Theme 9 Clinical trials and trial design

Acknowledgments:

We would like to thank patients and caregivers who participated in this study and sites who referred them. This study was funded by Genentech, Inc.

Acknowledgments:

We would like to thank the patients and staff of the ALS Center for Hope for their participation in this project. This project was funded by Genentech Inc. FM, WC, MM, AH, WC are employees/shareholders of Roceh Genentech.

Acknowledgments:

p-value communications provided editorial support. WA and SA are employees of MTPA.

Acknowledgments:

Data obtained from the Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT). Supported by grants from the ALS Association (LGCA-333 and 16-IIP-254) and the National Center for Advancing Translational Sciences (R43TR002047).

Acknowledgments:

p-value communications provided editorial support. JP, SA, and WA are employees of Mitsubishi Tanabe Pharma America, Inc.

Acknowledgments:

We thank the patients who participated in FORTITUDE-ALS and their families. Funding was provided by Cytokinetics, Inc.

Acknowledgments:

Funded by ALS Finding a Cure.

Acknowledgments:

CReATe Consortium funding from NCATS and NINDS (U54 NS090291); and PADL-ALS development funding from PCORI Program Award (#CDRN-1306-04631).

Acknowledgments:

This research is supported by a BMBF (the Bundesministerium für Bildung und Forschung) grant PYRAMID in the framework of the ERANET E-RARE program and and JPND (OnWebDUALS).

Acknowledgments:

This study was sponsored by AveXis, Inc., a Novartis company. Editorial assistance was provided by Ashfield Healthcare Communications, part of UDG Healthcare plc, and was funded by AveXis, Inc. The authors report the following potential conflicts of interest: employment by AveXis, Inc., a Novartis company, and potential ownership of Novartis stock or other equities; and employment by Precision Xtract or SSI Strategy, which has received consultancy fees from AveXis, Inc.

Acknowledgments:

Orphazyme A/S.

Acknowledgments:

The REFALS and REFALS-ES studies are sponsored by Orion Pharma, Orion Corporation.

Acknowledgments:

We thank the patients who participated in FORTITUDE-ALS and their families. Funding was provided by Cytokinetics, Inc.

Acknowledgments:

We thank the patients who participated in FORTITUDE-ALS and their families. Funding was provided by Cytokinetics, Inc.

Acknowledgments:

We thank the patients who participated in FORTITUDE-ALS and their families. Funding was provided by Cytokinetics, Inc.

Acknowledgments:

We thank the patients who participated in FORTITUDE-ALS and their families. Funding was provided by Cytokinetics, Inc.

Acknowledgments:

K. Matsuda, Y. Iwaki, M. Makhay, and J. Dojillo are employed by and own stock in MediciNova. S. Yasui declares no conflict in interest.

Acknowledgments:

All authors are employees of Orion Pharma. The study was sponsored by Orion Pharma.

Acknowledgments:

The machine learning models were created using PRO-ACT. This analysis was funded by Mallinckrodt.

Acknowledgments:

Our sincere thanks to the study participants and their carers for taking part in the study. Funding was provided by MNDRIA, grant GIA1558.

Acknowledgments:

This work was supported by the National Research Council, CNPq, Brazil (401922/2014-6).

Acknowledgments:

CNPq (401922/2014-6).

Acknowledgments:

This work was supported by the National Research Council, CNPq, Brazil (401922/2014-6).

Acknowledgments:

This study was supported by grants from the Korea Healthcare Technology R&D Project of the Ministry for Health & Welfare Affairs, Republic of Korea (HI10C1673). This study would not have been possible without the participation of the patients and their families. We also thank the ALS nurse specialists at the Hanyang ALS Clinic (Juyeon Oh and Yeoun Kyung Son) for their helpful support. K.-W.O. and S.H.K. had full access to all the data in this study and take responsibility for the integrity of the data and the accuracy of the data analysis.

Acknowledgments:

Study independently funded by Avera Research Institute with New Biotic providing RaphaLX™. KG owns stock in NBI and KG/MB have received consulting fees from NBI.

Acknowledgments:

Funded by FightMND.

Acknowledgments:

This study is graciously being supported by FightMND.

Acknowledgments:

This study was approved by the Institutional Review board of Kyungpook national university Chilgok hospital hospital.

Acknowledgments:

We would like to thank the patients and investigators for taking part in this post-marketing surveillance. The study was funded and conducted by Mitsubishi Tanabe Pharma Corporation, Inc., Tokyo, Japan (MTPC). MH, SY, KI, and HN are employees of MTPC.

Acknowledgments:

p-value communications provided editorial support. The authors are employees of MTPC (JP, MH, TY, FT, ME, and KT), MTDA (JP), or MTPA (SA and WA).

Acknowledgments:

p-value communications provided editorial support. The study was funded and conducted by MTPA. JH, SA, WA, CM, and SN are employees of MTPA.

Acknowledgments:

The studies were funded and conducted by Mitsubishi Tanabe Pharma Corporation, Tokyo, Japan (MTPC). All authors are employees of MTPC.

Acknowledgments:

The studies were funded and conducted by Mitsubishi Tanabe Pharma Corporation, Tokyo, Japan (MTPC).