Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas

Neuro-Oncology

Volumn 16, Issue 5, 2014, Pages 707-718

  Widemann, Brigitte C ^a   Dombi, Eva ^a   Gillespie, Andrea ^a   Wolters, Pamela L ^a   Belasco, Jean ^c   Goldman, Stewart ^d   Korf, Bruce R ^e   Solomon, Jeffrey ^f   Martin, Staci ^a   Salzer, Wanda ^j   Fox, Elizabeth ^a,c   Patronas, Nicholas ^b   Kieran, Mark W ^g   Perentesis, John P ^h   Reddy, Alyssa ^k   Wright, John J ^a   Kim, Aerang ⁱ   Steinberg, Seth M ^a   Balis, Frank M ^a,c  

^a NATIONAL CANCER INSTITUTE   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^d CHILDREN'S MEMORIAL HOSPITAL   (United States)

^e UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^f Expert Image Analysis LLC   (United States)

^g BOSTON CHILDREN S HOSPITAL   (United States)

^h CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

ⁱ CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^j U S ARMY MEDICAL RESEARCH AND MATERIEL COMMAND   (United States)

^k CHILDREN S HOSPITAL OF ALABAMA   (United States)

more..

Author keywords

Neurofibromatosis type 1; Phase 2 trial; Plexiform neurofibroma; RAS signaling; Trial design

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; BICARBONATE; BILIRUBIN; METHOTREXATE; NEUROFIBROMIN; PEGINTERFERON ALPHA2B; PIRFENIDONE; PLACEBO; RETINOIC ACID; THALIDOMIDE; TIPIFARNIB; VINBLASTINE; ANTINEOPLASTIC AGENT; ENZYME INHIBITOR; FARNESYL TRANS TRANSFERASE; QUINOLONE DERIVATIVE;

ABDOMINAL PAIN; ADOLESCENT; ADULT; ADULT DISEASE; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALLERGIC RHINITIS; ALOPECIA; ANOREXIA; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BEHAVIOR DISORDER; BICARBONATE BLOOD LEVEL; BILIRUBIN BLOOD LEVEL; BLURRED VISION; CANCER THERAPY; CHILD; CHILDHOOD DISEASE; CONSTIPATION; CONTROLLED STUDY; COUGHING; CROSSOVER PROCEDURE; DEPRESSION; DIARRHEA; DIZZINESS; DOUBLE BLIND PROCEDURE; DRUG DOSE REDUCTION; DRUG FEVER; DRUG TOLERABILITY; DRY SKIN; DYSURIA; EDEMA; EPISTAXIS; EVENING DOSAGE; EYE DISEASE; FATIGUE; FEMALE; FOLLOW UP; HALLUCINATION; HEADACHE; HEART PALPITATION; HEMATURIA; HUMAN; HYPERCALCEMIA; HYPERGLYCEMIA; HYPERKALEMIA; HYPERMAGNESEMIA; HYPERTENSION; HYPOALBUMINEMIA; HYPOCALCEMIA; HYPOGLYCEMIA; HYPOKALEMIA; HYPOMAGNESEMIA; HYPONATREMIA; HYPOPHOSPHATEMIA; INSOMNIA; LYMPHOCYTOPENIA; MAJOR CLINICAL STUDY; MALE; MOOD DISORDER; MORNING DOSAGE; MULTIPLE CYCLE TREATMENT; MUSCLE WEAKNESS; MYALGIA; NAUSEA; NECK PAIN; NEUROFIBROMA; NEUROFIBROMATOSIS; NEUROIMAGING; NEUTROPENIA; NUCLEAR MAGNETIC RESONANCE IMAGING; OTALGIA; PARTIAL THROMBOPLASTIN TIME; PERSONALITY DISORDER; PHARYNGITIS; PHASE 2 CLINICAL TRIAL; PROTEINURIA; PROTHROMBIN TIME; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; RASH; SEIZURE; SENSORY NEUROPATHY; SIDE EFFECT; SKIN BRUISING; SKIN PRURITUS; TASTE DISORDER; THORAX PAIN; TREATMENT DURATION; TUMOR GROWTH; TUMOR VOLUME; URINARY URGENCY; VOMITING; XEROSTOMIA; YOUNG ADULT; ANTAGONISTS AND INHIBITORS; CLINICAL TRIAL; DISEASE COURSE; NEUROFIBROMA, PLEXIFORM; NEUROFIBROMATOSIS 1; PRESCHOOL CHILD; TREATMENT OUTCOME;

ADOLESCENT; ADULT; ANTINEOPLASTIC AGENTS; CHILD; CHILD, PRESCHOOL; CROSS-OVER STUDIES; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; ENZYME INHIBITORS; FARNESYLTRANSTRANSFERASE; FEMALE; HUMANS; MALE; NEUROFIBROMA, PLEXIFORM; NEUROFIBROMATOSIS 1; QUINOLONES; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84899440574     PISSN: 15228517     EISSN: 15235866     Source Type: Journal    
DOI: 10.1093/neuonc/nou004     Document Type: Article

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