Long-term Safety of UCART19 for Leukemia

Not currently recruiting at 17 trial locations

Overseen By Institut de Recherches Internationales Servier

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Institut de Recherches Internationales Servier

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the safety and effectiveness of UCART19 for individuals with advanced lymphoid leukemia, a type of blood cancer. It examines the long-term effects of UCART19 treatment. Those who have previously received UCART19 in studies or special programs are eligible for this trial. Participants must commit to using effective birth control for a year after their last UCART19 treatment.

As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants an opportunity to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that UCART19 is likely to be safe for humans?

Research shows that UCART19 is generally safe for patients with relapsed B-cell acute lymphoblastic leukemia. These "off-the-shelf" CAR T cells involve modifying certain white blood cells to attack cancer cells. Studies have found that UCART19 can be used safely, with patients typically experiencing mild side effects that doctors can manage.

In various studies, both adults and children with difficult-to-treat leukemia have received UCART19. These studies suggest that UCART19 can be used without causing major harm. However, this treatment remains in the early stages of testing, so more research is needed to fully understand its long-term safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike traditional treatments for leukemia that often rely on chemotherapy or radiation, UCART19 offers a novel approach by using engineered T-cells to target and destroy cancer cells. This treatment is unique because it involves "off-the-shelf" CAR-T cell therapy, which means the T-cells are pre-manufactured and ready for use, potentially allowing for quicker administration compared to personalized CAR-T therapies that need to be tailored to each patient. Researchers are excited about UCART19 due to its potential to provide a more accessible and efficient treatment option with the ability to precisely target leukemia cells, minimizing harm to healthy cells.

What evidence suggests that UCART19 might be an effective treatment for leukemia?

Research has shown that UCART19, the treatment under study in this trial, holds promise for treating advanced lymphoid leukemia. In one study, 88% of patients who received UCART19 achieved complete or nearly complete remission within 28 to 42 days. Among these patients, 86% had no detectable cancer cells. The treatment generally did not cause severe side effects for most patients, indicating a manageable safety profile. These findings suggest that UCART19 could be an effective option for those with relapsed or difficult-to-treat B-cell acute lymphoblastic leukemia. ^{3 5 6 7 8}

Who Is on the Research Team?

Reuben Benjamin

Principal Investigator

King's College Hospital NHS Trust

Are You a Good Fit for This Trial?

This trial is for patients with advanced lymphoid leukemia who have previously received UCART19, either through a past study or compassionate use. Participants must agree to effective birth control for 12 months post-treatment and provide written consent.

Inclusion Criteria

Written informed consent obtained prior any study-specific procedure (patient or parent(s) or legal representative)

I have previously been treated with UCART19.

I am using effective birth control and will continue for 12 months after my last treatment.

Exclusion Criteria

No exclusion criteria for this study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants previously administered with UCART19 are monitored for safety and efficacy

12 months

Follow-up

Participants are monitored for long-term safety and effectiveness after treatment

Up to 15 years

What Are the Treatments Tested in This Trial?

Interventions

• UCART19

Trial Overview The study focuses on the long-term safety and effectiveness of UCART19 in individuals with advanced lymphoid leukemia. It's a follow-up evaluation for those who've already been treated with this intervention.

How Is the Trial Designed?

Group I: UCART19 follow-up Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Institut de Recherches Internationales Servier

Lead Sponsor

Trials

91

Recruited

67,100+

ADIR, a Servier Group company

Industry Sponsor

Trials

33

Recruited

4,300+

Published Research Related to This Trial

Over 20 years, 250 adults with acute lymphoblastic leukemia (ALL) were treated with various multidrug protocols, with the L10 and L10M protocols showing the highest long-term remission rates of 52% and 40%, respectively, compared to more recent protocols.

The overall long-term survival rate was 31%, with 35% of patients achieving complete remission remaining relapse-free for over 5 years, indicating that early remission is crucial for improving survival outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Importance of long-term follow-up in evaluating treatment regimens for adults with acute lymphoblastic leukemia. Clarkson, B., Gaynor, J., Little, C., et al.[2019]

The VALOR study, involving 711 patients, showed that the combination of vosaroxin and cytarabine resulted in better complete remission rates and survival for older patients (≥60 years) with relapsed/refractory acute myeloid leukemia (AML) compared to placebo/cytarabine.

Vosaroxin demonstrated a safety profile similar to existing AML treatments, making it a promising option for older patients who often face poor outcomes due to comorbidities and unfavorable cytogenetics.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosaroxin in relapsed/refractory acute myeloid leukemia: efficacy and safety in the context of the current treatment landscape. Sedov, V., Stuart, RK.[2020]

Since 2017, the FDA has approved nine new agents for treating acute myeloid leukemia (AML), marking significant progress in AML therapy after decades of slow advancement.

Specific treatments for subsets of AML, such as all-trans retinoic acid and arsenic trioxide for acute promyelocytic leukemia, have led to impressive 10-year survival rates of over 80%, demonstrating the efficacy of targeted therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acute myeloid leukemia: Treatment and research outlook for 2021 and the MD Anderson approach. Kantarjian, HM., Kadia, TM., DiNardo, CD., et al.[2021]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11575699/

UCART19, a first-in-class allogeneic anti-CD19 chimeric ... Relapsed or refractory B-cell acute lymphoblastic leukaemia in adults has a dismal prognosis, with 3-year overall survival of 4–15% after ...

2. ashpublications.org ashpublications.org/blood/article/132/Supplement%201/896/266300/Preliminary-Data-on-Safety-Cellular-Kinetics-and

Preliminary Data on Safety, Cellular Kinetics and Anti ... After UCART19 infusion, 88% of evaluable pts (14/16) achieved CR or CRi by D28 or D42 and 86% (12/14) of these pts were MRD negative (MRD- ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2352302622002459

UCART19, a first-in-class allogeneic anti-CD19 chimeric ... UCART19 had a manageable safety profile, and showed evidence of antileukaemic activity in heavily pretreated adult patients with relapsed or refractory B-cell ...

4. cellectis.com cellectis.com/uploads/files/ASH_CALM.pdf

Preliminary results of UCART19, an allogeneic anti ... - Cellectis Adult B-acute lymphoblastic leukemia (B-ALL). • B-ALL is incurable in ~60% of adult pa:ents. • At relapse, prognosis is very poor (<10% overall survival).

5. thelancet.com thelancet.com/journals/lanhae/article/PIIS2352-3026(22)00245-9/abstract

UCART19, a first-in-class allogeneic anti-CD19 chimeric ... UCART19 had a manageable safety profile, and showed evidence of antileukaemic activity in heavily pretreated adult patients with relapsed or ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36228643/

UCART19, a first-in-class allogeneic anti-CD19 chimeric ... This study shows that allogeneic off-the-shelf CAR T cells can be used safely to treat patients with relapsed B-cell acute lymphoblastic leukaemia.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT02808442

NCT02808442 | Study of UCART19 in Pediatric Patients ... This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S0006497119369459

Preliminary Data on Safety, Cellular Kinetics and Anti ... Preliminary safety/anti leukemic and cellular kinetics data of UCART19 administered to pediatric and adult patients with R/R B-ALL are reported.