Expanded Access to Provide Intramuscular Injections of PLX-PAD for the Treatment of Subjects With Critical Limb Ischemia (CLI) With Minor Tissue Loss Who Are Unsuitable for Revascularization
Expanded Access for Treatment Use of PLX-PAD in Critical Limb Ischemia (CLI)
Sponsors
Source
WideTrial, Inc.
Brief Summary
This is an Expanded Access program (EAP), sponsored by WideTrial for the treatment of
critical limb ischemia (CLI).
Widetrial, an Expanded Access specialist, has arranged to supply participating sites with
PLX-PAD for CLI patients who cannot participate in the ongoing research trial and who seek
exploratory treatment options. This program includes FDA-authorized cost recovery, meaning
payment is required to cover a portion of the costs of delivering product and complying with
regulatory obligations.
Overall Status
Available
Start Date
N/A
Completion Date
N/A
Primary Completion Date
N/A
Study Type
Expanded Access
Condition
Intervention
Intervention Type
Biological
Intervention Name
Description
PLX-PAD cell therapy to be administered via 30 IM injections (0.5 mL each) to the index leg leg per treatment, in each of two treatments, 8 weeks apart
Eligibility
Criteria
Inclusion Criteria:
1. Adult male or female subjects between ages 45 to 99 years of age at the time of
screening.
2. Subjects with a diagnosis of PAD due to atherosclerosis at the stage of CLI, with
minor tissue loss up to the ankle level (ulcer/s and/or necrosis).
3. Ankle pressure (AP) ≤70 mmHg or toe pressure (TP) ≤50 mmHg in the index leg or
transcutaneous oxygen pressure (TcPO2) ≤30 mmHg.
4. Subject unsuitable for revascularization (by any method) in the index leg based on
unfavorable risk-benefit assessment of the physician investigator. Unsuitability to
revascularization should be based on any of the following:
1. Anatomic considerations as: inappropriate target artery, diffuse/extensive tibial
and/or peroneal artery lesions, inadequate distal run-off.
2. Technical considerations as: inappropriate bypass conduit, failed recent
revascularization.
3. Medical considerations: subject's comorbidities.
5. Signed informed consent form.
6. Subjects are not eligible for the ongoing phase III study with PLX-PAD in CLI
(PLX-CLI-03) due to at least one of the following criteria:
1. Evidence of active localized osteomyelitis secondary to contiguous focus of
infection, unless amputation is expected within 1 month post PLX-PAD
administration. In case of osteomyelitis, patients must be treated with
antibiotics during screening and PLX-PAD administration or as long as there is
evidence of active infection.
2. Subject on renal replacement therapy or with eGFR <15 mL/min.
3. Current treatment with high dose systemic steroids (prednisone equivalent >7.5
mg/day) or topical steroids on the index leg.
4. History of autologous bone marrow transplantation (if not due to hematologic
malignancy) or solid organ transplantation, clinically stable.
5. Immunocompromised subjects due to disease for any reason, including
immunosuppressive therapy, at screening (for steroid therapy, refer to the
criterion c)
6. CLI with major tissue loss (Rutherford Category 6) in the contralateral leg.
7. Diabetes mellitus with glycosylated hemoglobin (HbA1c) >10% at Screening.
8. HIV controlled by antiretroviral therapy
9. Past drug or alcohol abuse. Known history of cancer is eligible if occurred
beyond 2 years before screening
Exclusion Criteria:
1. Non-atherosclerotic PAD and vasculitis (e.g., Buerger's disease [thromboangiitis
obliterans], Takayasu's arteritis, etc.).
2. CLI with major tissue loss (Rutherford Category 6) in the index leg. Ulcers from
venous or neuropathic origin if not associated with at least one ulcer from arterial
origin.
3. Evidence of active infection in either leg (e.g., cellulitis, myositis) except
localized osteomyelitis secondary to contiguous focus of infection, under antibiotic
treatment.
4. Subject having undergone surgical/endovascular revascularization or major/minor
amputation, in either leg, less than 1 month prior to Screening.
5. Planned or potential need for major/minor amputation or any revascularization of
either leg within 1 month of EAP entry upon physician's judgment.
6. Aortoiliac stenosis or common femoral artery stenosis ≥70%, or otherwise suspicion of
inadequate inflow to the index leg at the time of Screening.
7. Current evidence or sign supporting an assessment of life expectancy of less than 6
months.
8. Stroke or acute myocardial infarction within 3 months prior to Screening.
9. Severe congestive heart failure symptoms (New York Heart Association [NYHA] Stage IV)
at screening.
10. Life-threatening ventricular arrhythmia - except in subjects with an implantable
cardiac defibrillator at screening.
11. Uncontrolled severe hypertension during Screening.
12. Current or history of proliferative retinopathy.
13. Known active Hepatitis B virus or Hepatitis C virus infections at Screening.
14. Acquired immunodeficiency syndrome (AIDS), severe uncontrolled inflammatory disease,
or severe uncontrolled autoimmune disease (e.g., ulcerative colitis, Crohn's disease,
etc.).
15. Subjects at an increased risk of blood clotting or bleeding according to the
Physician's judgment.
16. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3×ULN. Subjects
with higher levels may be included if the condition associated with the increase in
those liver enzymes is known and is considered clinically stable.
17. Current drug or alcohol abuse.
18. Subject is currently enrolled in, or has not yet completed a period of at least 30
days since ending another investigational device or drug trial(s) unless in long-term
follow-up phase (in which there is no IP administration).
19. Current use or use within 30 days prior to screening of wound dressing containing
cells or growth factors like Apligraf®, or topical platelet derived growth factor.
20. Current use, planned use, or use within 15 days prior to treatment of hyperbaric
oxygen therapy, spinal cord stimulation, or lumbar sympathectomy.
21. Exposure to allogeneic cell based therapy in the past or exposure to autologous cell
therapy in the last 12 months before screening.
22. Known allergies to any of the following: dimethyl sulfoxide (DMSO), human serum
albumin, bovine serum albumin.
23. History of allergic/hypersensitivity reaction to any substance having required
hospitalization and/or treatment with intravenous steroids/epinephrine, known allergy
to more than 3 allergens, or in the opinion of the Physician the subject is at high
risk of developing severe allergic/hypersensitivity reactions.
24. History of severe atopic disease (including but not limited to chronic urticaria,
respiratory allergy requiring oral steroids), or history of uncontrolled Asthma
(Global Initiative for Asthma [GINA] III-IV).
1. Pulmonary disease requiring supplemental oxygen treatment on a daily basis.
2. History of acute transfusion reaction.
3. History of allogeneic bone marrow transplantation.
4. Active malignancy except for successfully resected skin basal cell carcinoma or
not located on the index leg.
5. Pregnant or lactation women
6. Inability to understand and provide an informed consent.
Gender
All
Minimum Age
45 Years
Maximum Age
99 Years
Overall Contact
Verification Date
2019-01-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keyword
Condition Browse
Firstreceived Results Date
N/A
Firstreceived Results Disposition Date
N/A
Expanded Access Info
Expanded Access Type Intermediate
Yes
Study First Submitted
November 14, 2018
Study First Submitted Qc
November 14, 2018
Study First Posted
November 20, 2018
Last Update Submitted
January 7, 2019
Last Update Submitted Qc
January 7, 2019
Last Update Posted
January 8, 2019
ClinicalTrials.gov processed this data on December 13, 2019
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Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
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that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.