Relapsed or Refractory B Cell Acute Lymphoblastic LeukemiaPhase 1NCT06173518
A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)
A Phase 1 study testing AUTO1 in people with Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia and Relapsed or Refractory B Cell Non-Hodgkin Lymphoma, run by Autolus Limited at 14 sites in 3 countries.
Trial summary
This is a Phase 1b/2 study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B… Read more about this study
- Who is it for?
- Ages 0 to 18 years
- Conditions the study is for
- Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia, Relapsed or Refractory B Cell Non-Hodgkin Lymphoma
- What is being tested?
- AUTO1
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 14 sitesin 3 countries
- Study dates
- 16 Nov 2023to Nov 2027
- Phase
- Phase 1
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 0 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- < 18 years old at screening
- ≥ 6 kg body weight at screening
- Pediatric patients with r/r B ALL
- r/r CD19-positive aggressive mature B including the B NHL subtypes: i) diffuse large B cell lymphoma, ii) Burkitt's lymphoma, iii) primary mediastinal large B cell lymphoma, iv) high-grade B cell lymphoma (not otherwise specified).
- Karnofsky (age ≥ 10 years) or Lansky (age < 10 year) performance status score ≥ 50%.
- In participants with B ALL, local documentation of CD19 expression on leukemic blasts in the BM, peripheral blood, or cerebrospinal fluid or biopsy done no more than 30 days prior to consent.
- Adequate renal, hepatic, pulmonary, and cardiac function.
Who cannot join
- Diagnosis of chronic myelogenous leukemia in lymphoid blast crisis.
- History or presence of clinically relevant central nervous system (CNS) pathology unrelated to CNS leukemia.
- Presence of active or uncontrolled fungal, bacterial, viral, or other infection requiring systemic antimicrobials for management.
- Received prior (< 3 months before obe cel infusion) stem cell transplantation.
- Prior CD19 targeted therapy other than blinatumomab.
- Experienced Grade ≥ 3 neurotoxicity following blinatumomab.
Trial locations and contacts
14 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 3 countries
United States9 sites, 9 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
- Recruiting
Lurie Children's Hospital
Chicago, Illinois, 60611
- Recruiting
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
- Recruiting
Lucile Packard Children's Hospital, Stanford University
Palo Alto, California, 94304
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Primary Children's Hospital
Salt Lake City, Utah, 84113
- Recruiting
Methodist Children's Hospital
San Antonio, Texas, 78229
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
United Kingdom3 sites, 3 recruiting
- Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust
London
- Recruiting
Royal Manchester Children's Hospital
Manchester
- Recruiting
Great North Children's Hospital
Newcastle upon Tyne
Spain2 sites, 2 recruiting
- Recruiting
Hospital Vall d'Hebron
Barcelona
- Recruiting
Hospital Nino Jesus
Madrid
About this study
Official title: A Single-Arm, Open-Label, Multicenter, Phase 1b/2 Study Evaluating the Safety and Efficacy of AUTO1 (Obecabtagene Autoleucel [Obe-cel]) in Pediatric Patients With CD19-positive Relapsed/Refractory (R/R) B Cell Acute Lymphoblastic Leukemia (B ALL) or R/R Aggressive Mature B Cell Non-Hodgkin Lymphoma (B NHL).
This is a Phase 1b/2 study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B NHL).
Read the detailed description
This is a single-arm, open-label, multi-center, Phase 1b/2 study to determine the safety and efficacy of obe-cel administered intravenously in pediatric patients < 18 years old with r/r B ALL and with r/r aggressive mature B NHL.
The safety and tolerability of obe cel in pediatric patients will be continually monitored by the Sponsor. Efficacy endpoints will be determined by an Independent Response Review Committee (IRRC).
The study will involve consented patients going through the following sequential study periods: screening, leukapheresis, bridging as necessary, lymphodepletion, treatment evaluation, and follow-up.
- Conditions
- Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia, Relapsed or Refractory B Cell Non-Hodgkin Lymphoma
- Being tested
- AUTO1
- Keywords
- B cell acute lymphoblastic leukemia, B cell Non-Hodgkin lymphoma, Relapsed B cell acute lymphoblastic leukemia, Relapsed B cell Non-Hodgkin lymphoma, Refractory B cell acute lymphoblastic leukemia, Refractory B cell Non-Hodgkin lymphoma, Aggressive mature B cell Non-Hodgkin lymphoma, Pediatric ALL, Pediatric NHL, Obecabtagene autoleucel, CD19-positive CAR T cell, Obe-cel
- Medical subject headings
- Recurrence, Burkitt Lymphoma, Lymphoma, B-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AUTO1Biological
Also called Obecabtagene autoleucel (obe-cel)
Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with anti-CD19 chimeric antigen receptor (CAR) T cells
| Study group | Role | What they receive |
|---|---|---|
| AUTO1 | Experimental | Not described Receives: Biological: AUTO1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
Measured: Up to 24 months
Incidence and duration of severe hypogammaglobulinemia
Measured: Up to 24 months
Proportion of pediatric participants with r/r B ALL at screening who achieve complete remission (CR) within 3 months of obe-cel infusion per Independent Response Review Committee (IRRC) assessment
Measured: 3 months
Secondary outcomes (10)
CR per IRRC assessment at any time in B ALL
Measured: Up to 24 months
Overall remission rate (ORR) (CR + complete remission with incomplete recovery of counts [CRi]) per IRRC assessment at any time in B ALL
Measured: Up to 24 months
Minimal residual disease (MRD)-negative ORR per IRRC assessment at any time in B ALL
Measured: Up to 24 months
Event-free survival in B ALL
Measured: Up to 24 months
Overall survival (OS) in B ALL
Measured: Up to 24 months
ORR (CR or partial response [PR]) per Investigator assessment occurring at any time in B NHL
Measured: Up to 24 months
Duration of response in B NHL
Measured: Up to 24 months
Progression-free survival in B NHL
Measured: Up to 24 months
OS in B NHL
Measured: Up to 24 months
Incidence of CD19-negative relapse at any time in B NHL
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2023
- Study start16 Nov 2023
- Primary completionNov 2027 (estimated)
- Study completionNov 2027 (estimated)
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 Relapsed or Refractory B Cell Acute Lymphoblastic LeukemiaStudies 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
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Autolus Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06173518
- Sponsor's protocol ID
- AUTO1-PY1
- Other IDs
- 2023-506307-26-00, 2023
- Status last verified
- Oct 2026
- First submitted
- 8 Dec 2023
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Davis KL, Yao CC, Zimmerman JAO, Rau RE. Immunotherapy in B-Cell Acute Lymphoblastic Leukemia. J Natl Compr Canc Netw. 2025 Dec;23(12):e257067. doi: 10.6004/jnccn.2025.7067.
PubMed 41671463