CTRI/2024/10/074924 [Registered on: 08/10/2024] Trial Registered Prospectively
Last Modified On:
27/01/2026
Post Graduate Thesis
No
Type of Trial
Interventional
Type of Study
Biological
Study Design
Single Arm Study
Public Title of Study
The purpose of the study is to assess the Safety and Efficacy of MyCART-01, a novel form of immunotherapy. Immunotherapy is a type of treatment that utilizes the bodys own immune system to fight diseases. In this case, MyCART-01 focuses on a specific protein called CD19
A Prospective, Single-Arm, Open-Label, Multi-Center, Phase 1/2 Study of the Safety
and Efficacy of MyCART-01 in Patients with CD19 positive Relapsed or Refractory B Cell Malignancies
Trial Acronym
CART-NAL, SUMM1T
Secondary IDs if Any
Secondary ID
Identifier
MCr_CART-NAL01_Version 2.0.0_03/06/2024
Protocol Number
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
Name
Dr Dinesh Pendharkar
Designation
Director and Head of Medical Oncology, Haemato-Oncology, Bone Marrow Transplantation.
Affiliation
Sarvodaya Hospital, Faridabad
Address
Sector-8, YMCA Rd, near Escorts Mujesar Metro Station, Sector 8, Faridabad, Haryana 121006.
Faridabad HARYANA 121006 India
Phone
9987736665
Fax
Email
drpendharkar@gmail.com
Details of Contact Person Scientific Query
Name
Dr Ashokkumar Thakkar
Designation
Head of the Department - Clinical Research and Medical Writing
320, Anna Salai, Rathna Nagar, Alwarpet, Chennai, Tamil Nadu 600035 Chennai TAMIL NADU
8148400246
dr.pkjayachandran@gmail.com
Dr Revathi Raj
Apollo Speciality Hospital, Chennai
320, Anna Salai, Rathna Nagar, Alwarpet, Chennai, Tamil Nadu 600035 Chennai TAMIL NADU
9841070249
revaraj@yahoo.com
Dr M Joseph John
Christian Medical College and Hospital, Ludhiana
Department of Clinical Haematology, Haemato-oncology & Bone marrow (stem cell) Transplantation, 4th Floor, Christian Medical College and Hospital Brown Road, Ludhiana, Punjab-141008, India Ludhiana PUNJAB
8054959525
mjosephjohn@cmcludhiana.in
Dr Suparno Chakrabarti
Dharamshila Narayana Superspeciality Hospital
Vasundhara Enclave, Near New Ashok Nagar Metro Station, New Delhi–110096 New Delhi DELHI
9871127809
supchak@gmail.com
Dr Reetu Jain
Jaslok Hospital & Research Centre, Mumbai
Dr. Gopalrao Deshmukh Marg, Pedder Road, Mumbai, Maharashtra - 400026, India Mumbai MAHARASHTRA
9819095659
reebun@yahoo.com
Dr Shrinath Manik Kshirsagar
K.J. somaiya hospital and research centre, Mumbai
CTRU Department, 3rd Floor, SS Building, K.J. Somaiya hospital and research centre, Somaiya Ayurvihar, SION( E ), Mumbai, 400022. Mumbai MAHARASHTRA
9821556030
shrinath@somaiya.edu
Dr Gauri Kapoor
Rajiv Gandhi Cancer Institute & Research Centre, Delhi
Sector-5, Rohini, Delhi-110085 New Delhi DELHI
9871114046
kapoor.gauri@gmail.com
Dr Dinesh Pendharkar
Sarvodaya Hospital, Faridabad
Department of Oncology, Sector-8, YMCA Rd, near Escorts Mujesar Metro Station, Sector 8, Faridabad, Haryana 121006 Faridabad HARYANA
9987736665
drpendharkar@gmail.com
Dr Harsha Pragnesh Panchal
The Gujarat Cancer and Research Institute
M.P Shah Cancer Hospital, New Civil Hospital Campus, Asarwa, Ahmedabad Gujarat- 380016 Ahmadabad GUJARAT
9825940769
harsha.panchal@gcriindia.org
Dr Sandip Shah
Vedanta Institute of Medical Sciences
First Floor, Nr Samved Hospital Stadium Commerce Road Navrangapura, Ahmedabad-380009 India Ahmadabad GUJARAT
MyCART-01 is a CD19-directed T cell immunotherapy.
A single dose of MyCART-01 contains 0.2 to 5.0 x 106 CAR-positive viable T cells per kg of body weight for patients 100 kg or less, suspended in a patient-specific infusion bag for intravenous infusion.
MyCART-01 administration will be single infusion process lasting for 1 hour and patient will be monitored for 2 year post MyCART-01 infusion.
Comparator Agent
NA
NA
Inclusion Criteria
Age From
18.00 Year(s)
Age To
90.00 Year(s)
Gender
Both
Details
Non Hodgkin’s Lymphoma:
1)Non Hodgkins Lymphoma Patients with age greater than or equal to 18 years.
2)Patients with relapsed or refractory B Cell NHL with histologically confirmed diagnosis of one of the following
a)DLBCL NOS
b)high grade B Cell lymphoma with MYC and BCL2 and or BCL6 rearrangements
c) transformed FL
d)mantle cell lymphoma
e)grade 3b FL
f)Richters transformation of CLL
g)Other forms of B cell NHL
Note:
i)For refractory disease patients must have progressive disease on last therapy, or have stable disease following at least 2 lines of therapy with duration of stable disease of less than or equal to 6 months
ii)For patients with transformed FL, patients must have received at least one line of chemotherapy for disease after transformation to DLBCL and have progressed or relapsed within 12 months
3) Refractory or relapsed disease, to prior first line therapy, including an anti CD20 monoclonal antibody and an anthracycline containing regimen
4) Patients with prior autologous hematopoietic stem cell transplant must not have traces of related toxicities.
5) At least 1 measurable lesion that is fluorodeoxy glucose positron emission tomography positive, as defined by Lugano criteria
Note: Previously irradiated lesions will be considered measurable only if progression is documented following g completion of radiation therapy. Acute Lymphoblastic leukemia
Acute Lymphoblastic Leukemia:
1)Adult patients of age greater than or equal to 18 years and children of age greater than or equal to 3 years
2)Diagnosed with the histologically confirmed CD19 positive relapsed or refractory adult or pediatric B Cell ALL
a)Refractory disease is defined by not achieving a CR after 2 cycles of a standard chemotherapy regimen or after completion of induction phase of pediatric regimens or
b)Relapsed disease as defined by bone marrow relapse at any point of time after achieving CR 1
3)Philadelphia chromosome positive Ph positive ALL patients
4)Confirmation from central pathology lab
i)Documentation of CD19 tumor expression in bone marrow by flow cytometryII
ii)Bone marrow with greater than or equal to 5 percent lymphoblasts
For all patients:
1)Eastern Cooperative Oncology Group performance status 0 to 1
2)Life expectancy greater than or equal to 12 weeks
3)Patients with absolute lymphocyte count of greater than or equal to 0.2 into 109 per L
4)Female patients of childbearing potential post menarcheal with an intact uterus and at least 1 ovary, who are less than 1 year postmenopausal must agree to use acceptable method of contraception from enrollment through at least 12 months after CAR T infusion. Male patients must agree to use effective contraception from enrollment through at least 12 months after CAR T infusion
5)Adequate organ function based on investigators discretion but not limited to the following points:
i)Renal: Estimated glomerular filtration rate greater than 50 ml min 1.73 m2
ii)Liver: Aspartate transaminase or alanine transaminase less than 3 upper limit of normal total bilirubin less than 1.5 ULN for patients with Gilberts syndrome total bilirubin less than 3 mg dL
iii)Cardiac: Hemodynamically stable and left ventricle ejection fraction greater than or equal to 45percent by echocardiogram
iv)Pulmonary Oxygen saturation level on room air greater than or equal to 91percent per pulse oximetry
6) Written voluntary informed consent (adults) or parental/guardian consent for children aged 3 to 11 and assent form for adolescent aged 12 to 17 years who are able to give written assent prior to performing any research related tests or procedures.
ExclusionCriteria
Details
1)Eligible for and agrees to allogeneic HSCT
2)Treatment with the following therapies as described below
a) Prior treatment with any gene therapy or genetically modified cell therapy, including CAR-T Cells
b) Prior treatment with a CD19-directed antibody, bispecific T Cell engager, or antibody drug conjugate, unless there is confirmed CD19 expression by immunohistochemistry or flow cytometry after progression or relapse following most recent CD19 directed treatment.
3) Prior allogenic HSCT
4)Prior anaphylactic reaction to Cyclophosphamide, Fludarabine or any of the excipients of CART product
5) History of a seizure disorder, cerebrovascular ischemia or hemorrhage, dementia, cerebellar disease, or any autoimmune disease with CNS involvement
6)Unstable angina, clinically significant arrhythmia, or myocardial infarction within 12 months of enrollment
7) Presence of active bacterial, viral, or fungal infection
8)Positive for presence of human immunodeficiency virus type 1 or 2, or active hepatitis B virus orhepatitis C virus infection or other viral infections. Patients with prior history of hepatitis B or C infection who have documented undetectable viral load by quantitative polymerase chain reaction or nucleic acid testing may be permitted
9) Patients have active SARS COV-2 positive RT PCR test infection at the time of screening.
10) Any condition leading to T Cell depletion, rendering patient unsuitable for CAR T Cell therapy
11) Previous or concurrent malignancy, except basal cell or squamous cell skin carcinoma, adequately resected and in situ carcinoma of cervix, or a previous malignancy that was completely treated and has been in remission for greater than or equal to 5 years
12) Patient underwent radiation therapy before 14 days of enrollment
13) Use of systemic anti tumor therapy or investigational agent within 14 days or 5 half-lives, whichever is longer, of enrollment. An exception is made for prior inhibitory or stimulatory immune checkpoint molecule therapy, which is prohibited within 3 half lives of enrollment
14) Primary immunodeficiency disorder or active autoimmune disease requiring steroids and or other immunosuppressive therapy
15) Diagnosis of significant psychiatric disorder or other medical condition that, in the opinion of the investigator, could impede the patient’s ability to participate in the study
16) Women who are pregnant or breastfeeding
17) Any condition, in opinion of the Investigator or Screening committee, would preclude safe participation of patient in the study
Method of Generating Random Sequence
Not Applicable
Method of Concealment
Not Applicable
Blinding/Masking
Not Applicable
Primary Outcome
Outcome
TimePoints
The incidence of adverse events, defined as safety evaluation
Evaluating the objective response rate (complete response (CR) + partial response(PR)) per the Lugano Response Criteria for malignant lymphoma
Evaluating the objective response rate in pediatric and young adult CD19+ relapsed or refractory B cell ALL, based on CR and complete remission with incomplete count recovery (CRi)
30 Days
Secondary Outcome
Outcome
TimePoints
Evaluating the objective response rate (complete response (CR) + partial response(PR)) per the Lugano Response Criteria for malignant lymphoma
3 Months
Duration of response
Progression-free survival (central read)
Disease control rate [CR+PR+ stable disease (SD)]
Only for ALL patient:
Overall survival
Frequency and severity of adverse events and clinically significant laboratory
abnormalities
2 Years
Target Sample Size
Total Sample Size="41" Sample Size from India="41" Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials" Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials"
Phase of Trial
Phase 1/ Phase 2
Date of First Enrollment (India)
28/10/2024
Date of Study Completion (India)
Applicable only for Completed/Terminated trials
Date of First Enrollment (Global)
Date Missing
Date of Study Completion (Global)
Applicable only for Completed/Terminated trials
Estimated Duration of Trial
Years="2" Months="0" Days="0"
Recruitment Status of Trial (Global)
Not Applicable
Recruitment Status of Trial (India)
Open to Recruitment
Publication Details
N/A
Individual Participant Data (IPD) Sharing Statement
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
Brief Summary
This study is a prospective, single-arm, open-label, multi-center clinical trial designed to evaluate the safety and efficacy of MyCART-01 in patients with CD19+ relapsed or refractory B cell malignancies. The trial encompasses two phases, I and II, with a total intended enrollment of 41 participants, including both ALL (Acute Lymphoblastic Leukemia) and NHL (Non-Hodgkin Lymphoma) patients.
Phase I involves the inclusion of 10 patients, comprising 7 adults (with at least 1 from either ALL or NHL) and 3 pediatric patients with ALL. Following Phase I, Phase II aims to enroll an additional 31 patients, distributed between Stage I (14 patients) and Stage II (17 patients).
The investigational product under evaluation is Mycabtagene Autoleaucel, denoted as MyCART-01. This therapy falls under the category of CAR-T (Chimeric Antigen Receptor T-cell) and is designed to target CD19, a protein expressed on the surface of B cells.
The study is currently in the I/II phase of development and is being conducted at approximately seven centers in India. The primary endpoint is to assess the safety and efficacy of MyCART-01 in treating CD19+ relapsed or refractory B cell malignancies.
Following the CAR-T infusion, participants will be monitored for a period of two years during the follow-up phase. The outcomes of this study will contribute valuable insights into the potential of MyCART-01 as a therapeutic intervention for patients with relapsed or refractory B cell malignancies.