News About Clinical Trials
NORD shares information about opportunities to participate in clinical trials so that patients and their physicians may decide whether specific studies are appropriate for them. We do not endorse or recommend participation in any specific clinical trials. If you have any questions for NORD or would like to post a clinical trial please contact us here.
The following is a brief sampling of current studies. For more comprehensive information about opportunities to participate in research, go to www.clinicaltrials.gov.
Castleman’s Disease
Castleman's disease is a rare, atypical lymphoproliferative disorder. An ongoing clinical research study is enrolling patients with Castleman’s disease. The purpose of this study is to evaluate different doses and schedules of CNTO328 to see which dose/schedule is safe. This research study will use a type of drug called anti-IL-6 antibody, also known as CNTO 328. This study is being conducted at 10 centers in the United States. For more information, go to www.castlemansresearch.com or ClinicalTrials.gov identifier: NCT01024036.
Chronic pain management clinical trials pediatric patients
If you are a parent with a child that experiences chronic pain who would like to know more information about pediatric chronic pain clinical studies being conducted, or a healthcare professional aware of pediatric patients requiring analgesia who might be appropriate for and interested in participating in clinical studies, please contact Purdue Pharma L.P.’s Medical Services Department. Contact details for the Medical Services department are as follows:
Email: purduepediatrics@pharma.com
Phone: (Toll-Free) 1-888-726-7535; Option#1
Business hours: Monday-Friday 8am-5pm EST (USA)
Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
Bayer is conducting the CHEST-1 study to assess the efficacy and safety of different doses of BAY63-2521, given orally for 16 weeks, in patients with CTEPH. Male and female patients age 18-80 with CTEPH either inoperable or with persistent or recurrent PH after surgery are eligible. ClinicalTrials.gov identifier: NCT00855465. Study website: http://www.phclinicalstudy.com.
Chronic Sarcoidosis
If you are experiencing symptoms despite your current therapy, you may be interested in learning more about a clinical research study that is being conducted to evaluate two investigational drugs for chronic sarcoidosis.
You may be eligible to participate if you are 18 to 85 years of age, have been told by your doctor that you have chronic sarcoidosis with lung and/or skin involvement, are experiencing symptoms despite your c urrent therapy. Study volunteers will be provided research related medical care and medication at no cost. To learn more about this research study, please visit ClinicalTrials.gov identifier: NCT00955279.
Craniosynostosis
Researchers at the University of California, Irvine Medical Center, are studying the causes of craniosynostosis with the ultimate goal of identifying genes and environmental factors causing this condition. They are recruiting families with at least one child with craniosynostosis. For information, contact Dr. Virginia Kimonis, MD (vkimonis@uci.edu; 949 824 0571 or fax: 714 456 5330) or research coordinator Dr. June-Anne Gold (goldj@uci.edu or 949 824-0521).
Ehlers-Danlos Syndrome Type IV (EDS type IV)
Researchers at the University of Washington are interested in learning more about EDS type IV (vascular EDS) pregnancies. Complications including uterine and arterial rupture, hemorrhage, and death have been reported during and after pregnancy in women with EDS type IV. A goal of the study is to develop recommendations for pregnancy management, counseling, and the route of delivery. Individuals with EDS type VI who have been pregnant may be eligible. Medical records from deceased relatives with EDS type IV who have been pregnant are also of interest. For additional information please contact: Melanie Pepin, MS, CGC at mpepin@uw.edu or 206-543-5464 or toll free (1-888-288-7362); Dru Leistritz, MS, CGC at Dru2@u.washington.edu or 206-543-5464 or Mitzi L. Murray, MD at mlmurray@uw.edu or 206-685-5495. (note: confidentiality of email communication can not be guaranteed).
Fibromuscular Dysplasia (FMD)
The Fibromuscular Dysplasia Society of America is collaborating with the Cleveland Clinic and Mayo Clinic on a research study to identify genetic factors that may influence susceptibility to certain vascular diseases like FMD. The objectives are to find new methods for prevention, diagnosis and treatment, and investigate the prevalence of traditional cardiovascular risk factors in individuals and family members with FMD. Patients with FMD and their first-degree family members (such as parents, siblings, and adult children) may volunteer to participate. Participation will involve giving consent, completing a medical questionnaire, and collecting a blood sample. If you wish to obtain more information about this study please feel free to contact pam.mace@fmdsa.org.
Fragile X Syndrome
The University of North Carolina at Chapel Hill and Washington University in St. Louis are currently recruiting for a study of Fragile X and brain development in infants with Fragile X. They are looking for children under the age of 12 months who are suspected of having Fragile X (parents who are expecting may also be eligible.) and need 30 participants in the US who meet the following criteria: 1) Are under 12 months of age (parents who are expecting may also be eligible) and 2) Have diagnosis of Fragile X (full or premutation) or are suspected of having Fragile X or have an older sibling with Fragile X. For more information visit http://www.ibisnetwork.org/fragilex/.
Freeman-Sheldon and Related Syndromes
The Freeman-Sheldon Research Group, Inc. is seeking participants for three studies to: (1) survey and review medical records for information about specific treatments and major problems in patients' medical history; (2) medically evaluate patients', their families', and other affected individuals' (who have spent considerable time with patients) to learn about quality of life and mental health issues relating to these syndromes; and (3) medically evaluate healthy persons and patients to compare their bodies' functioning. These studies are each designed to form a basis for improving the standard of care and developing new treatments. Persons with Freeman-Sheldon syndrome, Sheldon-Hall syndrome, distal arthrogryposis type 1, or distal arthrogryposis type 3 are eligible for all studies. For more information, contact the study coordinator, Maria Patterson (patterson_m@fsrgroup.org or 443-694-0057); research assistant, MI Poling (poling_mi@fsrgroup.org or 304- 460-9038); or lead researcher, RJ McCormick (mccormick_rj@fsrgroup.org or 304-472-0318); or visit the following websites:
http://fsrgroup.org
ClinicalTrials.gov identifier: NCT01144741
ClinicalTrials.gov identifier: NCT01307475
ClinicalTrials.gov identifier: NCT01306994
Hirschsprung Disease
Dr. Aravinda Chakravarti’s laboratory at Johns Hopkins University has been investigating the genetics of Hirschsprung disease (HSCR) for more than twenty years. The purpose of our study is to continue the search for genes involved in Hirschsprung disease and to further characterize the known genes and the interactions between them. Our study will hopefully lead to a better understanding of the genetics of HSCR and, further down the road, improved diagnosis, treatment, and genetic counseling.
We ask study volunteers to complete a medical/family history questionnaire, provide access to some medical records, and to submit blood samples from the individual(s) affected with Hirschsprung disease and his/her parents (if available). If you are interested, a kit containing all the materials necessary to participate can be sent to you. There will be no cost to you. For more information please contact the study coordinator, Courtney Berrios, at 410-502-7541 or hirschsprung@igm.jhmi.edu or visit https://aravindachakravartilab.org/pro/Hirschsprung_Study.html
Liposarcoma or Leiomyosarcoma
Janssen Research & Development, L.L.C. is conducting a Phase 3 clinical trial for a product under investigation in patients with liposarcoma or leiomyosarcoma. This study is designed to compare two different drugs to treat these conditions. The study will be divided into three phases: screening, treatment, and follow-up. During screening, potential patients will be assessed for study eligibility after providing signed informed consent. During the treatment phase, patients will receive study drug once every 3 weeks, until disease progression or signs of toxicity. Assessments will be performed to evaluate the effectiveness of the drug, and patient safety will be monitored. During the follow-up phase, after the last dose of study drug, clinical outcomes for patients will be evaluated. For more information on this study, please visit www.l-sarcomastudy.com or ClinicalTrials.gov identifier: NCT01343277.
Lupus Nephritis
If you have been diagnosed with lupus nephritis and are experiencing symptoms despite your current therapy, you may be interested in learning more about this study, currently seeking eligible patients, to evaluate the effectiveness and safety of an investigational medication for the treatment of active lupus nephritis. You may be eligible to participate if you are between 18 and 70 years of age and have persistently active lupus nephritis. To learn more about the study, view a list of site locations, and see if you pre-qualify, visit clinicaltrials.gov.
Neurofibromatosis Type 1 (NF1)
The Washington University Neurofibromatosis (NF) Center has launched a unique internet-based NF Registry (NF1 Patient Registry Initiative) that will collect medical information to help physicians and researchers better understand the spectrum of medical problems found in children and adults with NF1. All individuals with a diagnosis of NF1 are eligible to complete the 30-minute online questionnaire. The information collected from the NF1 Registry will be used to better characterize the relationships between specific problems in NF1. In particular, researchers hope to better understand correlations between NF1 and cancer, heart problems, allergies, depression, and learning disabilities. The Washington University NF1 Registry will enable studies that aim to identify children and adults at greatest risk for specific medical problems in NF1. These studies may help clinicians to develop personalized treatment strategies that improve the quality of life for individuals living with NF1. All information is housed on a secure server in compliance with established HIPAA guidelines. To access the registry, go to http://nf1registry.wustl.edu/.
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
The University of Florida is continuing to recruit patients for a clinical trial to investigate the safety and effectiveness of Coenzyme Q10 (CoQ10) as a treatment for children with specific mitochondrial diseases. CoQ10 is a mitochondrial cofactor and antioxidant in the process that cells use to convert food and oxygen into energy. To be considered for the trial, patients must be 12 months to 17 years of age and have a biochemical or molecular diagnosis of a deficiency of complex I, III or IV of the respiratory chain. For additional information contact Tracie Kurtz, RN, at Tracie.Kurtz@medicine.ufl.edu or 352-273-9016 or go to ClinicalTrials.gov identifier: NCT00432744.
Pediatric Medical Device Study
The FDA is initiating a study to identify scientific and medical issues (e.g., human factors, safety, and usability) with pediatric neurologic devices in children undergoing treatment. The ASK CHILDREN (Assess Specific Kinds of CHILDREN Challenges) study is being undertaken in order to develop more efficient strategies in evaluating products regulated by the FDA. Data will be collected from approximately 100 children including patients aged 7 to15 years old implanted with cerebral spinal fluid shunts, cochlear implants and deep brain stimulators, and patients aged 14 and 15 years old implanted with spinal cord stimulators. For more information about participating in this study please visit www.askchildrenstudy.org, ClinicalTrials.gov identifier: NCT01191307 or call Carlos Pena, PhD, MS at 301-827-6687 or Kristen Bowsher, PhD, at 240-276-3605.
Pulmonary Arterial Hypertension (PAH)
Bayer is conducting a study to assess the efficacy and safety of different doses of BAY63-2521 given orally for 12 weeks, in patients with symptomatic PAH. Male and female patients age 18-80 with symptomatic PAH (idiopathic, familial, associated PAH due to connective tissue disease, congenital heart disease, portal hypertension with liver cirrhosis, or due to anorexigen or amphetamine use) are eligible. Also eligible are patients who have never been treated or have been pre-treated with an endothelin antagonist or a prostacyclin analogue (except IV). ClinicalTrials.gov identifier: NCT00810693. Study website: http://www.phclinicalstudy.com.
Pulmonary Sarcoidosis
Individuals with stage II or stage III pulmonary sarcoidosis may be eligible to participate in a study to evaluate the effects of atorvastatin (Lipitor). This study will examine whether atorvastatin, a widely used cholesterol-lowering drug, may help patients with pulmonary sarcoidosis, replacing or reducing the need for steroids such as prednisone. This study is being conducted at the NIH Clinical Center in Bethesda, Md., and all participants will come to the Clinical Center. Travel assistance may be available. For information, call (866) 444-2214 (TTY: 866 411-1010) or go to ClinicalTrials.gov identifier: NCT00279708.
Sarcoidosis
The Worldwide Sarcoidosis Research Study (WISE) is designed to gain more information about the characteristics and clinical course of sarcoidosis in order to help physicians and researchers better understand how to treat the disease. Researchers at the University of Iowa Institute for Clinical and Translational Science will be gathering information from a large number of sarcoidosis patients localed all around the world using a web-based survey. Read More.
Urea Cycle Disorders (UCD)
A clinical trial called SELICA III sponsored by Cytonet is designed to evaluate the safety and efficacy of liver cell therapy in infants and children up to age 5 with UCD. Liver cell therapy involves collecting healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) which are then isolated and undergo complex processing. These cells are infused into the hepatic portal vein over six days. Researchers seek to enroll 20 patients in 12 medical centers. ClinicalTrials.gov identifier: NCT01195753.
Vaso-occlusive Crisis of Sickle Cell Disease
GlycoMimetics, Inc. is studying vaso-occlusive crisis of sickle cell disease as the first potential indication for GMI-1070, its lead clinical drug candidate. Inflammation is a key mediator of vaso-occlusive crisis, a condition which represents a significant unmet medical need. Sickle cell disease is a chronic condition with substantial morbidity and mortality, responsible for more than 75,000 hospitalizations per year in the U.S. for acute vaso-occlusive crisis. GMI-1070 has received both Orphan Drug and Fast Track status from the Food & Drug Administration for the treatment of vaso-occlusive crisis.
GMI-1070 is intended to treat vaso-occlusive crisis by inhibiting the cell activation and enhanced cell adhesion, which blocks microvascular blood flow and causes the ischemia and pain. In June 2010, GlycoMimetics, Inc. announced the start of a Phase 2 clinical trial of GMI-1070 for treatment of vaso-occlusive crisis of sickle cell disease. Nearly 20 sites are actively enrolling patients. For more information contact Dr. Helen Thackray at hthackray@glycomimetics.com or ClinicalTrials.gov identifier:NCT01119833.
Wegener's Granulomatosis
Wegener's granulomatosis is a multisystem disorder that causes inflammation in blood vessels (vasculitis), restricting blood flow to various organs. There is evidence to suggest that it may be caused by a combination of environmental and genetic factors. To better understand its causes, a study is in progress at Mount Sinai Hospital in Toronto to identify "susceptibility genes" that would increase one's risk for developing this disorder.
Blood samples are being collected for DNA analysis from WG patients and their immediate family members. Clinical data are also being reviewed. For information, contact Alida Pokoradi, the clinical research coordinator, at (416) 946-4501 ext. 3297 or clinicalgenet.research@gmail.com or go to: www.vasculitisfoundation.org/node/16.
