Correlation Between Haptoglobin Phenotypes and Infectious and Other Complications in Cystic Fibrosis Patients (Hp-in-CF)Mar 2013
Cystic Fibrosis is a genetic disease with variable severity, and a predisposition for lung infection. Usually severity is determined by the class of CF mutations, but even among patients with the same severity of mutations there is a variation of the severity of CF. Haptoglobin has several types (phenotypes), one of them was found to be related to infectious complications. In this study the investigators aim to find a correlation between Haptoglobin phenotypes in patients with CF and frequency of infectious complications. To this end the investigators will collect serum from CF patients, and determine their Haptoglobin protein phenotype. The investigators will correlate Haptoglobin phenotype to retrospectively gathered data on infectious complications.
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)Feb 2013
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease.
Congenital Transmission of Lineages I and II of Trypanosoma CruziFeb 2013
T. cruzi has been divided into two main lineages: T. cruzi I (TcI) and T. cruzi II (TcII, including all non-TcI). TcI is predominant in Mexico and Central America, while TcII (non-TcI) is predominant in most of South America, including Argentina. In recent studies from Argentina, the risk of congenital transmission has been estimated to vary between 2.6 percent and 7.9 percent. By contrast, we know very little about the congenital transmission of TcI. It has been suggested that congenital transmission of T. cruzi is strain related, and there is an urgent need to know if TcI transmits differently than TcII (non-TcI). Our primary hypothesis is that congenital transmission rates are different for TcI versus TcII. Our secondary hypothesis is that the characteristics of T. cruzi infected mothers (e.g., age, parity, transmission in previous pregnancies) and their exposure to vectors are different in regions where TcI is predominant versus regions where TcII (non-TcI) is predominant. To test these hypotheses, we propose to conduct a prospective study to enroll at delivery 13,000 women in Mexico, 7,500 women in Honduras, and 10,000 women in Argentina. We will measure transmitted maternal T. cruzi antibodies in cord blood, and, if the results are positive, we will identify infants who are congenitally infected by performing parasitological examinations on cord blood and at 4-8 weeks, and serological follow-up at 10 months. We will also perform standard PCR, real-time quantitative PCR, and T. cruzi genotyping on maternal blood, standard PCR and T. cruzi genotyping on the cord blood of congenitally infected newborns, and serological examinations on siblings. We will estimate the exposure to vectors in the household. In addition, we will measure prenatal outcomes among infected and uninfected infants with seropositive mothers, and the birth weight of their siblings. The specific aims of this study are: 1) To determine the rate of congenital transmission of TcI compared to TcII (non-TcI); 2) To compare the T. cruzi infected mothers' characteristics and exposure to vectors in regions where TcI is predominant and regions where TcII (non-TcI) is predominant; and 3) To describe the birth outcomes of infected and uninfected infants born to TcI and TcII seropositive women.
Bone Microarchitecture in Young Cystic Fibrosis PatientsFeb 2013
The aim of this study is to evaluate bone microarchitecture of paediatric CF patients matched to sex-age-pubertal status-healthy volonteers. In the meantime, biological markers will be collected and DXA (Dual-energy x-ray absorptiometry) will be performed in order to explore potential correlations HR-pQCT parameters.
Eficacy of Long-term Suplementation With Docosahexaenoic Acid in Patients With Cystic FibrosisJan 2013
Cystic Fibrosis (CF) is a congenital disease secondary to the abnormal function of CFTR. Patients with CF have an alteration of essential fatty acids, Arachidonic Acid (AA) is increased and Docosahexanoic Acid (DHA) is decrease and the ratio ω-6/ ω-3 is elevated, all these alterations stimulated a chronic and bad regulated state of inflammation. For this porpoise, a fase IV trial, multicentric, controlled, double blind, placebo and parallel in patients elder than two months old and randomized to received every day a dietetically supplement with DHA or placebo, will be done during 12 months. The trial has as a principal objective to proved if this long term supplementation could decrease in contrast with placebo.
Ext. Long-term Safety Study in CF Patients: Single Arm TIPJan 2013
The purpose of this extension study is to collect additional 48 weeks of safety data from patients taking TIP who have completed the core study CTBM100C2401. The purpose of collecting second year safety data through this study is to obtain long-term (2 years) safety data of TIP.
Normalization of dyrk1A and APP Function as an Approach to Improve Cognitive Performance and Decelerate AD Progression in DS Subjects: Epigallocatechin Gallate as Therapeutic ToolSep 2012
Epigallocatechin-3-gallate (EGCG), the major catechin in green tea, is postulated to modulate dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) and amyloid beta precursor protein (APP) gene overexpression in the brains of Down syndrome mouse models. The clinical study is aimed at demonstrating that normalization of Dyrk1A and APP functions is a therapeutic approach to improve cognitive performance and decelerate AD (Alzheimer's disease) like progression.
Azole Therapy in Cystic Fibrosis (ATCF) : Efficacy of itraconazole and of voriconazole in patients with cystic fibrosis and presenting with persistent positive sputums for AspergillusAug 2012
We wish to ascertain the effectiveness of the two possible treatments for Aspergillus infection in CF (voriconazole and itraconazole) and compare these to each other. Effectiveness will be assessed by the effect on growth of Aspergillus in the sputum, which we will also confirm with more sensitive techniques based on detecting Aspergillus DNA.
A Molecular and Functional Brain Imaging Study in Individuals With Down Syndrome and Healthy Controls Following Single Dose RG1662Aug 2012
This single-center, double-blind, placebo-controlled, parallel-group study with crossover component will evaluate the GABAAalpha5 receptor expression, occupancy and functional connectivity in the brains of individuals with Down syndrome and healthy controls following single dose RG1662. On two separate visits, no less than 96 hours and no more than 6 weeks apart, subjects will receive a single oral dose of either RG1662 or placebo followed by positron emission tomography (PET) and magnetic resonance imaging (MRI) scans.
A Phase II, Open-Label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults using FBS0701 (SSP-004184 ).Aug 2012
Measure the extent and durability of FBS0701 treatment effects; continue to assess the safety, tolerability and efficacy of FBS0701 (SSP-004184) in subjects who received FBS0701(SSP-004184) in prior Ferrokin sponsored study; allow access and assess response to FBS0701 (SSP-004184) in subjects randomised to another chelating therapy in a prior FerroKin-sponsored FBS0701(SSP-004184) study.
A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation TherapyAug 2012
Evaluate, in patients with transfusion-dependent iron overload, whose primary diagnosis is hereditary or congenital anemia: - Pharmacokinetic parameters provided a constant (steady state) of two doses of FBS0701; - Changes in the concentrations of iron in the liver, after 12 and 24 weeks following administration of two doses of FBS0701; - The safety and tolerability of two doses of FBS0701, administered daily for 24 weeks.
A randomized placebo-controlled study to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics of single and multiple ascending doses of QBW251 in healthy subjects and cystic fibrosis patients.May 2012
To assess the safety and tolerability of single and and multiple doses of QBW251 in healthy subjects and cystic fibrosis patients To evaluate the pharmacodynamic response to multiple doses of QBW251 in cystic fibrosis patients as reflected in changes in lung function (forced expiratory volume in one second, FEV1 - Part4 only)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis who Have the R117H-CFTR MutationApr 2012
To evaluate the efficacy of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CF transmembrane conductance regulator (CFTR) mutation
A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long-Term Ivacaftor Treatment in Subjects 6 Years of Age and Older with Cystic Fibrosis and a Non-G551D CFTR MutationApr 2012
To evaluate the safety of long-term ivacaftor treatment in subjects with cystic fibrosis (CF)
A Phase 2, multicenter, double-blinded, placebo-controlled, 3-part study to evaluate safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of VX 661 monotherapy and VX 661/VX 770 cotherapy in subjects with cystic fibrosis (CF), homozygous for the F508del-cystic fibrosis transmembrane conductance regulator gene (CFTR) mutationApr 2012
-To evaluate the safety and tolerability of VX 661 monotherapy and VX 661/VX 770 cotherapy -To evaluate the effect of VX 661 monotherapy and VX 661/VX 770 cotherapy on CFTR function
A Non-Drug Study of The Suitability of Neurocognitive Tests And Functioning Scales For The Measurement of Cognitive And Functioning Changes in Individuals With Down SyndromeMar 2012
This non-drug, longitudinal, multi-center, multi-national study will evaluate the suitability of neurocognitive tests and functioning scales for the measurement of cognitive and functioning changes in individuals with Down Syndrome. Tests will be administered at clinic visits in Weeks 1, (4) and 24. The duration of the study for each individual will be between 24 and 27 weeks.
Insulin therapy in non-diabetic adults with cystic fibrosisFeb 2012
This study aims to investigate the effect of low dose insulin therapy on nutritional status en body weight.
Open-Label Phase 3 Trial to Evaluate the Safety of Aztreonam 75 mg Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Children with Cystic Fibrosis (CF) and Chronic Pseudomonas aeruginosa (PA) in the Lower AirwaysDec 2011
The objective of this study is to evaluate safety of treatment with AZLI 75 mg 3 times daily (TID) for 3 courses of therapy (28 days on/28 days off) in female and male children less than 13 years of age with CF and chronic PA infection/colonization.
Prospective randomized, placebo-controlled, double blind, multicenter study (phase III) to evaluate clinical efficacy and safety of avian polyclonal anti-Pseudomonas antibodies (IgY) in prevention of recurrence of Pseudomonas aeruginosa infection in cystic fibrosis patientsDec 2011
The primary objective is to find out, if continuous long-term local application of specific egg yolk antibodies (IgY) directed against PA - initiated after successfully treated acute PA infection - can prolong the time to recurrence of a sputum culture positive for PA. The objective to prevent infections with PA is also to diminish the need of antibiotics and minimize the problem of bacterial resistance against antibiotics.
A Randomised Controlled Double-Blind Trial of Intranasal Fentanyl versus Intravenous Morphine in the Emergency Department Treatment of Severe Painful Sickle Cell Crises in ChildrenDec 2011
The primary objective of this study is to illustrate the comparative efficacy of intranasal fentanyl to IV morphine in reducing severe pain associated with Painful Sickle Cell Crises in the Emergency Department.
Relating Clinical Outcomes in Multiple Myeloma to Personal Assessment of Genetic Profile (CoMMpass)Oct 2011
The primary objective of this observational study is to identify the molecular profiles and clinical characteristics that define subsets of myeloma patients at initial diagnosis and at relapse of disease.
Exocrine Pancreatic Function Testing in Cystic FibrosisOct 2011
The purpose of this study is to develop and validate multimodal testing of exocrine pancreatic function. The investigators will be testing exocrine pancreatic function in patients with cystic fibrosis. Exocrine pancreatic function and imaging will be correlated to age group, genotype, nutritional status and quality of life. Earlier detection of exocrine pancreatic failure in the non classical form of CF may be of therapeutically benefit.
Optimizing Antidepressant Treatment by Genotype-dependent Adjustment of Medication according to the the ABCB1 GeneAug 2011
To evaluate the ABCB1-genotype dependent efficacy of a quick dose-escalation strategy within 21 days of treatment with approved antidepressants that are known substrates of the P-glycoprotein, an efflux pump of the blood-brain barrier expressed by ABCB1
Effect of Glycine in Cystic FibrosisAug 2011
The aim of this study is to evaluate if glycine, orally administered in a daily dose of 0.5 g/kg during 8 weeks, can ameliorate the airway inflammation in children with cystic fibrosis, as compared with placebo. During all of the study children will receive their usual treatment for cystic fibrosis.
Towards Onset Prevention of COGnitive decline in adults with Down syndrome (the TOP-COG study)Aug 2011
The aims of the study are to gather the data needed to design a full-scale multi-centred RCT of oral simvastatin 40mg a day.
Costimulatory Molecules as Biomarkers in Cystic FibrosisMay 2011
The purpose of this study is to investigate the expression of a certain class of molecules, called costimulatory molecules, in humans with Cystic Fibrosis. Cystic Fibrosis is a genetic disorder which renders the lung susceptible to persistent inflammation which, at times, can worsen, resulting in accelerated decline in lung function and eventually death or transplant. Our goal is to determine if the levels of costimulatory markers can be used to predict exacerbation and subsequent lung function decline in subjects with Cystic Fibrosis.
Genomic Testing for Primary Breast CancerApr 2011
The goal of this research study is to find out if researchers can use genetic testing on tumor samples to predict if tumors will respond to breast cancer treatments. The tumor sample will be tested to learn if certain genes are activated (turned on) in the tumor. Researchers hope that the activation of these genes may predict if the tumor will be sensitive or resistant to routine breast cancer treatments, such as chemotherapy or hormonal therapy.
Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood DisordersMar 2011
To evaluate the safety and effectiveness of topical sodium nitrite cream as a treatment for chronic leg ulcers in individuals with sickle cell disease or other red blood cell disorders.
Iron Mediated Vascular Disease in Sickle Cell Anemia PatientsNov 2010
The purpose of this research study is to determine the frequency and severity of iron overload in patients with Sickle Cell Anemia and its relationship to blood vessel function.
A Phase 2, Multicenter, Double Blinded, Placebo Controlled, Multiple Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of VX-809 Alone and in Combination with VX-770 in Subjects with Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR MutationOct 2010
To evaluate the safety and tolerability when VX-809 is administered alone and when VX-809 is coadministered with VX-770. To evaluate the effect of VX-809 administered alone and VX-809 coadministered with VX-770 on sweat chloride.
A 5-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) Patients (FISCO)Oct 2010
A long term observational study in sickle cell disease will enhance the understanding of the disease patterns, current transfusion practices, treatments and outcomes in sickle cell disease.
Genetic Contributions to Autism Spectrum DisordersJul 2010
This study is working towards gaining a better understanding of the genetic and environmental factors involved in autism spectrum disorders (ASD), which includes autism, pervasive developmental disorder (PDD), and Asperger's syndrome. The investigators hope that information gained from this study will lead to new ways of diagnosing and treating ASDs.
A Phase III, Multicentre, Randomised, Placebo-Controlled, Double Blind Study of the Incidence of Recurring Pulmonary Exacerbations in Cystic Fibrosis Patients using Two Different Doses of Inhaled Nacystelyn®May 2010
To determine the long-term safety and efficacy of inhaled Nacystelyn®, in the presence or absence of rhDNase, in patients with CF as reflected in a change per treatment group in the time to first pulmonary exacerbation (PE) during the study period.
A Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemiaMay 2010
The purpose of this research is to test the effects of L-glutamine on red blood cells of patients with Sickle Cell Anemia or Sickle ß0-Thalassemia.
Quantification de la régression de l'albuminurie et de l'atteinte endothéliale dans une population de patients drépanocytaires homozygotes hyperfiltrants traités par inhibiteurs du système rénine-angiotensine - Etude " RAND "May 2010
Le but premier de cette étude est de quantifier dans la néphropathie drépanocytaire (au stade d'une hyperfiltration glomérulaire associée à une micro/macroalbuminurie) l'effet de l'inhibition du système rénine angiotensine aldostérone sur la micro/macroalbuminurie.
An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX 770 in Subjects with Cystic FibrosisApr 2010
To evaluate the safety of long-term VX-770 treatment in subjects with cystic fibrosis (CF)
Trial of Doxycycline to Reduce Sputum MMP-9 Activity in Adult Cystic Fibrosis (CF) Patients (DOXY)Apr 2010
The purpose of this study is to examine the role of a well-known and well-tolerated antibiotic, doxycycline, in the treatment of cystic fibrosis patients who are hospitalized. This antibiotic does not effectively treat the bacteria in airways of cystic fibrosis patients, but may reduce the activity of inflammatory molecules in the disease.
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell DiseaseOct 2009
The purpose of this research study is to determine the effectiveness and safety of vorinostat when used to treat SCD.
Genetically determined brain abnormalities in Down’s Syndrome.- towards a treatment: A randomised, single-blind, placebo-controlled trial of lithium carbonate in Down’s Syndrome (DownsLit).Jul 2009
Our primary objective is to determine if brain myo-inositol concentration is significantly reduced in non-demented DS individuals by brief 4 week treatment with lithium carbonate at normal therapeutic doses with full monitoring of potential side effects in all participants.
A Phase 3, 2 Part, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX 770 in Subjects Aged 6 to 11 Years with Cystic Fibrosis and the G551D MutationJul 2009
Part A: To evaluate the pharmacokinetics of a single dose of orally administered VX 770 treatment in subjects 6 to 11 years of age with cystic fibrosis (CF) who have the G551D cystic fibrosis transmembrane conductance regulator (CFTR) mutation on at least 1 allele. Part B: To evaluate the efficacy of VX 770 after 24 weeks of treatment in subjects 6 to 11 years of age with CF who have the G551D CFTR mutation on at least 1 allele.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX 770 in Subjects with Cystic Fibrosis and the G551D MutationJun 2009
To evaluate the efficacy of VX 770 after 24 weeks of treatment in subjects with cystic fibrosis (CF) who have the G551D cystic fibrosis transmembrane conductance regulator (CFTR) mutation on at least 1 allele.
Study of the Molecular Genetics of Menstrual MigraineMay 2009
The aim of the study is to identify genes, genetic regions, and the effect of hormones on expression profiles in women with menstrual migraine compared to a control population with no personal or first-degree family history of migraine.
Tissue Sample Collection From Patients With Fanconi AnemiaMay 2009
This laboratory study is collecting and storing tumor tissue samples from patients with Fanconi anemia.
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis - A Safety and Efficacy StudyMar 2009
To determine whether inhaled mannitol compared to control improves FEV1 in patients with Cystic Fibrosis.
Genetic Susceptibility to Bladder CancerFeb 2009
This clinical research study will identify biologic and lifestyle factors which increase a person's risk of developing specific cancer. We propose to conduct a case-control study examining interindividual differences in susceptibility to tobacco carcinogenesis as predictors of bladder cancer risk. We will measure susceptibility to tobacco carcinogenesis and this will include studies of the genetic modulation of carcinogen activation and detoxification and of chromosome sensitivity to tobacco mutagens.
Exercise-Induced Bronchospasm in Cystic FibrosisDec 2008
Exercise is an important clinical feature in cystic fibrosis. Better exercise capacity has been associated with better patient outcomes and quality of life. Exercise-induced bronchospasm is a condition, often associated with asthma, which may make exercise difficult. The role that exercise-induced bronchospasm has in people with cystic fibrosis is unknown. This study is designed to determine how often exercise-induced bronchospasm occurs in cystic fibrosis.
A randomised, double-blind, placebo-controlled study to assess the safety and activity of pentosan polysulfate sodium on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain in patients with sickle cell diseaseJul 2008
To assess the safety and tolerability of pentosan polysulfate sodium administered daily for three months to patients with sickle cell disease
A randomized, double-blind, placebo-controlled parallel group study to investigate the safety and efficacy of two doses of tiotropium bromide (2.5 µg and 5 µg) administered once daily via the Respimat device for 12 weeks in patients with cystic fibrosis.Jun 2008
This study evaluates the effects of 12-week treatment with two doses of tiotropium bromide (2.5 μg q.d. and 5 μg q.d.) compared to placebo administered via the Respimat® device on lung function in patients with CF.
Glutamine supplementation for cystic fibrosis: a parallel group randomized controlled trialMay 2008
To assess the impact of glutamine supplementation on markers of infection and lung function in patients with cystic fibrosis and previous pseudomonas infection
Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or ThalassemiaApr 2008
Using sickle cell and thalassemia mouse models, researchers will evaluate the possibility of correcting these disorders by inserting healthy genetic material into the diseased blood cells.
Inheritance of Osteosarcoma & Paget's Disease Through Chromosome 18: Examination of Osteosarcoma Tissue Samples From Two Family Members for Loss of Heterozygosity in the Chromosome 18 Region, Genetically Linked With Paget's Disease of BoneFeb 2008
Researchers have previously demonstrated loss of heterozygosity in a region on chromosome 18q, associated with osteogenic sarcomas in bone affected by Paget's disease. The loci used in this study are specifically described by those authors as showing loss of heterozygosity in 6 of 7 affected families.
DNA Array Analysis of Patients With Cervical CancerAug 2007
The primary objectives of this study are: To obtain preliminary descriptive data on early changes in tumor DNA array expression following chemo-radiation of cervical cancer. These data will permit the design a future studies to correlate array expression changes with clinical outcome. To quantify the degree of therapy-induced apoptosis following chemo-radiation of cervical cancer in order to design future studies to correlate apoptosis levels with clinical outcome. To store material to later correlate the tumor DNA array expression with specific strains of tumor-related human papilloma virus (HPV) and finally to correlate changes in biomarker expression with clinical outcome and findings of the DNA array analyses.
Efficacy and safety of Intra-erythrocytes dexamethasone in cystic fibrosisJul 2007
The main objective is to determine the improvement or worsening lt; 2 of FEV1 in front of 18 months precedents the enrolnment.
Pharmacogenetic study of FcγRIIIa and HER2 genes in relation to treatment of breast cancerApr 2007
Primary objectives are: to determine the influence and frequency of polymorphisms in the FcγRIIIa, HER2 and other related genes on response to trastuzumab with respect to time-to-progression (TTP) and outcome (in patients with advanced/ metastatic breast cancer). To determine the frequency of polymorphisms in the HER2 gene and their association with HER2 expression in tumour samples in both the adjuvant and metastatic setting.
Identifying Genes That Predict Risk of Developing Cervical Intraepithelial Neoplasia or Invasive Cervical CancerApr 2007
This clinical trial is studying genes that may predict which patients are at risk of developing cervical intraepithelial neoplasia or invasive cervical cancer.
Peripheral targeting of inhaled rhDNase in stable CF patients.Apr 2007
To investigate the effect of treatment with nebulized rhDNase targeted to the peripheral airways compared to rhDNase targeted to the central airways on FEF75 (a lung function parameter for peripheral airflow limitation) in children with CF who are on maintenance treatment with rhDNase.
An open study on the pharmacokinetics and safety of oral voriconazole in adult patients with cystic fibrosisMar 2007
The primary endpoints of the trial are to determine the pharmacokinetic parameters for oral voriconazole and to determine if voriconazole serum levels >1.0mg L-1 are achieved in adult patients with cystic fibrosis using a standard oral dosing regimen.
An open-label study of two single oral doses of galantamine, examining the pharmacokinetics, safety, and tolerability in children with Down syndromeDec 2006
To determine the pharmacokinetic characteristics of glantamine after 2 single oral doses in children with Down syndrome.
Stem Cell Transplant in Sickle Cell Disease and ThalassemiaDec 2006
The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease.
N-Acetylcysteine for Treatment of Sickle Cell DiseaseNov 2006
To determine whether NAC therapy results in decreased red cell phosphatidylserine exposure, endothelial activation, inflammation, and reduction clotting activation in the steady state.
To investigate the effect of vitamin K supplementation on markers of bone turnover and bone density in adolescents and adults with cystic fibrosisSep 2006
To establish whether vitamin K supplementation in adolescent and adult patients with CF improves markers of bone formation.