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Endogenous Progenitors Cell Therapy for Diabetic Foot Ulcers (AMD3100)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01353937
Recruitment Status : Withdrawn (Never Activated)
First Posted : May 16, 2011
Last Update Posted : March 4, 2016
National Institutes of Health (NIH)
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
NYU Langone Health

Brief Summary:
Diabetic foot ulcers, a complication of diabetes leading to 80.000 lower limb amputations annually in the US, are a significant burden to our health system, costing more than a billion dollars annually. Here, we propose a novel combination of two drugs (Mozobil® and Regranex®Gel) to mobilize a specific sub-type of stem cells (endothelial progenitor cells) from the bone marrow and traffic them toward the wound, increasing the blood supply that subsequently improves wound healing. Because we are using the human body's own resources to regenerate itself by targeting and correcting the underlying pathophysiology, we believe that this novel therapy yields great promise in the treatment of diabetic foot ulcers.

Condition or disease Intervention/treatment Phase
Diabetic Ulcer Drug: AMD3100 injection + rhPDGF-BB topical Phase 1

Detailed Description:

Because diabetes impairs wound healing by altering fibroblast function, promotes chronic infection and diminishes blood supply to the skin, the lifetime risk of a person with diabetes developing a diabetic foot ulcer (DFU) is as high as 25%. Current strategies focus independently on the fibroblast dysfunction (growth factors such as PDGF/Regranex® Gel), on the chronic infection (debridement, antibacterial dressings) or on the blood supply (VAC®).

This project is different from the other projects because we propose to combine two drugs in a dual approach to first improve the fibroblast function using PDGF/Regranex® Gel and second to induce neovascularization in DFU by recruiting progenitor cells into the wound through a combination therapy of subcutaneous AMD3100 (Plerixafor/Mozobil®) with topical PDGF/Regranex® Gel. By contrast to novel stem cell therapies where cells are extracted, processed ex vivo and engrafted into the wound (exogenous stem cell therapy), here we propose to keep the stem cells in vivo (endogenous stem cell therapy).

Specifically, the first aim of the study will be to launch a prospective evaluator-blind pilot phase I/II safety and efficacy study to evaluate the clinical effect of AMD3100 (Plerixafor/Mozobil®) treatment with topical PDGF/Regranex® Gel compared to historical controls (standard of care and PDGF). AMD3100 (240 µg/kg SC) will be administered daily for 2 weeks. Our primary endpoint will be the measure of the percentage of change in area of the wound at 4 weeks (surrogate endpoint). In a second aim, we will measure the effect of AMD3100 treatment with PDGF using a quality-of-life index dedicated to DFU (DFS-SF).

Because we are addressing the underlying physiopathology in a dual approach, because we are avoiding the need for ex vivo processing and because both drugs are FDA approved, we believe that this novel therapy yields great promise in the treatment of DFUs.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: Endogenous Progenitors Cell Therapy for Diabetic Foot Ulcers
Study Start Date : April 2014
Estimated Primary Completion Date : March 2016
Estimated Study Completion Date : September 2016

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
No Intervention: Standard of Care
All patients will be receiving the Standard of Care treatments regardless of whether or not they are receiving study drug.
Active Comparator: Novel Combination Therapy
AMD3100 (Plerixafor) injection with Regranex Gel topical application
Drug: AMD3100 injection + rhPDGF-BB topical
drug therapy to be given for the first 2 week duration given on a daily basis initiated during the first visit (Day 0).
Other Names:
  • AMD3100 (Plerixafor)
  • rhPDGF-BB (Becaplermin)
  • Regranex Gel

Active Comparator: Becaplermin (Regranex Gel)
Topical application
Drug: AMD3100 injection + rhPDGF-BB topical
drug therapy to be given for the first 2 week duration given on a daily basis initiated during the first visit (Day 0).
Other Names:
  • AMD3100 (Plerixafor)
  • rhPDGF-BB (Becaplermin)
  • Regranex Gel

Primary Outcome Measures :
  1. Rate of Wound Closure [ Time Frame: 1 year ]
    The safety and efficacy of AMD3100 (Plerixafor) with rhPDGF-BB (Becaplermin) compared to two historical treatments group (Beclapermin versus standard of care (SOC) treatment) for the treatment of DFUs.[21] The central hypothesis to be tested is that a novel combination therapy will significantly increase the rate of closure of DFUs as compared to historical treatments groups, while presenting no major side effect.

  2. Quality of Life [ Time Frame: 4 weeks ]
    Test whether patients treated with the novel combination therapy will have an improvement in their quality of life, with higher scores on the DFS-SF than those of the historical control groups.

Secondary Outcome Measures :
  1. Glycosylated hemoglobin (HbA1C) [ Time Frame: 4 weeks ]
    long-term measure of diabetes control

  2. capillary blood glucose (ACCUCHEK Finger Stick) [ Time Frame: 4 weeks ]
    short-term measure of diabetes control

  3. Transcutaneous oxygen tension measurements on wound and 1 cm-radius periphery (Radiometer adult sensor) [ Time Frame: 4 weeks ]
    non-invasive measure of skin circulation

  4. Ankle-brachial index (ABI, Prestige sphygmomanometer and Summit doppler probe) [ Time Frame: 4 weeks ]
    measure of peripheral vascular disease

  5. pain (Visual-Analog Scale) [ Time Frame: 4 weeks ]
    measure of the subjective symptom of pain

  6. temperature of surrounding skin in a 1 cm-radius around the DFU (TempTouch Dermal Thermometer) [ Time Frame: 4 weeks ]
    to identify increased skin temperatures, intended as an early warning of inflammation, impending infection, and possible foot ulceration.

  7. sensation (Nk Pressure-Specified Sensory Device) [ Time Frame: 4 weeks ]
    Quantification of sensory nerve function in patients with symptoms of, or the potential for, neurologic damage or disease

  8. photogrammetry (Photoshop CS3, Adobe Systems) [ Time Frame: 4 weeks ]
    used to document wound appearance

  9. glomerular filtration rate (GFR, estimated by 24 hr. urine creatinine measurement) [ Time Frame: 4 weeks ]
    to estimate renal function

  10. diabetic retinopathy (digital ophthalmologic examination) [ Time Frame: 4 weeks ]
    to evaluate for development of nonproliferative and proliferative retinopathy

  11. cEPCs by FACS analysis [ Time Frame: 4 weeks ]
    to measure the extent of BM EPC mobilization into the circulation and correlate the number cEPCs to other primary and secondary endpoints

Information from the National Library of Medicine

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Ages Eligible for Study:   35 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Insulin-dependant type 2 diabetic patients
  2. Age between 35 and 60 years-old
  3. HbA1C between 6 and 12%
  4. Full-thickness diabetic neuropathic foot ulcers
  5. ≥ 2 weeks duration
  6. Following standard of care débridement, ulcer size must be between 1 and 6 cm2
  7. Adequate perfusion, defined as either transcutaneous oxygen measurements on the dorsum of the foot >30 mmHg or ankle brachial indexes 0.7<ABI<1.2, as well as toe pressure >30 mmHg.

Exclusion Criteria:

  1. Clinical infection at the studied ulcer site (bacterial and fungal)
  2. Clinically significant lower-extremity ischemia (as defined by an ankle/brachial index of <0.65)
  3. Active Charcot's foot as determined by clinical and radiographic examination
  4. Ulcer of a non-diabetic pathophysiology (e.g., rheumatoid, radiation-related, and vasculitis-related ulcers, and especially venous stasis ulcer)
  5. Significant medical conditions that would impair wound healing will also be excluded from the study. These conditions include liver disease, aplastic anemia, scleroderma and malignancy, treatment with immunosuppressive agents or steroids, myocardial infarcts, stroke, major surgery within 6 months of the study, usage of tobacco
  6. Subjects with cancerous or pre-cancerous lesions in the area to be treated
  7. Body weight > 160 kg (because of Plerixafor's pharmacokinetic limitation)
  8. Severe renal dysfunction (creatinine clearance < 50 ml/min)
  9. Severe non-proliferative or proliferative diabetic retinopathy
  10. Capillary blood glucose >350

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01353937

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United States, New York
Helen L. & Martin S. Kimmel Wound Healing Center at the NYU Hospital for Joint Diseases
New York, New York, United States, 10003
Sponsors and Collaborators
NYU Langone Health
National Institutes of Health (NIH)
Genzyme, a Sanofi Company
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Principal Investigator: Stephen Warren, MD NYU Langone Health

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Responsible Party: NYU Langone Health Identifier: NCT01353937    
Other Study ID Numbers: 10-02116
First Posted: May 16, 2011    Key Record Dates
Last Update Posted: March 4, 2016
Last Verified: March 2016
Keywords provided by NYU Langone Health:
wound healing
Additional relevant MeSH terms:
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Diabetic Foot
Foot Ulcer
Pathologic Processes
Diabetic Angiopathies
Vascular Diseases
Cardiovascular Diseases
Leg Ulcer
Skin Ulcer
Skin Diseases
Diabetes Complications
Diabetes Mellitus
Endocrine System Diseases
Diabetic Neuropathies
Foot Diseases
Plerixafor octahydrochloride
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Angiogenesis Inducing Agents
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs