Appendix D

Virtual Clinical Trials Presented by Speakers at the Workshop

REMOTE – See Appendix C

AT-HOME PD

• Year Posted: 2018
• Sponsor: National Institutes of Health
• Disease: Parkinson’s disease
• Intervention: Observational
• Phase: Not applicable (recruited participants from former Phase 3 Parkinson’s disease trials)¹
• Digital Health Technology: Video visits and smartphone

AT-HOME PD conducted longitudinal follow-up for participants in two prior Phase 3 Parkinson’s disease trials, using telemedicine and smartphone platforms for monitoring outcomes. This study has the potential to simplify long-term follow-up of large cohorts and will test the feasibility of new technology platforms to fulfill this purpose. For more information, please view the study record (ClinicalTrials.gov, 2019c).

KALM

• Year Posted: N/A
• Sponsor: 1747
• Disease: Anxiety and insomnia

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¹ AT-HOME PD recruited from STEADY PD III (ClinicalTrials.gov, 2019a) and SURE PD3 (ClinicalTrials.gov, 2018b), which were launched in 2014 and 2016, respectively.

• Intervention: Kava and Valerian (dietary supplements)
• Phase: Not applicable
• Digital Health Technology: Web-based

This study assessed efficacy of kava and valerian to treat anxiety and insomnia, with recruitment and consent taking place entirely online and study compounds being mailed directly to participants. KALM is likely the first study to test the feasibility of conducting a randomized, blinded trial over the Internet, and it was able to demonstrate the feasibility of such a model (Jacobs et al., 2005).

Omega-3 Fatty Acids for Hyperactivity Treatment in Autism Spectrum Disorder

• Year Posted: 2012
• Sponsor: Hugo Moser Research Institute at Kennedy Krieger, Inc.
• Disease: Autism spectrum disorder (ASD) and hyperactivity
• Intervention: Omega-3 fatty acids
• Phase: Phase 2
• Digital Health Technology: Web-based

This study evaluated the efficacy of an omega-3 fatty acid supplement in reducing hyperactivity in children with autism; it used e-mail invitations and Web-based enrollment and study management. It demonstrated the feasibility of conducting a Web-based clinical trial in children with ASD, with benefits including a fast enrollment rate (Bent et al., 2014). For more information, see the study record (ClinicalTrials.gov, 2018a).

TOPAZ (Trial of Parkinson’s Disease and Zoledronic Acid)

• Year Posted: 2019
• Sponsor: National Institute on Aging
• Disease: Osteoporosis and Parkinson’s disease
• Intervention: Zoledronic acid
• Phase: Phase 4
• Digital Health Technology: Web- and video-based

This study evaluated the efficacy of Zoledronic acid in reducing bone fracture risk in elderly Parkinson’s disease patients. The participants enroll through interactive electronic consent, and if deemed eligible to join the study will be scheduled for a video-based telemedicine assessment to confirm Parkinson’s disease diagnosis. A nurse home visit will also be scheduled to confirm final eligibility and administer the investigational drug. For more information, see the study record (ClinicalTrials.gov, 2019d).

Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-Term Effectiveness (ADAPTABLE)

• Year Posted: 2016
• Sponsor: Duke University
• Disease: Atherosclerotic cardiovascular disease
• Intervention: Aspirin
• Phase: Not applicable
• Digital Health Technology: Combination of electronic health records, a patient portal, and patient-reported outcomes

ADAPTABLE seeks to compare the effectiveness of two doses of aspirin in the secondary prevention of patients with established atherosclerotic cardiovascular disease and is using a combination of electronic health record searches and patient-reported outcomes for follow-up. An important component of ADAPTABLE is its aim to improve engagement with participants, their health care providers, and trial investigators within the trial infrastructure. For more information, see study record (ClinicalTrials.gov, 2019b).

Lithium Carbonate Treatment for Amyotrophic Lateral Sclerosis

• Year Posted: N/A
• Sponsor: PatientsLikeMe
• Disease: Amyotrophic lateral sclerosis (ALS)
• Intervention: Lithium carbonate
• Phase: N/A
• Digital Health Technology: Web-based

PatientsLikeMe tested the efficacy of lithium carbonate by capturing information on the study’s participants via an online data collection tool. Though this study did not show a treatment effect, it did note advantages of collecting patient-reported outcome data online, including speed, patient access, and availability of control participants (Wicks et al., 2011).

Lunasin Virtual Study (PatientsLikeMe and Duke ALS Clinic)

• Year Posted: 2016
• Sponsor: Duke University
• Disease: Amyotrophic lateral sclerosis (ALS)
• Intervention: Lunasin
• Phase: Phase 2
• Digital Health Technology: Hybrid (in-person and Internet-based)

This study investigated if Lunasin could slow progression of ALS and used in-person visits, virtual check-ups via the PatientsLikeMe online platform,

and telephone visits, which co-occurred with virtual visits (for troubleshooting and education on clinical scores). Though the study did not find evidence to support use of Lunasin to slow or reverse progression of ALS, the design of the trial did provide logistical benefits, such as cost efficiency, increased diversity of participants enrolled, and rapid enrollment (Bedlack et al., 2019). For more information, see the study record (ClinicalTrials.gov, 2016a).