Long-Term Follow-up Protocol for Subjects Treated with Gene-Modified T cells.

• Register: EUCTR
• Last refreshed on: 23 November 2020
• Main ID: EUCTR2017-001465-24-GB
• Date of registration: 17/06/2019
• Prospective Registration: No
• Primary sponsor: Celgene Corporation
• Public title: Long-Term Follow-up Protocol for Subjects Treated with Gene-Modified T cells.
• Scientific title: Long-Term Follow-up Protocol for Subjects Treated with Gene-Modified T cells.
• Date of first enrolment: 24/02/2019
• Target sample size: 525
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-001465-24
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: yes; Other trial design description: Long-Term Follow-up Protocol; If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 0
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, Canada, Finland, France, Germany, Italy, Japan, Netherlands, Norway, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

• Name: ClinicalTrialDisclosure
• Affiliation: Celgene Corporation

• Name: ClinicalTrialDisclosure
• Affiliation: Celgene Corporation

Key inclusion & exclusion criteria

Inclusion criteria:
1. All adult and pediatric subjects who received at least one gene-modified (GM) T cell infusion in a previous Celgene sponsored or Celgene alliance partner sponsored study, and have discontinued, or completed the post-treatment follow-up period in the parent treatment protocol, as applicable.
2. Subject (and, parental/legal representative, when applicable) must understand and voluntarily sign an ICF/IAF prior to any study-related assessments/procedures being
conducted.
3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
Are the trial subjects under 18? yes
Number of subjects for this age range: 91
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 238
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 196

Exclusion criteria:
Not Applicable.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Defined by parent protocol. The study will enroll all adult and paediatric subjects who received at least one genetically modified T cells infusion in a previous Celgene sponsored study.
MedDRA version: 21.0 Level: LLT Classification code 10025631 Term: Malignant lymphoid neoplasm NOS System Organ Class: 100000004864

Therapeutic area: Diseases [C] - Cancer [C04]

Intervention(s)

Product Name: Gene-Modified T cells
Product Code: JCAR017, bb2121
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: Gene-Modified T cells
Other descriptive name: Gene-Modified T cells
Concentration unit: Other
Concentration type: equal
Concentration number: 0-

Primary Outcome(s)

Main Objective: - To assess the risk of delayed adverse events (AEs) following exposure to gene-modified (GM) T cells
- To monitor for long-term persistence of GM T cells, including analysis of vector integration sites, as appropriate.
- To monitor for generation of replication competent retroviruses (RCR)
- To assess long-term efficacy following treatment with GM T cells
- Describe growth, developmental outcome, and sexual maturity status for subjects who were aged < 18 years at time of GM T cell treatment
- To assess long term health-related quality of life following treatment with GM T cells

Secondary Objective: None

Primary end point(s): Safety:
- Incidence of delayed Adverse Events suspected to be related to prior gene-modified (GM) T cell therapy, including:
- New malignancies (hematologic or solid)
- New neurologic disorder, or exacerbation of a pre-existing neurologic disorder
- New rheumatologic or autoimmune disorder, or exacerbation of a prior rheumatologic or other autoimmune disorder
- New hematologic disorder
- Other new clinical conditions considered related to the prior GM T cell therapy by the investigator.
Hospitalizations, regardless of relationship to prior treatment, including reasons and dates

- Persistence of GM T cells
- Analysis of vector integration sites
- Incidence of replication-competent retroviruses (RCR)
- Height, weight, growth, and organ development will be assessed for all pediatric subjects
- Sexual maturity status for pediatric subjects

Efficacy
Where applicable:
- Tumor Response Status
- Date of Disease Progression
- Date of Relapse
- Survival Status

HRQoL
Measurement of health-related quality of life (HRQoL) changes as assessed using instruments administered in the parent treatment protocol

Timepoint(s) of evaluation of this end point: HRQoL: Up to 5 years from last GM T cells infusion
Other endpoints: Up to 15 years from last GM T cells infusion

Secondary Outcome(s)

Secondary end point(s): Not Applicable.

Timepoint(s) of evaluation of this end point: Not Applicable.

Secondary ID(s)

GC-LTFU-001

2017-001465-24-BE

Source(s) of Monetary Support

Celgene Corporation

Ethics review

Status: Approved
Approval date: 24/02/2019
Contact: