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Primary CNS LymphomaPhase 1NCT04134117

Tisagenlecleucel In Primary CNS Lymphoma

A Phase 1 study testing Tisagenlecleucel in people with Primary CNS Lymphoma and Refractory Primary CNS Lymphoma, run by Matthew J. Frigault, M.D. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

In this study, is researching the safety of tisagenlecleucel in participants with primary central nervous system lymphoma. . -The name of the study intervention is tisagenlecleucel. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Tisagenlecleucel
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
1 siteUnited States
Study dates
1 Jan 2019to 14 Apr 2022
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

  • Primary CNS Lymphoma in high risk elderly patients
  • New diagnosis of primary CNS lymphoma.
  • Voluntarily sign informed consent form(s)
  • ≥60 years of age at the time of signing informed consent
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 - 2
  • Have failed or are unable to tolerate definitive first-line methotrexate based therapy as defined by:
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  • Grade 3+ AKI and/or transaminitis preventing repeat treatment exposure and/or,
  • Failure to achieve a complete response (per IPCG) following two cycles of first line therapy,
  • --- Definitive first-line therapies must include high dose methotrexate-based therapy but may also include temozolomide, high dose cytarabine, pemetrexed, lenalidomide, ibrutinib and rituximab.
  • Whole-brain irradiation, lenalidomide monotherapy and ibrutinib monotherapy are considered first line therapy if patient was not eligible for methotrexate-based chemotherapy at time of initial treatment but now meets study eligibility criteria.
  • Adequate absolute lymphocyte count (ALC > 500 cells/ul) within one week of apheresis.
  • Adequate bone marrow function defined by absolute neutrophil count (ANC) >1000 cells/mm3without growth factor support, and untransfused platelet count >50,000 mm3 within 7 days.
  • Left ventricular ejection fraction >40%
  • Adequate hepatic function defined by aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) <2.5 × upper limit of normal (ULN) and direct bilirubin <1.5 × ULN
  • Adequate renal function defined by creatinine clearance >30 ml/min using the Cockcroft-Gault formula
  • International ratio (INR) or partial thromboplastin time (PTT) <1.5 × ULN, unless on a stable dose of anticoagulant for a thromboembolic event.
  • The effects of tisagenlecleucel T cells on the developing human fetus are unknown. For this reason, women of child-bearing potential and men with partners of childbearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to leukapheresis for at least 1-year post tisagenlecleucel infusion and until CAR T cells are no longer present by qPCR on two consecutive tests. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men with partners of childbearing potential treated or enrolled on this protocol must also agree to use adequate contraception prior to leukapheresis and until 4 months after tisagenlecleucel T cells administration.
  • Ability and willingness to adhere to the study visit schedule and all protocol requirements
  • Relapsed/Refractory Primary CNS Lymphoma
  • Diagnosis of relapsed/refractory PCNSL having received at least one prior line of CNS directed therapy.
  • Voluntarily sign informed consent form(s)
  • ≥18 years of age at the time of signing informed consent
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2
  • Adequate absolute lymphocyte count (ALC > 500 cells/ul) within one week of apheresis.
  • Adequate bone marrow function defined by absolute neutrophil count (ANC) >1000 cells/mm3without growth factor support, untransfused platelet count >50,000 mm3, and untransfused hemoglobin >9 g/dL.
  • Left ventricular ejection fraction >40%
  • Adequate hepatic function defined by aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) <2.5 × upper limit of normal (ULN) and direct bilirubin <1.5 × ULN
  • Adequate renal function defined by creatinine clearance >30 ml/min using the Cockcroft-Gault formula
  • International ratio (INR) or partial thromboplastin time (PTT) <1.5 × ULN, unless on a stable dose of anticoagulant for a thromboembolic event.
  • The effects of tisagenlecleucel T cells on the developing human fetus are unknown. For this reason, women of child-bearing potential and men with partners of childbearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to leukapheresis for at least 1-year post tisagenlecleucel infusion and until CAR T cells are no longer present by qPCR on two consecutive tests. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men with partners of childbearing potential treated or enrolled on this protocol must also agree to use adequate contraception prior to leukapheresis and until 4 months after tisagenlecleucel T cells administration.
  • Ability and willingness to adhere to the study visit schedule and all protocol requirements
  • No Active, uncontrolled, systemic bacterial, viral, or fungal infection.
  • Adequate renal function defined by creatinine clearance >30 ml/min using the Cockcroft-Gault formula

Who cannot join

  • Prior treatment with an any investigational cellular therapy.
  • Ongoing treatment with chronic immunosuppressants (e.g., cyclosporine). Systemic steroids are allowed up to a dose of dexamethasone 4mg daily or equivalent.
  • Ongoing systemic immunosuppression for acute and/or chronic GVH as a result of previous allogeneic bone marrow transplant.
  • Significant co-morbid condition or disease which in the judgment of the Principal Investigator would place the subject at undue risk or interfere with the study; examples include, but are not limited to, cirrhotic liver disease, sepsis, and/or recent significant traumatic injury.
  • Active, uncontrolled, systemic bacterial, viral, or fungal infection.
  • Active hepatitis B or hepatitis C infection.
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  • HIV infection.
  • Subjects with a history of class III or IV congestive heart failure or non- ischemic cardiomyopathy.
  • Subjects with second malignancies if the second malignancy has required therapy in the last 3 years or is not in complete remission; exceptions to this criterion include successfully treated non-metastatic basal cell or squamous cell skin carcinoma, or prostate cancer that does not require therapy other than hormonal therapy.
  • Pregnant or lactating women
  • Live virus vaccines within 2 weeks prior to planned start of lymphodepleting chemotherapy.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Massachusetts General Hospital

    Boston, Massachusetts, 02115

Other trials in United States

About this study

Official title: Pilot Study of Tisagenlecleucel, CD19-targeted Chimeric Antigen Receptor (CAR) T Cells, in Patients With Primary Central Nervous System Lymphoma

In this study, is researching the safety of tisagenlecleucel in participants with primary central nervous system lymphoma. .

-The name of the study intervention is tisagenlecleucel.

Read the detailed description

This research study is a Pilot Study, which is the first time investigators are examining this intervention in people with primary central nervous system lymphoma.

  • The name of the study intervention is tisagenlecleucel. Tisagenlecleucel is an investigational treatment that uses the participants own immune cells, called T cells, to try to kill the cancerous cells
  • The research study procedures include screening for eligibility and study treatment including, leukapheresis, evaluations, and follow up visits.
  • The study treatment will be one day and participants will be followed for up to 2 years.
  • It is expected that about 12 people will take part in this research study
Being tested
Tisagenlecleucel
Keywords
Primary CNS Lymphoma, Refractory Primary CNS Lymphoma, Relapsed Primary CNS Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TisagenlecleucelBiological

    Also called KYMRIAH

    One time single predetermined dose level CAR-positive T cells will be utilized based on the FDA approved product label.

Study groups in NCT04134117
Study groupRoleWhat they receive
TisagenlecleucelExperimentalStudy procedures include screening for eligibility and study treatment including, leukapheresis, evaluations, and follow up visits. - Tisagenlecleucel will be administered intravenously as a one-time rapid infusion predetermined dose following lymphodepleting chemotherapy.
Receives: Biological: Tisagenlecleucel

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE Criteriaand ASTCT 2018 (CRS/NT)

    Measured: 12 Months

Secondary outcomes (6)

  1. Objective disease response to tisagenlecleucel

    IPCG response criteria.

    Measured: 1 Month

  2. Objective disease response to tisagenlecleucel

    IPCG response criteria.

    Measured: 3 Months

  3. Objective disease response to tisagenlecleucel

    IPCG response criteria.

    Measured: 6 months

  4. Objective disease response to tisagenlecleucel

    IPCG response criteria.

    Measured: 12 months

  5. Overall Survival Rate

    Kaplan-Meier method

    Measured: 15 years

  6. Progression Free Survival Rate

    Kaplan-Meier method

    Measured: from the date of assignment until the date of first documented progression or date of deathfrom any cause, whichever comes first, assessed up to 100 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Oct 2019
  2. Study start1 Jan 2019
  3. Primary completion14 Apr 2022
  4. Study completion14 Apr 2022

Resources

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
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04134117
Sponsor's protocol ID
19-319
Status last verified
Jan 2026
First submitted
18 Oct 2019
Last updated
9 Jan 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Tisagenlecleucel in people with Primary CNS Lymphoma and Refractory Primary CNS Lymphoma, run by Matthew J. Frigault, M.D. 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 33 inclusion and 11 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 9 Jan 2026. 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?

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?

Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE Criteriaand ASTCT 2018 (CRS/NT) (time frame: 12 Months). 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 NCT04134117.

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 9 Jan 2026; AzTrials last refreshed its copy on 3 Nov 2026.

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