PAVO: CAR-T Long Term Follow Up (LTFU) Study

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Recruiting

CT.gov ID

NCT02445222

Collaborator

University of Pennsylvania (Other)

1,400

Enrollment

Locations

Arm

243.7

Anticipated Duration (Months)

19.4

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all patients exposed to CAR-T therapied for 15 years following their last CAR-T (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.

Condition or Disease	Intervention/Treatment	Phase
Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program	Genetic: Previously treated CAR-T patients	N/A

Detailed Description

Patients are enrolled following completion or early discontinuation from a Novartis sponsored or supported study of CAR T-Cell treatment. Patients will be followed for 15 years post treatment from the last treatment. They will be monitored for safety and efficacy within the primary treatment protocols for the protocol defined duration. Patients can drop off treatment protocols at any time to enter this long term Follow up study. Patients discontinuing from the primary treatment protocols for any reason will be enrolled in this long term follow up (LTFU). This will allow collecting data on long term safety and efficacy (as applicable) as mandated by the health authorities of all patients treated with CAR-T therapy within the concept of a single protocol.

Collection of such long term effects of CAR-T cell therapy will help to further define the risk-benefit profile of CAR-T Therapies.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

1400 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Other

Official Title:

Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy

Actual Study Start Date :

Nov 2, 2015

Anticipated Primary Completion Date :

Feb 22, 2036

Anticipated Study Completion Date :

Feb 22, 2036

Arms and Interventions

Arm	Intervention/Treatment
Other: Previously treated CAR-T patients Patients who previously were exposed to lentiviral-based CART cell therapy	Genetic: Previously treated CAR-T patients Lentiviral-based CAR-T cell therapy

Arm

Intervention/Treatment

Other: Previously treated CAR-T patients

Patients who previously were exposed to lentiviral-based CART cell therapy

Genetic: Previously treated CAR-T patients

Lentiviral-based CAR-T cell therapy

Outcome Measures

Primary Outcome Measures

Percentage of patients with certain events (see description) [at M3 post treatment, M6, M9, M12 and then, every 6M up to year 5, yearly until year 15.]
The percentage of pts with listed categories: New secondary malignancies, new serious infection, new incidence of serious neurologic disorder, New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder, New incidence of a hematologic disorder

Secondary Outcome Measures

Percentage of patients with detectable CAR transgene levels in peripheral blood by q-PCR at pre- specified time points [at M3 post treatment, M6, M9, M12 and every 6M up to year 5, yearly until year 15.]
Percentage of patients with detectable RCL by VSV-G [at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15]
Percentage of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry;Incidence of death [at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.]
B- and T- lymphocyte count [at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.]
Height and weight, Tanner staging, menstruation status [at M3 post treatment then M6, M12 and every year until year 15.]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

All patients who have received a CAR-T therapy and completed or discontinued early from a Novartis sponsored treatment protocol that utilized CAR-T cells or from any CAR-T trial sponsored by the University of Pennsylvania with which Novartis has a contractual agreement to co-develop the CAR technology.
Patients who have provided informed consent for the long term follow up study prior to their study participation .

Exclusion Criteria:

There are no specific exclusion criteria for this study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mayo Clinic Arizona Mayo Clinic Building	Phoenix	Arizona	United States	85054
2	Childrens Hospital Los Angeles SC CTL019	Los Angeles	California	United States	90027
3	UCSF Medical Center	San Francisco	California	United States	94143
4	Stanford Universtiy Medical Center SC - CTL019B2205J - B2206	Stanford	California	United States	94304
5	Emory University School of Medicine/Winship Cancer Institute	Atlanta	Georgia	United States	30322
6	Children's Healthcare of Atlanta SC CTL019	Atlanta	Georgia	United States	30342
7	University of Chicago Medical Center, Hematology & Oncology	Chicago	Illinois	United States	60637
8	University of Kansas Cancer Center SC	Westwood	Kansas	United States	66205
9	University of Michigan	Ann Arbor	Michigan	United States	48109-2800
10	University of Michigan Health System SC CTL019	Ann Arbor	Michigan	United States	48109
11	University of Minnesota SC-4	Minneapolis	Minnesota	United States	55455
12	Children's Mercy Hospital SC	Kansas City	Missouri	United States	64108
13	Weill Cornell Medical College	New York	New York	United States	10065
14	Duke Unversity Medical Center SC - CTL019B2205J	Durham	North Carolina	United States	27705
15	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229-3039
16	Ohio State Comprehensive Cancer Center/James Cancer Hospital Ohio State Univ.	Columbus	Ohio	United States	43210
17	Oregon Health & Science University	Portland	Oregon	United States	97239
18	The Childrens Hospital of Philadelphia CHOP	Philadelphia	Pennsylvania	United States	19104
19	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
20	UTSW/Children's Medical Center	Dallas	Texas	United States	75235
21	MD Anderson Cancer Center SC	Houston	Texas	United States	77030
22	University of Utah Clinical Trials Office	Salt Lake City	Utah	United States	84108
23	University of Wisconsin Hospital and Clinics	Madison	Wisconsin	United States	53792
24	Novartis Investigative Site	Melbourne	Victoria	Australia	3000
25	Novartis Investigative Site	Parkville	Victoria	Australia	3052
26	Novartis Investigative Site	Camperdown		Australia	NSW
27	Novartis Investigative Site	Linz		Austria	4020
28	Novartis Investigative Site	Vienna		Austria	A-1090
29	Novartis Investigative Site	Wien		Austria	A 1090
30	Novartis Investigative Site	Gent		Belgium	9000
31	Novartis Investigative Site	Hamilton	Ontario	Canada	L8V 1C3
32	Novartis Investigative Site	Toronto	Ontario	Canada	M5G 1X8
33	Novartis Investigative Site	Montreal	Quebec	Canada	H1T 2M4
34	Novartis Investigative Site	Montreal	Quebec	Canada	H3T 1C5
35	Novartis Investigative Site	Copenhagen		Denmark	2100
36	Novartis Investigative Site	Helsinki		Finland	00029
37	Novartis Investigative Site	Paris Cedex 10		France	75475
38	Novartis Investigative Site	Paris Cedex 19		France	75935
39	Novartis Investigative Site	Pierre Benite Cedex		France	69495
40	Novartis Investigative Site	Regensburg	Bavaria	Germany	93053
41	Novartis Investigative Site	Berlin		Germany	13353
42	Novartis Investigative Site	Frankfurt		Germany	60590
43	Novartis Investigative Site	Hamburg		Germany	20246
44	Novartis Investigative Site	Koeln		Germany	50937
45	Novartis Investigative Site	Leipzig		Germany	04103
46	Novartis Investigative Site	Muenchen		Germany	81377
47	Novartis Investigative Site	Ulm		Germany	89081
48	Novartis Investigative Site	Wuerzburg		Germany	97080
49	Novartis Investigative Site	Monza	MB	Italy	20900
50	Novartis Investigative Site	Fukuoka city	Fukuoka	Japan	812-8582
51	Novartis Investigative Site	Sapporo city	Hokkaido	Japan	060 8648
52	Novartis Investigative Site	Sendai city	Miyagi	Japan	980 8574
53	Novartis Investigative Site	Chuo ku	Tokyo	Japan	104 0045
54	Novartis Investigative Site	Kyoto		Japan	606 8507
55	Novartis Investigative Site	Utrecht	CS	Netherlands	3584
56	Novartis Investigative Site	Amsterdam		Netherlands	1105 AZ
57	Novartis Investigative Site	Utrecht		Netherlands	3584CX
58	Novartis Investigative Site	Oslo		Norway	0424
59	Novartis Investigative Site	Oslo		Norway	NO 0424
60	Novartis Investigative Site	Singapore		Singapore	169608
61	Novartis Investigative Site	Sevilla	Andalucia	Spain	41013
62	Novartis Investigative Site	Esplugues de Llobregat	Barcelona	Spain	08950
63	Novartis Investigative Site	Salamanca	Castilla Y Leon	Spain	37007
64	Novartis Investigative Site	Barcelona	Catalunya	Spain	08035
65	Novartis Investigative Site	Hospitalet de LLobregat	Catalunya	Spain	08907
66	Novartis Investigative Site	Valencia	Comunidad Valenciana	Spain	46010
67	Novartis Investigative Site	Barcelona		Spain	08041
68	Novartis Investigative Site	Madrid		Spain	28009
69	Novartis Investigative Site	Madrid		Spain	28041
70	Novartis Investigative Site	Madrid		Spain	28046
71	Novartis Investigative Site	Taipei		Taiwan	10002
72	Novartis Investigative Site	London		United Kingdom	SE5 9RS

Sponsors and Collaborators

Novartis Pharmaceuticals
University of Pennsylvania

Investigators

Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Novartis Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT02445222

Other Study ID Numbers:

CCTL019A2205B
2014-001673-14

First Posted:

May 15, 2015

Last Update Posted:

Jun 28, 2022

Last Verified:

Jun 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Study Results

No Results Posted as of Jun 28, 2022