Navigation Links
Edison Pharmaceuticals Announces Initiation of NIH-Sponsored EPI-743 Clinical Trial with NIH Undiagnosed Diseases Program Patients
Date:9/24/2013

MOUNTAIN VIEW, Calif., Sept. 24, 2013 /PRNewswire/ -- Edison Pharmaceuticals announced today initiation of a phase 2 clinical trial titled "Therapeutic Trial of EPI-743 in Patients with Disorders of Energy Utilization or Oxidation-Reduction."

The trial is a double-blind, placebo-controlled crossover study in which subjects will receive either EPI-743 treatment or placebo for six months. They will then be crossed over to the other arm (treatment or placebo). Eligible subjects must possess defined or undefined genetic diagnoses that feature cellular oxidation/reduction or energy production defects.

Those children absent a genetic diagnosis will be recruited through the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP). The principle enrollment criterion stipulates that all subjects must have a neuromuscular disorder of suspected mitochondrial or metabolic etiology. All subjects will also be enrolled in a clinical trial studying the natural history of metabolic disorders. Prior to enrollment, patient cells will be analyzed in the laboratory to verify that the cells display biochemical abnormalities associated with mitochondrial disease and can be rescued by EPI-743. 

The primary endpoints in this study are improvement in disease severity and quality of life as measured by the Newcastle Pediatric Mitochondrial Disease Scale. Secondary outcome measures include neuromuscular function and disease-related biochemical markers. More information on study specifics is available on www.ClinicalTrials.gov.

Of the approximately 4 million children born each year in the United States, an estimated 4,000 will have a mitochondrial disease. However, this may be an underestimation of true disease prevalence, since many pediatric neuromuscular patients present with laboratory and clinical findings consistent with energy or mitochondrial dysfunction absent a genetic diagnosis. The objective of the study is to evaluate responsiveness to EPI-743, including subjects who do not possess a genetic diagnosis.

"This is a pioneering study for the National Human Genome Research Institute (NHGRI)," said NHGRI Clinical Director William Gahl, MD, PhD, who also is director of the UDP. "If successful, it will offer the potential for treatment for clinically devastating pediatric neurological disorders. The study can also provide diagnostic clues regarding the causes of rare and undiagnosed diseases."

UDP was started in 2008 to address the unmet medical need of patients with complex, multisystem disorders. Details of the program and its accomplishments were recently reported in Genetics in Medicine (The National Institutes of Health Undiagnosed Diseases Program: Insights into rare disease, 2012;13:51-59).  The UDP multidisciplinary team combines sophisticated cutting-edge basic science and molecular diagnostic techniques with high-level clinical care to provide diagnoses and therapies for patients with previously undiagnosed diseases.

Edison Pharmaceuticals
Edison Pharmaceuticals is a biotechnology company dedicated to developing treatments for children and adults with mitochondrial diseases. More information can be found at www.edisonpharma.com.


'/>"/>
SOURCE Edison Pharmaceuticals
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Edison initiates coverage of Derma Sciences
2. Edison expands North American healthcare sector coverage with initiation of coverage on Alexza Pharmaceuticals
3. Edison Expands North American Healthcare Sector Coverage With Initiation of Coverage on Bellus Health
4. Edison Pharmaceuticals, Inc. signs licensing agreement with Dainippon Sumitomo Pharma Co., Ltd. for development & commercialization of orphan mitochondrial and adult central nervous system disease drugs
5. Edison Pharmaceuticals & Bambino Gesu Childrens Hospital Announce Initiation of EPI-743 Phase 2 Cobalamin C Deficiency Syndrome Clinical Trial
6. Samsung Medison Showcases New Medical Equipment and Technology with Samsung Electronics at RSNA 2012
7. Edison Pharmaceuticals Closes $20M Series F Financing
8. Edison Pharmaceuticals Announces Initiation of EPI-743 Phase 2B Leigh Syndrome Clinical Trial
9. Massive Hiring Event for Life Science Professionals in Edison, NJ on 9/18/12
10. Edison Pharmaceuticals Announces Positive Results of EPI-743 Phase 2A Leigh Syndrome Clinical Trial
11. EMA Grants Orphan Designation to Edison Pharmaceuticals for EPI-743 for Treatment of Leigh Syndrome
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/23/2016)... , June 23, 2016 The vast ... an outpatient dialysis facility.  Treatments are usually 3 times ... hours per visit, including travel time, equipment preparation and ... patient, but especially grueling for patients who are elderly ... a skilled nursing and rehabilitation centers for some duration ...
(Date:6/23/2016)... , June 23, 2016  Experian ... integrating and transforming the patient payment and ... several innovative new products and services that ... its revenue cycle offerings. These award-winning solutions ... efficient workflows, remain compliant in an ever-changing ...
(Date:6/23/2016)... June 23, 2016  The National Pharmaceutical Council ... the health policy research organization as its newest ... MD, senior vice president and chief scientific officer, ... on the NPC Board of Directors. ... Mallinckrodt has joined us in support of our ...
Breaking Medicine Technology:
(Date:6/25/2016)... ... ... Austin residents seeking Mohs surgery services, can now turn to Dr. Jessica ... Peckham for medical and surgical dermatology. , Dr. Dorsey brings specialization to include Mohs ... Mohs Micrographic Surgery completed by Dr. Dorsey was under the direction of Glenn Goldstein, ...
(Date:6/25/2016)... ... ... First Choice Emergency Room , the largest network of independent freestanding emergency ... its new Mesquite-Samuell Farm facility. , “We are pleased to announce Dr. Ogunleye ... M. Muzzarelli, Executive Medical Director of First Choice Emergency Room. , Dr. Ogunleye ...
(Date:6/25/2016)... ... 25, 2016 , ... On Friday, June 10, Van Mitchell, Secretary of the ... to iHire in recognition of their exemplary accomplishments in worksite health promotion. , The ... Health & Wellness Symposium at the BWI Marriott in Linthicum Heights. iHire was one ...
(Date:6/24/2016)... ... June 24, 2016 , ... Those who have experienced traumatic events may suffer ... unhealthy avenues, such as drug or alcohol abuse, as a coping mechanism. To avoid ... healthy coping following a traumatic event. , Trauma sufferers tend to feel a range ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Pulmonary ... Dallas that it will receive two significant new grants to support its work ... marked its 25th anniversary by recognizing patients, medical professionals and scientists for their ...
Breaking Medicine News(10 mins):