Treatment of Middle East respiratory syndrome with a combination of lopinavir/ritonavir and interferon-β1b (MIRACLE trial): Statistical analysis plan for a recursive two-stage group sequential randomized controlled trial

Trials

Volumn 21, Issue 1, 2020, Pages

  Arabi, Yaseen M ^a,b   Asiri, Ayed Y ^c   Assiri, Abdullah M ^d   Aziz Jokhdar, Hani A ^d   Alothman, Adel ^a,b   Balkhy, Hanan H ^a,b   Aljohani, Sameera ^a,b   Al Harbi, Shmeylan ^b,e   Kojan, Suleiman ^a,b   Al Jeraisy, Majed ^b,e   Deeb, Ahmad M ^b,e   Memish, Ziad A ^f,g   Ghazal, Sameeh ^c   Al Faraj, Sarah ^c   Al Hameed, Fahad ^b,h   Alsaedi, Asim ^b,h   Mandourah, Yasser ⁱ   Al Mekhlafi, Ghaleb A ⁱ   Sherbeeni, Nisreen Murad ⁱ   Elzein, Fatehi Elnour ⁱ   Almotairi, Abdullah ^j   Al Bshabshe, Ali ^k   Kharaba, Ayman ^l   Jose, Jesna ^e   Al Harthy, Abdulrahman ^m   Al Sulaiman, Mohammed ^m   Mady, Ahmed ^m,n   Fowler, Robert A ^o,p   Hayden, Frederick G ^q   Al Dawood, Abdulaziz ^a,b   Abdelzaher, Mohamed ^r,s   Bajhmom, Wail ^t   Hussein, Mohamed A ^b,e   more..

^a KING SAUD BIN ABDULAZIZ UNIVERSITY FOR HEALTH SCIENCES   (Saudi Arabia)

^b KING ABDULLAH INTERNATIONAL MEDICAL RESEARCH CENTER   (Saudi Arabia)

^c Paediatric Department Prince Mohamed Bin Abdulaziz Hospital National Guard Health Authority   (Saudi Arabia)

^d MINISTRY OF HEALTH   (Saudi Arabia)

^e KING SAUD BIN ABDULAZIZ UNIVERSITY FOR HEALTH SCIENCES   (Saudi Arabia)

^f ALFAISAL UNIVERSITY   (Saudi Arabia)

^g EMORY UNIVERSITY   (United States)

^h KING SAUD BIN ABDULAZIZ UNIVERSITY FOR HEALTH SCIENCES   (Saudi Arabia)

ⁱ The General Directorate of Armed Forces Health Services   (Saudi Arabia)

^j KING FAHAD MEDICAL CITY   (Saudi Arabia)

^k KING KHALID UNIVERSITY   (Saudi Arabia)

^l KING FAHAD HOSPITAL   (Saudi Arabia)

^m KING SAUD MEDICAL CITY   (Saudi Arabia)

ⁿ TANTA UNIVERSITY HOSPITAL   (Egypt)

^o UNIVERSITY OF TORONTO   (Canada)

^p SUNNYBROOK RESEARCH INSTITUTE   (Canada)

^q UNIVERSITY OF VIRGINIA   (United States)

^r KING SAUD BIN ABDULAZIZ UNIVERSITY FOR HEALTH SCIENCES   (Saudi Arabia)

^s CAIRO UNIVERSITY   (Egypt)

^t KING FAHAD GENERAL HOSPITAL   (Saudi Arabia)

more..

Author keywords

Antiviral; Clinical trial; Coronavirus; Interferon 1b; Lopinavir ritonavir; MERS; Protocol; Statistical analysis plan

Indexed keywords

INTERFERON BETA SERINE; LOPINAVIR PLUS RITONAVIR; ANTIVIRUS AGENT; LOPINAVIR; LOPINAVIR-RITONAVIR DRUG COMBINATION; RITONAVIR;

ARTICLE; CLINICAL OUTCOME; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; HOSPITAL PATIENT; HUMAN; INTENTION TO TREAT ANALYSIS; MATHEMATICAL MODEL; MIDDLE EAST RESPIRATORY SYNDROME; MORTALITY; MULTICENTER STUDY; RANDOMIZED CONTROLLED TRIAL; STATISTICAL ANALYSIS; CORONAVIRUS INFECTION; DRUG COMBINATION; DRUG EFFECT; HOST PATHOGEN INTERACTION; MIDDLE EAST RESPIRATORY SYNDROME CORONAVIRUS; MULTICENTER STUDY (TOPIC); PATHOGENICITY; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAUDI ARABIA; TIME FACTOR; TREATMENT OUTCOME; VIROLOGY;

ANTIVIRAL AGENTS; CORONAVIRUS INFECTIONS; DATA INTERPRETATION, STATISTICAL; DOUBLE-BLIND METHOD; DRUG COMBINATIONS; HOST-PATHOGEN INTERACTIONS; HUMANS; INTERFERON BETA-1B; LOPINAVIR; MIDDLE EAST RESPIRATORY SYNDROME CORONAVIRUS; MULTICENTER STUDIES AS TOPIC; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RITONAVIR; SAUDI ARABIA; TIME FACTORS; TREATMENT OUTCOME;

EID: 85077479986     PISSN: None     EISSN: 17456215     Source Type: Journal    
DOI: 10.1186/s13063-019-3846-x     Document Type: Article

References (17)

1. Arabi YM, Balkhy HH, Hayden FG, Bouchama A, Luke T, Baillie JK, Al-Omari A, Hajeer AH, Senga M, Denison MR, et al. Middle East respiratory syndrome. N Engl J Med. 2017;376:584-94.
2. Arabi Y, Shalhoub S, Mandourah Y, Al-Hameed F, Al-Omari A, Al Qasim E, Jose J, Alraddadi B, Almotairi A, Al Khatib K, Abdulmomen A, Qushmaq I, Sindi AA, Mady A, Solaiman O, Al-Raddadi R, Maghrabi K, Ragab A, Al Mekhlafi GA, Balkhy HH, Al Harthy A, Kharaba A, Gramish JA, Al-Aithan AM, Al-Dawood A, Merson L, Hayden FG, Fowler R, Saudi Critical Care Trials Group. Ribavirin and interferon therapy for critically ill patients with the Middle East respiratory syndrome: a multicenter observational study, Clin Infect Dis 2019; ciz544. doi: https://doi.org/10.1093/cid/ciz544.
3. Arabi YM, Alothman A, Balkhy HH, Al-Dawood A, AlJohani S, Al Harbi S, Kojan S, Al Jeraisy M, Deeb AM, Assiri AM, et al. Treatment of Middle East respiratory syndrome with a combination of lopinavir-ritonavir and interferon-beta1b (MIRACLE trial): study protocol for a randomized controlled trial. Trials. 2018;19:81.
4. Chang M. Adaptive design theory and implementation using SAS and R. 2nd ed. Boca Raton: Chapman & Hall/CRC; 2014. p. 153-80.
5. Liu Q, Anderson KM, Pledger GW. Benefit-risk evaluation of multi-stage adaptive designs. Seq Anal. 2004;23:317-31.
6. US Food and Drug Administration (FDA). Draft guidance for industry on enrichment strategies for clinical trials to support approval of human drugs and biological products; availability. 2012. https://www.federalregister.gov/documents/2012/12/17/2012-30274/draft-guidance-for-industry-on-enrichment-strategies-for-clinical-trials-to-support-approval-of. Accessed 26 June 2019.
7. Wassmer G, Eisebitt R, Coburger S. Flexible interim analyses in clinical trials using multistage adaptive test designs. Drug Inf J. 2001;35:1131-46.
8. Schafer H, Muller HH. Modification of the sample-size and the schedule of interim analyses in survival trials based on data inspections. Stat Med. 2001;20:3741-51.
9. Moher D, Hopewell S, Schulz KF, Montori V, Gotzsche PC, Devereaux PJ, Elbourne D, Egger M, Altman DG. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c869.
10. Schulz KF, Altman DG, Moher D, Group C. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c332.
11. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use: Good Clinical Practice (GCP) guideline. https://database.ich.org/sites/default/files/E6_R2_Addendum.pdf.
12. The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH): statistical principles for clinical trials. https://database.ich.org/sites/default/files/E9_Guideline.pdf.
13. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use: E3-structure and content of clinical study reports. https://database.ich.org/sites/default/files/E3_Guideline.pdf.
14. Gamble C, Krishan A, Stocken D, Lewis S, Juszczak E, Dore C, Williamson PR, Altman DG, Montgomery A, Lim P, et al. Guidelines for the content of statistical analysis plans in clinical trials. JAMA. 2017;318:2337-43.
15. Srivastava DK, Rai SN, Pan J. Robustness of an odds-ratio test in a stratified group sequential trial with a binary outcome measure. Biom J. 2007;49:351-64.
16. Rubin DB. Multiple imputation after 18+ years. J Am Stat Assoc. 1996;91:473-89.
17. Benjamini Y, Hochberg Y. Controlling the false discovery rate: a practical and powerful approach to multiple testing. J R Stat Soc Ser B Methodol. 1995;57:289-300.