Clinical trials: Syndrome X
Rett Syndrome, RTT
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.
- Trial name
- ARCH (NCT03848832)
- Testing
- GWP42003-P
- Where
- 26 sites in United States, Italy and 2 more
- Volunteers
- 29 people
- Sponsor
- Jazz Pharmaceuticals
Metabolic Syndrome X
Effect of Dapagliflozin Administration on Metabolic Syndrome, Insulin Sensitivity, and Insulin Secretion
The Metabolic Syndrome is a high prevalence disease worldwide. About a quarter of the adult population suffers the disease. Dapagliflozin is an inhibitor of the sodium-glucose co-transporter SGLT2 in the kidney and is a novel…
- Trial name
- NCT02113241
- Testing
- Dapagliflozin
- Where
- Mexico
- Volunteers
- 24 people
- Sponsor
- University of Guadalajara
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
Obesity, Metabolic Syndrome X
Drug Therapy Induced Weight Loss to Improve Blood Vessel Function in Subjects With Obesity
Obesity is common (>30% of US adults), contributes to substantial morbidity and mortality, but is difficult to treat. Partly this is due to the transient, arduous and modest nature of lifestyle interventions.
- Trial name
- REVIVE (NCT01351753)
- Testing
- Metformin, Orlistat, Topiramate
- Where
- United States
- Volunteers
- 128 people
- Sponsor
- University of Iowa
Rett Syndrome
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
- Trial name
- NCT04181723
- Testing
- Trofinetide
- Where
- 21 sites in United States
- Volunteers
- 187 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Exercise Therapy, Metabolic Syndrome X
Exercise Training Effects on Metabolic Syndrome: Interactions With Medication
To analyze the effects of different exercise training modalities (continuous, intervallic, and resistance training) on cardiorespiratory and metabolic fitness of metabolic syndrome patients when this training interacts with their…
- Conditions
- Exercise Therapy, Metabolic Syndrome X, Cardiorespiratory Fitness
- Trial name
- METSYND (NCT03019796)
- Testing
- MEDICATION AND EXERCISE TRAINING
- Where
- Spain
- Volunteers
- 40 people
- Sponsor
- University of Castilla-La Mancha
NLRC4-MAS, XIAP Deficiency
Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency
This is a Phase 3 study to assess the safety and efficacy of Tadekinig alfa in patients with monogenic, interleukin-18 (IL 18) driven autoinflammation due to Nucleotide-binding oligomerization domain, leucine-rich repeat and…
- Trial name
- NCT03113760
- Testing
- Tadekinig alfa, 0.9% sodium chloride
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- AB2 Bio Ltd.
Microvascular Angina
Microvascular Assessment of Ranolazine in Non-Obstructive Atherosclerosis (MARINA)
The purpose of this study is to look at the effects of the drug Ranolazine compared to Placebo on symptoms of chest pain or chest tightness (known as angina), exercise endurance and ability, and changes in blood flow to the very…
- Trial name
- MARINA (NCT02147067)
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Emory University
Fragile x Syndrome
Ganaxolone Treatment in Children With Fragile X Syndrome
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS).
- Trial name
- NCT01725152
- Testing
- Ganaxolone
- Where
- 2 sites in United States, Belgium
- Volunteers
- 59 people
- Sponsor
- Marinus Pharmaceuticals
Metabolic Syndrome X
Diurnal Variation of Plasminogen Activator Inhibitor-1
To determine if nighttime administration of an aldosterone antagonist would effectively lower peak plasma Plasminogen Activator Inhibitor-1 (PAI-1) levels more effectively than morning administration.
- Trial name
- NCT00515021
- Testing
- Eplerenone (Morning), Eplerenone (Night-time)
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Vanderbilt University Medical Center
Wiskott-Aldrich Syndrome (WAS)
Gene Therapy for Wiskott-Aldrich Syndrome
This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.
- Trial name
- TIGET-WAS (NCT01515462)
- Testing
- TLT003
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Fondazione Telethon
Fragile X Syndrome
Trial of Minocycline to Treat Children With Fragile X Syndrome
This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant.
- Trial name
- NCT01053156
- Testing
- minocycline hydrochloride
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- University of California, Davis
Schizophrenia, Metabolic Syndrome X
An Observational Study On Metabolic Syndrome Parameters In Schizophrenia Patients Treated With Atypical Antipsychotics
The purpose of the study is to find and follow-up the metabolic syndrome parameters on patients administering atypical antipsychotics.
- Trial name
- MESSAGE (NCT00448630)
- Testing
- No intervention
- Where
- 47 sites in Turkey (Türkiye)
- Volunteers
- 328 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire
Fragile X Tremor/Ataxia Syndrome
Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
The objective of this study is to determine if citocoline is effective for balance abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor ataxia syndrome.
- Trial name
- NCT02197104
- Testing
- citocoline
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Rush University Medical Center
Metabolic Syndrome X, Insulin Resistance
Oral Contraceptives in the Metabolic Syndrome
Oral contraceptives (OCs) are the most widely used method of reversible birth control. However, the long-term cardiovascular safety of the widely used low-dose OCs (ethinyl-estradiol < 50 mcg) is still debated.
- Conditions
- Metabolic Syndrome X, Insulin Resistance, Obesity, Cardiovascular Diseases
- Trial name
- NCT00205504
- Testing
- Ortho Tri Cyclen
- Where
- United States
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Virginia Commonwealth University
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Fragile X Syndrome
Riluzole in Fragile X Syndrome
The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.
- Trial name
- NCT00895752
- Testing
- Riluzole
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Indiana University
Leukemia, Hodgkin Lymphoma
Study of Stem Cell Transplantation for Hematologic Malignancies Using Clofarabine and Busulfan Regimen
The goals of the study are (Phase I) to determine the appropriate dose for Clofarabine with Busulfan as a full-intensity conditioning (Clo/BU4 regimen) prior to transplant and then (Phase II) to investigate the safety and…
- Conditions
- Leukemia, Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma
- Trial name
- NCT00556452
- Testing
- Clofarabine/Busulfan x 4, Peripheral blood stem cell transplant, Total Lymphoid Irradiation
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- University of Michigan Rogel Cancer Center
Microvascular Angina
Study to Evaluate Effect of Nebivolol on Angina in Women With Microvascular Disease
Women have less significant blockages of coronary arteries, however have greater symptoms and worse outcomes compared to their age-matched male counterparts.
- Trial name
- NIRVANA (NCT01665508)
- Testing
- Nebivolol
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Massachusetts General Hospital