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Analysis of Participant Withdrawal in Huntington Disease Clinical Trials.
J Huntingtons Dis. 2017; 6(2):149-156.JH

Abstract

BACKGROUND

Excellent retention in Huntington disease (HD) clinical trials is essential for testing new therapies. The stage of disease, cognitive status, and availability of a care partner may influence retention in HD clinical trials.

OBJECTIVE

We sought to analyze reasons for early withdrawal in three HD clinical trials, and evaluated if either baseline characteristics or follow-up assessments were associated with time to withdrawal.

METHODS

Analyses of participant withdrawal were performed for three randomized, double-blind, placebo-controlled trials including the CARE-HD (coenzyme Q10 and remacemide in HD, n = 347), DOMINO (pilot study of minocycline in HD, n = 114), and 2CARE (coenzyme Q10 in HD, n = 609) trials. Reasons for withdrawal were obtained by review of textual data in the study databases. Participant demographic and clinical characteristics were analyzed as potential predictors of time to withdrawal using Cox-proportional hazards models.

RESULTS

Estimated probabilities of withdrawal at 12 months were 2.9% for CARE-HD, 10.5% for DOMINO, and 5.9% for 2CARE. The top reasons for withdrawal (202 in total), expressed as mean percentage across the three trials, were loss to follow-up (23.2%), death (15.9%), and loss of interest/desire to participate (15.2%). Baseline and time-dependent variables associated with time to withdrawal were mainly motor, behavioral, and functional scores. Age, gender, ethnicity, and educational level were not associated with time to withdrawal in any of the three studies.

CONCLUSIONS

The estimated withdrawal probability at 12 months ranged from 2.9% to 10.5% in the three HD trials considered here. A possible strategy to improve retention of participants in future HD clinical trials is to enroll individuals with higher baseline functional and behavioral status.

Authors+Show Affiliations

Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Nagoya University Graduate School of Medicine, Nagoya, Japan.Department of Neurology, University of Rochester, Rochester, NY, USA. Department of Neurology, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, The State University of New York, Buffalo, NY, USA.Department of Biostatistics and Computational Biology, University of Rochester, Rochester, NY, USA. Department of Neurology, Center for Human Experimental Therapeutics, University of Rochester, Rochester, NY, USA.Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.Department of Biostatistics and Computational Biology, University of Rochester, Rochester, NY, USA.Department of Neurology, Center for Human Experimental Therapeutics, University of Rochester, Rochester, NY, USA.Department of Neurology, Center for Human Experimental Therapeutics, University of Rochester, Rochester, NY, USA.Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

28671136

Citation

Banno, Haruhiko, et al. "Analysis of Participant Withdrawal in Huntington Disease Clinical Trials." Journal of Huntington's Disease, vol. 6, no. 2, 2017, pp. 149-156.
Banno H, Andrzejewski KL, McDermott MP, et al. Analysis of Participant Withdrawal in Huntington Disease Clinical Trials. J Huntingtons Dis. 2017;6(2):149-156.
Banno, H., Andrzejewski, K. L., McDermott, M. P., Murphy, A., Majumder, M., de Blieck, E. A., Auinger, P., Cudkowicz, M. E., & Atassi, N. (2017). Analysis of Participant Withdrawal in Huntington Disease Clinical Trials. Journal of Huntington's Disease, 6(2), 149-156. https://doi.org/10.3233/JHD-170246
Banno H, et al. Analysis of Participant Withdrawal in Huntington Disease Clinical Trials. J Huntingtons Dis. 2017;6(2):149-156. PubMed PMID: 28671136.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Analysis of Participant Withdrawal in Huntington Disease Clinical Trials. AU - Banno,Haruhiko, AU - Andrzejewski,Kelly L, AU - McDermott,Michael P, AU - Murphy,Alyssa, AU - Majumder,Madhurima, AU - de Blieck,Elisabeth A, AU - Auinger,Peggy, AU - Cudkowicz,Merit E, AU - Atassi,Nazem, PY - 2017/7/4/entrez PY - 2017/7/4/pubmed PY - 2018/3/29/medline KW - Huntington disease KW - clinical trial KW - patient selection KW - retention SP - 149 EP - 156 JF - Journal of Huntington's disease JO - J Huntingtons Dis VL - 6 IS - 2 N2 - BACKGROUND: Excellent retention in Huntington disease (HD) clinical trials is essential for testing new therapies. The stage of disease, cognitive status, and availability of a care partner may influence retention in HD clinical trials. OBJECTIVE: We sought to analyze reasons for early withdrawal in three HD clinical trials, and evaluated if either baseline characteristics or follow-up assessments were associated with time to withdrawal. METHODS: Analyses of participant withdrawal were performed for three randomized, double-blind, placebo-controlled trials including the CARE-HD (coenzyme Q10 and remacemide in HD, n = 347), DOMINO (pilot study of minocycline in HD, n = 114), and 2CARE (coenzyme Q10 in HD, n = 609) trials. Reasons for withdrawal were obtained by review of textual data in the study databases. Participant demographic and clinical characteristics were analyzed as potential predictors of time to withdrawal using Cox-proportional hazards models. RESULTS: Estimated probabilities of withdrawal at 12 months were 2.9% for CARE-HD, 10.5% for DOMINO, and 5.9% for 2CARE. The top reasons for withdrawal (202 in total), expressed as mean percentage across the three trials, were loss to follow-up (23.2%), death (15.9%), and loss of interest/desire to participate (15.2%). Baseline and time-dependent variables associated with time to withdrawal were mainly motor, behavioral, and functional scores. Age, gender, ethnicity, and educational level were not associated with time to withdrawal in any of the three studies. CONCLUSIONS: The estimated withdrawal probability at 12 months ranged from 2.9% to 10.5% in the three HD trials considered here. A possible strategy to improve retention of participants in future HD clinical trials is to enroll individuals with higher baseline functional and behavioral status. SN - 1879-6400 UR - https://www.unboundmedicine.com/medline/citation/28671136/Analysis_of_Participant_Withdrawal_in_Huntington_Disease_Clinical_Trials_ L2 - https://content.iospress.com/openurl?genre=article&id=doi:10.3233/JHD-170246 DB - PRIME DP - Unbound Medicine ER -