A checklist for clinical trials in rare disease: Obstacles and anticipatory actions-lessons learned from the FOR-DMD trial

Trials

Volumn 19, Issue 1, 2018, Pages

  Crow, Rebecca A ^a   Hart, Kimberly A ^b   McDermott, Michael P ^b   Tawil, Rabi ^b   Martens, William B ^b   Herr, Barbara E ^b   McColl, Elaine ^c   Wilkinson, Jennifer ^c   Kirschner, Janbernd ^d   King, Wendy M ^b   Eagle, Michele ^a   Brown, Mary W ^b   Hirtz, Deborah ^e   Lochmuller, Hanns ^a   Straub, Volker ^a   Ciafaloni, Emma ^b   Shieh, Perry B ^f   Spinty, Stefan ^g   Childs, Anne Marie ^h   Manzur, Adnan Y ⁱ   Morandi, Lucia ^j   Butterfield, Russell J ^k   Horrocks, Iain ^l   Roper, Helen ^m   Flanigan, Kevin M ⁿ   Kuntz, Nancy L ^o   Mah, Jean K ^p   Morrison, Leslie ^q   Darras, Basil T ^r   von der Hagen, Maja ^s   Schara, Ulrike ^t   Wilichowski, Ekkehard ^u   Mongini, Tiziana ^v   McDonald, Craig M ^w   Vita, Giuseppe ^x   Barohn, Richard J ^y   Finkel, Richard S ^z   Wicklund, Matthew ^aa   McMillan, Hugh J ^ab   Hughes, Imelda ^ac   Pegoraro, Elena ^ad   Bryan Burnette, W ^ae   Howard, James F ^af   Thangarajh, Mathula ^ag   Campbell, Craig ^ah   Griggs, Robert C ^b   Bushby, Kate ^a   Guglieri, Michela ^a   more..

^a NEWCASTLE UNIVERSITY   (United Kingdom)

^b UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^c NEWCASTLE UNIVERSITY   (United Kingdom)

^d UNIVERSITY OF FREIBURG   (Germany)

^e NATIONAL INSTITUTES OF HEALTH   (United States)

^f UNIVERSITY OF CALIFORNIA   (United States)

^g ALDER HEY CHILDREN S HOSPITAL   (United Kingdom)

^h LEEDS TEACHING HOSPITALS NHS TRUST   (United Kingdom)

ⁱ UNIVERSITY COLLEGE LONDON   (United Kingdom)

^j DEPARTMENT OF NEUROSURGERY   (Italy)

^k UNIVERSITY OF UTAH   (United States)

^l NHS GREATER GLASGOW AND CLYDE   (United Kingdom)

^m BIRMINGHAM HEARTLANDS HOSPITAL   (United Kingdom)

ⁿ NATIONWIDE CHILDREN S HOSPITAL   (United States)

^o CHILDREN'S MEMORIAL HOSPITAL   (United States)

^p UNIVERSITY OF CALGARY   (Canada)

^q UNIVERSITY OF NEW MEXICO   (United States)

^r BOSTON CHILDREN S HOSPITAL   (United States)

^s DRESDEN UNIVERSITY OF TECHNOLOGY   (Germany)

^t UNIVERSITY OF DUISBURG ESSEN   (Germany)

^u UNIVERSITY MEDICAL CENTER GÖTTINGEN   (Germany)

^v UNIVERSITY OF TURIN   (Italy)

^w UNIVERSITY OF CALIFORNIA   (United States)

^x UNIVERSITY OF MESSINA   (Italy)

^y UNIVERSITY OF KANSAS MEDICAL CENTER   (United States)

^z NEMOURS CHILDREN S HOSPITAL   (United States)

^aa PENN STATE COLLEGE OF MEDICINE   (United States)

^ab CHILDREN S HOSPITAL OF EASTERN ONTARIO   (Canada)

^ac ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^ad UNIVERSITY OF PADOVA   (Italy)

^ae MONROE CARELL JR CHILDREN S HOSPITAL AT VANDERBILT   (United States)

^af UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^ag CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^ah LONDON HEALTH SCIENCES CENTRE   (Canada)

more..

Author keywords

Academic led clinical trial; Clinical trial; Clinical trial regulations; Duchenne muscular dystrophy; Rare disease

Indexed keywords

STEROID;

CHECKLIST; CLINICAL OUTCOME; CLINICAL PROTOCOL; CLINICAL RESEARCH; DRUG DEALING; DRUG MANUFACTURE; DRUG MARKETING; DRUG USE; DUCHENNE MUSCULAR DYSTROPHY; FINANCIAL MANAGEMENT; FUNDING; HEALTH CARE DELIVERY; HEALTH INSURANCE; HUMAN; INTERPERSONAL COMMUNICATION; NATIONAL HEALTH ORGANIZATION; RARE DISEASE; REVIEW; BUDGET; CLINICAL TRIAL (TOPIC); CONTRACT; ECONOMICS; INTERNATIONAL COOPERATION; LEGISLATION AND JURISPRUDENCE; METHODOLOGY; MULTICENTER STUDY (TOPIC); PATIENT SELECTION; PROCEDURES; SUPPLY AND DISTRIBUTION; TIME FACTOR; TREATMENT OUTCOME;

BUDGETS; CHECKLIST; CLINICAL TRIALS AS TOPIC; CONTRACTS; HUMANS; INTERNATIONAL COOPERATION; MULTICENTER STUDIES AS TOPIC; MUSCULAR DYSTROPHY, DUCHENNE; PATIENT SELECTION; RARE DISEASES; RESEARCH DESIGN; RESEARCH SUPPORT AS TOPIC; STEROIDS; TIME FACTORS; TREATMENT OUTCOME;

EID: 85047553551     PISSN: None     EISSN: 17456215     Source Type: Journal    
DOI: 10.1186/s13063-018-2645-0     Document Type: Review

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