Abstract Details

Title Barriers to participation in a prospective clinical trial for schwannomatosis-related pain

Topic Neuro-oncology

Presentation(s) P7 - Poster Session 7 (8:00 AM-9:00 AM)

Poster/Presentation
Number 11-003

Objective To understand barriers to patient participation in the first therapeutic trial for schwannomatosis (SWN)

Background

SWN is a rare neurogenetic condition characterized by severe chronic pain. A clinical trial of tanezumab, an anti-nerve growth factor antibody, was initiated for SWN-related pain (NCT04163419).

Design/Methods

We prospectively tracked recruitment outcomes for this single-institution, phase 2, randomized, double-blind, placebo-controlled trial. Eligible subjects were ≥18 years old with moderate-to-severe SWN-related pain (11-point Numerical Rating Scale (NRS-11) score ≥5) despite treatment with alternate pain medications.

Major exclusion criteria include inability to discontinue non-steroidal anti-inflammatory drugs (NSAIDs) and diagnosis of osteoarthritis.

Results Enrollment began in October 2020 with a target of forty-six participants. Fifty-two subjects were screened as of March 2022, including twenty-five (48%) from the trial institution, seven (13%) from external institutions, and fifteen (29%) through the Children’s Tumor Foundation. Eight subjects enrolled as of March 2022. The most common reasons for non-enrollment included not meeting eligibility criteria (n=18, 41%), time/travel/financial burden (n=10, 23%), and pursuit of other treatment options (n=3, 7%) offering more immediate pain relief (surgery). The most common eligibility criteria which prevented subjects from participating included NRS-11 score <5 (N=6, 33%), diagnosis of osteoarthritis (N=5, 28%), and no confirmed diagnosis of SWN (N=5, 28%).

Conclusions We identified disease-specific, design-related, and drug-specific barriers to participation in this trial. Disease-specific barriers include the rarity of SWN and challenges confirming a SWN diagnosis. Design-related barriers include the use of a single center which amplified economic and logistical barriers of participation, requirements to discontinue NSAIDs, requirements to maintain stable dosing of other pain medications, and short duration of treatment. Lastly, drug-specific barriers included ineligibility due to diagnosis of osteoarthritis. Importantly, participants did not cite the double-blind design as a reason for non-participation. These results will inform design of future prospective clinical trials for SWN-related pain.

Authors/Disclosures
Scott R. Plotkin, MD, PhD, FAAN (Mass General Cancer Center) PRESENTER	Dr. Plotkin has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NFlection Therapeutics. Dr. Plotkin has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Akouos. Dr. Plotkin has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for SonALAsense. Dr. Plotkin has stock in NFlection Therapeutics. Dr. Plotkin has stock in NF2 Therapeutics. Dr. Plotkin has stock in SonALAsense. Dr. Plotkin has received research support from Children's Tumor Foundation. Dr. Plotkin has received research support from Department of Defense. Dr. Plotkin has received research support from National Institutes of Health. Dr. Plotkin has received intellectual property interests from a discovery or technology relating to health care.
Jennifer Da (Massachusetts General Hospital)	No disclosure on file
Vanessa Merker (Massachusetts General Hospital)	The institution of Vanessa Merker has received research support from Children's Tumor Foundation . The institution of Vanessa Merker has received research support from NF Northeast. The institution of Vanessa Merker has received research support from Patient Centered Outcomes Research Institute. The institution of Vanessa Merker has received research support from U.S. Department of Defense . The institution of Vanessa Merker has received research support from Neurofibromatosis Therapeutic Accleration Project. Vanessa Merker has received personal compensation in the range of $10,000-$49,999 for serving as a Science Writer with Neurofibromatosis Network.
Justin T. Jordan, MD, MPH, FAAN (University of Texas Southwestern Medical Center)	Dr. Jordan has received personal compensation for serving as an employee of City of Beverly. An immediate family member of Dr. Jordan has received personal compensation for serving as an employee of Genesis HR Solutions. An immediate family member of Dr. Jordan has received personal compensation for serving as an employee of Children's Hospital. Dr. Jordan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Shepherd Therapeutics. Dr. Jordan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Jordan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Springworks Therapeutics. Dr. Jordan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AstraZeneca. Dr. Jordan has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Continental Casualty Company. Dr. Jordan has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for HennLesperance. Dr. Jordan has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Rhoades McKee. Dr. Jordan has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Wheeler Trigg O'Donnell. Dr. Jordan has or had stock in Navio Theragnostics. An immediate family member of Dr. Jordan has or had stock in OldGate.Dr. Jordan has or had stock in Shepherd Therapeutics.Dr. Jordan has or had stock in Khora TX. The institution of Dr. Jordan has received research support from NIH. The institution of Dr. Jordan has received research support from Department of Defense. The institution of Dr. Jordan has received research support from PCORI. Dr. Jordan has received publishing royalties from a publication relating to health care. Dr. Jordan has received publishing royalties from a publication relating to health care.
Ina Ly, MD (Pappas Center for Neuro-Oncology)	An immediate family member of Dr. Ly has received personal compensation for serving as an employee of Enanta Pharmaceuticals. Dr. Ly has received research support from Department of Defense. An immediate family member of Dr. Ly has received intellectual property interests from a discovery or technology relating to health care.
Alona Muzikansky	No disclosure on file
Michael Parsons (Massachusetts General Hospital)	No disclosure on file
Pamela Wolters (National Cancer Institute)	No disclosure on file
Lei Xu, PhD (Vertex Pharmaceuticals)	No disclosure on file
Mark T. Brown, MD (Pfizer Inc.)	Dr. Brown has received personal compensation for serving as an employee of Pfizer Inc. Dr. Brown has received stock or an ownership interest from Pfizer Inc.
	No disclosure on file

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