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

NCT03002519 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PLX-R18-HCT-01
• Study Type: interventional
• Target: 21
• Locations: 2 countries
• Sites: 10

Brief Summary

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

Conditions

Incomplete HCT (Hematopoietic Cell Transplantation)

Outcome Measures

Primary Outcomes (4)

• Emergent Adverse Events (AEs)

  Recorded after time of consent throughout the study until the last visit (~1 year)

• Safety laboratory values (including immunological testing)

  Blood samples will be collected on each visit, through study completion (~1 year)

• Vital signs

  Will be assessed during each visit, through study completion (~1 year)

• ECG

  Will be assessed during the screening visit, before each IP treatment visit and on the 14th day of participation (visit 5).

Study Arms (1)

PLX-R18

EXPERIMENTAL

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.

Biological: PLX-R18

Interventions

PLX-R18 BIOLOGICAL

Intramuscular (IM) administration of PLX-R18

PLX-R18

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 years.
• At least 3 months after HCT, either autologous or allogeneic (of any source, with any preparatory regimen, for any indication), prior to study treatment.
• 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).
• 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).
• 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.
• General performance status evaluated by Eastern Cooperative Oncology Group 0-2 scale.
• Signed written informed consent.

You may not qualify if:

• Age ≥18 years.
• At least 3 months after HCT, either autologous or allogeneic (of any source, with any preparatory regimen, for any indication), prior to study treatment.
• 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).
• \. 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).
• \. 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.
• \. General performance status evaluated by Eastern Cooperative Oncology Group 0-2 scale.
• Evidence of developing malignancy since the HCT, or any evidence of malignancy at the time of screening.
• Current active infection requiring systemic treatment (if infection resolved but antibiotic coverage continues, patient may be included).
• Acute graft versus host disease (GvHD) Grade III or IV, or severe chronic GvHD at the time of screening.
• 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.
• 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).
• 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).
• Known allergies to any of the following: dimethyl sulfoxide (DMSO), human serum albumin, bovine serum albumin, gentamicin, or antihistamine.

Sponsors & Collaborators

• Pluristem Ltd.: lead

Study Sites (10)

The University of Chicago Medical Center

Chicago, Illinois, 60637-1447, United States

Location

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

Westwood, Kansas, 66205-2005, United States

Location

University of Maryland Medical Center,22 South Greene Street

Baltimore, Maryland, 21201, United States

Location

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

Rochester, Minnesota, 55905, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

University Hospitals Case Medical Center

Cleveland, Ohio, 44106, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Rambam Medical Center

Haifa, Israel

Location

Hadassah Ein Karem Medical Center

Jerusalem, Israel

Location

Sheba Medical Center

Ramat Gan, Israel

Location

Related Publications (1)

• 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.

  PMID: 37553467 DERIVED

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2016
• First Posted: December 23, 2016
• Study Start: February 8, 2017
• Primary Completion: September 30, 2021
• Study Completion: October 30, 2021
• Last Updated: December 16, 2022

Record last verified: 2022-12

Locations

US (7); IL (3)