Clinical trials: Syndrome X
Dyskeratosis Congenita, Aplastic Anemia
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant…
- Trial name
- NCT00455312
- Testing
- Campath 1H, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
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
Metabolic Syndrome X, Overweight
Atheroma Reduction With Chloroquine in Patients With the Metabolic Syndrome (ARCH-MS)
Metabolic syndrome consists of a group of co-occuring conditions that increase an individual's risk of developing heart disease, stroke, and diabetes.
- Conditions
- Metabolic Syndrome X, Overweight, Hypertension, Dyslipidemias
- Trial name
- ARCH-MS (NCT00455403)
- Testing
- Chloroquine
- Where
- United States
- Volunteers
- 357 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
Schizophrenia, Metabolic Syndrome X
Ziprasidone for Improving Insulin Sensitivity in People With Schizophrenia Who Are at Risk for Diabetes
This study will evaluate the effectiveness of ziprasidone treatment versus treatment with a standard atypical antipsychotic drug in improving insulin sensitivity and reducing excess abdominal fat storage in people with…
- Conditions
- Schizophrenia, Metabolic Syndrome X, Insulin Resistance
- Trial name
- NCT00338949
- Testing
- Switch, Control
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- Veterans Medical Research Foundation
Rett Syndrome
Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
The investigators are recruiting children for a research study using a medication known as IGF-1 (mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT).
- Trial name
- NCT01253317
- Testing
- rhIGF-1
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Boston Children's Hospital
Dry Eye Syndrome
Study to Assess the Safety and Efficacy of BOL-303242-X Ophthalmic Suspension in Dry Eye Syndrome
The objective of this study is to identify the concentration and daily dosing frequency of BOL-303242-X ophthalmic suspension in treating dry eye syndrome over a 12 week dosing period.
- Trial name
- NCT01163643
- Testing
- 0.3% BOL-303242-X ophthalmic suspension, 2% BOL-303242-X ophthalmic suspension, 1% BOL-303242-X ophthalmic suspension
- Where
- United States
- Volunteers
- 356 people
- Sponsor
- Bausch & Lomb Incorporated
Small Fiber Neuropathy, Fabry Disease
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Sudoscan™ (Impeto Medical, Paris France) uses electrochemical skin conductance as a novel noninvasive method to detect sudomotor dysfunction.
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Trial name
- NCT02985710
- Testing
- Sudoscan, Skin biopsy, QSART
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Primary Immune Deficiency Disease
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
- Trial name
- NCT00634569
- Testing
- Flebogamma 5% DIF
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Instituto Grifols, S.A.
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.
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
This study is to discover whether children with severe combined immunodeficiency disease (SCID) or other primary immunodeficiency disorder (PID) for which no satisfactory treatment other than stem cell transplantation (SCT)…
- Conditions
- Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
- Trial name
- MASCI (NCT00579137)
- Testing
- Campath -1H, Fludarabine, Anti-CD45
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Lesch-Nyhan Disease
Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam…
- Trial name
- NCT01065558
- Testing
- Ecopipam
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Psyadon Pharma
Fragile X Syndrome (FXS)
Decisional Capacity and Informed Consent in Fragile X Syndrome
The Decisional Capacity and Informed Consent in Fragile X Syndrome (FXS) project is for the Eunice Kennedy Shriver National Institute of Child Health \& Human Development, 1R01HD071987-01A1, and will provide the first…
- Trial name
- NCT02465931
- Testing
- Paper and Pencil Informed Consent, Digital Informed Consent Tool
- Where
- United States
- Volunteers
- 152 people
- Sponsor
- RTI International
Primary Immunodeficiency, Common Variable Hypogammaglobulinemia
Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
The main objective of this study is to see if GAMMAPLEX is efficacious with respect to Food and Drug Administration (FDA) minimal requirements (no more than 1 serious, acute, bacterial infection per subject per year) in subjects…
- Conditions
- Primary Immunodeficiency, Common Variable Hypogammaglobulinemia, X-linked Hypogammaglobulinemia, Hypogammaglobulinemia
- Trial name
- NCT00278954
- Testing
- Gammaplex (Intravenous immunoglobulin)
- Where
- 7 sites in United States
- Volunteers
- 50 people
- Sponsor
- Bio Products Laboratory
Rett Syndrome
Open-Label Extension Study of Trofinetide for Rett Syndrome
To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome
- Trial name
- NCT04776746
- Testing
- trofinetide
- Where
- 21 sites in United States
- Volunteers
- 77 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
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
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
HIV-Associated Lipodystrophy Syndrome, Insulin Resistance
Growth Hormone and/or Rosiglitazone for HIV-Associated Increased Abdominal Fat and Insulin Resistance
The purpose of the study is to determine if the combination of recombinant human growth hormone plus rosiglitazone (an insulin-sensitizing drug) is safe and more effective than either drug alone (or no active therapy) for the…
- Conditions
- HIV-Associated Lipodystrophy Syndrome, Insulin Resistance, HIV Infections, Metabolic Syndrome X
- Trial name
- NCT00130286
- Testing
- Rosiglitazone, Recombinant human growth hormone + rosiglitazone
- Where
- 4 sites in United States
- Volunteers
- 77 people
- Sponsor
- Weill Medical College of Cornell University
Fragile X Syndrome, Autism Spectrum Disorders
Acamprosate in Youth With Fragile X Syndrome
Fragile X syndrome (FXS) is the most common inherited form of developmental disability. FXS is inherited from the carrier parent, most often the mothers.
- Trial name
- NCT01300923
- Testing
- Acamprosate
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Indiana University