therapies), Healthy Subjects (Treated With no Diabetes Therapies)
A Retrospective Cohort Study of Acute Pancreatitis in Relation to Use of Exenatide and Other Antidiabetic Agents
The purpose of this research was to assess the absolute and relative incidence of acute pancreatitis in persons initiating exenatide compared with persons initiating a different antidiabetic agent, and secondarily, persons…
- Trial name
- NCT01077323
- Testing
- exenatide, Other antidiabetic therapies, No diabetes therapy
- Where
- United States
- Volunteers
- 363,766 people
- Sponsor
- AstraZeneca
Type 2 Diabetes
Characteristics of Patients With Type 2 Diabetes Mellitus (CVD-RNDR)
This observational retrospective study will describe characteristics of patients with type 2 diabetes mellitus (T2DM) who were initiating use or treatment with sodium-glucose co-transporter-2 (SGLT-2) inhibitors and other…
- Trial name
- NCT03694470
- Where
- Russia
- Volunteers
- 10,000 people
- Sponsor
- AstraZeneca
Schizophrenia
Study of Aripiprazole in Patients With Schizophrenia- Effects on Glucose Metabolism-
The objective of this study is to examine the effects of aripiprazole on glucose metabolism in schizophrenic patients without hyperglycemia and diabetes mellitus or any history thereof.
- Trial name
- NCT00392197
- Testing
- Aripiprazole
- Where
- 5 sites in Japan
- Volunteers
- 111 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Hypercholesterolemia, Familial Hypercholesterolemia
A 12-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05244)
This study is a non-interventional (observational) study in Japan to confirm the safety and efficacy of Zetia when administered alone or in combination with other lipid-lowering drugs in daily medical practice throughout a…
- Conditions
- Hypercholesterolemia, Familial Hypercholesterolemia, Homozygous Sitosterolemia
- Trial name
- NCT00704444
- Testing
- Ezetimibe, Ezetimibe + other lipid-lowering medication(s)
- Volunteers
- 11,332 people
- Sponsor
- Organon and Co
Diabetes Mellitus Type 2
Comparative Effectiveness of Cardiovascular Outcomes in New Users of SGLT-2 Inhibitors
CVD-REAL is a multinational, observational cohort study in patients with type 2 diabetes mellitus evaluating the comparative effectiveness of initiating treatment with a sodium-glucose co-transporter-2 (SGLT-2) inhibitor versus…
- Trial name
- CVD-REAL (NCT02993614)
- Where
- 17 sites in United States, Australia and 15 more
- Volunteers
- 99,999 people
- Sponsor
- AstraZeneca
Diabetes, Diabetes Mellitus, Type 1
Observational Study on Levemir® in Obese Diabetic Patients
This study is conducted in Europe. The aim of this observational study is to investigate the effect of switch from other basal insulin treatments to Levemir® on body weight in obese diabetic patients.
- Conditions
- Diabetes, Diabetes Mellitus, Type 1, Diabetes Mellitus, Type 2
- Trial name
- KILOS (NCT00849342)
- Testing
- insulin detemir
- Where
- 2 sites in Netherlands, Slovenia
- Volunteers
- 580 people
- Sponsor
- Novo Nordisk A/S
MPS IV A
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
- Trial name
- NCT01275066
- Testing
- BMN 110 Weekly, BMN 110 Every Other Week
- Where
- 28 sites in United States, Argentina and 15 more
- Volunteers
- 177 people
- Sponsor
- BioMarin Pharmaceutical
Diabetes, Diabetes Mellitus, Type 2
Observational Study on the Effect of NovoMix® 30, Levemir® or NovoRapid® (Alone or Combined) in Type 2 Diabetics Previously Treated With Anti-diabetic Medication
This study is conducted in Africa, Asia, South America and Europe. The aim of this observational study is to document the experience with the study insulins when used in routine clinical practice.
- Trial name
- A1chieve® (NCT00869908)
- Testing
- insulin aspart, insulin detemir, biphasic insulin aspart
- Where
- 22 sites in Algeria, Argentina and 20 more
- Volunteers
- 66,726 people
- Sponsor
- Novo Nordisk A/S
Anemia, Hemosiderosis
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older.
- Trial name
- NCT00303329
- Testing
- Deferasirox
- Where
- 28 sites in United States, Belgium and 5 more
- Volunteers
- 184 people
- Sponsor
- Novartis Pharmaceuticals
Methylmalonic Acidemia
An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
The primary objective of this study is to evaluate the long-term safety and clinical activity of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT)…
- Trial name
- NCT05295433
- Testing
- mRNA-3705
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 56 people
- Sponsor
- ModernaTX, Inc.
Weight Gain
Study to Examine the Effect of Betahistine on Body Weight Gain Due to Olanzapine Treatment
This is a randomized, double-blind, placebo-controlled, multicenter, multinational study. Approximately 78 subjects (39 per treatment group) will be randomized into this 16 week study.
- Trial name
- NCT00428168
- Testing
- Betahistine
- Where
- 10 sites in Canada, Israel
- Volunteers
- 36 people
- Sponsor
- OBEcure Ltd.
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Schizophrenia, Bipolar Disorder
Physician Survey on Monitoring of Patients Treated With Quetiapine
A physician survey to document receipt of metabolic educational materials and assess behavior of physicians in following messages communicated through the educational materials
- Conditions
- Schizophrenia, Bipolar Disorder, Major Depressive Disorder
- Trial name
- NCT01795326
- Volunteers
- 800 people
- Sponsor
- AstraZeneca
Iron Overload, Sickle Cell Disease
Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body.
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Trial name
- FIRST (NCT02041299)
- Testing
- Deferiprone, Deferoxamine
- Where
- 33 sites in United States, Brazil and 6 more
- Volunteers
- 230 people
- Sponsor
- ApoPharma
Gaucher Disease
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1.
- Trial name
- NCT04094181
- Where
- Canada
- Volunteers
- 2 people
- Sponsor
- Shire