Clinical trials: Metabolism Changes
- Condition Metabolism Changes
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Diabetes
Self-Monitoring of Blood Glucose Study#1: The Ability of People With Diabetes to Use OneTouch(R) Ultrasmart(tm)
Insulin therapy requires monitoring and frequent review of glucose levels to optimize dosing. Effective presentation and data analysis are essential.
- Trial name
- NCT00333853
- Testing
- OneTouch(r) UltraSmart(r)
- Where
- 7 sites in United States
- Volunteers
- 250 people
- Sponsor
- LifeScan
Diabetes, Diabetes Mellitus, Type 2
Observational Study of Type 2 Diabetes Patients Failing on Oral Anti-diabetic Agents Initiated on Levemir® or Insulatard®
This trial is conducted in Europe. The aim of this observational study is to evaluate the change in weight in type 2 diabetes patients using Levemir® or Insulatard® under normal clinical practice conditions.
- Trial name
- EVIDENCE (NCT00658099)
- Testing
- insulin detemir, insulin NPH
- Where
- Romania
- Volunteers
- 699 people
- Sponsor
- Novo Nordisk A/S
Chronic Adrenal Insufficiency
A European Post-Authorisation Observational Study (Registry) of Patients With Chronic Adrenal Insufficiency (AI)
As a post-approval requirement of the European Medicines Agency, this European patient post authorization safety study is an observational study being conducted to monitor the safety of long-term treatment with Plenadren and…
- Trial name
- NCT01661387
- Where
- 24 sites in Germany, Italy and 3 more
- Volunteers
- 3,258 people
- Sponsor
- Shire
Gaucher Disease, Type 1, MRI
Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
This study with standardized reading MRIs, will provide for the first time objective and quantified data on organomegaly (liver and spleen volumes) as well as bone alteration (Bone Marrow Burden11) of French patients treated with…
- Trial name
- EIROS (NCT03333447)
- Testing
- MRI
- Where
- France
- Volunteers
- 20 people
- Sponsor
- CEN Biotech
Obesity, Weight Gain
A Study of Tirzepatide in Adolescents With Obesity and Weight-Related Comorbidities (SURMOUNT-ADOLESCENTS-2)
The goal of the study is to assess how tirzepatide impacts bodyweight and cardiovascular risk factors when used in conjunction with healthy nutrition and physical activity in adolescents with obesity and multiple weight related…
- Trial name
- NCT06439277
- Testing
- Tirzepatide
- Where
- 69 sites in United States, Argentina and 10 more
- Volunteers
- 300 people
- Sponsor
- Eli Lilly and Company
Fabry Disease
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201…
- Trial name
- NCT04999059
- Testing
- Safety and Efficacy Assessments
- Where
- 3 sites in Australia, Brazil
- Volunteers
- 5 people
- Sponsor
- AVROBIO
Obesity
Study to Evaluate the Efficacy and Safety of Belviq XR® in Conjunction With Lifestyle Modification for Weight Loss in Obese Adolescents, Age 12 to 17 Years
This study will be conducted to demonstrate weight loss efficacy by change in body mass index (BMI) and safety in adolescents age 12 to 17 years (inclusive) during 52 weeks of treatment with Belviq XR 20 milligrams (mg)…
- Trial name
- NCT03338296
- Testing
- lorcaserin hydrochloride XR
- Where
- 16 sites in United States
- Volunteers
- 278 people, healthy volunteers welcome
- Sponsor
- Eisai Inc.
Hunter Syndrome
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth…
- Trial name
- NCT02455622
- Testing
- Elaprase for intravenous (IV) infusion
- Where
- 8 sites in United States, Dominican Republic and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Mucopolysaccharidosis Type II, Metabolic Diseases
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
- Trial name
- NCT05208281
- Testing
- GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg
- Where
- 5 sites in Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Dehydration
Transdermal Optical Imaging (TOI) as a Non-invasive Measure to Detect Changes in Hydration Status in Healthy Adults and Athletes
The primary objective of this study is to analyze smart phone-based Transdermal Optical Imaging (TOI) features to develop a model that can discern hydration status, including: 1) at various timepoints throughout a 24-hour period…
- Trial name
- NCT06212518
- Testing
- GENPOP ad libitum fluid intake, GENPOP fluid restriction, ATHLETE ad libitum fluid intake
- Where
- 4 sites in United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- PepsiCo Global R&D
Post Menopausal Osteoporosis
A Study of Ibandronate (Boniva) to Evaluate Bone Turnover Markers in Women With Treatment-Naive Postmenopausal Osteoporosis
This study will evaluate whether an early positive response to once-monthly oral ibandronate in treatment-naive participants with postmenopausal osteoporosis is predictive of efficacy later in treatment.
- Trial name
- NCT02598934
- Testing
- Ibandronate
- Where
- 53 sites in United States, Puerto Rico
- Volunteers
- 308 people
- Sponsor
- Hoffmann-La Roche
Adolescent Obesity, Obesity in Adolescence
A Phase IV Safety and Efficacy Study of VI-0521 in Adolescents With Obesity
This study is being conducted to assess weight loss efficacy, as determined by changes in body mass index (BMI), and safety of VI-0521 (Qsymia®) or placebo, taken for 56 weeks accompanied by a lifestyle modification program in…
- Conditions
- Adolescent Obesity, Obesity in Adolescence, Adolescent Overweight
- Trial name
- NCT03922945
- Testing
- VI-0521 oral capsule, Lifestyle Modification
- Where
- United States
- Volunteers
- 223 people
- Sponsor
- VIVUS LLC
Inflammatory Bowel Diseases, Irritable Bowel Syndrome
Fecal Calprotectin Collection Protocol
The primary objective is to obtain stool samples from subjects diagnosed with , and displaying signs and/or symptoms of IBD and/or IBS will be evaluated in this study.
- Conditions
- Inflammatory Bowel Diseases, Irritable Bowel Syndrome, Ulcerative Colitis, Crohn Disease
- Trial name
- CALFE (NCT03143517)
- Testing
- Stool collection
- Where
- 13 sites in United States
- Volunteers
- 240 people
- Sponsor
- DiaSorin Inc.
Type 2 Diabetes Mellitus
Effects of Exenatide Long-Acting Release on Glucose Control and Safety in Subjects With Type 2 Diabetes Mellitus(DURATION - 1)
A Randomized, Open-Label, Multicenter, Comparator-Controlled Study to Examine the Effects of Exenatide Long-Acting Release (LAR) on Glucose Control (HbA1c) and Safety in Subjects with Type 2 Diabetes Mellitus Managed with Diet…
- Trial name
- NCT00308139
- Testing
- exenatide, long acting release, exenatide
- Where
- 25 sites in United States, Canada
- Volunteers
- 303 people
- Sponsor
- AstraZeneca
Diabetes, Diabetes Mellitus, Type 1
Observational Study of the Predictability of Levemir® in Terms of Metabolic Control, Change of Body Weight and Hypos
This trial is conducted in Europe. The aim of this trial is to evaluate the effect of Levemir® (insulin detemir) on glycaemic control, weight and incidence of hypoglycaemic events in insulin treated subjects with type 1 or type 2…
- Conditions
- Diabetes, Diabetes Mellitus, Type 1, Diabetes Mellitus, Type 2
- Trial name
- NCT01542489
- Testing
- insulin detemir, insulin aspart, human soluble insulin
- Where
- Slovakia
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Mucopolysaccharidosis IH
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of…
- Trial name
- TigetT10_MPSIH (NCT03488394)
- Testing
- Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Orchard Therapeutics
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.