Incomplete HCT (Hematopoietic Cell Transplantation)Phase 1NCT03002519
Safety Evaluation of Intramuscular Injections of PLX-R18 in Subjects With Incomplete Hematopoietic Recovery Following Hematopoietic Cell Transplantation
A Phase 1 study testing PLX-R18 in people with Incomplete HCT (Hematopoietic Cell Transplantation), run by Pluristem Ltd. at 10 sites in 2 countries.
Trial summary
This study aims to evaluate the safety of intramuscular (IM) administration of PLX-R18 in subjects with incomplete hematopoietic recovery following HCT. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Incomplete HCT (Hematopoietic Cell Transplantation)
- What is being tested?
- PLX-R18
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 8 Feb 2017to 30 Oct 2021
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
- Subjects must meet all of the inclusion criteria listed below to be eligible for the study:
- 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).
- 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. Exclusion Criteria
- 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.
- 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).
- A known history of allergic/hypersensitivity reactions to 3 or more allergens.
- History of uncontrolled asthma (Global Initiative for Asthma Grade III IV).
- History of severe atopic disease (including but not limited to chronic urticaria, allergic reaction with respiratory symptoms requiring systemic steroids).
- Medical history of human immunodeficiency virus or syphilis infection.
- Known active hepatitis B or hepatitis C infection at the time of screening.
- 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:
- Oral/intravaginal/transdermal combined estrogen and progestogen containing hormonal contraception for at least 3 months prior to screening.
- Oral/injectable/implantable progestogen-only hormonal contraception for at least 3 months prior to screening.
- An intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
- 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).
- Serum glutamic pyruvic transaminase (alanine aminotransferase), serum oxaloacetic pyruvic transaminase (aspartate aminotransferase) >2.5 x upper limit of normal range.
- 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.
- Severe or uncontrolled/unstable cardiac, pulmonary, or renal disease, including myocardial infarction or cerebrovascular accident within 3 months prior to treatment.
- History of solid organ transplantation.
- Signs and symptoms of active central nervous system disease.
- Life expectancy <6 months as assessed by the Investigator.
- Subject has participated in a clinical interventional study and received the last treatment within 30 days prior to screening.
- In the opinion of the Investigator, the subject is unsuitable for participating in the study.
Who cannot join
None listed.
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States7 sites
University of Maryland Medical Center,22 South Greene Street
Baltimore, Maryland, 21201
The University of Chicago Medical Center
Chicago, Illinois, 60637-1447
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
Baylor University Medical Center
Dallas, Texas, 75246
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Mayo Clinic Cancer Center (MCCC) - Rochester,200 1st Street SW
Rochester, Minnesota, 55905
University of Kansas Cancer Center - The Richard and Annette Bloch Cancer Care Pavilion,2330 Shawnee Mission Parkway
Westwood, Kansas, 66205-2005
Israel3 sites
Rambam Medical Center
Haifa
Hadassah Ein Karem Medical Center
Jerusalem
Sheba Medical Center
Ramat Gan
About this study
Official title: A Phase I Open-label Dose-escalation Study to Evaluate the Safety of Intramuscular Injections of PLX-R18 in Subjects With Incomplete Hematopoietic Recovery Following Hematopoietic Cell Transplantation
This study aims to evaluate the safety of intramuscular (IM) administration of PLX-R18 in subjects with incomplete hematopoietic recovery following HCT.
- Being tested
- PLX-R18
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PLX-R18Biological
Intramuscular (IM) administration of PLX-R18
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: PLX-R18 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Emergent Adverse Events (AEs)
Measured: Recorded after time of consent throughout the study until the last visit (~1 year)
Safety laboratory values (including immunological testing)
Measured: Blood samples will be collected on each visit, through study completion (~1 year)
Vital signs
Measured: Will be assessed during each visit, through study completion (~1 year)
ECG
Measured: Will be assessed during the screening visit, before each IP treatment visit and on the 14th day of participation (visit 5).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2016 (estimated)
- Study start8 Feb 2017
- Primary completion30 Sep 2021
- Study completion30 Oct 2021
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 Incomplete HCT (Hematopoietic Cell Transplantation)Studies 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pluristem Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03002519
- Sponsor's protocol ID
- PLX-R18-HCT-01
- Status last verified
- Dec 2022
- First submitted
- 13 Dec 2016
- Last updated
- 16 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 37553467
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Open-label Dose-escalation Study to Evaluate the Safety of Intramuscular Injections of PLX-R18 in Subjects With Incomplete Hematopoietic Recovery Following Hematopoietic Cell Transplantation
- ClinicalTrials.gov ID
- NCT03002519
- Acronym / sponsor ID
- PLX-R18-HCT-01
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Pluristem Ltd.Industry (pharma, biotech, devices)
- Study start
- 8 Feb 2017 (actual)
- Primary completion
- 30 Sep 2021 (actual)
- Study completion
- 30 Oct 2021 (actual)
- First posted
- 23 Dec 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 study aims to evaluate the safety of intramuscular (IM) administration of PLX-R18 in subjects with incomplete hematopoietic recovery following HCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PLX-R18 | Experimental | Biological: PLX-R18Dose 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (45 listed)
- 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).
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| PLX-R18Intramuscular (IM) administration of PLX-R18 | Biological | PLX-R18 |
Primary outcome
Emergent Adverse Events (AEs)
Time frame: Recorded after time of consent throughout the study until the last visit (~1 year)
Safety laboratory values (including immunological testing)
Time frame: Blood samples will be collected on each visit, through study completion (~1 year)
Vital signs
Time frame: Will be assessed during each visit, through study completion (~1 year)
ECG
Time frame: Will be assessed during the screening visit, before each IP treatment visit and on the 14th day of participation (visit 5).
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 16 Dec 2022.
- Derived from this study: PubMed 37553467McGuirk 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.
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.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PLX-R18 in people with Incomplete HCT (Hematopoietic Cell Transplantation), run by Pluristem Ltd. at 10 sites in 2 countries.
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 45 inclusion and 0 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 16 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Israel. 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 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?
Emergent Adverse Events (AEs) (time frame: Recorded after time of consent throughout the study until the last visit (~1 year)). The study lists 4 primary outcomes in all. 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 NCT03002519, 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 16 Dec 2022.