A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, Umbilical Cord Blood-derived, Stem and Progenitor Cells, versus Unmanipulated Umbilical Cord Blood for Patients with Hematological Malignancies | Aplastic Anemia & MDS International Foundation

ClinicalTrials.gov Identifier:

NCT03258359

Company

PersImmune Logo

Contact Info

Kimberly Aguilar
k1aguilar@ucsd.edu
(858) 534-5201

Dates

Start: February 2018
End: December 2020

Official Title

Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)

Purpose

Patients with higher risk myelodysplastic syndromes (MDS) who fail current approved therapy or become refractory, have few therapeutic options other than bone marrow transplant, which may not be an option due to age, infirmity or lack of a donor. This is a Phase 1 open label, dose escalation clinical trial of Personalized, Adoptive immunotherapy by Cytotoxic T cells that are targeted to patient-specific cancer cell Neoantigens (PACTN) for such patients. It’s primary goal is to learn the safety and tolerability of PACTN infusion and secondarily, if PACTN show signs of being effective. The trial is currently recruiting patients at the University of California, San Diego Moores Cancer Center. Patients will first participate in a blood collection protocol to ensure the availability of sufficient blood cells to conduct the study. Eligible patient may then participate in the clinical trial and receive an infusion of their T cells that have been shown to eliminate MDS cells in the laboratory. Patient will be intensively followed for adverse effects for 6 weeks after PACTN infusion and periodically for up to 1 year.

Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)

Clinical Trial: NCT03258359

Instructions

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Status: 
Recruiting
Phase: 
Phase 1
Gender: 
Female
Male
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
No
Inclusion Criteria: 
  1. Confirmed diagnosis of MDS according to the French-American-British (FAB) criteria. Subjects with MDS must have intermediate, high, or very high risk IPSS-R scores and cytopenia of at least one lineage.
  2. Relapsed/refractory disease, or inadequate response to at least 4-6 cycles of hypomethylating (HMA) therapy or subjects who decline HMA therapy. Subjects must not have received any MDS or AML directed therapy for >28 days prior to receiving the study treatment.
  3. Subjects who have opted not to undergo allogeneic hematopoietic stem cell transplantation or for whom no donor is available and who are not deemed eligible for high intensity chemotherapy.
  4. Age >18 year at the time of obtaining informed consent, male or female.
  5. An Eastern Cooperative Oncology Grou (ECOG) performance status score of 0, 1, or 2. Adequate organ function.
  6. Seronegative test for HIV-1/2 and hepatitis C antibodies (HCV), and a negative test for Hepatitis B antigen (HBsAg). If hepatitis C antibody test is positive, then the subject must betested for the presence of antigen by RT-PCR and be HCV RNA negative.
  7. Women of childbearing potential must have negative pregnancy test prior to initiating study treatment.
  8. Life expectancy >6 months at time of screening.
  9. Ability to adhere to the protocol requirements and study visit schedule.
  10. Prior participation in PersImmune’s pre-clinical collection study, NCT03072498
Exclusion Criteria: 
  1. Subjects who anticipate use of other investigational or non-investigational agents for the treatment of MDS during the study period, aside from a stable dose of erythropoietin stimulating agent started >8 weeks prior to screening for this study.
  2. Subjects who have received investigational agents, cytotoxic chemotherapy, or radiotherapy within 28 days prior to entering the study, or who have not recovered from AEs due to agents administered more than 28 days earlier.
  3. Subjects who are less than 21 days from surgery or have insufficient recovery from surgical-related trauma or wound healing.
  4. Prior history of allogeneic hematopoietic stem cell transplantation.
  5. Current use of granulocyte colony-stimulating factory (G-CSF) or GM-CSF.
  6. History of major organ autoimmune disease.
  7. Concurrent immunosuppressive therapy. A stable dose of prednisone <10 mg daily or inhaled corticosteroids are allowed.
  8. Any form of primary immunodeficiency.
  9. Active bacillus tuberculosis (TB) or any other active or uncontrolled infection.
  10. Prior history of treated malignancy in the past 2 years. Subjects with non-melanoma skin cancer, localized prostate cancer, and carcinoma in situ of the breast of cervix are allowed.
  11. Impaired cardiac function.
  12. Pregnant women are excluded from this study as the proposed treatment has not been well studied in pregnant subjects.
  13. Any other medical or psychiatric disorders, or social situation, that would, in the investigator's opinion, place the subject at unacceptable risk if he/she participates in the study.
Intervention: 
Biological: PACTN To treat patients with MDS who have failed treatment with hypomethylating agents or have relapsed after treatment with hypomethylating agents or have declined hypomethylating therapy.

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