Diabetic UlcerPhase 1AMD3100NCT01353937
Endogenous Progenitors Cell Therapy for Diabetic Foot Ulcers
A Phase 1 study testing AMD3100 injection + rhPDGF-BB topical in people with Diabetic Ulcer, run by NYU Langone Health at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 35 to 60 years
- Conditions the study is for
- Diabetic Ulcer
- What is being tested?
- AMD3100 injection + rhPDGF-BB topical
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2014to Sep 2016
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Insulin-dependant type 2 diabetic patients
- Age between 35 and 60 years-old
- HbA1C between 6 and 12%
- Full-thickness diabetic neuropathic foot ulcers
- ≥ 2 weeks duration
- Following standard of care débridement, ulcer size must be between 1 and 6 cm2
- 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.
Who cannot join
- Clinical infection at the studied ulcer site (bacterial and fungal)
- Clinically significant lower-extremity ischemia (as defined by an ankle/brachial index of <0.65)
- Active Charcot's foot as determined by clinical and radiographic examination
- Ulcer of a non-diabetic pathophysiology (e.g., rheumatoid, radiation-related, and vasculitis-related ulcers, and especially venous stasis ulcer)
- 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
- Subjects with cancerous or pre-cancerous lesions in the area to be treated
Show all 10Show fewer
- Body weight > 160 kg (because of Plerixafor's pharmacokinetic limitation)
- Severe renal dysfunction (creatinine clearance < 50 ml/min)
- Severe non-proliferative or proliferative diabetic retinopathy
- Capillary blood glucose >350
Trial locations and contacts
1 site in 1 country.
United States1 site
Helen L. & Martin S. Kimmel Wound Healing Center at the NYU Hospital for Joint Diseases
New York, New York, 10003
About this study
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.
Read the 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.
- Conditions
- Diabetic Ulcer
- Being tested
- AMD3100 injection + rhPDGF-BB topical
- Keywords
- wound healing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AMD3100 injection + rhPDGF-BB topicalDrug
Also called AMD3100 (Plerixafor), rhPDGF-BB (Becaplermin), Regranex Gel
drug therapy to be given for the first 2 week duration given on a daily basis initiated during the first visit (Day 0).
| Study group | Role | What they receive |
|---|---|---|
| Standard of Care | No intervention | All patients will be receiving the Standard of Care treatments regardless of whether or not they are receiving study drug. |
| Novel Combination Therapy | Active comparator | AMD3100 (Plerixafor) injection with Regranex Gel topical application Receives: Drug: AMD3100 injection + rhPDGF-BB topical |
| Becaplermin (Regranex Gel) | Active comparator | Topical application Receives: Drug: AMD3100 injection + rhPDGF-BB topical |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Wound Closure
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.
Measured: 1 year
Quality of Life
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.
Measured: 4 weeks
Secondary outcomes (11)
Glycosylated hemoglobin (HbA1C)
long-term measure of diabetes control
Measured: 4 weeks
capillary blood glucose (ACCUCHEK Finger Stick)
short-term measure of diabetes control
Measured: 4 weeks
Transcutaneous oxygen tension measurements on wound and 1 cm-radius periphery (Radiometer adult sensor)
non-invasive measure of skin circulation
Measured: 4 weeks
Ankle-brachial index (ABI, Prestige sphygmomanometer and Summit doppler probe)
measure of peripheral vascular disease
Measured: 4 weeks
pain (Visual-Analog Scale)
measure of the subjective symptom of pain
Measured: 4 weeks
temperature of surrounding skin in a 1 cm-radius around the DFU (TempTouch Dermal Thermometer)
to identify increased skin temperatures, intended as an early warning of inflammation, impending infection, and possible foot ulceration.
Measured: 4 weeks
sensation (Nk Pressure-Specified Sensory Device)
Quantification of sensory nerve function in patients with symptoms of, or the potential for, neurologic damage or disease
Measured: 4 weeks
photogrammetry (Photoshop CS3, Adobe Systems)
used to document wound appearance
Measured: 4 weeks
glomerular filtration rate (GFR, estimated by 24 hr. urine creatinine measurement)
to estimate renal function
Measured: 4 weeks
diabetic retinopathy (digital ophthalmologic examination)
to evaluate for development of nonproliferative and proliferative retinopathy
Measured: 4 weeks
cEPCs by FACS analysis
to measure the extent of BM EPC mobilization into the circulation and correlate the number cEPCs to other primary and secondary endpoints
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2011 (estimated)
- Study startApr 2014
- Primary completionMar 2016 (estimated)
- Study completionSep 2016 (estimated)
Why it stopped: Never Activated
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetic UlcerStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Stephen Warren, MD, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01353937
- Sponsor's protocol ID
- 10-02116
- Acronym
- AMD3100
- Status last verified
- Mar 2016
- First submitted
- 12 May 2011
- Last updated
- 4 Mar 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Endogenous Progenitors Cell Therapy for Diabetic Foot Ulcers
- ClinicalTrials.gov ID
- NCT01353937
- Acronym / sponsor ID
- AMD3100 / 10-02116
- Conditions
- Diabetic Ulcer
- Keywords
- wound healing
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Never Activated
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), Genzyme, a Sanofi Company
- Study start
- Apr 2014
- Primary completion
- Mar 2016 (estimated)
- Study completion
- Sep 2016 (estimated)
- First posted
- 16 May 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Standard of Care | No intervention | –All patients will be receiving the Standard of Care treatments regardless of whether or not they are receiving study drug. |
| Novel Combination Therapy | Active comparator | Drug: AMD3100 injection + rhPDGF-BB topicalAMD3100 (Plerixafor) injection with Regranex Gel topical application |
| Becaplermin (Regranex Gel) | Active comparator | Drug: AMD3100 injection + rhPDGF-BB topicalTopical application |
Who may qualify
- Ages
- 35 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Insulin-dependant type 2 diabetic patients
- Age between 35 and 60 years-old
- HbA1C between 6 and 12%
- Full-thickness diabetic neuropathic foot ulcers
- ≥ 2 weeks duration
Key exclusion criteria (10 listed)
- Clinical infection at the studied ulcer site (bacterial and fungal)
- Clinically significant lower-extremity ischemia (as defined by an ankle/brachial index of <0.65)
- Active Charcot's foot as determined by clinical and radiographic examination
- Ulcer of a non-diabetic pathophysiology (e.g., rheumatoid, radiation-related, and vasculitis-related ulcers, and especially venous stasis ulcer)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AMD3100 injection + rhPDGF-BB topicalAlso: AMD3100 (Plerixafor), rhPDGF-BB (Becaplermin), Regranex Geldrug therapy to be given for the first 2 week duration given on a daily basis initiated during the first visit (Day 0). | Drug | Becaplermin (Regranex Gel), Novel Combination Therapy |
Primary outcome
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.
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.
11 secondary outcomes
Glycosylated hemoglobin (HbA1C)
Time frame: 4 weeks
capillary blood glucose (ACCUCHEK Finger Stick)
Time frame: 4 weeks
Transcutaneous oxygen tension measurements on wound and 1 cm-radius periphery (Radiometer adult sensor)
Time frame: 4 weeks
Ankle-brachial index (ABI, Prestige sphygmomanometer and Summit doppler probe)
Time frame: 4 weeks
pain (Visual-Analog Scale)
Time frame: 4 weeks
temperature of surrounding skin in a 1 cm-radius around the DFU (TempTouch Dermal Thermometer)
Time frame: 4 weeks
sensation (Nk Pressure-Specified Sensory Device)
Time frame: 4 weeks
photogrammetry (Photoshop CS3, Adobe Systems)
Time frame: 4 weeks
glomerular filtration rate (GFR, estimated by 24 hr. urine creatinine measurement)
Time frame: 4 weeks
diabetic retinopathy (digital ophthalmologic examination)
Time frame: 4 weeks
cEPCs by FACS analysis
Time frame: 4 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2016.
Related trials for Diabetic Ulcer
Not yet recruiting
Efficacy Evaluation of Turmeric-Derived Nanoparticle Hydrogel for the Treatment of Diabetic Foot Ulcer Wounds
Diabete Mellitus
Active, not recruiting
Evaluation of Personalized Nutritional Intervention on Wound Healing of Cutaneous Ulcers in Diabetics
Foot, Diabetic
Active, not recruiting
Wearable Insoles for Recurrent Diabetic Ulcer Prevention
Diabetes, Peripheral Neuropathy
Not yet recruiting
Better Care Closer to Home: Enhancing Primary and Community Care
Diabetic Foot Disease, Recurrence
Recruiting
Continuous Monitoring of Plantar Temperature Variations Using Connected Insoles and a Mobile Application for Diabetic Foot.
Diabetic Foot Disease, Diabetic Foot Infection
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephen Warren, MD principal investigator
NYU Langone Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AMD3100 injection + rhPDGF-BB topical in people with Diabetic Ulcer, run by NYU Langone Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 4 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Wound Closure (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2016.