Peripheral Arterial DiseaseEarly Phase 1NCT02756884
Safety and Preliminary Efficacy of Adipose Derived Stem Cells and Low Frequency Ultrasound in PAD
A Early Phase 1 study testing LoFU and aADSC and adipose derived stem cells in people with Peripheral Arterial Disease, run by Arkansas Heart Hospital at 2 sites in United States.
Trial summary
This is a single center, open label, prospective, randomized, controlled study in patients with non- revascularizable moderate or severe lower extremity Peripheral Arterial Disease (PAD) receiving autologous stem cells therapy (minimally manipulated). This study will enroll a maximum of 10 subjects in this study. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Peripheral Arterial Disease
- What is being tested?
- LoFU and aADSC and adipose derived stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- 3 Jun 2015to 31 Jan 2019
- Phase
- Early 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 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and Females ≥18 years
- Patients with a Rutherford classification of 3 or 4.
- Non revascularizable lesions by balloon or stent angioplasty of the lower extremity SFA (Superficial femoral artery), Popliteal, AT (anterior tibial), PT (posterior tibial) or peroneal arteries. Non-revascularizable lesions will be determined based on a CT angiogram and/ or angiography.
- Life expectancy greater than 6 months.
- Ability to understand and provide signed informed consent.
- Reasonable expectation that patient will receive standard post-treatment care and attend all scheduled safety follow-up visits
- Written informed consent
Who cannot join
- Immunosuppressive agents, including but not limited to, corticosteroids and steroidal anti- inflammatory agents (SAIDS)
- Non-steroidal anti- inflammatory agents (NSAIDS)
- Patients taking currently P2Y12 inhibitors or calcium channel blockers.
- Uncontrolled seizure disorder
- Dementia
- Evidence or presence of immune deficiency.
Show all 11Show fewer
- Presence of any other clinically-significant medical condition, psychiatric condition, or laboratory abnormality that in the judgment of the Investigator or Sponsor would pose a safety risk to the subject
- Participation in another study with an investigational drug or device within one month prior to treatment
- Females known to be pregnant, lactating or having a positive pregnancy test (will be tested during screening) or planning to become pregnant during the study
- Inability to comply with the conditions of the protocol.
- Allergy to sodium citrate or any "caine" type of local anesthetic
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Arkansas Heart Hospital
Little Rock, Arkansas, 72211
Arkansas Site Management Services LLC
Little Rock, Arkansas, 72211
About this study
Official title: Phase I/Phase II Safety and Preliminary Efficacy Study Using Low Frequency Ultrasound in Addition to Adipose Derived Stem Cells (ADSCs) in Patients With Moderate to Severe Lower Extremity Peripheral Arterial Disease (PAD)
This is a single center, open label, prospective, randomized, controlled study in patients with non- revascularizable moderate or severe lower extremity Peripheral Arterial Disease (PAD) receiving autologous stem cells therapy (minimally manipulated). This study will enroll a maximum of 10 subjects in this study. The patients will be divided into two groups. One group will receive Adipose Derived Stem Cells (ADSCs) alone and the other group will receive low frequency ultrasound before the addition of ADSCs. The randomization will be 1:1. If the results of this pilot study are not clearly different, there will be a need for a phase II study to support the results.
Baseline, 6 week, 3 month and 6 month assessments of 6 min walking distance (6MWT), Ankle Brachial Indexes (ABIs)/Toe Brachial Indexes (TBIs), Transcutaneous Partial Oxygenation (TcPO2) and Rutherford assessments will be performed for the study group. Prior to the stem cell treatment, the ultrasound group patient will receive noninvasive transcutaneous pulsed focused ultrasound around the involved vessel(s) in the affected extremity. All patients will then receive 200 million autologous stromal vascular fraction cells containing adipose derived stem cell therapy: 1/3 of the cells will be delivered intra-venous proximal to the lesion (not retrograde), 1/3 of the cells will be delivered intra- adventitia proximal to the lesion and 1/3 of the cells will be delivered by intra- muscular injection along the vessel path (20-30 injections each separated by1.5 inch). Standard therapy for PAD patients will not be interrupted for the duration of the study. Standard therapy for PAD is defined as cardiovascular general measures like lifestyle changes, smoke cessation, exercise, cardiovascular rehabilitation, etc. All participants have already received maximal non-surgical therapy. Safety will be monitored on an ongoing basis.
Read the detailed description
This is a single center, open label, prospective, randomized, controlled study in patients with non- revascularizable moderate or severe lower extremity PAD receiving autologous stem cells therapy. We will enroll a maximum of 10 subjects in this study. The patients will be divided into two groups. One group will receive ADSCs alone and the other group will receive low frequency ultrasound before the addition of ADSCs. The randomization will be 1:1. If the results of this pilot study are not clearly different, there will be a need for a phase II study to support the results.
Baseline, 6 week, 3 month and 6 month assessments of 6 min walking distance, ABIs/TBIs, TcPO2, Rutherford assessments will be performed for the study group. Prior to the stem cell treatment, the ultrasound group patient will receive noninvasive transcutaneous pulsed focused ultrasound around the involved vessel(s) in the affected extremity. All patients will then receive 100 to 200 million autologous adipose derived stem cell therapy: 1/3 of the cells will be delivered intra-venous proximal to the lesion (not retrograde), 1/3 of the cells will be delivered intra- adventitia proximal to the lesion and 1/3 of the cells will be delivered by intra- muscular injection along the vessel path (20-30 injections each separated by 1.5 inch). Standard therapy for PAD patients will not be interrupted for the duration of the study. Standard therapy for PAD is defined as cardiovascular general measures like lifestyle changes, smoke cessation, exercise, cardiovascular rehabilitation, etc. All participants have already received maximal non-surgical therapy. Safety will be monitored on an ongoing basis.
SUBJECT PARTICIPATION A screening evaluation to determine if a potential subject is suitable for participation in this trial will be conducted prior to enrollment and treatment by the Investigator. This includes obtaining written informed consent, collection of a complete medical history, including current and past medications, physical examination and clinical laboratory tests, PAD related testing (ABI/TBI, 6MWT, TcPO2, QoL questionnaires, etc.).
Eligible subjects will be randomized and treated at the Sponsor hospital as approved by the sponsor. Study medical staff will evaluate the patients upon admission according to the scheduled medical assessments to ensure eligibility of the subjects. Ultrasound group will receive LFUS over the affected limb (see page XIV). Intra-venous, intra-adventitia and intramuscular dosing of the autologous ADSC will occur on the morning of Day 0. Conscious sedation (I.V.) may be used during the tissue harvesting and intra-adventitia administration of the cells.. Subjects must remain in the clinic/hospital until they are clinically stable for discharge; subjects that are clinically stable will be discharged into the care of a responsible individual. Follow-up visits will occur at 6 weeks and 3 and 6 months post-treatment, and may occur with the patient's personal physician who will be provided the follow-up assessment forms. If this occurs, the Investigator and/or Sponsor or sponsor's designee may also contact the patient by telephone to collect follow-up safety information. Adverse events, concomitant medications, vital signs, clinical laboratory test results and physical examinations will be collected or conducted at follow-up visits according to the schedule of assessments (Appendix B). All previous medical care, including medications and rehabilitation, for the patient's current medical conditions will be continued as prescribed by the patient's personal physician(s). Use of other investigational agents or treatments is not allowed during this study.
- Conditions
- Peripheral Arterial Disease
- Being tested
- LoFU and aADSC, adipose derived stem cells
- Medical subject headings
- Peripheral Arterial Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LoFU and aADSCBiological
Administration of low frequency ultrasound prior to administration of autologous adipose derived stem cells.
adipose derived stem cellsBiological
control arm will only received the adipose derived stem cells.
| Study group | Role | What they receive |
|---|---|---|
| LoFU and aADSC | Experimental | Low Frequency Ultrasound LFUS will be delivered in a non-sterile manner using a custom modified LFUS combined imaging/therapy probe. Adipose derived stem cells from the patient will be harvested through lipoaspiration. This solution will be injected intra-venous, intra-adventitia and intramuscular in the affected vessel and area after the administration of the low frequency ultrasound Receives: Biological: LoFU and aADSC |
| Adipose Derived Stem Cells | Active comparator | Adipose derived stem cells from the patient will be harvested through lipoaspiration. This solution will be injected intra-venous, intra-adventitia and intramuscular in the affected vessel and area without the administration of the low frequency ultrasound Receives: Biological: adipose derived stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety measured by incidence of adverse events
The primary outcome will be the safety/tolerability of treatment with aADSC in patients with PAD as determined by the incidence and severity of adverse events and serious adverse events.
Measured: 6 months
Secondary outcomes (5)
Efficacy of stem cells in improving ABI
Changes in ABI reported as an entire number with two decimals (e.g. 1.00) from baseline
Measured: 6 months
Efficacy of stem cells in improving 6MWT
Improvement in six minute walk test (6MWT) in meters when compared to baseline.
Measured: 6 months
Efficacy of stem cells in improving the TcPO2
improvement in Transcutaneous oxygen pressure (TcPO2) in mmHg as compared to baseline
Measured: 6 months
Efficacy of stem cells in improving Quality of Life using EQ-5D 5L questionnaire
Improvement in overall quality of life as measured using the EQ-5D 5L questionnaire as compared to baseline.
Measured: 6 months
Efficacy of stem cells in improving Quality of Life using the VascuQol questionnaire
Improvement in overall quality of life as measured using the VascuQol questionnaire as compared to baseline.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2016 (estimated)
- Study start3 Jun 2015
- Primary completion31 Jan 2019
- Study completion31 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Peripheral Arterial DiseaseStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arkansas Heart Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- SonaCare Medical
- Responsible party
- Sponsor
- Investigators
- Wilson Wong, MD, Principal investigator, Arkansas Heart Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02756884
- Sponsor's protocol ID
- AHH-2015-10
- Status last verified
- Jun 2019
- First submitted
- 25 Apr 2016
- Last updated
- 26 Jun 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Tebebi PA, Burks SR, Kim SJ, Williams RA, Nguyen BA, Venkatesh P, Frenkel V, Frank JA. Cyclooxygenase-2 or tumor necrosis factor-alpha inhibitors attenuate the mechanotransductive effects of pulsed focused ultrasound to suppress mesenchymal stromal cell homing to healthy and dystrophic muscle. Stem Cells. 2015 Apr;33(4):1173-86. doi: 10.1002/stem.1927.
PubMed 25534849
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/Phase II Safety and Preliminary Efficacy Study Using Low Frequency Ultrasound in Addition to Adipose Derived Stem Cells (ADSCs) in Patients With Moderate to Severe Lower Extremity Peripheral Arterial Disease (PAD)
- ClinicalTrials.gov ID
- NCT02756884
- Acronym / sponsor ID
- AHH-2015-10
- Conditions
- Peripheral Arterial Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Arkansas Heart HospitalUniversity, hospital or other
- Collaborators
- SonaCare Medical
- Study start
- 3 Jun 2015 (actual)
- Primary completion
- 31 Jan 2019 (actual)
- Study completion
- 31 Jan 2019 (actual)
- First posted
- 29 Apr 2016 (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.
This is a single center, open label, prospective, randomized, controlled study in patients with non- revascularizable moderate or severe lower extremity Peripheral Arterial Disease (PAD) receiving autologous stem cells therapy (minimally manipulated). This study will enroll a maximum of 10 subjects in this study. The patients will be divided into two groups. One group will receive Adipose Derived Stem Cells (ADSCs) alone and the other group will receive low frequency ultrasound before the addition of ADSCs. The randomization will be 1:1. If the results of this pilot study are not clearly different, there will be a need for a phase II study to support the results.
Baseline, 6 week, 3 month and 6 month assessments of 6 min walking distance (6MWT), Ankle Brachial Indexes (ABIs)/Toe Brachial Indexes (TBIs), Transcutaneous Partial Oxygenation (TcPO2) and Rutherford assessments will be performed for the study group. Prior to the stem cell treatment, the ultrasound group patient will receive noninvasive transcutaneous pulsed focused ultrasound around the involved vessel(s) in the affected extremity. All patients will then receive 200 million autologous stromal vascular fraction cells containing adipose derived stem cell therapy: 1/3 of the cells will be delivered intra-venous proximal to the lesion (not retrograde), 1/3 of the cells will be delivered intra- adventitia proximal to the lesion and 1/3 of the cells will be delivered by intra- muscular injection along the vessel path (20-30 injections each separated by1.5 inch). Standard therapy for PAD patients will not be interrupted for the duration of the study. Standard therapy for PAD is defined as cardiovascular general measures like lifestyle changes, smoke cessation, exercise, cardiovascular rehabilitation, etc. All participants have already received maximal non-surgical therapy. Safety will be monitored on an ongoing basis.
Detailed description
This is a single center, open label, prospective, randomized, controlled study in patients with non- revascularizable moderate or severe lower extremity PAD receiving autologous stem cells therapy. We will enroll a maximum of 10 subjects in this study. The patients will be divided into two groups. One group will receive ADSCs alone and the other group will receive low frequency ultrasound before the addition of ADSCs. The randomization will be 1:1. If the results of this pilot study are not clearly different, there will be a need for a phase II study to support the results.
Baseline, 6 week, 3 month and 6 month assessments of 6 min walking distance, ABIs/TBIs, TcPO2, Rutherford assessments will be performed for the study group. Prior to the stem cell treatment, the ultrasound group patient will receive noninvasive transcutaneous pulsed focused ultrasound around the involved vessel(s) in the affected extremity. All patients will then receive 100 to 200 million autologous adipose derived stem cell therapy: 1/3 of the cells will be delivered intra-venous proximal to the lesion (not retrograde), 1/3 of the cells will be delivered intra- adventitia proximal to the lesion and 1/3 of the cells will be delivered by intra- muscular injection along the vessel path (20-30 injections each separated by 1.5 inch). Standard therapy for PAD patients will not be interrupted for the duration of the study. Standard therapy for PAD is defined as cardiovascular general measures like lifestyle changes, smoke cessation, exercise, cardiovascular rehabilitation, etc. All participants have already received maximal non-surgical therapy. Safety will be monitored on an ongoing basis.
SUBJECT PARTICIPATION A screening evaluation to determine if a potential subject is suitable for participation in this trial will be conducted prior to enrollment and treatment by the Investigator. This includes obtaining written informed consent, collection of a complete medical history, including current and past medications, physical examination and clinical laboratory tests, PAD related testing (ABI/TBI, 6MWT, TcPO2, QoL questionnaires, etc.).
Eligible subjects will be randomized and treated at the Sponsor hospital as approved by the sponsor. Study medical staff will evaluate the patients upon admission according to the scheduled medical assessments to ensure eligibility of the subjects. Ultrasound group will receive LFUS over the affected limb (see page XIV). Intra-venous, intra-adventitia and intramuscular dosing of the autologous ADSC will occur on the morning of Day 0. Conscious sedation (I.V.) may be used during the tissue harvesting and intra-adventitia administration of the cells.. Subjects must remain in the clinic/hospital until they are clinically stable for discharge; subjects that are clinically stable will be discharged into the care of a responsible individual. Follow-up visits will occur at 6 weeks and 3 and 6 months post-treatment, and may occur with the patient's personal physician who will be provided the follow-up assessment forms. If this occurs, the Investigator and/or Sponsor or sponsor's designee may also contact the patient by telephone to collect follow-up safety information. Adverse events, concomitant medications, vital signs, clinical laboratory test results and physical examinations will be collected or conducted at follow-up visits according to the schedule of assessments (Appendix B). All previous medical care, including medications and rehabilitation, for the patient's current medical conditions will be continued as prescribed by the patient's personal physician(s). Use of other investigational agents or treatments is not allowed during this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LoFU and aADSC | Experimental | Biological: LoFU and aADSCLow Frequency Ultrasound LFUS will be delivered in a non-sterile manner using a custom modified LFUS combined imaging/therapy probe. Adipose derived stem cells from the patient will be harvested through lipoaspiration. This solution will be injected intra-venous, intra-adventitia and intramuscular in the affected vessel and area after the administration of the low frequency ultrasound |
| Adipose Derived Stem Cells | Active comparator | Biological: adipose derived stem cellsAdipose derived stem cells from the patient will be harvested through lipoaspiration. This solution will be injected intra-venous, intra-adventitia and intramuscular in the affected vessel and area without the administration of the low frequency ultrasound |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Males and Females ≥18 years
- Patients with a Rutherford classification of 3 or 4.
- Non revascularizable lesions by balloon or stent angioplasty of the lower extremity SFA (Superficial femoral artery), Popliteal, AT (anterior tibial), PT (posterior tibial) or peroneal arteries. Non-revascularizable lesions will be determined based on a CT angiogram and/ or angiography.
- Life expectancy greater than 6 months.
- Ability to understand and provide signed informed consent.
Key exclusion criteria (11 listed)
- Immunosuppressive agents, including but not limited to, corticosteroids and steroidal anti- inflammatory agents (SAIDS)
- Non-steroidal anti- inflammatory agents (NSAIDS)
- Patients taking currently P2Y12 inhibitors or calcium channel blockers.
- Uncontrolled seizure disorder
- Dementia
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 |
|---|---|---|
| LoFU and aADSCAdministration of low frequency ultrasound prior to administration of autologous adipose derived stem cells. | Biological | LoFU and aADSC |
| adipose derived stem cellscontrol arm will only received the adipose derived stem cells. | Biological | Adipose Derived Stem Cells |
Primary outcome
Safety measured by incidence of adverse events
Time frame: 6 months
The primary outcome will be the safety/tolerability of treatment with aADSC in patients with PAD as determined by the incidence and severity of adverse events and serious adverse events.
5 secondary outcomes
Efficacy of stem cells in improving ABI
Time frame: 6 months
Efficacy of stem cells in improving 6MWT
Time frame: 6 months
Efficacy of stem cells in improving the TcPO2
Time frame: 6 months
Efficacy of stem cells in improving Quality of Life using EQ-5D 5L questionnaire
Time frame: 6 months
Efficacy of stem cells in improving Quality of Life using the VascuQol questionnaire
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2019.
- Background: PubMed 25534849Tebebi PA, Burks SR, Kim SJ, Williams RA, Nguyen BA, Venkatesh P, Frenkel V, Frank JA. Cyclooxygenase-2 or tumor necrosis factor-alpha inhibitors attenuate the mechanotransductive effects of pulsed focused ultrasound to suppress mesenchymal stromal cell homing to healthy and dystrophic muscle. Stem Cells. 2015 Apr;33(4):1173-86. doi: 10.1002/stem.1927.
- Individual participant dataSharing planned: No
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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.
Wilson Wong, MD principal investigator
Arkansas Heart Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing LoFU and aADSC and adipose derived stem cells in people with Peripheral Arterial Disease, run by Arkansas Heart Hospital at 2 sites 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 11 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: finished, as of the sponsor's last update on 26 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety measured by incidence of adverse events (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02756884, which also lists 1 linked publication.
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 26 Jun 2019.