Rare Diseases

All Conditions

Dendritic Cell Tumor

Blastic Plasmacytoid Dendritic Cell

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Other Dietary Supplements

Cyclophosphamide

Fludarabine

Fludarabine phosphate

Mitogens

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Creatine

The study drug, MB-102 consists of adoptively transferred T cells that are genetically modified using a self-inactivating (SIN) lentiviral vector to express a CD123-specific, CD28-costimulatory chimeric antigen receptor (CAR) as well as a truncated human epidermal growth factor receptor (EGFRt) (CD123.CD28.CD3ζ.EGFRt+T cells) derived from autologous leukapheresis which is administered after a lymphodepletion regimen.

Single dose of MB-102 up to 600 x 10 6 CART-T+ cells (Day 0) as defined by Phase 1 will be administered.

MB-102

Fludarabine 30 mg/m2/day IV (3 days) on days -5, -4, and -3

* A 20% dose reduction (24 mg/m2/day IV (3 days) on days -5, -4, and -3) is required for patients with moderately impaired renal function (creatine clearance ≤ 70 mL/min).

Cyclophosphamide 300 - 500 mg/m2/day IV (3 days) on days -5, -4, and -3

Lihua E Budde, MD

A phase 1/2 study to assess the safety and efficacy of MB-102 in patients with relapsed or refractory BPDCN

The Phase 1 portion of the study will determine the maximum tolerated dose of MB-102.

The Phase 2 portion of the trial will evaluate the efficacy of MB-102 in relapsed or refractory BPDCN.

Trial to Evaluate the Safety and Efficacy of MB-102 in Patients With BPDCN.

A Phase 1/2, Open Label, Multicenter Trial to Assess the Safety and Efficacy of MB-102 in Patients With Relapsed or Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 in Phase 1

To determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose of MB-102

Relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm is measured by a response rate which consists of Complete Response and clinical Complete Response and Complete Response with incomplete hematologic recovery (CR + CRc + CRi) at day 28 post infusion

CR MRD- Response Rate for patients with CR and CRi

Incidence of treatment-emergent AEs (TEAEs), including SAEs, therapy-related AEs or death.

The European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0 is an integrated, modular approach for evaluating the quality of life of patients participating in international clinical trials. The questionnaire is designed to measure cancer patients' physical, psychological and social functions. The questionnaire is composed of 5 multi-item scales (physical, role, social, emotional and cognitive functioning) and 9 single items (pain, fatigue, financial impact, appetite loss, nausea/vomiting, diarrhea, constipation, sleep disturbance and quality of life).

It utilizes a four-point scales for the first 28 questions which are coded with response categories as "Not at all", "A little", "Quite a bit" and "Very much.". the final two question consist of an overall physical condition questions which have employed a 7-point response scale where the higher number indicates a better overall health.

The Functional Assessment of Cancer treatment for cancer, and a transplant-specific module, bone marrow transplant (BMT) concerns, that addresses disease and treatment-related questions specific to BMT.

It utilizes a 5 point scale assessing physical, social, emotional, functional and other well-being concerns. Response categories are coded as "Not at all", "A little bit", "Somewhat", "Quite a bit" and "Very much.".

To confirm the absence of replication competent lentivirus

Mustang Bio

Neoplasms

Blood and Lymph Conditions

Immune System Diseases

Leukemia

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Hematologic Neoplasms

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Primary Myelofibrosis

Neoplasms by Histologic Type

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Lorvotuzumab mertansine

Maytansine

Tubulin Modulators

Antimitotic Agents

Antineoplastic Agents, Immunological

Immunotoxins

Immunoconjugates

Lorvotuzumab mertansine (IMGN901) administered intravenously at a dose of 100 mg/m2 on Day 1 and 8 of a 21-day cycle.

100 mg/m2 by vein on Day 1 and 8 of a 21-day cycle.

Lorvotuzumab Mertansine (IMGN901)

Naval Daver, MD

The goal of this clinical research study is to learn if lorvotuzumab mertansine can help to control blood cancers that have the CD56 tumor marker. The safety of this drug will also be studied.

Study Treatment:

If you are found to be eligible to take part in this study, you will receive lorvotuzumab by vein on Days 1 and 8 (+/- 2 days) of each cycle. It may take a few minutes or several hours to receive the study drug. Your doctor will discuss this with you.

All treatments with IMGN901 must be administered at MD Anderson.

Each cycle is 21 days (+/-6 days). However, they may be longer or shorter depending on if/how the disease responds to the treatment, how your bone marrow reacts to treatment, and what the doctor thinks is in your best interest.

Your dose of lorvotuzumab may be raised, lowered, and/or delayed if the doctor thinks it is in your best interest.

Study Visits:

On Day 1 of each cycle, you will have a physical exam.

One (1) time each week during the first 4 cycles, blood (about 1 tablespoon) will be drawn for routine tests. After Cycle 4, you will have these blood draws 1 time every 2-4 weeks. If your doctor thinks it is needed, more blood may need to be drawn. The study doctor or study staff will discuss this with you if more blood is needed.

During the first cycle, all the laboratory evaluations will be done at MD Anderson. After Cycle 1, you may be able to have these blood draws performed at a local lab or clinic that is closer to your home, and the results reported to the research nurse of the study at MD Anderson. The study doctor or study staff will discuss this option with you. The results of these blood draws will be sent to the study doctor for review.

On Day 21 of Cycle 1 (+/- 7 days), then every 1-3 cycles after that, you will have a bone marrow aspiration/biopsy to check the status of the disease. If the doctor thinks it is needed, these may be done more or less often, depending on your response to the study drug.

You will have blood draws and/or bone marrow aspirations at any time that the doctor thinks it is needed while you are on study.

If you stay on study for more than 6 months and you are not having side effects, you may have some of the above tests more or less frequently. For example, you may only have a bone marrow aspiration 1 time every 6-12 months and blood draws 1 time each cycle. The study doctor will discuss this with you.

Length of Study:

You may take up to 12 cycles of lorvotuzumab. If the doctor thinks it is in your best interest, you may be able to continue receiving the study drug beyond Cycle 12. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

Your participation on the study will be over after follow-up.

End-of-Study Visit:

If you leave the study before the end of Cycle 12, within 30 days (+/- 7 days) after the last dose of study drug:

* Blood (about 2-3 tablespoons) will be drawn for routine tests.
* If the doctor thinks it is needed, you will have a bone marrow aspirate/biopsy to check the status of the disease.

Long Term Follow-Up:

If the disease appears to get better and you are responding well to the study drug, you will return to MD Anderson every 3-6 months for up to 5 years after your last dose of study drug for the study staff to check how you are doing.

If you cannot return to MD Anderson for these visits, you may be called by a member of the study staff. The call should last about 10 minutes.

This is an investigational study. Lorvotuzumab is not FDA approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the drug is designed to work.

Up to 60 participants will take part in this study. All will be enrolled at MD Anderson.

An Open-label Phase II Study of Lorvotuzumab Mertansine

An Open-label Phase II Study of Lorvotuzumab Mertansine (IMGN901) in CD56 Expressing Hematological Malignancies

ORR, defined as CR (complete remission) + CRp (complete remission with incomplete platelet recovery) + CRi (complete remission with incomplete count recovery) within 3 cycles of therapy with IMGN901.

ImmunoGen, Inc.

M.D. Anderson Cancer Center

Zero participants were registered on Cohort 2 (Myelofibrosis arm) or Cohort 3 (Blastic Plasmacytoid Dendritic Cell Neoplasm arm).

Lorvotuzumab mertansine (IMGN901) administered intravenously at a dose of 100 mg/m2 on Day 1 and 8 of a 21-day cycle.

Lorvotuzumab Mertansine (IMGN901): 100 mg/m2 by vein on Day 1 and 8 of a 21-day cycle.

Cohort 1: CD56 Expressing Hematological Malignancies

Cohort 2: Myelofibrosis

Cohort 3: Blastic Plasmacytoid Dendritic Cell Neoplasm

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Naval Daver, MD/ Associate Professor

Overall Response Rate (ORR) of IMGN901 in Participants CD56 Expressing Hematological Malignancies

Overall Study

Lymphoma

Lymphoma, T-Cell

Hodgkin Disease

Lymphoma, B-Cell

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Lymphoma, Non-Hodgkin

B-cell Lymphoma

Lymphosarcoma

Hodgkin Lymphoma

Acute Leukemia of Ambiguous Lineage

Anti-Inflammatory Agents

Antiemetics

Gastrointestinal Agents

Anti-Infective Agents

Dermatologic Agents

Reproductive Control Agents

Micronutrients

Hematinics

Vitamins

Dexamethasone

Hydrocortisone

Hydrocortisone 17-butyrate 21-propionate

Hydrocortisone acetate

Hydrocortisone hemisuccinate

Cytarabine

Azacitidine

Methotrexate

Vincristine

Dexamethasone acetate

BB 1101

Glucocorticoids

Hormones

Hormone Antagonists

Antineoplastic Agents, Hormonal

Antimetabolites

Enzyme Inhibitors

Folic Acid

Vitamin B Complex

Folic Acid Antagonists

Antiviral Agents

Folinic Acid

Folate

Vitamin B9

Tagraxofusp

-Days 1-5

IT Therapy (may include methotrexate, cytarabine, or triple IT)

* Day 1
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

Tagraxofsup

-Days 4-8

Fludarabine -Days 1-5

Cytarabine

-Days 1-5

IT Therapy (may include methotrexate, cytarabine, or triple IT) CNS1 IT Therapy

* Day 1
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

CNS2/3 IT Therapy

* Days 1, 8, 15, and 22
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

Tagraxofsup

-Days 8-12

Dexamethasone -Days 1-5

Vincristine

-Days 1, 8, 15, and 22

IT Therapy (may include methotrexate, cytarabine, or triple IT) CNS1 IT Therapy

* Day 1
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

CNS2/3 IT Therapy

* Days 1, 8, 15, and 22
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

Tagraxofsup -Days 1-5

Azacitidine

-Days 1-5

IT Therapy (may include methotrexate, cytarabine, or triple IT) CNS1 IT Therapy

* Day 1
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

CNS2/3 IT Therapy

* Days 1, 8, 15, and 22
* Patients may receive additional IT therapy with their end-of-cycle disease re-evaluation at the discretion of the treating investigator

Dose will be assigned at study entry. Give IV over 15 minutes.

Tagraxofusp

30 mg/m\^2 will be given IV over 30 minutes on days 1-5.

Infusion will start 30 minutes after start of tagraxofusp on days 4 and 5.

2000 mg/m2 intravenously will be given daily over 1-3 hours for 5 days on days 1 through 5. Infusion will begin 4 hours after start of fludarabine. Because of an increased risk of neurotoxicity, it is recommended that IT cytarabine be separated from high dose IV cytarabine administration by at least 24 hours on C1D1.

* 20 mg/m2/day divided BID (max 40 mg/day) given orally on days 1 through 5 and 15 through 19. The two doses should be separated by at least 8 hours.
* Any oral formulation of dexamethasone is acceptable.
* IV may be given if oral formulation is not tolerated

* 1.5 mg/m2 (maximum dose 2 mg) given intravenously as an IV push over 1-5 minutes or infusion via minibag as per institutional policy on days 1, 8, 15, and 22.
* Infusion will start 30 minutes after start of tagraxofusp on day 8.

* 75 mg/m2 subcutaneously or intravenously will be given daily over 15 minutes for 5 days on days 1 through 5.
* Azacitidine will be given 30-60 minutes before beginning the tagraxofusp infusion.

Give intrathecally:

* 8 mg for patients age 1-1.99
* 10 mg for patients age 2-2.99
* 12 mg for patients 3-8.99 years of age
* 15 mg for patients ≥9 years of age

Give intrathecally:

* 30 mg for patients age 1-1.99
* 50 mg for patients age 2-2.99
* 70 mg for patients ≥3 years of age

If given as part of Triple IT Therapy:

AML Patients:

Age 1-1.99 - 24 mg Age 2-2.99 - 30 mg Age ≥3 years of age - 36 mg

AML Patients:

Age 1-1.99 - 16 mg Age 2-2.99 - 20 mg Age 3-8.99 - 24 mg Age ≥9 years of age - 30 mg

Cytarabine IT

Given intrathecally.

AML Patients:

Age 1-1.99 - 16 mg Age 2-2.99 - 20 mg Age ≥3 years of age - 24 mg

AML Patients:

Age 1-1.99 - 8 mg Age 2-2.99 - 10 mg Age 3-8.99 - 12 mg Age ≥9 years of age - 15 mg

Benjamin N Brookhart

Ellynore Florendo

Alan Wayne, MD

Van Thu Huynh, MD

Michelle Hermiston, MD

Margaret Macy, MD

Reuven Schore, MD

Julio Barredo, MD

Melinda Pauly, MD

Jenna Rossoff, MD

Sandeep Batra, MD

Pat Brown, MD

Nirali Shah, MD

Andrew Place, MD

Rajen Mody, MD

Nathan Gossai, MD

Nobuko Hijiya, MD

Maria Sulis, MD, MS

Joel Kaplan, MD

Robin Norris, MD

Rachel Egler, MD

Susan Vear-Colace, MD

Bill Chang, MD

Sue Rheingold, MD

Jeffrey Rubnitz, MD

Tamra Slone, MD

Kenneth Heym, MD

Eric Schafer, MD

Anupam Verma, MD

Adam Lamble, MD

Michael Burke, MD

Luciano Dalla-Pozza, MD

Tracey O'Brien, MD

Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). This trial aims to examine the safety of this novel agent in pediatric patients with relapsed/refractory hematologic malignancies.

The mechanism by which tagraxofusp kills cells is distinct from that of conventional chemotherapy. Tagraxofusp directly targets CD123 that is present on tumor cells, but is expressed at lower or levels or absent on normal hematopoietic stem cells. Tagraxofusp also utilizes a payload that is not cell cycle dependent, making it effective against both highly proliferative tumor cells and also quiescent tumor cells.

The rationale for clinical development of tagraxofusp for pediatric patients with hematologic malignancies is based on the ubiquitous and high expression of CD123 on many of these diseases, as well as the highly potent preclinical activity and robust clinical responsiveness in adults observed to date.

This trial includes two parts: a monotherapy phase and a combination chemotherapy phase. This design will provide further monotherapy safety data and confirm the FDA approved pediatric dose, as well as provide safety data when combined with chemotherapy.

The goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to describe the toxicities, pharmacokinetics, and pharmacodynamic properties of tagraxofusp in pediatric patients.

About 54 children and young adults will participate in this study. Patients with Down syndrome will be included in part 1 of the study.

Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies

A Phase I Study of Tagraxofusp With or Without Chemotherapy in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies

The incidence of dose limiting toxicity (DLT) will be measured at different dose levels.

Therapeutic Advances in Childhood Leukemia Consortium

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Precancerous Conditions

Venetoclax

Tin Fluorides

SL-401 will be administered every 4 weeks, on a 28 day cycle; SL-401 will be given intravenously; Azacitidine will be administered every 4 weeks, on a 28 day cycle; Azacitidine will be given intravenously or subcutaneously

SL-401 will be administered every 4 weeks, on a 28 day cycle; SL-401 will be given intravenously; Azacitidine will be administered every 4 weeks, on a 28 day cycle; Azacitidine will be given intravenously or subcutaneously; Venetoclax will be administered for 21 days on a 28 day cycle; Venetoclax will be taken orally

SL-401 works by targeting leukemia cells (blasts), and also possibly by stopping or slowing the growth of cancer stem cells, which are the undeveloped cells which can develop into cancer cells.

SL-401

Venetoclax is a BH3-mimetic. Venetoclax blocks the anti-apoptotic B-cell lymphoma-2 (Bcl-2) protein, leading to programmed cell death of CLL cells. Overexpression of Bcl-2 in some lymphoid malignancies has sometimes shown to be linked with increased resistance to chemotherapy.

Andrew Lane, MD, PhD

Anthony Stein, MD

Naveen Pemmaraju, MD

This research study is studying a drug as a possible treatment for diagnosis of AML, BPDCN and high-risk MDS.

The interventions involved in this study are:

* SL-401
* Azacitidine
* Venetoclax

This research study is a Phase I clinical trial, which tests the safety of an investigational intervention and also tries to define the appropriate dose of the investigational intervention to use for further studies. "Investigational" means that the intervention is being studied.

The FDA (the U.S. Food and Drug Administration) has approved azacitidine and venetoclax as a treatment option for AML. However, the combination of these two drugs with SL-401 has not been FDA approved.

The combination of SL-401, azacitidine and venetoclax has not been FDA approved for BPDCN. However, SL-401 has been FDA approved for BPDCN.

The combination of SL-401 and azacitidine has not been FDA approved for BPDCN.

In this research study, the study drug SL-401 will be combined with the standard dose of azacitidine (for MDS patients) or azacitidine/venetoclax (for AML and BPDCN patients). The goal of this research study is to try and determine the safest, highest dose of study drug, SL-401, in combination with azacitidine or azacitidine/venetoclax that can be given to patients with AML, BPDCN or high-risk MDS. SL-401 works by stopping or slowing the growth of cancer stem cells, which are the undeveloped cells which can develop into cancer cells. The goals of this research study are to look at if this combination works to help treat your cancer and if there is any lasting effect of this combination. This study will also look at how the SL-401, in combination with azacitidine or azacitidine/venetoclax, affects certain proteins in your blood and bone marrow. SL-401 has been given to patients with AML, and MDS in the past, but this is the first time it will be given in combination with another drug.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Trial to Evaluate the Safety and Efficacy of MB-102 in Patients With BPDCN.	NCT04109482	Blastic Plasmac...	MB-102 Fludarabine Cyclophosphamid...	18 Years -	Mustang Bio
An Open-label Phase II Study of Lorvotuzumab Mertansine	NCT02420873	Leukemia	Lorvotuzumab Me...	18 Years -	M.D. Anderson Cancer Center
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies	NCT05476770	Hematologic Mal... AML ALL BPDCN MDS Lymphoblastic L... Lymphoma, B-Cel... Lymphoma, T-Cel... Hodgkin Lymphom... Mixed Phenotype... Acute Undiffere...	Tagraxofusp Fludarabine Cytarabine Dexamethasone Vincristine Azacitidine Methotrexate Cytarabine IT Hydrocortisone	1 Year - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)	NCT03113643	Acute Myeloid L... Myelodysplastic... Blastic Plasmac...	Azacitidine SL-401 Venetoclax	18 Years -	Dana-Farber Cancer Institute
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies	NCT05476770	Hematologic Mal... AML ALL BPDCN MDS Lymphoblastic L... Lymphoma, B-Cel... Lymphoma, T-Cel... Hodgkin Lymphom... Mixed Phenotype... Acute Undiffere...	Tagraxofusp Fludarabine Cytarabine Dexamethasone Vincristine Azacitidine Methotrexate Cytarabine IT Hydrocortisone	1 Year - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies	NCT05476770	Hematologic Mal... AML ALL BPDCN MDS Lymphoblastic L... Lymphoma, B-Cel... Lymphoma, T-Cel... Hodgkin Lymphom... Mixed Phenotype... Acute Undiffere...	Tagraxofusp Fludarabine Cytarabine Dexamethasone Vincristine Azacitidine Methotrexate Cytarabine IT Hydrocortisone	1 Year - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
Study of Venetoclax, a BCL2 Antagonist, for Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)	NCT03485547	Blastic Plasmac...	Venetoclax	18 Years -	Dana-Farber Cancer Institute
SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)	NCT03113643	Acute Myeloid L... Myelodysplastic... Blastic Plasmac...	Azacitidine SL-401 Venetoclax	18 Years -	Dana-Farber Cancer Institute

Spots Global Cancer Trial Database for bpdcn

Every month we try and update this database with for bpdcn cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for bpdcn

Every month we try and update this database with for bpdcn cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for bpdcn cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.