Clinical trial NCT06003049: A Study to Evaluate Safety and Efficacy of BOTOX in Adults With Moderate COPD
Official title: An Investigator-Initiated, Single Center, Prospective, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate Safety and Efficacy of OnabotulinumtoxinA (BOTOX®) in Adults With Moderate Chronic Obstructive Pulmonary Disease (COPD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: COPD
Lead sponsor: RMW Testing
Sponsor class: INDUSTRY
First posted: 2023-08-21
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is an Investigator-initiated a clinical trial to determine the safety and preliminary efficacy of subcutaneous injections of BOTOX in participants with moderate COPD.

Participants will be treated with either one series of subcutaneous injections of BOTOX or of normal saline solution in the back in the T-1 to T-5 dermatome regions as indicated in Appendix A.

Participants' COPD status will be measured using the COPD Assessment Test (CAT), Pulmonary Function Test (Spirometry), and 6 Minute Walk Test (6MWT).
Primary purpose: TREATMENT

Brief summary: The aim of this clinical trial to determine the safety and effectiveness of treating the COPD symptoms with subcutaneous BOTOX injections in participants with moderate COPD.

Detailed description: This is a prospective, double-blind, randomized, placebo-controlled, investigator-initiated clinical trial to assess the safety, tolerability, and efficacy using an approved drug, BOTOX, in moderate COPD participants.

Forty participants with moderate COPD, defined as mMRC Dyspnea Scale scores of grades 2 or 3 and CAT scores of 12 to 20, will be included. If eligibility criteria are met, subjects will be randomized to either the treatment arm (BOTOX) or placebo, treated on day one and observed for 90 days for safety and efficacy.

Eligibility criteria: Inclusion Criteria:

1. 18 years of age or older
2. History of moderate COPD as diagnosed by a physician
3. FEV1 /FVC Ratio \< 0.7 with Post-Bronchodilator FEV1 ≥50% of predicted value at Screening
4. Modified Medical Research Council (mMRC) Dyspnea Scale score of Grade 2 or 3 (Appendix B) at Screening
5. CAT score of 12-20 (Appendix C) measured at Screening and confirmed at Baseline
6. Ability to consent
7. Ability to receive subcutaneous injections along the spine/back in the T-1 \> T-5 region
8. Females of reproductive potential must have a negative pregnancy test at screening and use of highly effective contraception method (refer to section 12.3.9) for 4 weeks after study drug administration. Women are generally considered to be of reproductive potential if a non-menopausal female has not had a hysterectomy, bilateral oophorectomy, or medically documented ovarian failure.
9. Males of reproductive potential must use condoms with spermicide or other methods to ensure effective contraception with partner for 4 weeks after study drug administration. Men are generally considered to be of reproductive potential if they can produce sperm.

Exclusion Criteria:

1. Any medical condition that may put the participant at increased risk with exposure to botulinum toxin type A (BTX-A), including, but not limited to diagnoses of myasthenia gravis (MG), Eaton-Lambert syndrome, amyotrophic lateral sclerosis, multiple sclerosis, or any other significant disease that might interfere with neuromuscular function
2. Treatment of study target treatment area using acupuncture, transcutaneous electrical stimulation (TENS), cranial traction or injection of anesthetics/steroids within 4 weeks prior to the screening visit (oral or inhaled steroids are allowed)
3. Known allergy to BTX-A
4. Known history of pulmonary hypertension
5. Documented COPD exacerbation within 4 weeks of Baseline (Day 1)
6. Post-bronchodilator FEV1 value ≤30% of the predicted value at screening
7. Current smoker (e-cigarettes or cigarettes, cigars, pipe tobacco, vape)
8. Prior or current lung cancer
9. Post COVID symptoms resulting in breathing difficulties beyond pre-COVID COPD symptoms
10. Allergy to local anesthetics
11. History of botulism
12. Has an active pulmonary infection requiring systemic therapy within 14 days prior to the baseline visit
13. Known/documented medical history of clotting disorders
14. Pregnant or breastfeeding
15. Requires immediate admission to the hospital during screening that would render the participant unable to comply with the protocol
16. Any serious medical condition, such as kidney, liver, or cardiac disease, asthma, angioedema, bleeding disorder, malignancy, seizure, neutropenia, AIDS, that in the judgement of the Investigator could confound study assessments
17. Any condition (physical, mental, or social), occupational reason or other responsibility that, in the judgment of the Investigator, would jeopardize the safety or rights of the study participant, or render the participant unable to comply with the protocol, including participant's return for follow-up visits on schedule
18. Any chronic conditions which may cause diffusion of excess of Substance P in the cerebrospinal fluid (CSF), including but not limited to diabetes, chronic kidney failure, cirrhosis, pancreatitis, osteoporosis, ongoing clinical depression, migraines and fibromyalgia, that would compromise the participant's safety as determined by the Investigator.
19. History of keloid formation after injury.
20. Treatment with another investigational drug or other investigational intervention within 2 weeks of study start and throughout study duration
21. Treatment with any botulinum toxin (BoNT) products within 6 months of the baseline visit.
22. Any abnormal laboratory value greater than 3 times upper limit of normal (ULN) for albumin, total protein, alanine transaminase (ALT), aspartate transaminase (AST), alkaline phosphatase (ALP), and bilirubin), and 5 times the upper limit of normal (ULN) for all other lab values.

Primary outcomes:
measure: CAT (Average)
timeFrame: 28 days
Secondary outcomes:
measure: CAT (Change in Units)
timeFrame: 28 days
measure: FEV1
timeFrame: 28 days
measure: FEV1/FVC
timeFrame: 28 days
measure: 6MWT
timeFrame: 28 days
measure: COPD Exacerbations
timeFrame: 90 days
measure: Adverse Reactions
timeFrame: 90 days
Other outcomes:
measure: CAT Score
timeFrame: 90 days
measure: FEV1
timeFrame: 90 days
measure: FEV1/FVC
timeFrame: 90 days
measure: Vital Signs: Oxygen saturation
timeFrame: 90 days
measure: 6MWT
timeFrame: 90 days
Locations:
city: Webster
country: United States
facility: Tranquil Clinical Research
state: Texas
Study officials:
affiliation: Investigator
name: Victoria Do
role: PRINCIPAL_INVESTIGATOR
References: