A Phase 2 Multicenter Study of Axicabtagene Ciloleucel in Subjects With Relapsed/Refractory Indolent Non-Hodgkin Lymphoma

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Kite Pharma Inc.

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

7 Sep 2018 → 5 Dec 2024

Decision date (initial)

2023-09-26

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Kite Pharma, Inc

External identifiers

EU CT number

2023-505169-10-00

EudraCT number

2017-001912-13

ClinicalTrials.gov

NCT03105336

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

The primary objective is to evaluate the efficacy of axicabtagene ciloleucel, as measured by objective response rate (ORR), in subjects with relapsed or refractory (r/r) B-cell iNHL.

Secondary objectives 1

1. Key secondary objectives are to characterize the safety profile, complete response (CR) rate, ORR among those subjects with 3 or more lines of prior therapy, CR rate among those subjects with 3 or more lines of prior therapy, and to determine duration of response (DOR), progressionfree survival (PFS), and overall survival (OS). Additional secondary objectives will include additional safety and pharmacokinetic/pharmacodynamic endpoints and the time to next therapy endpoint.

Conditions and MedDRA coding

Follicular Lymphoma, Marginal Zone Lymphoma, Indolent Non-Hodgkin Lymphoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 18 Years and older.
2. Individual has [follicular lymphoma or marginal zone lymphoma that has progressed after at least 2 lines of treatment with combination chemoimmunotherapy] (e.g. R-bendamustine, R-CHOP).
3. Individual has [measurable disease].
4. Individual has no known presence or history of central nervous system (CNS) involvement by lymphoma.
5. If individual is on conventional systemic therapy or systemic inhibitory/stimulatory immune checkpoint therapy, individual is able to stop conventional therapy 2 weeks or 5 half-lives, whichever is shorter, or immune checkpoint therapy 3 half-lives prior to planned leukapheresis.
6. Individual has Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 and adequate renal, hepatic, pulmonary, and cardiac function
7. Individual is not pregnant or breastfeeding (female individuals only) and is willing to use birth control from the time of consent through 12 months following chimeric antigen receptor (CAR) T cell infusion (both male and female individuals).
8. Females of childbearing potential must have a negative serum or urine pregnancy test (females who have undergone surgical sterilization or who have been postmenopausal for at least 2 years are not considered to be of childbearing potential).

Exclusion criteria 5

1. Transformed follicular lymphoma (FL) or marginal zone lymphoma (MZL).
2. Small lymphocytic lymphoma.
3. Histological Grade 3b FL.
4. Individual will have undergone autologous transplant within 6 weeks of planned leukapheresis or has undergone allogeneic transplant.
5. Individual has evidence of involvement of the heart by lymphoma or requirement for urgent therapy due to ongoing or impending oncologic emergency (e.g. mass effect, tumor lysis syndrome, etc.).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Objective response rate (ORR) per central read for up to 15 years

Secondary endpoints 14

1. CR Rate per central read for up to 15 years.
2. DOR for up to 15 years.
3. PFS for up to 15 years.
4. Percentage of Participants Experiencing Treatment-Emergent Adverse Events for up to 15 years.
5. Levels of anti-CD19 CAR T cells in blood at enrollment, Day 7, Week 2, Week 4, Month 3, Month 6, Month 12, Month 18, Month 24, annually up to year 5.
6. Levels of cytokines in serum at enrollment, prior to axicabtagene ciloleucel infusion on Day 0, Day 3, Day 7, Week 2, Week 4.
7. Percentage of Participants experiencing anti-axicabtagene ciloleucel antibodies at Week 4, Month 3, every 3 months up to Month 12.
8. Percentage of Participants Experiencing clinically significant changes in lab values for up to 15 years
9. Time to next therapy for up to 15 years.
10. Objective response rate among participants with 3 or more lines of prior therapy for up to 15 years.
11. Complete response rate among those participants with 3 or more lines of prior therapy for up to 15 years.
12. Objective Response Rate as Determined by the Investigator Read for up to 15 years.
13. Best Objective Response per Central Read or Investigator Read for up to 15 years.
14. Overall survival for up to 15 years

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 8

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Kite Pharma Inc.

Sponsor organisation

Kite Pharma Inc.

Address

2225 Colorado Avenue

City

Santa Monica

Postcode

90404-3505

Country

United States

Scientific contact point

Organisation

Kite Pharma Inc.

Contact name

EU CT Support

Public contact point

Organisation

Kite Pharma Inc.

Contact name

EU CT Support

Third parties 14

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 3 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications