CAR T-cell therapy long term follow-up

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma Services AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20], Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

19 Jul 2017 → ongoing

Decision date (initial)

2024-03-14

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma Services AG

External identifiers

EU CT number

2023-508128-37-00

EudraCT number

2014-001673-14

ClinicalTrials.gov

NCT02445222

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Pharmacokinetic

Describe selected, delayed AEs that are suspected to be related to previous CAR T-cell therapy as outlined in current Health Authority guidelines

Secondary objectives 5

1. Monitor the persistence of modified T-cells in peripheral blood
2. Monitor for RCL (Replication Competent Lentivirus)
3. Assess the long-term efficacy of CAR T-cell therapy for hematology/oncology patients
4. Monitor lymphocyte levels
5. Describe the growth, development, and female reproductive status for patients who were aged < 18 years at the time of the initial CAR T cell therapy

Conditions and MedDRA coding

All patients who have been treated with Novartis or Penn CAR T-cell therapy for any indication

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Patients must have received CAR T-cell therapy within one of the following: • Novartis or Penn sponsored CAR T-cell treatment trials where CAR T-cell therapy was given as monotherapy or as combination therapy. • Novartis managed access programs (MAPs) outside of the commercial setting, i.e. where CAR T-cell therapy was intended to be given in the setting of a Novartis or Penn sponsored CAR T-cell treatment trial
2. Patients must provide informed consent/assent prior to their entry into this study

Exclusion criteria 1

1. There are no specific exclusion criteria for this study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of patients with events in each of the following categories: For participants treated for non-hematology/non-oncology indications: New primary malignancies•For participants with hematology/oncology indications: New secondary malignancies•New serious infections•New incidence of serious neurologic disorder•New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder•New incidence of a hematologic disorder•Other adverse events considered related to CAR T-cell thera

Secondary endpoints 5

1. Proportion of patients with detectable CAR transgene levels in peripheral blood by qPCR at pre-specified time points
2. Proportion of patients with detectable RCL by VSV-G qPCR in peripheral blood at pre-specified time points
3. • Proportion of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry • Incidence of death Note: For non-hematology/non-oncology patients, though there will be no formal assessments for efficacy, data on disease indication management will be captured in the Concomitant Medication CRF (e.g., rescue medication including immunosuppressive medications).
4. B and T lymphocyte counts
5. Height and weight, Tanner staging, menstruation status The pregnancy status for female patients will be captured in the CRF. For female partners of male patients if female partners provide consent, pregnancy outcome and data on live births for infants at birth, 3 months and 1 year of age is captured in Novartis safety forms.

Investigational products

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

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma Services AG

Sponsor organisation

Novartis Pharma Services AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma Services AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma Services AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 9

Locations

12 EU/EEA countries · 53 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

Documents 314 files

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

Application history

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