Safety Evaluation of Intramuscular Injections of PLX-R18 in Subjects With Incomplete Hematopoietic Recovery Following Hematopoietic Cell Transplantation

NCT ID: NCT03002519

Last Updated: 2022-12-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-02-08

Study Completion Date

2021-10-30

Brief Summary

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This study aims to evaluate the safety of intramuscular (IM) administration of PLX-R18 in subjects with incomplete hematopoietic recovery following HCT.

Detailed Description

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Conditions

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Incomplete HCT (Hematopoietic Cell Transplantation)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PLX-R18

Dose Escalation- first three subjects will be enrolled in the low dose cohort, 6 subjects in the intermediate-dose cohort, and 15 subjects in the high dose cohort.

Group Type EXPERIMENTAL

PLX-R18

Intervention Type BIOLOGICAL

Intramuscular (IM) administration of PLX-R18

Interventions

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PLX-R18

Intramuscular (IM) administration of PLX-R18

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. Age ≥18 years.
2. At least 3 months after HCT, either autologous or allogeneic (of any source, with any preparatory regimen, for any indication), prior to study treatment.
3. Sustained platelet count ≤50,000/µL, and/or sustained Hb ≤8 g/dL and/or sustained ANC ≤1000/mm3, attributed to graft failure as a major contributor, as evident by hypocellular bone marrow.

Cytopenia should be confirmed by at least 2 consecutive blood counts, at least one of them within 28 days prior to treatment (higher transient levels following occasional blood product transfusions are allowed).
4. Stable donor cell chimerism in at least 3 consecutive tests prior to treatment (the most recent test should be within 28 days prior to treatment).
5. If the subject had allogeneic HCT for a malignant disease, the subject should have complete donor chimerism.

\*complete donor chimerism should be determined by the investigator per site's standards.
6. General performance status evaluated by Eastern Cooperative Oncology Group 0-2 scale.
7. Signed written informed consent.



1. Age ≥18 years.
2. At least 3 months after HCT, either autologous or allogeneic (of any source, with any preparatory regimen, for any indication), prior to study treatment.
3. Sustained platelet count ≤50,000/µL, and/or sustained Hb ≤8 g/dL and/or sustained ANC ≤1000/mm3, attributed to graft failure as a major contributor, as evident by hypocellular bone marrow.

Cytopenia should be confirmed by at least 2 consecutive blood counts, at least one of them within 28 days prior to treatment (higher transient levels following occasional blood product transfusions are allowed).

4\. Stable donor cell chimerism in at least 3 consecutive tests prior to treatment (the most recent test should be within 28 days prior to treatment).

5\. If the subject had allogeneic HCT for a malignant disease, the subject should have complete donor chimerism.

\*complete donor chimerism should be determined by the investigator per site's standards.

6\. General performance status evaluated by Eastern Cooperative Oncology Group 0-2 scale.

Exclusion Criteria

1. Evidence of developing malignancy since the HCT, or any evidence of malignancy at the time of screening.
2. Current active infection requiring systemic treatment (if infection resolved but antibiotic coverage continues, patient may be included).
3. Acute graft versus host disease (GvHD) Grade III or IV, or severe chronic GvHD at the time of screening.
4. Subject has received prophylactic treatment with donor lymphocyte infusion (DLI) within 6 months prior to treatment, or any other cell therapy within 3 months prior to treatment.
5. History of malignancy (other than the disease that required the HCT) within 2 years prior to screening (except for skin basal cell carcinoma or squamous cell carcinoma lesions that were fully resected with no need for further treatment, and not located at the injection site).
6. History of significant transfusion reaction including: Transfusion related acute lung injury (pulmonary edema), shock, severe disturbances of liver function tests, renal dysfunction, or hemolytic anemia (as part of the transfusion reaction).
7. Known allergies to any of the following: dimethyl sulfoxide (DMSO), human serum albumin, bovine serum albumin, gentamicin, or antihistamine.
8. History of allergic/hypersensitivity reaction to any substance having required hospitalization and/or treatment with intra-venous steroids/epinephrine or in the opinion of the Investigator the subject is at high risk of developing severe allergic/hypersensitivity reactions (does not apply to transfusion reactions - see exclusion criterion 8).
9. A known history of allergic/hypersensitivity reactions to 3 or more allergens.
10. History of uncontrolled asthma (Global Initiative for Asthma Grade III IV).
11. History of severe atopic disease (including but not limited to chronic urticaria, allergic reaction with respiratory symptoms requiring systemic steroids).
12. Medical history of human immunodeficiency virus or syphilis infection.
13. Known active hepatitis B or hepatitis C infection at the time of screening.
14. A pregnant or lactating woman or a woman who plans to become pregnant during the study. In addition, any woman of childbearing potential (not sterile or postmenopausal), who is unwilling to adhere to the use of a highly effective contraception method for the duration of the study:

1. Oral/intravaginal/transdermal combined estrogen and progestogen containing hormonal contraception for at least 3 months prior to screening.
2. Oral/injectable/implantable progestogen-only hormonal contraception for at least 3 months prior to screening.
3. An intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
15. Subjects on renal replacement therapy or with estimated glomerular filtration rate (eGFR) \<15 mL/min/1.73m2 (based on Modification of Diet in Renal Disease \[MDRD\] equation).
16. Serum glutamic pyruvic transaminase (alanine aminotransferase), serum oxaloacetic pyruvic transaminase (aspartate aminotransferase) \>2.5 x upper limit of normal range.
17. International normalized ratio (INR) \>2 or subjects who are on oral anticoagulant therapy with INR \>2 unless anticoagulation treatment can be safely interrupted/discontinued around each investigational product (IP) treatment upon primary care physician and/or Investigator's discretion.
18. Severe or uncontrolled/unstable cardiac, pulmonary, or renal disease, including myocardial infarction or cerebrovascular accident within 3 months prior to treatment.
19. History of solid organ transplantation.
20. Signs and symptoms of active central nervous system disease.
21. Life expectancy \<6 months as assessed by the Investigator.
22. Subject has participated in a clinical interventional study and received the last treatment within 30 days prior to screening.
23. In the opinion of the Investigator, the subject is unsuitable for participating in the study.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pluristem Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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The University of Chicago Medical Center

Chicago, Illinois, United States

Site Status

University of Kansas Cancer Center - The Richard and Annette Bloch Cancer Care Pavilion,2330 Shawnee Mission Parkway

Westwood, Kansas, United States

Site Status

University of Maryland Medical Center,22 South Greene Street

Baltimore, Maryland, United States

Site Status

Mayo Clinic Cancer Center (MCCC) - Rochester,200 1st Street SW

Rochester, Minnesota, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

University Hospitals Case Medical Center

Cleveland, Ohio, United States

Site Status

Baylor University Medical Center

Dallas, Texas, United States

Site Status

Rambam Medical Center

Haifa, , Israel

Site Status

Hadassah Ein Karem Medical Center

Jerusalem, , Israel

Site Status

Sheba Medical Center

Ramat Gan, , Israel

Site Status

Countries

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United States Israel

References

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McGuirk JP, Metheny L 3rd, Pineiro L, Litzow M, Rowley SD, Avni B, Tamari R, Lazarus HM, Rowe JM, Sheleg M, Rothenstein D, Halevy N, Zuckerman T. Placental expanded mesenchymal-like cells (PLX-R18) for poor graft function after hematopoietic cell transplantation: A phase I study. Bone Marrow Transplant. 2023 Nov;58(11):1189-1196. doi: 10.1038/s41409-023-02068-3. Epub 2023 Aug 8.

Reference Type DERIVED
PMID: 37553467 (View on PubMed)

Other Identifiers

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PLX-R18-HCT-01

Identifier Type: -

Identifier Source: org_study_id