Clinical trials: Syndrome, Metabolic
Cytomegalovirus Retinitis, HIV Infections
A Study of Valganciclovir in the Treatment of Cytomegalovirus (CMV) Retinitis in Patients With AIDS
The purpose of this study is to see if valganciclovir is a safe treatment for CMV retinitis in patients who have been treated for this condition in the past.
- Trial name
- NCT00002222
- Testing
- Valganciclovir
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Hoffmann-La Roche
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.
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
This will be a double blind, randomised, placebo controlled, single and multiple oral dose study conducted in 3 parts: Part A, Part B and Part C.
- Conditions
- Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies, MELAS Syndrome, Mitochondrial Myopathies
- Trial name
- NCT03888716
- Testing
- KL1333
- Where
- 2 sites in United Kingdom
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Abliva AB
Hypertension, Metabolic Syndrome X
Phase IV, 9 Weeks Comparison Between MICARDIS 80 mg and Amlodipine 10 mg on Biological PPAR Gamma Activities
The objective of the study is to compare PPAR activities (increase of adiponectin level) between MICARDIS and amlodipine after 6 weeks of treatment in hypertensive patients with metabolic syndrome.
- Trial name
- NCT00242814
- Testing
- Micardis, Amlodipine, Abdominal biopsy
- Where
- 31 sites in France
- Volunteers
- 100 people
- Sponsor
- Boehringer Ingelheim
COVID-19
Mapping Organ Health Following COVID-19 Disease Due to SARS-CoV-2 Infection
A prospective, longitudinal, observational cohort study looking at patients following COVID-19 disease using multi-parametric magnetic resonance imaging (MRI) to assess the degree and prevalence of organ injury.
- Trial name
- COVERSCAN (NCT04369807)
- Testing
- Outpatient MRI
- Where
- 2 sites in United Kingdom
- Volunteers
- 693 people
- Sponsor
- Perspectum
Bile Acid Synthesis Disorders
The REPLACE Registry for Cholbam® (Cholic Acid)
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using…
- Trial name
- NCT03115086
- Testing
- Cholbam
- Where
- 24 sites in United States
- Volunteers
- 55 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
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
Obesity Hypoventilation Syndrome
Obesity Hypoventilation Syndrome Prevalence Study
This is an observational study to characterize the prevalence of Obesity Hypoventilation Syndrome in patients referred to the sleep lab.
- Trial name
- NCT01964339
- Testing
- BMI greater than or equal to 30kg/m2 scheduled for PSG. Undergo blood draw (venous blood gas and metabolic panel) and data collection from PSG study., BMI greater than or equal to 30kg/m2 scheduled for PSG. Undergo blood draw (arterial blood gas and metabolic panel) and data collection from PSG study.
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Philips Respironics
HIV Infections
Pharmacokinetics and Metabolism of 14 Carbon [14C]-GSK3640254
This is an open-label, single-center, single group, non-randomized, two-period, single sequence, mass balance study which will enroll 6 healthy male participants.
- Trial name
- NCT04507321
- Testing
- GSK3640254 Oral tablet, [14C]-GSK3640254 intravenous infusion, [14C]-GSK3640254 powder
- Where
- United Kingdom
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Prader-Willi Syndrome, Hyperphagia
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).
- Trial name
- ZEPHYR (NCT03790865)
- Testing
- Livoletide
- Where
- 37 sites in United States, Australia and 6 more
- Volunteers
- 158 people
- Sponsor
- Millendo Therapeutics SAS
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03811028
- Testing
- SOBI003
- Where
- 3 sites in United States, Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
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
CAH - Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a…
- Trial name
- NCT04045145
- Testing
- Crinecerfont
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences
Celiac Disease
Safety and Tolerability Study of Larazotide Acetate in Celiac Disease Subjects
This study was run to determine the safety, tolerance, and efficacy of multiple doses of larazotide acetate in subjects with celiac disease following a gluten challenge.
- Trial name
- NCT00362856
- Testing
- larazotide acetate
- Where
- 9 sites in United States
- Volunteers
- 80 people
- Sponsor
- 9 Meters Biopharma, Inc.
Overweight, Obesity
Individual Factors Related to Chronic Low-grade Inflammation and Cardiometabolic Disease Risk
The goal of this observational study is to learn about low-grade inflammation in healthy individuals and individuals with overweight or obesity.
- Conditions
- Overweight, Obesity, Metabolic Syndrome, Healthy
- Trial name
- PINEAPPL (NCT06355544)
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Integrative Phenomics
Leigh Syndrome
ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
A phase 2a, open-label study to evaluate the safety, tolerability, and clinical activity of ABI-009 (nab-sirolimus) in patients with genetically-confirmed Leigh or Leigh-like syndrome
- Trial name
- NCT03747328
- Testing
- ABI-009
- Sponsor
- Aadi Bioscience, Inc.
Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency
The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome
An open-label study of volanesorsen (ISIS 304801) administered subcutaneously to participants with FCS.
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Trial name
- NCT02658175
- Testing
- Volanesorsen
- Where
- 34 sites in United States, Brazil and 9 more
- Volunteers
- 68 people
- Sponsor
- Akcea Therapeutics
Cytomegalovirus Retinitis, HIV Infections
A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS
To compare the time to progression of CMV retinitis between oral ganciclovir and IV ganciclovir during 20 weeks of maintenance treatment.
- Trial name
- NCT00002257
- Testing
- Ganciclovir
- Where
- 13 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Hoffmann-La Roche
Celiac Disease, Coeliac Disease
A Study of Efficacy, Safety, and Tolerability of KAN-101 in People With Celiac Disease
The study goal is to evaluate the efficacy, safety, and tolerability of KAN-101 in participants with Celiac Disease (CeD)
- Trial name
- SynCeD (NCT06001177)
- Testing
- KAN-101
- Where
- 28 sites in United States, Canada and 6 more
- Volunteers
- 55 people
- Sponsor
- Kanyos Bio, Inc., a wholly-owned subsidiary of Anokion SA