Systematically Testing the Evidence on Marijuana

The overall median survival of nonresectable malignant hilar obstruction in most series has been less than 6 months. Most patients with malignant hilar obstruction present with advanced disease, making allative endoscopic drainage the principal therapeutic option. However, the optimal endoscopic management strategy is contentious. In malignant hilar obstruction, exclusively endoscopic placement of bilateral metal stents has been considered very difficult and complex, and it may require multiple procedures, with an increased risk of complications and mortality. To overcome this difficulty and strategize plan of management, the investigators evaluated the technical and clinical efficacy of endoscopic bilateral placement of newly designed stents, Y-configuration, followed by side-by-side insertion in failure of stent-in-stent method for the management of malignant hilar obstruction.

First Posted: June 10, 2010

Condition(s): Malignant Hilar Stricture, Bilateral Stent Insertion

Intervention(s): Bilateral stent-in-stent insertion, Bilateral side-by-side insertion

Status: Recruiting

Enrollment (expected or actual): 80

Allocation: Randomized

Sponsor: Soon Chun Hyang University

Principal Investigator: Tae Hoon Lee, MD, PhD

Completion Date (primary or actual): March 30, 2018

CT.gov Identifier: NCT01141088

The purpose of this study is to determine the relative bioavailability (rBA; Period 1) and bioequivalence (BE; Period 2 and 3) of various strengths and formulations of niraparib and abiraterone acetate (AA) at steady state under modified fasted conditions in participants with metastatic castration-resistant prostate cancer (mCRPC).

First Posted: October 8, 2020

Condition(s): Prostatic Neoplasms

Intervention(s): Niraparib, Abiraterone Acetate (AA), Prednisone

Status: Active, not recruiting

Enrollment (expected or actual): 136

Allocation: Randomized

Sponsor: Janssen Research & Development, LLC

Principal Investigator:

Completion Date (primary or actual): October 15, 2021

CT.gov Identifier: NCT04577833

Dental fluorosis is a clinical manifestation of chronic exposure to high intakes of fluoride during the tooth development. The present study is planned to evaluate the efficacy of Resin Infiltration technique in improving the esthetics of non-pitted fluorosis stains on permanent anterior teeth. Resin infiltration is a novel method that has shown its efficacy in improving the esthetics of various types of white spot lesions (WSLs), particularly post orthodontics WSLs, in in-situ and in-vivo studies. The Resin Infiltration technique is also known to stop the progression of caries in non cavitated lesions in both, smooth surface and interproximal lesions. Therefore, the following trial will be conducted to assess the esthetic improvement of fluorosed permanent anterior teeth after resin infiltration and compare it with standard bleaching procedure and a combination of bleaching and Resin infiltration therapy in children with fluorosed teeth.

First Posted: November 27, 2012

Condition(s): Caries Infiltration, Fluorosis, Dental Care

Intervention(s): Office Bleaching, Resin Infiltration, Resin Infiltration twice, Bleaching + Resin Infiltration

Status: Completed

Enrollment (expected or actual): 80

Allocation: Randomized

Sponsor: DMG Dental Material Gesellschaft mbH

Principal Investigator:

Completion Date (primary or actual): December 2013

CT.gov Identifier: NCT01733888

This is a randomized, parallel group, multicentre, multinational, prospective, open-label exploratory study in Parkinson's disease (PD) patients to evaluate the add-on efficacy of opicapone 50 mg or an extra dose of levodopa (L-DOPA) 100 mg as first strategy for the treatment of wearing-off.

First Posted: August 4, 2021

Condition(s): Parkinson Disease

Intervention(s): Opicapone, L-DOPA/DDCI

Status: Active, not recruiting

Enrollment (expected or actual): 106

Allocation: Randomized

Sponsor: Bial - Portela C S.A.

Principal Investigator:

Completion Date (primary or actual): June 30, 2023

CT.gov Identifier: NCT04990284

Interactions between genes and environment, are likely to be crucial in the development of the common diseases such as type 2 diabetes. Recently, the investigators have obtained data that genotypes of genes encoding for fatty acid desaturases 1 and 2 (FADS1 and FADS2) are the strongest genes in a genome-wide analysis regulating serum fatty acid profile.The aim of this study is to test if subjects with different genotypes of the FADS2 gene respond differently to a diet supplemented with linoleic acid or alpha-linolenic acid (substrates for FADS2). The study hypothesizes that subjects will be more sensitive to the dietary modifications according to their genotype leading to more robust differences in serum FA profile, tissue inflammation and serum lipids.

First Posted: June 28, 2018

Condition(s): Fatty Acids, Genetics

Intervention(s): LA, ALA

Status: Completed

Enrollment (expected or actual): 140

Allocation: Randomized

Sponsor: University of Eastern Finland

Principal Investigator:

Completion Date (primary or actual): December 19, 2018

CT.gov Identifier: NCT03572205

Background: - Researchers have been studying patterns of mood and drug use in specific neighborhoods. This study will look at environmental factors that may affect drug use, addiction, and treatment seeking in Baltimore neighborhoods. The results could inform prevention efforts, enhance treatment interventions, and improve substance use outcomes. Objectives: - To better understand why some people start to use drugs, why some people who use drugs become addicted, and why some people who become addicted enter treatment. Eligibility: - Individuals at least 18 years of age who are living in the neighborhoods participating in the study. Design: Participants will be screened with a physical exam and medical history. They will be separated into one of four groups: (1) people who do not use drugs, (2) people who have used drugs in the past, (3) people who are using drugs and want treatment, and (4) people who are using drugs and do not want treatment. This study will include two outpatient visits about 12 months apart. Each visit will last about 5 hours. Each study visit may be done in 1 day or in 2 days. At each study visit, participants will provide blood, breath, urine, and saliva samples. They will also have a heart function test and body measurements. They will complete questionnaires about personal and family history. There will be monthly follow-up phone calls between the two visits.

First Posted: April 5, 2012

Condition(s): Stigma, Activity Space, Social Networks, HIV Status, Drug Abuse/Dependence

Intervention(s):

Status: Completed

Enrollment (expected or actual): 1651

Allocation:

Sponsor: National Institute on Drug Abuse (NIDA)

Principal Investigator:

Completion Date (primary or actual):

CT.gov Identifier: NCT01571752

This is an investigator-initiated trial. In this 6-month open label, single arm, exploratory pilot study, the investigator intends to enroll a pilot sample of 25 patients with Parkinson's disease who are being started on the novel FDA approved medication "Istradefylline'' as an add on therapy for management of their motor symptoms (based on the judgment of the movement disorders specialist, if patients are in sub-optimal management of their symptoms on maximum tolerable dose of their dopaminergic medications). The investigator will evaluate their motor symptoms before starting the new medication, including their MDS-UPDRS total and MDS-UPDRS-III scores.

First Posted: June 1, 2023

Condition(s): Parkinson Disease, Tremor

Intervention(s): Istradefylline Pill

Status: Recruiting

Enrollment (expected or actual): 25

Allocation: N/A

Sponsor: Georgetown University

Principal Investigator:

Completion Date (primary or actual): September 2023

CT.gov Identifier: NCT05885360

This is a Phase IV, prospective, observational, post-marketing study designed to obtain additional data on the effect of XADAGO on motor and non-motor symptoms in Parkinson's Disease patients newly prescribed XADAGO.

First Posted: May 10, 2019

Condition(s): Idiopathic Parkinson Disease

Intervention(s): XADAGO (safinamide)

Status: Completed

Enrollment (expected or actual): 164

Allocation:

Sponsor: Supernus Pharmaceuticals, Inc.

Principal Investigator:

Completion Date (primary or actual): December 23, 2019

CT.gov Identifier: NCT03944785

The purpose of the study is to investigate these effects of Second-Generation Antipsychotic (SGAs) on glucose and lipid metabolic parameters in patients with schizophrenia, and explore the relationship between genes polymorphisms (such as drug metabolic enzyme, Endogenous Cannabinoid Receptor Type 1(CB1) and so on) and the SGAs-induced glucose and lipid metabolic disorder in Chinese Han persons with schizophrenia who are taking one of the SGAs(olanzapine, risperidone or ziprasidone).

First Posted: November 16, 2016

Condition(s): Schizophrenia

Intervention(s):

Status: Active, not recruiting

Enrollment (expected or actual): 300

Allocation:

Sponsor: Xijing Hospital

Principal Investigator:

Completion Date (primary or actual): June 2017

CT.gov Identifier: NCT02964923

The hypothesis is that the differential extent of microstructural damages in the affected brain regions can be specific to the disease of interest and could reflect the clinical severity. Therefore, we propose that the whole brain parcellation of diffusion MRI can be used to improve the diagnosis and prediction of clinical outcomes in Parkinson's Disease. A regression model between clinical severity and two-year clinical outcomes and diffusion properties from multiple parcellated regions will be developed. Blind validation will be performed.

First Posted: July 15, 2020

Condition(s): Parkinson Disease

Intervention(s):

Status: Completed

Enrollment (expected or actual): 288

Allocation:

Sponsor: Chang Gung Memorial Hospital

Principal Investigator: Wang . Jiun-Jie, Study Principal Investigator

Completion Date (primary or actual): August 1, 2018

CT.gov Identifier: NCT04472325

The goal of this trial is to evaluate the safety and tolerability of lithium in people with progressive supranuclear palsy or corticobasal degeneration.

First Posted: June 23, 2008

Condition(s): Progressive Supranuclear Palsy, Corticobasal Degeneration

Intervention(s): Lithium

Status: Completed

Enrollment (expected or actual): 17

Allocation: Non-Randomized

Sponsor: Westat

Principal Investigator:

Completion Date (primary or actual): January 2010

CT.gov Identifier: NCT00703677

The study will investigate the biomarkers of Aβ and Tau seeds in plasma detected by Alzheimer's disease (AD) related seeds quantitative detector (AD-seeds-detector), and their sensitivity and specificity in diagnosing AD, compared with those from age-matched cognitively normal controls, and those with other types of dementia. To perform a high throughput analysis of the amount of Aβ and Tau seeds, the investigators have developed an AD-seeds-detector, in which a fluorescence microplate reader was combined with an oscillating mixer or water-bath-type ultrasonicator.

First Posted: April 20, 2021

Condition(s): Alzheimer's Disease

Intervention(s):

Status: Recruiting

Enrollment (expected or actual): 1500

Allocation:

Sponsor: Capital Medical University

Principal Investigator: Jianping Jia, Chief Director

Completion Date (primary or actual): December 31, 2023

CT.gov Identifier: NCT04850053

ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) represents the formalized integration of ARTFL (U54 NS092089; funded through 2019) and LEFFTDS (U01 AG045390; funded through 2019) as a single North American research consortium to study FTLD for 2019 and beyond.

First Posted: April 27, 2020

Condition(s): Frontotemporal Lobar Degeneration (FTLD), Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Behavioral Variant Frontotemporal Dementia (bvFTD), Semantic Variant Primary Progressive Aphasia (svPPA), Nonfluent Variant Primary Progressive Aphasia (nfvPPA), FTD With Amyotrophic Lateral Sclerosis (FTD/ALS), Amyotrophic Lateral Sclerosis, Oligosymptomatic PSP (oPSP), C9orf72, GRN Related Frontotemporal Dementia, MAPT Gene Mutation, TBK1 Gene Mutation, Oligosymptomatic Progressive Supranuclear Palsy

Intervention(s):

Status: Recruiting

Enrollment (expected or actual): 2100

Allocation:

Sponsor: Mayo Clinic

Principal Investigator: Bradley Boeve, Principal Investigator

Completion Date (primary or actual): July 2024

CT.gov Identifier: NCT04363684

This project aims to develop a minimally invasive microneedle sensor to monitor levodopa levels in real time. We will test the accuracy, tolerability, and safety of this device in people with Parkinson disease.

First Posted: February 3, 2021

Condition(s): Parkinson Disease

Intervention(s): Levodopameter

Status: Recruiting

Enrollment (expected or actual): 20

Allocation: N/A

Sponsor: University of California, San Diego

Principal Investigator: Irene Litvan, Director of the UCSD Parkinson and Other Movement Disorders Center

Completion Date (primary or actual): December 2027

CT.gov Identifier: NCT04735627

This is an analytical validation observational cohort study is designed to provide evidence of: safety and reliability of Body Surface Gastric Mapping using the Gastric Alimetry System (GAS), normal reference values, and correlation of metrics with patient symptoms among healthy adults and patients diagnosed with upper abdominal motility disorders. GAS is intended to record, store, view and process gastric myoelectrical activity. This is a proprietary system consisting of multiple electrodes arranged on an array that is placed precisely over the stomach, a reader to collect the electrode measurements and a smart tablet application to track patient reported symptoms. Participants meeting inclusion and exclusion criteria will continue fasting for 30 minutes after the Gastric Alimetry System has been applied and begun measuring, eat a standard study meal within 10 minutes and remain quietly seated, reclining, for 4 hours as the GAS continues to collect data. The array is removed and the abdomen is examined for evidence of skin effects.

First Posted: April 13, 2023

Condition(s): Motility Disorder, Gastroparesis, Functional Dyspepsia, Cyclical Vomiting, Cannabinoid Hyperemesis Syndrome, Diabetic Gastroparesis

Intervention(s): Gastric Alimetry System

Status: Enrolling by invitation

Enrollment (expected or actual): 200

Allocation:

Sponsor: University of Calgary

Principal Investigator:

Completion Date (primary or actual): July 31, 2024

CT.gov Identifier: NCT05812339

Parkinson's disease (PD) is a neurological condition, which affects the brain. PD gets worse over time, but how quickly it progresses varies a lot from person to person. Some symptoms of PD are tremors, stiffness, and slowness of movement. This study will assess how safe and effective ABBV-951 is in adult participants with PD. Adverse events and change in disease activity is evaluated. ABBV-951 is an investigational (unapproved) drug containing Levodopa Phosphate/Carbidopa Phosphate (LDP/CDP) given as an infusion under the skin for the treatment of Parkinson's Disease. Adult participants with advanced PD and who have completed M15-736 or M20-339 study will be enrolled. Approximately 130 participants will be enrolled in the study in approximately 60 sites in the United States and Australia. Participants will receive continuous subcutaneous infusion (CSCI) (under the skin) of ABBV-951 for 96 weeks during the Primary Treatment Period and during the optional Extended Treatment Period. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical and remote telephone assessments, blood tests, checking for side effects, and completing questionnaires.

First Posted: February 11, 2021

Condition(s): Parkinson's Disease (PD)

Intervention(s): ABBV-951

Status: Active, not recruiting

Enrollment (expected or actual): 118

Allocation: N/A

Sponsor: AbbVie

Principal Investigator:

Completion Date (primary or actual): May 16, 2025

CT.gov Identifier: NCT04750226

This is a double-blind, randomized, placebo-controlled single-center clinical trial to explore the safety and efficacy of a full cannabis flower formulation, rich in cannabinoids and terpenes formulation, Xltran Plus™ and Xltran™, both compared to placebo for the treatment of Long COVID patients with prolonged symptoms caused by COVID-19.

First Posted: July 21, 2022

Condition(s): Post-acute COVID-19 Syndrome

Intervention(s): Xltranplus, Xltran

Status: Not yet recruiting

Enrollment (expected or actual): 111

Allocation: Randomized

Sponsor: LUCINDA BATEMAN, MD

Principal Investigator: LUCINDA BATEMAN, MD, Medical Director

Completion Date (primary or actual): July 2023

CT.gov Identifier: NCT05467904

The purpose of this study is to investigate whether speed-dependent measures of gait can be identified in patients with neurological conditions that affect gait, particularly in subjects with parkinsonian disorders.

First Posted: December 16, 2016

Condition(s): Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Corticobasal Degeneration, Gait, Frontal, Huntington Disease

Intervention(s): Anti-Parkinson medication, Deep Brain Stimulation

Status: Active, not recruiting

Enrollment (expected or actual): 120

Allocation:

Sponsor: Beth Israel Deaconess Medical Center

Principal Investigator: Veronique Vanderhorst, Associate Professor of Neurology

Completion Date (primary or actual): June 20, 2024

CT.gov Identifier: NCT02994719

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

First Posted: January 18, 2023

Condition(s): Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young, Cystic Fibrosis, Hypophosphatasia, Infantile, Congenital Hypothyroidism, DAVID, Pituitary Hormone Deficiency, Combined, Diamond Blackfan Anemia, Wiskott-Aldrich Syndrome, Fanconi Anemia, Hemophilia A, Hemophilia B, Glucose 6 Phosphate Dehydrogenase Deficiency, Alpha-Thalassemia, Sickle Cell Disease, Shwachman-Diamond Syndrome, Alpha 1-Antitrypsin Deficiency, Inflammatory Bowel Disease 25, Autosomal Recessive, Wilson Disease, Progressive Familial Intrahepatic Cholestasis, Crigler-Najjar Syndrome, DIAR4, Familial Chylomicronemia, Lysosomal Acid Lipase Deficiency, Familial Hemophagocytic Lymphocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Severe Congenital Neutropenia, SCID, Chronic Granulomatous Disease, Menkes Disease, X-ALD, Smith-Lemli-Opitz Syndrome, Ataxia With Vitamin E Deficiency, THMD5, THMD4, Thiamine-Responsive Megaloblastic Anemia, Thiamine Metabolism Dysfunction Syndrome 2, GOT2 DEFICIENCY, Cerebral Folate Transport Deficiency, Segawa Syndrome, Autosomal Recessive, Congenital Myasthenic Syndrome, Metachromatic Leukodystrophy, Sepiapterin Reductase Deficiency, Dopamine Beta Hydroxylase Deficiency, Glut1 Deficiency Syndrome, Late-Infantile Neuronal Ceroid Lipofuscinosis, Aromatic L-amino Acid Decarboxylase Deficiency, Charcot-Marie-Tooth Disease, Type 6C, Hereditary Hyperekplexia, Brain Dopamine-Serotonin Vesicular Transport Disease, Very Long Chain Hydroxy Acyl Dehydrogenase Deficiency, Tyrosinemia, Type I, Disaccharide Intolerance I, Beta Ketothiolase Deficiency, Phosphoglycerate Dehydrogenase Deficiency, Succinyl-Coa 3-Oxoacid Transferase Deficiency, Pyridoxine-5'-Phosphate Oxidase Deficiency, Pyridoxine-Dependent Epilepsy, Propionic Acidemia, Pompe Disease, Phenylalanine Hydroxylase Deficiency, Ornithine Transcarbamylase Deficiency, N Acetyl Glutamate Synthethase Deficiency, Riboflavin Deficiency, Maple Syrup Urine Disease, Medium Chain Acyl CoA Dehydrogenase Deficiency, Malonic Acidemia, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Isovaleric Acidemia, Phosphoserine Aminotransferase Deficiency, Phosphoserine Phosphatase Deficiency, Spatccm, Hyperornithinemia-Hyperammonemia-Homocitrullinuria, MRT8, FORMERLY, S-Adenosylhomocysteine Hydrolase Deficiency, Mucopolysaccharidosis VII, Mucopolysaccharidosis VI, Mucopolysaccharidosis IV A, Mucopolysaccharidosis II, Mucopolysaccharidosis I, Transcobalamin Deficiency, Isolated Methylmalonic Acidemia, Cobalamin Deficiency, Homocystinuria, Holocarboxylase Synthetase Deficiency, Fanconi Bickel Syndrome, Glycogen Storage Disease, Glycine Encephalopathy, Glutaric Acidemia I, Glucose Galactose Malabsorption, Gaucher Disease, Type 1, Galactosemias, Fructosemia, Fructose-1,6-Diphosphatase Deficiency, Carbamoyl Phosphate Synthase 1 Deficiency, Citrullinemia Type II, Citrullinemia 1, Creatine Deficiency Syndrome, Systemic Primary Carnitine Deficiency, Carnitine Palmitoyltransferase Deficiency 2, Carnitine Palmitoyltransferase Deficiency 1, Carnitine Acylcarnitine Translocase Deficiency, Riboflavin Transporter Deficiency, Branched-Chain Keto Acid Dehydrogenase Kinase Deficiency, Andersen Tawil Syndrome, Timothy Syndrome, Jervell-Lange Nielsen Syndrome, Catecholaminergic Polymorphic Ventricular Tachycardia, Familial Hypertrophic Cardiomyopathy Type 4, Pseudohypoaldosteronism, Type II, Pseudohypoaldosteronism Type 1, Primary Hyperoxaluria, X Linked Hypophosphatemia, Hereditary Nephrogenic Diabetes Insipidus, Cystinosis, Congenital Nephrotic Syndrome, Finnish Type, Alport Syndrome, Hereditary Retinoblastoma, Biotinidase Deficiency, Aciduria, Argininosuccinic, Arginemia, ACAD9 Deficiency, 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency

Intervention(s):

Status: Recruiting

Enrollment (expected or actual): 40000

Allocation:

Sponsor: Laurent Servais

Principal Investigator: Laurent Servais, Professor

Completion Date (primary or actual): August 31, 2023

CT.gov Identifier: NCT05687474

From previous parabolic flight campaigns completed by our research group investigators gathered data showing important changes in neuro-endocrine and also immunological changes. This experiment now will complete the data set-up obtained during the MARS500 study on how these respective individuals will re-act to highly physically and emotionally challenging situations during a parabolic flight. This add-on experiment to MARS500 will enable answering to the following questions i) if the stress response systems - when compared for two conditions but in the same individuals - do react uniformly and ii) if those subjects prone to have adequate stress response pattern will show gradually less immune modulation effects. The understanding of the complex interactions of stress and immunity under chronic and acute stress conditions might help to enable adequate health and immune monitoring and might as well suggest suitable countermeasures for the prevention of the unwanted immunological effects during long-term confinement and space missions.

First Posted: August 6, 2015

Condition(s): Healthy

Intervention(s): Parabolic flight, neuro-endocrine measurements, immunological measurements, cellular energy metabolism measurements

Status: Completed

Enrollment (expected or actual): 6

Allocation: N/A

Sponsor: University Hospital, Caen

Principal Investigator:

Completion Date (primary or actual): January 2014

CT.gov Identifier: NCT02517190

The hypothesis is that the differential extent of microstructural damages in the affected brain regions can be specific to the disease of interest and could reflect the clinical severity. Therefore, the investigator propose that whole brain parcellation of diffusion MRI can be used to improve diagnosis and prediction of clinical outcomes in Parkinson's Disease. A regression model between clinical severity and two year clinical outcomes and diffusion properties from multiple parcellated regions will be developed. Blind validation will be performed.

First Posted: June 17, 2020

Condition(s): Alzheimer Disease, Parkinson Disease

Intervention(s):

Status: Completed

Enrollment (expected or actual): 212

Allocation:

Sponsor: Chang Gung Memorial Hospital

Principal Investigator: Wang . Jiun-Jie, Study Principal Investigator

Completion Date (primary or actual): October 1, 2018

CT.gov Identifier: NCT04434898

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.

First Posted: May 1, 2018

Condition(s): Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy

Intervention(s): Thiotepa--single daily dose, Thiotepa--escalated dose

Status: Active, not recruiting

Enrollment (expected or actual): 6

Allocation: Randomized

Sponsor: University of Florida

Principal Investigator:

Completion Date (primary or actual): February 17, 2022

CT.gov Identifier: NCT03513328

This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.

First Posted: August 7, 2013

Condition(s): Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease

Intervention(s): Abatacept

Status: Completed

Enrollment (expected or actual): 10

Allocation: N/A

Sponsor: Emory University

Principal Investigator: John Horan, Associate Professor

Completion Date (primary or actual): September 19, 2019

CT.gov Identifier: NCT01917708

This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient?s immune cells and help destroy any remaining cancer cells.

First Posted: November 7, 2017

Condition(s): Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome

Intervention(s): Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation

Status: Active, not recruiting

Enrollment (expected or actual): 31

Allocation: N/A

Sponsor: Roswell Park Cancer Institute

Principal Investigator:

Completion Date (primary or actual): August 28, 2022

CT.gov Identifier: NCT03333486

Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked lymphoproliferative disease, and metabolic diseases affecting hematopoiesis) are sublethal diseases caused by mutations that adversely affect the development or function of different types of blood cells. Although pathophysiologically diverse, these genetic diseases share a similar clinical course of significant progressive morbidity, overall poor quality of life, and ultimate death from complications of the disease or its palliative treatment. Supportive care for these diseases includes chronic transfusion, iron chelation, and surgery (splenectomy or cholecystectomy) for the hemoglobinopathies; prophylactic antibiotics, intravenous immunoglobulin, and immunomodulator therapies for the immune deficiencies; and enzyme replacement injections and dietary restriction for some of the metabolic diseases. The suboptimal results of such supportive care measures have led to efforts to implement more aggressive therapeutic interventions to cure these lymphohematopoietic diseases. The most logical strategies for cure of these diseases have been either replacement of the patient's own hematopoietic stem cells (HSC) with those derived from a normal donor allogeneic bone marrow transplant (BMT) or hematopoietic stem cell transplant (HSCT), or to genetically modify the patient's own stem cells to replace the defective gene (gene therapy).

First Posted: July 31, 2015

Condition(s): Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation, Genetic Diseases, Thalassemia, Pediatrics, Diamond-Blackfan Anemia, Combined Immune Deficiency, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Lymphoproliferative Disease, Metabolic Diseases

Intervention(s): Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3, Cyclophosphamide Dose Level 4

Status: Terminated

Enrollment (expected or actual): 20

Allocation: Non-Randomized

Sponsor: Children's Hospital Los Angeles

Principal Investigator: Neena Kapoor, M.D., Principal Investigator

Completion Date (primary or actual): September 2013

CT.gov Identifier: NCT02512679