Treadmill Exercise and GM-CSF Study to Improving Functioning in Peripheral Artery Disease (PAD)

NCT ID: NCT01408901

Last Updated: 2020-01-21

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

210 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-30

Study Completion Date

2017-08-15

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The PROPEL study will test the hypothesis that GM-CSF combined with supervised treadmill exercise will significantly improve functional performance in patients with PAD more than GM-CSF alone or supervised treadmill exercise alone. In addition to identifying novel therapeutic options for patients with PAD, the current proposal is expected to identify mechanisms by which functional impairment is improved in patients with PAD.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Eight million men and women in the United States have lower extremity peripheral arterial disease (PAD). PAD is expected to be increasingly common as the population survives longer with chronic disease. Patients with PAD have greater functional impairment and faster functional decline compared to those without PAD. However, currently there are only two FDA approved medications for improving functional performance in patients with PAD. Furthermore, these FDA approved medications are only modestly beneficial for improving walking performance in patients with PAD.

Preliminary evidence suggests that increasing circulating levels of CD34+ cells with granulocyte macrophage colony stimulating factor (GM-CSF) or other therapies may improve walking performance in patients with PAD. However, results of small clinical trials testing the ability of GM-CSF to improve walking performance in patients with PAD are mixed. The association of GM-CSF with improved walking performance in PAD is not definitively established. Preliminary data also suggest that lower extremity ischemia, induced during walking exercise, may increase circulating CD34+ cell levels, enhance homing of CD34+ cells to ischemic sites, and augment the ability of GMCSF to improve walking performance in PAD. However, it is currently unknown whether the combination of GM-CSF and supervised treadmill exercise significantly improve functional performance more than either therapy alone.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Peripheral Arterial Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

FACTORIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

A: GM-CSF + supervised treadmill exercise therapy

Group Type EXPERIMENTAL

Supervised Treadmill Exercise Therapy

Intervention Type BEHAVIORAL

Exercise intervention will be delivered three times weekly for 26 weeks. In the first week, participants will be asked to exercise 15 minutes per session (excluding rest periods). Walking exercise duration will be increased to 25 minutes minutes per session during week 2. Week 3 and 4 sessions will also be 25 minutes long, but the intensity will be increased either to produce leg symptoms or at a target rate of perceived exertion (RPE)of 12-14 on the Borg's 6-20 scale. For weeks 5-8, walking duration will be increased to 40 to 50 minutes while maintaining intensity. For weeks 9-26, exercise duration will continue to be 40 to 50 minutes but we will increase intensity up to a maximum of 4.0 miles per hour at 10% grade.

granulocyte macrophage colony stimulating factor (GM-CSF)

Intervention Type DRUG

The dose of GM-CSF will be 250 ug/M\^2 subcutaneously three times weekly for two weeks.

B: GM-CSF + attention control group

Group Type ACTIVE_COMPARATOR

Health education sessions (Control)

Intervention Type OTHER

Participants randomized to the attention control group will attend weekly one-hour educational sessions at Northwestern University for six months. These educational sessions are on topics of interest to the typical PAD patient and are led by physicians and other health care workers. Topics include Medicare Part D, nutritional supplements, C-reactive protein, and hypertension. Sessions do not include information about exercise.

granulocyte macrophage colony stimulating factor (GM-CSF)

Intervention Type DRUG

The dose of GM-CSF will be 250 ug/M\^2 subcutaneously three times weekly for two weeks.

C: placebo + supervised exercise therapy

Group Type ACTIVE_COMPARATOR

Supervised Treadmill Exercise Therapy

Intervention Type BEHAVIORAL

Exercise intervention will be delivered three times weekly for 26 weeks. In the first week, participants will be asked to exercise 15 minutes per session (excluding rest periods). Walking exercise duration will be increased to 25 minutes minutes per session during week 2. Week 3 and 4 sessions will also be 25 minutes long, but the intensity will be increased either to produce leg symptoms or at a target rate of perceived exertion (RPE)of 12-14 on the Borg's 6-20 scale. For weeks 5-8, walking duration will be increased to 40 to 50 minutes while maintaining intensity. For weeks 9-26, exercise duration will continue to be 40 to 50 minutes but we will increase intensity up to a maximum of 4.0 miles per hour at 10% grade.

D: placebo + attention control group

Group Type PLACEBO_COMPARATOR

Health education sessions (Control)

Intervention Type OTHER

Participants randomized to the attention control group will attend weekly one-hour educational sessions at Northwestern University for six months. These educational sessions are on topics of interest to the typical PAD patient and are led by physicians and other health care workers. Topics include Medicare Part D, nutritional supplements, C-reactive protein, and hypertension. Sessions do not include information about exercise.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Supervised Treadmill Exercise Therapy

Exercise intervention will be delivered three times weekly for 26 weeks. In the first week, participants will be asked to exercise 15 minutes per session (excluding rest periods). Walking exercise duration will be increased to 25 minutes minutes per session during week 2. Week 3 and 4 sessions will also be 25 minutes long, but the intensity will be increased either to produce leg symptoms or at a target rate of perceived exertion (RPE)of 12-14 on the Borg's 6-20 scale. For weeks 5-8, walking duration will be increased to 40 to 50 minutes while maintaining intensity. For weeks 9-26, exercise duration will continue to be 40 to 50 minutes but we will increase intensity up to a maximum of 4.0 miles per hour at 10% grade.

Intervention Type BEHAVIORAL

Health education sessions (Control)

Participants randomized to the attention control group will attend weekly one-hour educational sessions at Northwestern University for six months. These educational sessions are on topics of interest to the typical PAD patient and are led by physicians and other health care workers. Topics include Medicare Part D, nutritional supplements, C-reactive protein, and hypertension. Sessions do not include information about exercise.

Intervention Type OTHER

granulocyte macrophage colony stimulating factor (GM-CSF)

The dose of GM-CSF will be 250 ug/M\^2 subcutaneously three times weekly for two weeks.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

treadmill exercise attention control group sargramostim leukine

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Participants with an ankle brachial index (ABI) ≤ 0.90 will be eligible for participation.
2. Participants with an ABI \> 0.90 but ≤ 1.00 who experience a 20% drop in ankle pressure after the heel-rise exercise will be eligible.
3. Participants with an ABI \> 0.90 who have medical record evidence of prior lower extremity revascularization and experience a 20% drop in ankle pressure after the heel-rise exercise will be eligible for inclusion.
4. Participants with an ABI \> 0.90 who have medical record evidence of a non-invasive vascular laboratory test result consistent with PAD. Note that a screen-positive test from Lifeline Screening is not sufficient for inclusion in the study.

Exclusion Criteria

1. Below or above-knee amputation.
2. Wheelchair confinement.
3. Use of a walking aid other than a cane (i.e. people using walkers).
4. Non-English speaking.
5. Significant hearing impairment.
6. Significant visual impairment.
7. Diagnosis of Parkinson's disease.
8. Inability to return to the medical center at the required visit frequency (three times per week).
9. \> Class II New York Heart Association heart failure or angina (symptoms at rest or with minimal exertion).
10. Any increase in angina pectoris symptoms during the previous 6 months or angina at rest.
11. Foot ulcer. (Participants with a foot ulcer will be excluded by telephone and/or during a baseline study visit).
12. Lower extremity revascularization in the last three months or major orthopedic surgery during the previous three months.
13. Myocardial infarction, stroke, or coronary artery bypass grafting during the previous 3 months.
14. Major medical illnesses including end stage renal disease requiring dialysis and chronic lung disease requiring oxygen, since these individuals may not be able to adhere to study requirements. Participants who only use oxygen at night may still qualify.
15. Potential participants who have received G-CSF, GM-CSF, or erythropoietin within the past year will be excluded because these interventions may influence study outcomes independently of the interventions.
16. Pre-menopausal women will be excluded because cyclic estrogen changes can influence progenitor cell levels.
17. Potential participants with diabetes and documented proliferative retinopathy will be excluded because GM-CSF may exacerbate this condition.
18. Potential participants with a history of myeloid malignancy will be excluded because GM-CSF may exacerbate these conditions.
19. Potential participants who have been treated for late stage cancer during the past three years, since GM-CSF may theoretically activate quiescent cancer cells.
20. Planned lower extremity revascularization within the next 6 months.
21. Current participation in another clinical trial. If a participant recently completed a clinical trial, at least three months must have passed before they can be considered for the PROPEL Trial. However, for a clinical trial of stem cell or gene therapy intervention, potential participants will be potentially eligible immediately after the final study visit of the stem cell or gene therapy clinical trial, so long as at least six months has passed since the participant received their final treatment in the stem cell or gene therapy intervention.
22. Walking for exercise at a level comparable to that targeted in our intervention.
23. Current participation in or completion of a cardiac rehabilitation program within the last six months.

24. Severe aortic stenosis identified by physical exam at the study visit.
25. Critical limb ischemia identified by physical exam at the study visit.
26. Coronary ischemia during exercise, defined as ST segment depression \> 1 mm during the baseline exercise treadmill test, with or without associated chest discomfort, without a perfusion stress test demonstrating no reversible ischemia within the previous 3 months.
27. Left-bundle branch block or significant ST-T wave changes on the baseline ECG without a perfusion stress test demonstrating no reversible ischemia within the previous 3 months.
28. Stopping during the treadmill stress test for shortness of breath, chest pain, hip pain, knee pain, or another symptom that may not represent ischemic leg pain.
29. Stopping during the six-minute walk test for symptoms other than ischemic leg symptoms.
30. Foot ulcer identified at the study visit.
31. Mini-Mental Status Examination (MMSE) score \< 23 or disabling psychiatric disease.
32. Failure to complete a study run-in period.
33. Walking impairment due to a cause other than PAD.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Northwestern University

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Mary McDermott

Professor of Medicine and Preventive Medicine at Northwestern University Feinberg School of Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Mary M McDermott, MD

Role: PRINCIPAL_INVESTIGATOR

Northwestern University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Northwestern University Feinberg School of Medicine

Chicago, Illinois, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Domanchuk K, Ferrucci L, Guralnik JM, Criqui MH, Tian L, Liu K, Losordo D, Stein J, Green D, Kibbe M, Zhao L, Annex B, Perlman H, Lloyd-Jones D, Pearce W, Taylor D, McDermott MM. Progenitor cell release plus exercise to improve functional performance in peripheral artery disease: the PROPEL Study. Contemp Clin Trials. 2013 Nov;36(2):502-9. doi: 10.1016/j.cct.2013.09.011. Epub 2013 Sep 27.

Reference Type BACKGROUND
PMID: 24080099 (View on PubMed)

Saber R, Liu K, Ferrucci L, Criqui MH, Zhao L, Tian L, Guralnik JM, Liao Y, Domanchuk K, Kibbe MR, Green D, Perlman H, McDermott MM. Ischemia-related changes in circulating stem and progenitor cells and associated clinical characteristics in peripheral artery disease. Vasc Med. 2015 Dec;20(6):534-43. doi: 10.1177/1358863X15600255. Epub 2015 Aug 31.

Reference Type BACKGROUND
PMID: 26324152 (View on PubMed)

McDermott MM, Guralnik JM, Criqui MH, Liu K, Kibbe MR, Ferrucci L. Six-minute walk is a better outcome measure than treadmill walking tests in therapeutic trials of patients with peripheral artery disease. Circulation. 2014 Jul 1;130(1):61-8. doi: 10.1161/CIRCULATIONAHA.114.007002. No abstract available.

Reference Type BACKGROUND
PMID: 24982117 (View on PubMed)

McDermott MM. Lower extremity manifestations of peripheral artery disease: the pathophysiologic and functional implications of leg ischemia. Circ Res. 2015 Apr 24;116(9):1540-50. doi: 10.1161/CIRCRESAHA.114.303517.

Reference Type BACKGROUND
PMID: 25908727 (View on PubMed)

McDermott MM, Ferrucci L, Tian L, Guralnik JM, Lloyd-Jones D, Kibbe MR, Polonsky TS, Domanchuk K, Stein JH, Zhao L, Taylor D, Skelly C, Pearce W, Perlman H, McCarthy W, Li L, Gao Y, Sufit R, Bloomfield CL, Criqui MH. Effect of Granulocyte-Macrophage Colony-Stimulating Factor With or Without Supervised Exercise on Walking Performance in Patients With Peripheral Artery Disease: The PROPEL Randomized Clinical Trial. JAMA. 2017 Dec 5;318(21):2089-2098. doi: 10.1001/jama.2017.17437.

Reference Type RESULT
PMID: 29141087 (View on PubMed)

McDermott MM, Zhang D, Zhao L, Polonsky T, Ferrucci L, Ho KJ, Kibbe MR, Guralnik JM, Criqui MH, Tian L. Duration of Supervised Exercise Necessary for Meaningful Improvement in Peripheral Artery Disease. J Am Heart Assoc. 2025 Sep 16;14(18):e040058. doi: 10.1161/JAHA.124.040058. Epub 2025 Sep 5.

Reference Type DERIVED
PMID: 40913279 (View on PubMed)

Hammond MM, Tian L, Zhao L, Zhang D, McDermott MM. Attendance at Supervised Exercise Sessions and Walking Outcomes in Peripheral Artery Disease: Results From 2 Randomized Clinical Trials. J Am Heart Assoc. 2022 Dec 20;11(24):e026136. doi: 10.1161/JAHA.122.026136. Epub 2022 Dec 19.

Reference Type DERIVED
PMID: 36533626 (View on PubMed)

McDermott MM, Tian L, Criqui MH, Ferrucci L, Greenland P, Guralnik JM, Kibbe MR, Li L, Sufit R, Zhao L, Polonsky TS. Perceived Versus Objective Change in Walking Ability in Peripheral Artery Disease: Results from 3 Randomized Clinical Trials of Exercise Therapy. J Am Heart Assoc. 2021 Jun 15;10(12):e017609. doi: 10.1161/JAHA.120.017609. Epub 2021 Jun 2.

Reference Type DERIVED
PMID: 34075780 (View on PubMed)

Patel K, Polonsky TS, Kibbe MR, Guralnik JM, Tian L, Ferrucci L, Criqui MH, Sufit R, Leeuwenburgh C, Zhang D, Zhao L, McDermott MM. Clinical characteristics and response to supervised exercise therapy of people with lower extremity peripheral artery disease. J Vasc Surg. 2021 Feb;73(2):608-625. doi: 10.1016/j.jvs.2020.04.498. Epub 2020 May 19.

Reference Type DERIVED
PMID: 32416309 (View on PubMed)

Kosmac K, Gonzalez-Freire M, McDermott MM, White SH, Walton RG, Sufit RL, Tian L, Li L, Kibbe MR, Criqui MH, Guralnik JM, S Polonsky T, Leeuwenburgh C, Ferrucci L, Peterson CA. Correlations of Calf Muscle Macrophage Content With Muscle Properties and Walking Performance in Peripheral Artery Disease. J Am Heart Assoc. 2020 May 18;9(10):e015929. doi: 10.1161/JAHA.118.015929. Epub 2020 May 9.

Reference Type DERIVED
PMID: 32390569 (View on PubMed)

McDermott MM, Polonsky TS, Guralnik JM, Ferrucci L, Tian L, Zhao L, Stein J, Domanchuk K, Criqui MH, Taylor DA, Li L, Kibbe MR. Racial Differences in the Effect of Granulocyte Macrophage Colony-Stimulating Factor on Improved Walking Distance in Peripheral Artery Disease: The PROPEL Randomized Clinical Trial. J Am Heart Assoc. 2019 Jan 22;8(2):e011001. doi: 10.1161/JAHA.118.011001.

Reference Type DERIVED
PMID: 30661439 (View on PubMed)

Berroug J, Korcarz CE, Mitchell CK, Weber JM, Tian L, McDermott MM, Stein JH. Brachial artery intima-media thickness and grayscale texture changes in patients with peripheral artery disease receiving supervised exercise training in the PROPEL randomized clinical trial. Vasc Med. 2019 Feb;24(1):12-22. doi: 10.1177/1358863X18804050. Epub 2018 Nov 12.

Reference Type DERIVED
PMID: 30418100 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

R01HL107510

Identifier Type: NIH

Identifier Source: secondary_id

View Link

STU00049675

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Self-Managed Walking Improves Function
NCT00611988 COMPLETED PHASE2
Exercise and Weight Loss in PAD
NCT02607033 COMPLETED NA