Advances in clinical trials for movement disorders

Movement Disorders

Volumn 30, Issue 11, 2015, Pages 1580-1587

  Kieburtz, Karl ^a   Olanow, C Warren ^b  

^a UNIVERSITY OF ROCHESTER SCHOOL OF MEDICINE AND DENTISTRY   (United States)

^b MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Clinical Trials; Huntington; Parkinson

Indexed keywords

CARBIDOPA PLUS LEVODOPA; DOPAMINE RECEPTOR STIMULATING AGENT; LEVODOPA; MONOAMINE OXIDASE B INHIBITOR; PLACEBO; PRAMIPEXOLE; RASAGILINE;

CLINICAL TRIAL (TOPIC); COGNITIVE DEFECT; GAIT DISORDER; HUMAN; MEDICAL DEVICE; METHODOLOGY; MOTOR DYSFUNCTION; PARKINSON DISEASE; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; STUDY DESIGN; WEARABLE DEVICE; DISABLED PERSON; MOVEMENT DISORDERS; PATHOPHYSIOLOGY; PROCEDURES;

CLINICAL TRIALS AS TOPIC; DISABLED PERSONS; HUMANS; MOVEMENT DISORDERS;

EID: 84941600728     PISSN: 08853185     EISSN: 15318257     Source Type: Journal    
DOI: 10.1002/mds.26371     Document Type: Review

References (51)

1. Hauser RA, Hsu A, Kell S, et al. Extended-release carbidopa-levodopa (IPX066) compared with immediate-release carbidopa-levodopa in patients with Parkinson's disease and motor fluctuations: a phase 3 randomised, double-blind trial. Lancet Neurol 2013;12:346-356.
2. Olanow CW, Kieburtz K, Odin P, Espay AJ, Standaert DG, Fernandez HH, et al. Continuous intrajejunal infusion of levodopa-carbidopa intestinal gel for patients with advanced Parkinson's disease: a randomised, controlled, double-blind, double-dummy study. Lancet Neurol. 2014; 13:141-9.
3. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research. Guidance for industry applications covered by section 505(b)(2). 1999. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM079345.pdf. 20 Aug 2015.
4. Alterman RL, Tagliati M, Olanow CW. Open-label surgical trials for Parkinson disease: time for reconsideration. Ann Neurol 2011;70:5-8.
5. Schuepbach WM, Rau J, Knudsen K, et al. Neurostimulation for Parkinson's disease with early motor complications. N Engl J Med 2013;368:610-622.
6. Marks WJ, Jr., Ostrem JL, Verhagen L, et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol 2008;7:400-408.
7. Marks WJ, Jr., Bartus RT, Siffert J, et al. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol 2010;9:1164-1172.
8. Bartus RT, Herzog CD, Chu Y, et al. Bioactivity of AAV2-neurturin gene therapy (CERE-120): differences between Parkinson's disease and nonhuman primate brains. Mov Disord 2011;26:27-36.
9. Bartus RT, Baumann TL, Siffert J, et al. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology 2013;80:1698-1701.
10. Olanow CW, Bartus OT, Baumann TL, et al. Gene delivery of AAV2-neurturin to putamen and substantia nigra in Parkinson's disease: a double-blind, randomized, controlled trial. Ann Neurol 2015;78:248-257.
11. Palfi S, Gurruchaga JM, Ralph GS, et al. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet 2014;383:1138-1146.
12. Paganini M, Biggeri A, Romoli AM, et al. Fetal striatal grafting slows motor and cognitive decline of Huntington's disease. J Neurol Neurosurg Psychiatry 2014;85:974-981.
13. Freed CR, Greene PE, Breeze RE, et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med 2001;344:710-719.
14. Olanow CW, Goetz CG, Kordower JH, et al. A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol 2003;54:403-414.
15. Deep-Brain Stimulation for Parkinson's Disease Study Group. Deep-brain stimulation of the subthalamic nucleus or the pars interna of the globus pallidus in Parkinson's disease. N Engl J Med 2001;345:956-963.
16. Olanow CW, Hauser RA, Jankovic J, et al. A randomized, double-blind, placebo-controlled, delayed start study to assess rasagiline as a disease modifying therapy in Parkinson's disease (the ADAGIO study): rationale, design, and baseline characteristics. Mov Disord 2008;23:2194-2201.
17. Olanow CW, Rascol O, Hauser R, et al. A double-blind, delayed-start trial of rasagiline in Parkinson's disease. N Engl J Med 2009;361:1268-1278.
18. Schapira AH, McDermott MP, Barone P, et al. Pramipexole in patients with early Parkinson's disease (PROUD): a randomised delayed-start trial. Lancet Neurol 2013;12:747-755.
19. PD Med Collaborative Group, Gray R, Ives N, et al. Long-term effectiveness of dopamine agonists and monoamine oxidase B inhibitors compared with levodopa as initial treatment for Parkinson's disease (PD MED): a large, open-label, pragmatic randomised trial. Lancet 2014;384:1196-1205.
20. Writing Group for the NET-PD Investigators, Kieburtz K, Tilley BC, et al. Effect of creatine monohydrate on clinical progression in patients with Parkinson disease: a randomized clinical trial. JAMA 2015;313:584-593.
21. Olanow CW, Kieburtz K. Defining disease-modifying therapies for PD-a road map for moving forward. Mov Disord 2010;25:1774-1779.
22. NET-PD Investigators. S453. WIP pioglitazone as a potential disease modifying therapy in early Parkinson's disease. Net-PD Investigators. Ann Neurol 2014;76:(Suppl 18):S1-S151.
23. Tabrizi SJ, Scahill RI, Owen G, et al. Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data. Lancet Neurol 2013;12:637-649.
24. Long JD, Paulsen JS, Marder K, et al. Tracking motor impairments in the progression of Huntington's disease. Mov Disord 2014; 29:311-319.
25. Paulsen JS, Long JD, Ross CA, et al. Prediction of manifest Huntington's disease with clinical and imaging measures: a prospective observational study. Lancet Neurol 2014;13:1193-1201.
26. Dorsey ER, Beck CA, Darwin K, et al. Natural history of Huntington disease. JAMA Neurol 2013;70:1520-1530.
27. Padowski JM, Weaver KE, Richards TL, et al. Neurochemical correlates of caudate atrophy in Huntington's disease. Mov Disord 2014;29:327-335.
28. Rosas HD, Doros G, Gevorkian S, et al. PRECREST: a phase II prevention and biomarker trial of creatine in at-risk Huntington disease. Neurology 2014;82:850-857.
29. Parkinson Study Group PRECEPT Investigators. Mixed lineage kinase inhibitor CEP-1347 fails to delay disability in early Parkinson disease. Neurology 2007;69:1480-1490.
30. Ravina B, Tanner C, Dieuliis D, et al. A longitudinal program for biomarker development in Parkinson's disease: a feasibility study. Mov Disord 2009;24:2081-2090.
31. Tanner CM, Meng CC, Ravina B, et al. A practical approach to remote longitudinal follow-up of Parkinson's disease: the FOUND study. Mov Disord 2014;29:743-749.
32. Stocchi F, Hauser RA, Olanow W, Rascol O. Incidence of key clinical milestones in rasagiline treated PD patients 5-8 years after diagnosis. Mov Disord 2014;29:S268-S269.
33. Rascol O, Olanow W, Stocchi F, Hauser RA. UPDRS progression in rasagiline treated PD patients 5-8 years from diagnosis. Mov Disord 2014;29(Suppl 1):700.
34. Kang JH, Irwin DJ, Chen-Plotkin AS, et al. Association of cerebrospinal fluid beta-amyloid 1-42, T-tau, P-tau181, and alpha-synuclein levels with clinical features of drug-naive patients with early Parkinson disease. JAMA Neurol 2013;70:1277-1287.
35. O'Neill RT, Temple R. The prevention and treatment of missing data in clinical trials: an FDA perspective on the importance of dealing with it. Clin Pharmacol Ther 2012;91:550-554.
36. Hauser RA, Olanow CW, Kieburtz KD, et al. Tozadenant (SYN115) in patients with Parkinson's disease who have motor fluctuations on levodopa: a phase 2b, double-blind, randomised trial. Lancet Neurol 2014;13:767-776.
37. Hauser RA, Cantillon M, Pourcher E, et al. Preladenant in patients with Parkinson's disease and motor fluctuations: a phase 2, double-blind, randomised trial. Lancet Neurol 2011;10:221-229.
38. Rascol O, Hauser R, Stocchi F, et al. Post-hoc analyses of phase-3 data with preladenant, an adenosine 2a antagonist, in patients with Parkinson's disease (P7.088). Neurology 2014;82: P7.088.
39. Dorsey ER, Venkataraman V, Grana MJ, et al. Randomized controlled clinical trial of "virtual house calls" for Parkinson disease. JAMA Neurol 2013;70:565-570.
40. Achey MA, Beck CA, Beran DB, et al. Virtual house calls for Parkinson disease (Connect.Parkinson): study protocol for a randomized, controlled trial. Trials 2014;15:465.
41. Tsanas A, Little MA, McSharry PE, Ramig LO. Accurate telemonitoring of Parkinson's disease progression by noninvasive speech tests. IEEE Trans Biomed Eng 2010;57:884-893.
42. Tsanas A, Little MA, McSharry PE, Spielman J, Ramig LO. Novel speech signal processing algorithms for high-accuracy classification of Parkinson's disease. IEEE Trans Biomed Eng 2012;59:1264-1271.
43. Arora S, Venkataraman V, Zhan A, et al. Detecting and monitoring the symptoms of Parkinson's disease using smartphones: a pilot study. Parkinsonism Relat Disord 2015;21:650-653.
44. Dragalin V. An introduction to adaptive designs and adaptation in CNS trials. Eur Neuropsychopharmacol 2011;21:153-158.
45. Gallo P, Anderson K, Chuang-Stein C, et al. Viewpoints on the FDA draft adaptive designs guidance from the PhRMA working group. J Biopharm Stat 2010;20:1115-1124.
46. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Guidance for industry considerations for the design of early-phase clinical trials of cellular and gene therapy products. 2013. Available at: http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM359073.pdf. 20 Aug 2015.
47. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research. Guidance for Industry expedited programs for serious conditions-drugs and biologics. 2014. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf. 20 Aug 2015.
48. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research. Guidance for industry. Alzheimer's disease: developing drugs for the treatment of early stage. 2013. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM338287.pdf. 20 Aug 2015.
49. Kozauer N, Katz R. Regulatory innovation and drug development for early-stage Alzheimer's disease. N Engl J Med 2013;368:1169-1171.
50. Siddiqui O, Hung HM, O'Neill R. MMRM vs. LOCF: a comprehensive comparison based on simulation study and 25 NDA datasets. J Biopharm Stat 2009;19:227-246.
51. Dorsey ER, Venuto C, Venkataraman V, Harris DA, Kieburtz K. Novel methods and technologies for 21st-century clinical trials: a review. JAMA Neurol 2015;72:582-588.