The BEST Trial: Biomarkers for Evaluating Spine Treatments

NCT ID: NCT05396014

Last Updated: 2025-07-23

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

1014 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-09-12

Study Completion Date

2024-10-22

Brief Summary

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The BEST Trial (Biomarkers for Evaluating Spine Treatments) is a National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)-sponsored clinical trial being conducted through the NIH Helping to End Addiction Long-term (HEAL) Initiative's Back Pain Consortium (BACPAC) Research Program. The primary objective of this trial is to inform a precision medicine approach to the treatment of Chronic Low-Back Pain by estimating the optimal treatment or combination of treatments based on patient features and response to the initial treatment. Interventions being evaluated in this trial are: (1) enhanced self-care (ESC), (2) acceptance and commitment therapy (ACT), (3) evidence-based exercise and manual therapy (EBEM), and (4) duloxetine.

Detailed Description

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Each participant will complete an initial screening call and enrollment visit, followed by a 2-week run-in period, two consecutive 12-week treatment periods, and a 12-week post-treatment follow-up period. Upon completion of the run-in period, participant eligibility will be reassessed based on adherence to study protocol. Participants who no longer meet eligibility criteria will be considered screen failures and discontinued from the study.

All participants will undergo phenotyping assessments at Visit 0, 1 and 2 corresponding to baseline, the end of the first 12-week intervention period, and the end of the second 12-week intervention period, respectively. A subset of participants will undergo additional phenotyping, consisting of a more comprehensive set of phenotyping assessments, at the same visits.

Pain, Enjoyment of Life, and General Activity (PEG) and Patient Global Impressions Scale (PGIC) will be assessed at 6 weeks (midpoint of intervention period one), 12 weeks (Visit 1), 18 weeks (midpoint of intervention period two), 24 weeks (Visit 2), and 36 weeks post-baseline (12 weeks after intervention period two). Basic safety assessments will also be performed at these time points to assess participant tolerability to their current study intervention(s). Patients who are unable to tolerate their assigned study treatment will be educated on how to safely discontinue their current treatment plan but will otherwise remain in the study.

Conditions

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Chronic Low-back Pain

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Sequential Multiple Assignment Randomized Trial (SMART)
Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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Treatment Period 1: Enhanced Self-Care (ESC)

This arm includes participants who are randomized to ESC in Treatment Period 1.

Depending on their response to Treatment 1 measured at 12 Weeks post-initial randomization, participants in this arm will stay on ESC or be randomized to augment ESC with an additional treatment during Treatment Period 2.

Group Type ACTIVE_COMPARATOR

Enhanced Self-Care (ESC)

Intervention Type BEHAVIORAL

The Enhanced Self-Care intervention will be comprised of educational modules on evidence-based cognitive-behavioral self-management skills for pain. These modules will be provided digitally for self-administration over a period of 12 weeks. There will be no therapist associated with the delivery of these educational materials; however, after the first four modules, email or text messages will be used to make personalized recommendations for accessing additional modules based upon identified problems from the baseline assessment. Additionally, the walking program module will utilize Fitbit step tracking to allow participants to monitor their walking progress.

Treatment Period 1: Acceptance and Commitment Therapy (ACT)

This arm includes participants who are randomized to ACT in Treatment Period 1.

Depending on their response to Treatment 1 measured at 12 Weeks post-initial randomization, participants in this arm will stay on ACT, augment ACT with an additional treatment, or switch to a new treatment during Treatment Period 2.

Group Type ACTIVE_COMPARATOR

Acceptance and Commitment Therapy (ACT)

Intervention Type BEHAVIORAL

ACT is a form of cognitive behavioral therapy that is well established for the treatment of chronic pain. The goal of ACT is to build psychological flexibility thereby interrupting pain avoidance behavior patterns. Participants randomized to ACT will take part in 12 sessions over the course of 12 weeks. Sessions will be delivered as a combination of 4 remote face-to-face visits with a therapist and 8 therapist-supported online sessions (self-directed online modules supported by provider coaching). Online sessions will focus on helping participants accept pain, connect with negative thoughts and emotions, develop mindfulness and identify and commit to values and goals that are important to them. During face-to-face sessions with the therapist, participants will be encouraged to share their experience of skills practice and mastery, provide examples of skill use at home, and describe what barriers they encountered.

Treatment Period 1: Evidence-Based Exercise and Manual Therapy (EBEM)

This arm includes participants who are randomized to EBEM in Treatment Period 1.

Depending on their response to Treatment 1 measured at 12 Weeks post-initial randomization, participants in this arm will stay on EBEM, augment EBEM with an additional treatment, or switch to a new treatment during Treatment Period 2.

Group Type ACTIVE_COMPARATOR

Evidence-Based Exercise and Manual Therapy (EBEM)

Intervention Type BEHAVIORAL

Licensed physical therapists (PTs) or Doctors of Chiropractic (DCs) will rely on evidence-based guidance to direct decision-making on the particular type of manual and exercise therapy that may be best suited to an individual study participant. Special attention will be paid to the clinician's choice of language in regard to the purpose and expected outcomes of manual therapy in order to avoid enhancing catastrophizing ideations or preference for passive interventions. A total of 10 sessions will be provided over an 8-week treatment period. Two sessions per week are provided in the first two weeks followed by weekly sessions over the next 6 weeks. Treatment sessions will last approximately 60 minutes each.

Treatment Period 1: Duloxetine

This arm includes participants who are randomized to Duloxetine in Treatment Period 1.

Depending on their response to Treatment 1 measured at 12 Weeks post-initial randomization, participants in this arm will stay on Duloxetine, augment Duloxetine with an additional treatment, or switch to a new treatment during Treatment Period 2.

Group Type ACTIVE_COMPARATOR

Duloxetine

Intervention Type DRUG

Duloxetine is a serotonin norepinephrine reuptake inhibitor (SNRI) that is FDA-approved for use in Chronic Low-Back Pain, and, as such, is included as a recommended therapy in nearly all current treatment guidelines for low back pain. Study participants will be treated with duloxetine for 12 weeks during the active treatment phase. At the time of randomization, the approved drug pharmacy at each study site will dispense between 185 and 192 duloxetine 30 mg capsules and provide to participants. This will ensure enough capsules to maintain up to a 60 mg dosage through the 12-week intervention phase and to taper the dose in the 13th week if needed.

Interventions

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Enhanced Self-Care (ESC)

The Enhanced Self-Care intervention will be comprised of educational modules on evidence-based cognitive-behavioral self-management skills for pain. These modules will be provided digitally for self-administration over a period of 12 weeks. There will be no therapist associated with the delivery of these educational materials; however, after the first four modules, email or text messages will be used to make personalized recommendations for accessing additional modules based upon identified problems from the baseline assessment. Additionally, the walking program module will utilize Fitbit step tracking to allow participants to monitor their walking progress.

Intervention Type BEHAVIORAL

Acceptance and Commitment Therapy (ACT)

ACT is a form of cognitive behavioral therapy that is well established for the treatment of chronic pain. The goal of ACT is to build psychological flexibility thereby interrupting pain avoidance behavior patterns. Participants randomized to ACT will take part in 12 sessions over the course of 12 weeks. Sessions will be delivered as a combination of 4 remote face-to-face visits with a therapist and 8 therapist-supported online sessions (self-directed online modules supported by provider coaching). Online sessions will focus on helping participants accept pain, connect with negative thoughts and emotions, develop mindfulness and identify and commit to values and goals that are important to them. During face-to-face sessions with the therapist, participants will be encouraged to share their experience of skills practice and mastery, provide examples of skill use at home, and describe what barriers they encountered.

Intervention Type BEHAVIORAL

Evidence-Based Exercise and Manual Therapy (EBEM)

Licensed physical therapists (PTs) or Doctors of Chiropractic (DCs) will rely on evidence-based guidance to direct decision-making on the particular type of manual and exercise therapy that may be best suited to an individual study participant. Special attention will be paid to the clinician's choice of language in regard to the purpose and expected outcomes of manual therapy in order to avoid enhancing catastrophizing ideations or preference for passive interventions. A total of 10 sessions will be provided over an 8-week treatment period. Two sessions per week are provided in the first two weeks followed by weekly sessions over the next 6 weeks. Treatment sessions will last approximately 60 minutes each.

Intervention Type BEHAVIORAL

Duloxetine

Duloxetine is a serotonin norepinephrine reuptake inhibitor (SNRI) that is FDA-approved for use in Chronic Low-Back Pain, and, as such, is included as a recommended therapy in nearly all current treatment guidelines for low back pain. Study participants will be treated with duloxetine for 12 weeks during the active treatment phase. At the time of randomization, the approved drug pharmacy at each study site will dispense between 185 and 192 duloxetine 30 mg capsules and provide to participants. This will ensure enough capsules to maintain up to a 60 mg dosage through the 12-week intervention phase and to taper the dose in the 13th week if needed.

Intervention Type DRUG

Other Intervention Names

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Cymbalta

Eligibility Criteria

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Inclusion Criteria

* Ability to read and understand English
* Provision of signed and dated informed consent form(s)
* Willing and able to receive study-related messages and survey links via email
* Willing and able to receive study-related phone calls
* Age 18 years old or older
* Low-back pain for at least 3 months and occurring on at least half the days in the past 6 months
* Contraindicated to no more than one of the study interventions at the time of eligibility assessment(s)
* Eligible to receive at least three of the four study interventions and willing to receive any intervention for which they are eligible
* A PEG score 4 or higher prior to the Run-in period
* Willing and able to undergo required phenotyping
* Regular reliable access to an internet-enabled device such as a smart phone, tablet, or laptop computer
* Meet Run-in period engagement eligibility criteria:

o Completion of two Run-in study information modules prior to period 1 randomization (Visit 0)
* Low-back pain more severe than pain in other parts of the body
* Available to complete the full study protocol (approximately 9 months)

Exclusion Criteria

An individual who meets any of the following criteria will be excluded from participation in this study:

* Pregnant at the time of Visit 0 (Baseline)
* Affirmative participant response to any of the following conditions:

* Progressive neurodegenerative disease
* History of discitis osteomyelitis (spine infection) or spine tumor
* History of ankylosing spondylitis, rheumatoid arthritis, polymyalgia rheumatica, psoriatic arthritis, or lupus
* History of cauda equina syndrome or spinal radiculopathy with functional motor deficit (strength \<4/5 on manual motor testing)
* Diagnosis of any vertebral fracture in the last 6 months
* Osteoporosis requiring pharmacologic treatment other than vitamin D, calcium supplements, or bisphosphonates.
* History of any bone-related cancer or cancer that metastasized to the bone
* Currently in treatment for any non-skin cancer or plan to start non-skin cancer treatment in the next 12 months
* History of any non-skin cancer treatment in the last 24 months
* Visual or hearing difficulties that would preclude participation
* Uncontrolled drug/alcohol addiction
* Individuals actively pursuing disability or workers compensation or involved in active personal injury-related litigation
* Currently participating in another interventional pain study
* Any condition that, in the opinion of the investigator, would preclude the patient from being able to safely participate in in the trial
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

NIH

Sponsor Role collaborator

University of North Carolina, Chapel Hill

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kevin Anstrom, PhD

Role: PRINCIPAL_INVESTIGATOR

UNC Chapel Hill

Matthew Mauck, PhD

Role: PRINCIPAL_INVESTIGATOR

UNC Chapel Hill

Gwendolyn Sowa, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh

Daniel Clauw, MD

Role: PRINCIPAL_INVESTIGATOR

University of Michigan

Locations

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Stanford University

Redwood City, California, United States

Site Status

University of California, San Diego

San Diego, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

University of Kansas Health System

Kansas City, Kansas, United States

Site Status

Massachusetts General Hospital/Brigham Women's Hospital, Harvard Medical School

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

University of North Carolina Hospital Pain Management Clinic

Chapel Hill, North Carolina, United States

Site Status

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Countries

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United States

References

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Rowland B, Barth KS, Bell KM, Brooks AK, Chadwick AL, Cleven A, Hurley RW, Mackey S, Patel KV, Piva SR, Schneider MJ, Al-Kadhi F, Asante-Nketiah B, Bagaason S, Batorsky A, Borckardt JJ, Bowden AE, Carey TS, Castellanos J, Chen L, Chidgey B, Dalton D, Dufour JS, Eberting JL, Eller SM, Fields AJ, Fritz JM, Fu A, Ghulamhussain I, Goolsby RW, Greco CM, Grim S, Gunn CA, Hanes L, Harris RE, Harte SE, Hassett AL, Helton K, Hoffmeyer A, Ivanova A, Jones Berkeley S, Kaplan C, Kidwell KM, Knapik GG, Kosorok MR, Kurillo G, Li D, Lobo R, Long J, Lotz JC, Mageswaran P, Majumdar S, Mao J, Marras WS, McCracken LM, McCumber M, McLean SA, McMillan M, Mehling W, Mendoza R, Mitchell UH, Napadow V, O'Neill C, Pearson S, Peltier S, Rundell SD, Ryser S, Schrepf A, Schulze E, Sperger J, Vo N, Wallace MS, Wampler AM, Wasan AD, Weaver TE, Weber KA 2nd, Wilcox L, Williams DA, Wilson L, Woo JE, Zeidan F, Zhao B, Zhou B, Anstrom KJ, Clauw DJ, Sowa GA, Mauck MC. Baseline characteristics of participants in the Biomarkers for Evaluating Spine Treatments clinical trial: a sequential multiple assignment randomized trial for chronic low back paindagger. Pain Med. 2025 Aug 5:pnaf073. doi: 10.1093/pm/pnaf073. Online ahead of print.

Reference Type DERIVED
PMID: 40794568 (View on PubMed)

Jones Berkeley S, Wedin S, Patidar SM, Margolies SO, Goetzinger AM, Mauck MC, Wasan AD, McCracken LM. Design and implementation of online acceptance and commitment therapy with enhanced therapist support for chronic low back pain (ACT for PAIN). Pain Med. 2025 Aug 1;26(8):451-458. doi: 10.1093/pm/pnaf026.

Reference Type DERIVED
PMID: 40249098 (View on PubMed)

Mauck MC, Barth KS, Bell KM, Brooks AK, Chadwick AL, Gunn CA, Hurley RW, Ivanova A, Piva SR, Schneider MJ, Bailey JF, Bagaason S, Batorsky A, Borckardt JJ, Bowden AE, Carey TS, Castellanos J, Chen L, Chidgey B, Dalton D, Dufour JS, Fields AJ, Fritz JM, Goolsby RW, Greco CM, Harris RE, Harte S, Hassett AL, Hoffmeyer A, Jones Berkeley S, Kaplan C, Kidwell KM, Knapik GG, Kosorok MR, Kurillo G, Lobo R, Lotz JC, Mackey S, Mageswaran P, Majumdar S, Mao J, Marras WS, McCumber M, McLean SA, Mehling W, Mitchell UH, Napadow VJ, O'Neill C, Patel KV, Peltier S, Psioda M, Rowland B, Rundell SD, Schrepf A, Sperger J, Vo N, Wallace MS, Wasan AD, Weaver TE, Weber KA 2nd, Williams DA, Wilson L, Zeidan F, Zhao B, Anstrom KJ, Clauw DJ, Sowa GA. The design and rationale of the Biomarkers for Evaluating Spine Treatments trial: a sequential multiple assignment randomized trial. Pain Med. 2025 Sep 1;26(9):538-553. doi: 10.1093/pm/pnaf032.

Reference Type DERIVED
PMID: 40205455 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

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1U24AR076730

Identifier Type: NIH

Identifier Source: secondary_id

View Link

21-1972

Identifier Type: -

Identifier Source: org_study_id

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