Investigational New Drug (IND) Applications: What You Need to Know for Successful Interactions with the FDA

An educational program of the NIH Clinical Center-FDA CDER Joint Task Force emphasizing regulatory requirements for clinical trials of new and repurposed drugs, with a special focus on rare diseases. Target Audience: Clinical investigators, clinical research study coordinators, clinical and translational research scientists and trainees. Objectives: • To provide training in FDA regulations for NIH investigators involved in phase I and phase II clinical trials of investigational drugs, emphasizing safety considerations in the early phases of drug development. • To describe the IND-enabling elements of non-clinical research that are required to implement a first-in-human clinical trial, including chemistry, manufacturing, and controls (CMC) conducted under good manufacturing practice (GMP) conditions, and non-clinical pharmacology and toxicology studies. • To address special considerations applicable to drug development for rare diseases, including limited number of patients and the need for well-designed, efficient, and informative phase I-II clinical trials to support further development of novel therapies for rare diseases. • To inform the NIH clinical research community about new web-based tools that will facilitate IND submissions. Air date: 12/12/2013 8:00:00 AM
Source: Videocast - All Events - Category: Journals (General) Tags: Upcoming Events Source Type: video

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AbstractHereditary angioedema (HAE) with C1 esterase inhibitor (C1-INH) deficiency (C1-INH-HAE) is a rare disease characterized by diminished levels or dysfunctional activity of C1-INH, leading to dysregulated plasma kallikrein activity within the kallikrein –kinin pathway. Symptoms manifest as painful, potentially life-threatening swelling of subcutaneous tissues throughout the body and/or submucosal edema in the upper airway or gastrointestinal tract. Attacks recur with unpredictable frequency, intensity, and duration, placing a heavy burden on pati ents’ daily lives. Despite improved availability of medicati...
Source: BioDrugs - Category: Drugs & Pharmacology Source Type: research
(Aarhus University) For the first time ever, biomedical researchers from Aarhus University, Denmark, have unveiled cellular defects that lead to the rare disease hereditary angioedema (HAE), where patients experience recurrent episodes of swelling that requires immediate treatment. This new understanding is an important step towards gene therapy for patients.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
By MIKE STOBBE NEW YORK (AP) — This year has seen a record number of cases of a mysterious paralyzing illness in children, U.S. health officials said Monday.It's still not clear what's causing the kids to lose the ability to move their face, neck, back, arms or legs. The symptoms tend to occur about a week after the children had a fever and respiratory illness.No one has died from the rare disease this year, but it was blamed for one death last year and it may have caused others in the past.What's more, Centers for Disease Control and Prevention officials say many children have lasting paralysis. And close to half th...
Source: JEMS Operations - Category: Emergency Medicine Authors: Tags: Operations News Source Type: news
Pulmonary Arterial Hypertension (PAH) is a chronic rare disease that can lead to serious cardiovascular problems and death. Additional treatments that increase effectiveness, that are safe and with a convenien...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Authors: Riera-Mestre A, Ribas J, Castellote J Abstract Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant inherited Rare Disease that causes a systemic anomalous vascular overgrowth. The approach and follow-up of these patients should be from multidisciplinary units. Its diagnosis is carried out according to Curaçao clinical Criteria. Telangiectasia in the nasal mucosa cause recurrent epistaxis, the main symptom of HHT and difficult to control. The three types of hepatic shunting, hepatic artery to hepatic vein, hepatic artery to portal vein or to portal vein to hepatic vein, can cause hi...
Source: Medicina Clinica - Category: General Medicine Tags: Med Clin (Barc) Source Type: research
AbstractLaryngeal leiomyosarcoma is a rare smooth muscle malignancy of the head and neck region. Diagnosis is based on immunohistochemistry. Here we present a case of laryngeal leiomyosarcoma that was diagnosed and treated in our center, focusing on the clinical features, histological diagnosis and management of this rare disease.
Source: Indian Journal of Otolaryngology and Head and Neck Surgery - Category: ENT & OMF Source Type: research
AbstractHere, we report brain white matter alterations in individuals clinically and genetically diagnosed with periodontal Ehlers –Danlos syndrome, a rare disease characterized by premature loss of teeth and connective tissue abnormalities. Eight individuals of two families clinically diagnosed with periodontal Ehlers–Danlos syndrome were included in the present study and underwent general physical, dental, and neurologica l examination. Whole exome sequencing was performed, and all patients included in the study underwent MRI of the brain. Whole exome sequencing revealed heterozygousC1R mutations c.926G>T ...
Source: Neurogenetics - Category: Genetics & Stem Cells Source Type: research
Authors: Monagle P, Newall F Abstract Venous thrombosis (VTE) in children and neonates presents numerous management challenges. Although increasing in frequency, VTE in children and neonates is still uncommon compared with adults. The epidemiology of VTE is vastly different in neonates vs children vs adolescents vs adults. In reality, pediatric thrombosis should be viewed as a multitude of rare diseases (eg, renal vein thrombosis, spontaneous thrombosis, catheter-related thrombosis, cerebral sinovenous thrombosis), all requiring different approaches to diagnosis and with different short- and long-term consequences,...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
The new tool "KIT" in advanced systemic mastocytosis. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):127-136 Authors: Shomali W, Gotlib J Abstract Mastocytosis is a rare disease characterized by KIT-driven expansion and accumulation of neoplastic mast cells in various tissues. Although mediator symptoms related to mast cell activation can impose a symptom burden in cutaneous disease and across the spectrum of systemic mastocytosis subtypes, the presence of an associated hematologic neoplasm and/or organ damage denotes advanced disease and the potential for increased morbidity and...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
ConclusionsOur results corroborate that cxSVs cause Mendelian disease, and we recommend their consideration during clinical investigations. We show that resolution of breakpoints can be critical to interpret pathogenicity and present evidence of replication-based mechanisms in cxSV formation.
Source: Genome Medicine - Category: Genetics & Stem Cells Source Type: research
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