Myelodysplastic SyndromesPhase 1NCT03258359
Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)
A Phase 1 study testing PACTN in people with Myelodysplastic Syndromes, run by PersImmune, Inc at 1 site in United States.
Trial summary
This study will evaluate the safety of autologous T cells that have been immunized ex vivo with patient-specific MDS stem cell neoantigens in patients with MDS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- PACTN
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2018to 1 Dec 2020
- 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
- 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.
- Relapsed/refractory disease, or inadequate response to at least 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.
- 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.
- Age >18 year at the time of obtaining informed consent, male or female.
- An Eastern Cooperative Oncology Grou (ECOG) performance status score of 0, 1, or 2.
- Adequate organ function.
Show all 10Show fewer
- 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 be tested for the presence of antigen by RT-PCR and be HCV RNA negative.
- Women of childbearing potential must have negative pregnancy test prior to initiating study treatment.
- Life expectancy >6 months at time of screening.
- Ability to adhere to the protocol requirements and study visit schedule.
Who cannot join
- 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.
- 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 dur to agents administered more than 28 days earlier.
- Subjects who are less than 21 days from surgery or have insufficient recovery from surgical-related trauma or wound healing.
- Prior history of allogeneic hematopoietic stem cell transplantation.
- Current use of granulocyte colony-stimulating factory (G-CSF) or GM-CSF.
- History of major organ autoimmune disease.
Show all 13Show fewer
- Concurrent immunosuppressive therapy. A stable dose of prednisone <10 mg daily or inhaled corticosteroids are allowed.
- Any form of primary immunodeficiency.
- Active bacillus tuberculosis (TB) or any other active or uncontrolled infection.
- Pior 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.
- Impaired cardiac function.
- Pregnant women are excluded from this study as the proposed treatment has not been well studied in pregnant subjects.
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Diego
San Diego, California, 92093
About this study
Official title: A Phase 1 Clinical Trial of Personalized, Adoptive Cellular Immunotherapy Targeting Patient-specific Neoplastic Stem Cell Neoantigens (PACTN) in Patients With Myelodysplastic Syndromes (MDS)
This study will evaluate the safety of autologous T cells that have been immunized ex vivo with patient-specific MDS stem cell neoantigens in patients with MDS.
Read the detailed description
PACTN is manufactured by a novel method to employ cancer-specific somatic variants (mutations) as a means to immunize autologous T lymphocytes to specifically kill cancer cells bearing the protein products of the mutations.
The PACTN method is based on the premise that somatic DNA mutations that cause cancer often give rise to proteins with an altered amino acid sequence. Peptides derived from these proteins, if expressed in the context of MHC Class I or II may be perceived as "non-self" by the immune system; that is, they may be perceived as neoantigens (aka, neoepitopes). Such neoantigens could therefore serve as immunogenic targets for the development of patient-specific, personalized T cell mediated immunotherapy.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- PACTN
- Keywords
- MDS
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PACTNBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| PACTN | Experimental | Open label 3+3 dose escalation phase 1 trial; 200 to 1000 mL of immunized T cells infused at 0.3, 1, and 3 x 10e7 nucleated cells/kg body weight. Receives: Biological: PACTN |
What is being measured
The primary outcome is the main question the study is designed to answer.
Acute and subacute toxicities and AEs
The incidence of dose limiting toxicities (DLTs) after PACTN infusion will be used to determine the maximum tolerated dose (MTD). Adverse effects (AEs) and in particular cytokine release syndrome (CRS) and potential autoimmune AEs will be monitored.
Measured: baseline to four weeks after infusion
Secondary outcomes (3)
Persistence, abundance, and activity of PACTN
Determined by quantity of PACTN in the subject's blood sample, assessed by the unique phenotype of PACTN lymphocytes and by functional measurement of PACTN activity (antigen-specific cytotoxicity)
Measured: Samples will be collected on days 1, 4, 8, 15, 36, and 57, and then 3, 6, and 12 months
Disease Response
Disease response will be assessed by International Working Group (IWG) criteria on bone marrow aspiration
Measured: Samples will be collected between day 29 and 43, and then at 3, 6, and 12 months
Overall and progression-free survival of subjects who receive PACTN
Incidence of subjects who are alive, and both alive and disease - free will be assessed at 6 and 12 months
Measured: Six and 12 months after PACTN infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2017
- Study start1 Jan 2018
- Primary completion1 Dec 2020 (estimated)
- Study completion1 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies 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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PersImmune, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of California, San Diego
- Responsible party
- Sponsor
- Investigators
- Antonella Vitiello, PhD, Study director, PersImmune, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03258359
- Sponsor's protocol ID
- PACTN-02
- Status last verified
- Mar 2020
- First submitted
- 18 Aug 2017
- Last updated
- 4 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Clinical Trial of Personalized, Adoptive Cellular Immunotherapy Targeting Patient-specific Neoplastic Stem Cell Neoantigens (PACTN) in Patients With Myelodysplastic Syndromes (MDS)
- ClinicalTrials.gov ID
- NCT03258359
- Acronym / sponsor ID
- PACTN-02
- Conditions
- Myelodysplastic Syndromes
- Keywords
- MDS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- PersImmune, IncIndustry (pharma, biotech, devices)
- Collaborators
- University of California, San Diego
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 1 Dec 2020 (estimated)
- Study completion
- 1 Dec 2020 (estimated)
- First posted
- 23 Aug 2017 (actual)
- 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 will evaluate the safety of autologous T cells that have been immunized ex vivo with patient-specific MDS stem cell neoantigens in patients with MDS.
Detailed description
PACTN is manufactured by a novel method to employ cancer-specific somatic variants (mutations) as a means to immunize autologous T lymphocytes to specifically kill cancer cells bearing the protein products of the mutations.
The PACTN method is based on the premise that somatic DNA mutations that cause cancer often give rise to proteins with an altered amino acid sequence. Peptides derived from these proteins, if expressed in the context of MHC Class I or II may be perceived as "non-self" by the immune system; that is, they may be perceived as neoantigens (aka, neoepitopes). Such neoantigens could therefore serve as immunogenic targets for the development of patient-specific, personalized T cell mediated immunotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PACTN | Experimental | Biological: PACTNOpen label 3+3 dose escalation phase 1 trial; 200 to 1000 mL of immunized T cells infused at 0.3, 1, and 3 x 10e7 nucleated cells/kg body weight. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
- Relapsed/refractory disease, or inadequate response to at least 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.
- 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.
- Age >18 year at the time of obtaining informed consent, male or female.
- An Eastern Cooperative Oncology Grou (ECOG) performance status score of 0, 1, or 2.
Key exclusion criteria (13 listed)
- 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.
- 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 dur to agents administered more than 28 days earlier.
- Subjects who are less than 21 days from surgery or have insufficient recovery from surgical-related trauma or wound healing.
- Prior history of allogeneic hematopoietic stem cell transplantation.
- Current use of granulocyte colony-stimulating factory (G-CSF) or GM-CSF.
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 |
|---|---|---|
| PACTNTo treat patients with MDS who have failed treatment with hypomethylating agents or have relapsed after treatment with hypomethylating agents or have declined hypomethylating therapy. | Biological | PACTN |
Primary outcome
Acute and subacute toxicities and AEs
Time frame: baseline to four weeks after infusion
The incidence of dose limiting toxicities (DLTs) after PACTN infusion will be used to determine the maximum tolerated dose (MTD). Adverse effects (AEs) and in particular cytokine release syndrome (CRS) and potential autoimmune AEs will be monitored.
3 secondary outcomes
Persistence, abundance, and activity of PACTN
Time frame: Samples will be collected on days 1, 4, 8, 15, 36, and 57, and then 3, 6, and 12 months
Disease Response
Time frame: Samples will be collected between day 29 and 43, and then at 3, 6, and 12 months
Overall and progression-free survival of subjects who receive PACTN
Time frame: Six and 12 months after PACTN infusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2020.
Related trials for Myelodysplastic Syndromes
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingObservational study
Natural History Study of GATA2 Deficiency and Related Disorders
GATA2 Deficiency
RecruitingObservational study
Cancer Experience Registry (CER) for Cancer Patients and Caregivers
Neoplasms, Cancer
RecruitingPhase 2
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
RecruitingPhase 2
Trial of 2 Step ATG for Prevention of Acute GVHD Post Allogeneic Stem Cell Transplant
Acute Leukemia, Myelodysplastic Syndrome
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.
Antonella Vitiello, PhD study director
PersImmune, Inc
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PACTN in people with Myelodysplastic Syndromes, run by PersImmune, Inc at 1 site in United States.
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 10 inclusion and 13 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: status not confirmed, as of the sponsor's last update on 4 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
Results are not available in the source record.
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?
Acute and subacute toxicities and AEs (time frame: baseline to four weeks after infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Mar 2020; AzTrials last refreshed its copy on 30 Oct 2026.