Corporate Studies 2/19/2021

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NEW DM ONBOARDING FEB 2021 |1.

The CIBMTR®(Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program®(NMDP)/Be The Match®and the Medical College of Wisconsin (MCW).

Corporate Studies and Registries

February 19, 2021

There are no conflicts of interest to disclose.

Agenda

• Corporate studies • Registry studies: – BMT CTN – PIDTC – CMS

• Cell therapy studies overview • Actively enrolling studies

Corporate Study vs. Registry Study

Corporate study: CIBMTR is approached by Corporate sponsor to collect data on their behalf

– This may include supplemental data that would require participation agreement

Registry Study: Do not require any additional efforts or consent – These studies are done on data already submitted to the CIBMTR

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Consent for Corporate and Registry Studies

• No additional consent from the recipient is required • These studies are a part of the CIBMTR research database

protocol

CIBMTR Corporate Membership

The CIBMTR Corporate Membership Program provides a variety of resource materials to corporations related to the most current and comprehensive data

– There are five levels of membership offered

– Members are invited to participate in the annual CIBMTR meetings and educational forums

– Ability to contract with us to develop their projects

Steps of a Corporate Study

Corporate Membership established Study proposal submitted for review Internal study team assigned Center contact for participation

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• Upon agreement to

participate, you will receive an agreement form

• You will then be notified once enrollment starts; marking the study active in the systems

Center participation agreement

How does a patient get enrolled?

• This is automated so you don’t have to do anything! – Study coordinator works with the FN3 team to put in place the

events and actions based on the study inclusion criteria

• Consent, disease, age, center location

– Center completes the F2400 and F2402 and based on what is reported, the study ID will populate in the recipient grid

• Data collection for corporate & registry studies uses the standard CIBMTR

Comprehensive Report Forms (CRF)

– Some studies may have an additional supplemental form – Some studies may allow TED

level submission with disease forms

Data Collection

2400 2402

• Comprehensive Report Form

F2004/05/ 06

• The donor, recipient, and product forms will come due, if necessary

Baseline Forms

• F2000 – additional pre-transplant data • Disease specific baseline

Follow-up Forms

• Includes post forms (2100) and disease specific forms

Patient • Patient’s death is reported on F2900

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Data Collection

• Studies are often backdated to drug approval date • Example:

– study contract signed July 2020

– Drug of interest was FDA approved March 2020

• CIBMTR may identify eligible CRIDs that already have forms completed

CPI Reminder:

• The CPI program assures timely and accurate submission of data to help CIBMTR achieve its goal of promoting collaborative research that increases access and improves outcomes of all cellular therapies. • Form completion of at least 90% of

the required forms at each of three time periods

Corporate and Registry Studies and CPI

• Study forms are track by DUE date

• Two ways to identify study forms that are due:

Weekly Recipient CPI Summary and Forms Due Reports

Weekly Study Forms Due

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Corporate and Registry Studies and CPI

• Weekly Recipient CPI Summary and Forms Due Reports are automatically

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Corporate and Registry Studies and CPI

• Additional forms due list sent weekly that is just the study forms

– Remember: Study forms due are a subset of big CPI list • Study forms are due by form due date

BMT-CTN

• Established in October of 2001

• Conduct large multicenter clinical trials to

improve the outcomes of HCT for patients

facing life-threatening disorders

• Jointly funded by the NHLBI and NCI

• Network = >100 transplant centers

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What is BMT CTN

Centers

• 20 core centers

• includes consortia made of 2 or more centers • 75 affiliated centers

Funding • 2017-2024 is the 4

th_{cycle of funding}

Accrual • In total, more than 13,200 patients enrolled

Studies and Publications

Studies

PIs

TCT 2021

• Completed accrual to 44 trials • 5 studies in development • Accruing to 11 trials

• 1 released to centers

• 116 manuscripts including 29 Primary study results papers from 34 trials • 112 abstracted from 39 trials

• Late Breaking Abstract for BMT CTN 1301 Progress II Trial by Dr. Perales • Best Abstract for BMT CTN 1102 by Dr. Nakamura

• Poster for study design & statistics by Dr. Vasu for BMT CTN 1308 • Oral Abstracts by Dr. Putta and Dr. Khera

https://www.cibmtr.org/Studies/ClinicalTrials/BMT_CTN/pages/index.aspx

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Data Collection for BMT CTN

• Advantage EDC system • eClinical system

By Emmes

• FormsNet3

• ePRO data collection system • RAVE system

By CIBMTR

CIBMTR Data Collection for BMT CTN

Trial Planning Compare enrolled vs. non-enrolled population DSMB, FDA and other safety/regulato ry reporting Long-term follow up data Data not captured on study forms* Ancillary studies CMS protocols

*Reduces data collection burden for sites

CIBMTR Data Collection for BMT CTN (cont.)

Minimum of TED level

• Centers participating in BMT CTN trials must provide at least TED-level data to the CIBMTR on all (i.e. auto & allo) consecutive HCTs while actively enrolling patients.

CRF usually required

• Patients consenting for BMT CTN protocols consent to the treatment specified & the data collection for the clinical protocol. In nearly all cases, data collection on CIBMTR CRFs is required.

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CIBMTR Data Collection for BMT CTN (cont.)

Chapter 4 of every BMT CTN protocol will have details on CIBMTR Data Reporting

BMT CTN protocol where enrollment occurs

PRE-TRANSPLANT

• Comprehensive Report Form (CRF) data required

• regardless of the center’s CIBMTR data reporting status

• TED level only reporting status does not apply to BMT CTN studies

POST-TRANSPLANT

• Reporting track will stay the same

• An example is CTN 1705 (randomized trial for treatment of GVHD), where patient is not eligible or enrolled until the post transplant event had occurred.

CIBMTR Data Collection for BMT CTN (cont.)

• When CIBMTR CRF level data is required and CIBMTR Research Consent is No; data will only be used for BMT CTN related research and not for CIBMTR Observational Research.

• Therefore, All patients SHOULD be approached at the same time to provide consent for BOTH, as this increases the usefulness of their data for other research without increasing the data submission burden.

BMT CTN Research

Consent CIBMTR Research Consent

Patient ID/ Emmes ID

CIBMTR Data Collection for BMT CTN (cont.)

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Accruing and upcoming studies

Accruing and upcoming studies Reporting track

1507 Haplo Sickle Cell (Accruing) CRF

1702 Donor Source (Accruing) TED/CRF

1703 PTCy vs. Tac/Mtx 1801 Mi-Immune (Accruing) CRF

1704 CHARM (Accruing) CRF

1705 AAT (Accruing) TED/CRF

1706 Myeloma Maintenance (S1803) (Accruing) N/A

1905 BEAT-MS (ITN077AI) (Accruing) CRF

1803 Haplo NK Cell (Accruing) CRF

1901 MM CAR T – High Risk (In Development) TBD

1902 MM CAR T – Upgrade Response (In Development) TBD

1903 HIV T Cell (In Development) CRF

1904 Treo BM Failure Syndromes (In Development) TED/CRF

2001 Gene Therapy SCD (Release to Centers) CRF

2002 Acute GVHD Treatment T Guard2 (In Development) TBD

CIBMTR Data Compliance

From Center Performance Report:

From Center Performance Metrics:

PIDTC Studies

• CIBMTR CRF level data are required

• PIDTC sites participating in SCID study 6901 – continue as 6907 for current funding cycle

• Should be sharing CRID with PIDTC coordinator at your center to help link data collected by CIBMTR & PIDTC for analysis and future use.

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Cellular Therapy Corporate Studies

• Registry Study: Do not require any additional efforts or consent

• Corporate study: CIBMTR is approached by Corporate sponsor to collect data on their behalf

Cellular Therapy Corporate Studies

Cell

therapy Corporate Registry

Commercial CAR-T reporting

• The Food and Drug Administration (FDA) required

companies to have a structure in place to follow up

recipients long term

– Recipients of CAR-T cell therapy be followed for a

minimum of 15 years post infusion

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Commercial CAR-T reporting

• Companies have partnered with the CIBMTR to collect and analyze data during long term follow-up of several commercial CAR-T cell recipients

– Data collection will utilize Cellular Therapy Essential Data (CTED) forms, no additional study forms are necessary – The CTED forms have been developed to capture essential

data elements for recipients of CAR-T cells and many other upcoming cellular therapies

Commercial CAR-T reporting

• Data collection by the center remains voluntary but strongly recommended for commercial CAR T-cell recipients • Patients have the right not to consent to share data for

research

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Industry-sponsored Projects

Project Sponsor Objective Timeline/Duration

Yescarta LTFU (Axicabtagene ciloleucel)

Kite Safety and efficacy outcomes (PASS) N=1,500 (Completed 07/2020) Diseases: NHL 07/2018 2years of accrual 15 years of follow up Kymriah LTFU

(Tisagenlecleucel) Novartis Safety and efficacy outcomes (PASS)N=2,500 (Current N=1000) Diseases: NHL and ALL

08/2018 5years of accrual 15years of follow up Lisocabtagene maraleucel BMS Safety and efficacy outcomes (PASS)

N=1,000 Disease: NHL 5years15years of follow up Idecabtagene vecleucel BMS Safety and efficacy outcomes (PASS)

N=1,000 Diseases: Multiple Myeloma

5years 15years of follow up Tecartus

(Brexucatagene autoleucel) Kite Safety and efficacy outcomes (PASS)N=500 Disease: Mantle Cell Lymphoma 5years15years of follow up Ciltacabtagene autoleucel Janssen/

Legend Safety and efficacy outcomes (PASS)N=TBD Disease: Multiple Myeloma 5years15years of follow up Under Development

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Cellular therapy corporate studies

How does a patient get enrolled?

• This is automated so you don’t have to do anything! – Study coordinator works with the FN3 team to put in place the

events and actions based on the study inclusion criteria

• Consent, product name

– Center completes the F4000 and based on product name, the study ID will populate in the recipient grid

Active Studies

Amgen Blinatumomab SC18-10

Study Objective:

Evaluate overall survival and incidence of transplant-related adverse events in R/R B-cell ALL patients after allo HCT: induction with blinatumomab vs. chemotherapy

Patient Eligibility:

Children and adults in United States and Canada Relapsed, refractory or MRD+ (CR1/CR2) B-cell ALL Blinatumomab treatment vs. standard of care chemotherapy prior to HCT

Start Date: January 14, 2019

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Amgen Blinatumomab SC18-10

Duration: Enrollment: 5 yearsFollow-Up: 5 years post HCT (with potential follow up to 10 years)

Accrual (as 1/15/2021):

Current: 238 (blinatumomab) Current: 419 (non-blinatumomab) Goal: 200-500 (blinatumomab)

Reporting Track: TED or CRF: if on TED, disease inserts (F2011 and F2111) will generate in FN3 and reimbursement rates apply

Adienne Tepadina SC17-03

Study Objective: Determine the effectiveness and safety of Tepadina as a conditioning treatment prior to HCT in patients with CNS Lymphoma

Consenting patients with CNS Lymphoma receiving Tepadina prior to auto HCT from a US transplant center

Canadian patients can receive auto HCT for any disease

Start Date: September 2017

Adienne Tepadina SC17-03

Duration: Enrollment: 5 years_{Follow-Up: 5 years post HCT}

Accrual (as 1/15/2021): US centers = 100 (accrual complete) CNS Lymphoma Patients Other diseases = 15 (goal is 50)

Reporting Track: CRF _{F2540 supplemental data collection form}

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Kyowa Kirin Mogamulizumab SC18-09

Assess toxicities/complications of transplant among patients with cutaneous cell lymphoma or adult T-cell Leukemia/Lymphoma treated with Mogamulizumab alone or in combination within 1 year prior to or 18 months post allo HCT

Consenting adult patients with either CTCL or ATLL and an alloHCT performed from January 2012 onward with or without being treated with Mogamulizumab

Start Date: February 1, 2020

Kyowa Kirin Mogamulizumab SC18-09

Duration: Enrollment timeframe: January 2020 until accrual goal is met. Follow-Up timeframe: 2 years post HCT

Accrual (as 1/15/2021): Cases: 2 exposed to Moga within one year prior to HCT (goal = 50) Controls: 84 not exposed to Moga (goal = 100)

Reporting Track: TED or CRF_{F2542 supplemental data collection form}

Pfizer Inotuzumab Ozogamicin SC17-10

Evaluate adult and pediatric patients with B-cell ALL who received Inotuzumab Ozogamicin and proceeded to HCT

Consenting patients with B-cell precursor ALL diagnosis who received at least 1 dose of Inotuzumab Ozogamicin prior to proceeding to HCT from a US transplant center

Start Date: August 18, 2017

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Pfizer Inotuzumab Ozogamicin SC17-10

Duration: Enrollment: Through 08/17/2022_{Follow-Up: 5 years post HCT}

Accrual (as 1/15/2021): Current: 237

Reporting Track: CRF_{F2541 supplemental data collection form}

Pfizer Mylotarg SC19-12

Characterize toxicity following Gemtuzumab Ozogamicin (GO) treatment and hematopoietic stem cell transplantation in adult patients with Acute Myeloid Leukemia (AML)

Patient Eligibility: Consenting adult AML patients receiving GO prior to or at the time of transplant from a US transplant center

Start Date: December 2, 2020

Pfizer Mylotarg SC19-12

Duration: Enrollment: until accrual is met _{Follow up: 1 year post HCT}

Accrual (as 1/15/2021): Current: 51_{Goal: 200}

Reporting Track: TED and CRF_{F2543 supplemental data collection form}

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Jazz Pharmaceuticals VOD Registry SP16-02

Develop a veno-occlusive disease data registry to capture additional data on hematopoietic stem cell transplantation patients who develop VOD

Patient Eligibility: Adult and pediatric patients who develop VOD post transplant

Start Date: 2016

Jazz Pharmaceuticals VOD Registry SP16-02

Duration: Enrollment: Through January 31, 2022

Accrual (as 1/15/2021): Current: 1,532

Reporting Track: TED and CRF_{F2553 VOD supplemental data collection form}

Novartis Kymriah SC17-08

Study Objective: Evaluate the safety of patients with B-lymphocyte malignancies treated with tisagenlecleucel

Patients who were prescribed commercial tisagenlcecleucel by their treating physician, which includes those who were treated with tisagenlecleucel under a managed access program, and who consent to have data reported to the CIMBTR

Start Date: August 30, 2017

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Novartis Kymriah SC17-08

Duration: Enrollment timeframe: 5 years_{Follow up timeframe:15 years post-infusion.}

Accrual:

Current: 1114 (as of 2/1/2020) Goal: 2500 patients (1000 pediatric and 1500 adults)

Reporting Track: CTED

Kite Yescarta SC17-07

Study Objective: To evaluate the development of subsequent neoplasms after administration of axicabtagene ciloleucel

Recipient of axicabtagene ciloleucel for treatment of relapsed or refractory large B-cell lymphoma at participating centers and who consent to have data reported to the CIMBTR

Start Date: October 17, 2017

Kite Yescarta SC17-07

Duration: Enrollment timeframe: 5 years_{Follow up timeframe:15 years post-infusion.}

Accrual:

Current :1500 Goal:1500 patients

Reporting Track: CTED

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NEW DM ONBOARDING FEB 2021 |52. CMS Myelodysplastic Syndrome 10‐CMSMDS Study Objective: To prospectively examine outcomes of allogeneic HCT in adults >= 65 years of age with MDS to determine whether their outcomes are similar to those in younger patients. https://www.cibmtr.org/Studies/ClinicalTrials/mct/HCT‐MDS/Pages/index.aspx CMS Myelofibrosis 16‐CMS‐MF Study Objective: Compare the five‐year survival probabilities from DIPSS assessment between the two study arms: alloHCT recipients and non‐HCT therapies recipients. https://www.cibmtr.org/Studies/ClinicalTrials/mct/mms/Pages/default.aspx

CMS studies

CMS Multiple Myeloma 17‐CMS‐MM Study Objective: To compare five‐year OS probabilities between the alloHCT cohort and an age and disease risk matched cohort of autoHCT patients. https://www.cibmtr.org/Studies/ClinicalTrials/mct/mmms/Pages/default.aspx CMS Sickle Cell Disease 17‐CMS‐SCD Study Objective: Compare the 5‐year survival probabilities between patients with Sickle Cell Disease (SCD) who received allogeneic HCT to those who received standard of care https://www.cibmtr.org/Studies/ClinicalTrials/mct/Pages/Sickle‐Cell‐Disease‐CED.aspx

CMS studies

Submitting questions about a study

/

Cell therapy corporate study questions are submitted separately

Cell therapy corporate study IDs not included in this list

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