Neoplasms

Nervous System Diseases

Immune System Diseases

All Conditions

Musculoskeletal Diseases

Symptoms and General Pathology

Rare Diseases

Myasthenia Gravis

Muscle Weakness

Paresis

Asthenia

Muscular Diseases

Neuromuscular Manifestations

Neurologic Manifestations

Syndrome

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

Neuromuscular Diseases

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Anti-Inflammatory Agents

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Cyclophosphamide

Prednisolone

Methylprednisolone

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Mesna

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Glucocorticoids

Hormones

Hormone Antagonists

Protective Agents

Antineoplastic Agents, Hormonal

Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning

Autologous Hematopoietic Stem Cell Transplantation

Hematopoietic Stem Cell Transplantation

ATG (rabbit)

G-CSF

Richard Burt, MD

MG may be neonatal, congenital, or autoimmune. Neonatal MG arises from transplacental transfer of ACh receptor antibodies from a mother with autoimmune MG to the fetus. Neonatal MG resolves with post delivery clearance of maternal antibodies. Congenital MG results from a genetic defect in the ACh receptor. Patients with congenital MG do not have ACh receptor antibodies. Both neonatal and congenital MG are excluded from this study. Autoimmune MG, which is the most common form of MG, affects approximately 25,000 Americans. Like most autoimmune diseases, it is associated with particular HLA genotypes, has a female predominance, and environmental factors involved in breaking tolerance to the ACh receptor are unknown. Patients with refractory and severe autoimmune MG will be considered candidates for this study.

The purpose of this study is to assess the toxicity/feasibility (phase I) of autologous hematopoietic stem cell transplantation for refractory myasthenia gravis.

There will be no randomization in this study. All subjects who are determined to be eligible for the study treatment will receive high dose cyclophosphamide and ATG followed by infusion of autologous peripheral blood stem cells. The procedures the subject will undergo are as follows:

1. Physician visit to determine potential eligibility for the study. Subjects will be evaluated by a transplant physician and a neurologist. They will have a complete physical examination and will provide a full medical history at these visits. The study will be described in detail by the transplant physician and nurse and the consent form will be provided to be taken home to read.
2. Insurance verification. Subjects who remain interested in pursuing the study treatment and who have refractory myasthenia gravis will proceed to the insurance verification phase. Third party payment or self-pay must be verified before subjects can proceed.
3. Consent form. Prior to proceeding, the appropriate signatures will be obtained on the consent form. Subjects will be given an opportunity to ask further questions of the attending physician and transplant nurse prior to signing the consent form.
4. Pre-transplant testing. To determine final eligibility for the study, subjects will undergo a series of tests/procedures. These include: CXR; electrocardiogram; MUGA or echocardiogram; pulmonary function test; CT scan of the sinuses; CT scan of the chest to r/o thymoma; dental examination; urinalysis; and blood testing to include CBC, chemistries, liver and kidney function tests, coagulation studies, viral studies and, for females, a pregnancy test. All pre-transplant testing is routine medical testing done to verify diagnosis and to insure adequate organ function and absence of viral illnesses which would preclude a safe transplant course.
5. Autologous peripheral blood stem cell collection. Subjects who are proceeding to transplant will undergo a routine procedure for the mobilization and collection of peripheral blood stem cells. This includes the administration of IV cyclophosphamide, given as an inpatient requiring an overnight stay, followed by the subcutaneous administration of G-CSF, to be self-administered as an outpatient. Approximately ten days after the administration of IV cyclophosphamide, the peripheral blood stem cells will be collected as an outpatient in the Blood Center. A pheresis catheter will be placed for this purpose on the first day of leukopheresis. Leukopheresis will be continued on a daily basis until an adequate number of peripheral blood stem cells have been collected (a maximum of four leukophereses may be performed). G-CSF will continue to be administered until leukopheresis is completed. The pheresis catheter will be discontinued when stem cell harvesting is completed. Processed cells will be frozen and stored until they are reinfused after the conditioning regimen.
6. PICC line placement. Subjects will have a double lumen PICC line placed prior to the administration of study treatment for the administration of chemotherapy, IV fluids, blood products and the withdrawal of blood samples. The placement of a PICC line is a routine medical procedure.
7. Study treatment. Subjects will undergo conditioning which will include four days of intravenous high dose cyclophosphamide and three days of intravenous anti-thymocyte globulin (ATG). Both cyclophosphamide and ATG are common immune suppressive agents. The previously collected peripheral blood stem cells will be reinfused following the completion of the conditioning regimen.
8. Post-treatment follow-up. Subjects will have a history and physical by the transplant physician and neurologist at 3 months, 6 months, 12 months, and yearly for 5 years. In addition, routine urinalysis and blood testing will be performed at these same intervals to include CBC, chemistries, kidney and liver function tests.

Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis

Northwestern University

Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning

Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation

Age, Categorical

Age, Continuous

Sex: Female, Male

United States

Region of Enrollment

Dr. Richard Burt

Survival both treatment related and non-treatment related

Survival

Overall Study

Blood and Lymph Conditions

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Anti-Infective Agents

Cytarabine

Fludarabine

Fludarabine phosphate

Decitabine

Mycophenolic Acid

Tacrolimus

Thymoglobulin

Busulfan

Idarubicin

Clofarabine

Antilymphocyte Serum

Antimetabolites

Antiviral Agents

Calcineurin Inhibitors

Enzyme Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Antitubercular Agents

Salvage Chemotherapy Before Transplant: Decitabine 20 mg/m2 by vein on Days 1 - 5. Cytarabine 1 g/m2 by vein on Days 6 - 10. Idarubicin 10 mg/m2 by vein on Days 6 - 8. Clofarabine 15 mg/m2 by vein on Days 6 - 9.

Stem Cell Transplant: Test dose Busulfan 32 mg/m2 by vein on Day -8. Busulfan AUC 5,000 by vein on Days -6 to -3. Fludarabine 10 mg/m2 by vein on Days -6 to -3. Clofarabine 40 mg/m2 by vein on Days -6 to -3. Stem cell infusion performed on Day 0.

Salvage Chemotherapy Before Transplant: Decitabine 20 mg/m2 by vein on Days 1 - 5. Cytarabine 1 g/m2 by vein on Days 6 - 10. Idarubicin 10 mg/m2 by vein on Days 6 - 8. Clofarabine 15 mg/m2 by vein on Days 6 - 9.

Stem Cell Transplant: Test dose Busulfan 32 mg/m2 given by vein on Day -8. Busulfan AUC 5,000 by vein on Days -6 to -3. Fludarabine 10 mg/m2 by vein on Days -6 to -3. Clofarabine 40 mg/m2 by vein on Days -6 to -3. Total body irradiation (TBI) delivered at 2Gy on Day -2. Stem cell infusion performed on Day 0. Cyclophosphamide 50 mg/kg by vein on Days +3 and +4. Tacrolimus 0.015 mg/kg/day by vein or mouth on Day +5. Mycophenolate mofetil 15 mg/kg/dose by vein or by mouth three times a day from Day +5 to Day+100.

Salvage Chemotherapy Before Transplant: Decitabine 20 mg/m2 by vein on Days 1 - 5. Cytarabine 1 g/m2 by vein on Days 6 - 10. Idarubicin 10 mg/m2 by vein on Days 6 - 8. Clofarabine 15 mg/m2 by vein on Days 6 - 9.

Stem Cell Transplant: Test dose Busulfan 32 mg/m2 given by vein on Day -8. Busulfan AUC 5,000 by vein on Days -6 to -3. Fludarabine 10 mg/m2 by vein on Days -6 to -3. Clofarabine 40 mg/m2 by vein on Days -6 to -3. Thymoglobulin 2.0 mg/Kg by vein on Days -3 and -2. Stem cell infusion performed on Day 0.

Test dose Busulfan 32 mg/m2 given by vein on Day -8. Busulfan AUC 5,000 by vein on Days -6 to -3.

Fludarabine 10 mg/m2 by vein on Days -6 to -3.

Salvage Chemotherapy Before Transplant: Clofarabine 15 mg/m2 by vein on Days 6 - 9.

Stem Cell Transplant: Clofarabine 40 mg/m2 by vein on Days -6 to -3.

Total body irradiation (TBI) delivered at 2Gy on Day -2.

Total Body Irradiation (TBI)

Thymoglobulin 2.0 mg/Kg by vein on Days -3 and -2.

Fresh or cryopreserved bone marrow or peripheral blood (PB) progenitor cells infused on Day 0. Goal is to infuse 4 X 106 CD34+ cells/kg if PB or \>3.0 X 108 marrow mononuclear cells/kg if bone marrow.

Stem Cell Infusion

Cyclophosphamide 50 mg/kg by vein on Days +3 and +4.

Tacrolimus 0.015 mg/kg/day by vein or mouth on Day +5.

Mycophenolate mofetil 15 mg/kg/dose by vein or by mouth three times a day from Day +5 to Day+100.

Mycophenolate mofetil

Stefan Ciurea, MD

Objectives:

Primary Objectives:

1. To determine the safety and feasibility of allogeneic hematopoietic stem cell transplantation (AHSCT) as initial salvage treatment for patients with primary induction failure (PIF) acute myeloid leukemia (AML).
2. To determine efficacy of AHSCT following decitabine, clofarabine, idarubicin, and cytarabine (DCIA) salvage chemotherapy evaluated by overall response rate (RR), defined as complete response (CR) or CR without platelet recovery (CRp) or CR with insufficient hematological recovery (CRi).

Secondary Objectives:

1. To determine the percentage of patients with PIF AML eligible for AHSCT after up to 2 courses of induction chemotherapy.
2. To determine the early treatment-related mortality (TRM) (within first 4 weeks of first salvage chemotherapy regimen with DCIA and day 100 TRM after AHSCT.
3. To determine the efficacy DCIA regimen as salvage chemotherapy for patients with PIF AML (% of patients who achieve \</=5% bone marrow blasts prior to AHSCT.
4. To determine the TRM at 1 year, relapse rate (RR), overall survival (OS) and event-free survival (EFS) for patients with PIF AML treated with DCIA followed by early AHSCT.

Salvage Chemotherapy Before Transplant:

Study Drug Administration:

On Days 1-5, you will receive decitabine 1 time each day by vein over about 1-3 hours.

On Days 6-10:

* You will receive cytarabine 1 time a day by vein over about 1-3 hours.
* On Days 6-8 only, you will receive idarubicin 1 time a day by vein over about 30 minutes.
* On Days 6-9 only, you will receive clofarabine 1 time a day by vein over about 1-2 hours.

Study Visits:

After your last study drug dose:

* Blood (about 2 teaspoons) will be drawn to check your kidney and liver function.
* You will have an echocardiogram (ECHO) or multigated acquisition (MUGA) scan to check your heart function.
* You will have a lung function test.

On Day 21 (+/- 7 days), you will have a bone marrow biopsy/aspirate to check the status of the disease. To collect a bone marrow biopsy/aspirate, an area of the hip is numbed with anesthetic, and a small amount of bone and bone marrow is withdrawn through a large needle.

If the results of your bone marrow biopsy/aspirate, blood tests, and heart and lung function tests show that you are eligible to receive an allogeneic stem cell transplant, you will be asked to sign a separate informed consent for the transplant.

If the results of your bone marrow aspirate/biopsy, blood tests, and heart and lung function tests show that you are not eligible to receive an allogeneic stem cell transplant you will not receive it. The study staff will call you and ask how you are feeling and about any other drugs you may be taking every 3 months for 2 years after your last study drug dose. These calls should last about 5 minutes each.

If you are found to NOT be eligible to receive an allogeneic stem cell transplant, your doctor will discuss other treatment options with you.

Stem Cell Transplant:

Study Drug Administration, Pharmacokinetic (PK) Testing, and Stem Cell Transplant:

For a stem cell transplant, the days before you receive your stem cells are called minus days. The day you receive the stem cells is called Day 0. The days after you receive the stem cells are called plus days.

You will receive a dose of busulfan by vein over about 45 minutes to 1 hour as an outpatient or as an inpatient on Day -8. With the first busulfan infusion, blood (about 1 teaspoon each time) will be drawn for pharmacokinetic (PK) testing about 11 times over 11 hours before and after you receive your first dose of busulfan. PK testing measures the amount of study drug in the body at different time points. The study staff will tell you the blood testing schedule. Test doses are used to study how your body breaks down busulfan and decide the dose of busulfan that you will receive on Days -6 through -3.

A heparin lock line will be placed in your vein before the PK testing to lower the number of needle sticks needed for these draws. If for any reason it is not possible for the PK tests to be performed, you will receive the standard dose of busulfan.

On Day -7, you will rest.

On Days -6 through -3, you will receive fludarabine by vein over 1 hour, clofarabine by vein over 1 hour, and then busulfan by vein over 3 hours.

On Days -3 and -2, if you will receive stem cells from a matched unrelated donor, you will receive ATG by vein over 4 hours each day.

On Day -2, if you will receive stem cells from a haploidentical donor, you will receive total body irradiation (TBI) one time. TBI involves the delivery of high doses of radiation designed to destroy cancer cells and/or lower the immune system in order to lower the risk of the body rejecting the new stem cells.

On Day -2, you will receive tacrolimus by vein over 24 hours every day until you are able to take it by mouth. Tacrolimus is designed to weaken the immune system and lower the risk of graft-versus-host-disease (GVHD - a reaction of the donor's immune cells against your body). After you are able to take tacrolimus by mouth, you will take it every day for about 6 months, or until the doctor thinks it is safe to stop.

On Day -1, you will rest. If you will receive stem cells from a matched sibling donor, you will rest on Days -2 and -1.

On Day 0, you will receive the donor's stem cells by vein. The infusion will last anywhere from about 30 minutes to several hours.

If you will receive stem cells from a haploidentical donor:

After the stem cell infusion, you will receive tacrolimus to help lower the risk of GVHD. Tacrolimus will be given by vein non-stop for about 2 weeks. After the 2 weeks of taking tacrolimus by vein, you will take tacrolimus by mouth as a pill for at least 4 months after the transplant.

On Days +3 and +4, you will receive cyclophosphamide by vein over 3 hours. Cyclophosphamide is given to lower the immune system in order to lower the risk of GVHD.

If you receive stem cells from a matched sibling or matched unrelated donor:

On Days +1, +3, +6, and +11, you will receive methotrexate by vein over 30 minutes. Methotrexate is given to help prevent GVHD.

Study Testing:

Before you are sent home from the hospital and/or clinic, you will receive additional written instructions. These instructions will include how often you will come to the hospital/clinic, which standard drugs you will take at home, and what side effects you may have and what to do for them.

After finishing the chemotherapy and transplant, your follow-up care will be routine standard of care follow-up that all patients receiving allogeneic stem cell transplantation receive. At each visit, you will have a physical exam. You will be asked about any side effects you may have had. Blood (about 1 tablespoon) will be drawn for routine tests. If the doctor thinks it is needed, you will have a bone marrow aspiration to check the status of the disease. To collect a bone marrow aspirate, an area of the hip or other site is numbed with anesthetic, and a small amount of bone marrow is withdrawn through a large needle.

Length of Treatment:

After 2 years, your participation in this study will be over. You may be taken off study early if the disease gets worse, if your transplant does not "take" (graft failure), if you are unable to follow study directions, if your doctor thinks it is in your best interest, if the study is stopped, or if you choose to leave the study early.

If for any reason you want to leave the study early, you must talk to the study doctor. It may be life-threatening to leave the study after you have started to receive the study drugs but before you receive the stem cell transplant because your blood cell counts will be dangerously low.

Allogeneic Hematopoietic Stem Cell Transplantation (AlloSCT) Initial Salvage Therapy for Induction Failure Acute Myeloid Leukemia (AML)

Allogeneic Hematopoietic Stem Cell Transplantation as Initial Salvage Therapy for Patients With Primary Induction Failure Acute Myeloid Leukemia Refractory to High-Dose Cytarabine-Based Induction Chemotherapy

Number of participants classified as achieving overall response of complete remission(CR: \< 5% blast in a bone marrow/and platelet \>100/and ANC \>1) or CR without platelet recovery (CRp: \< 5% blast in a bone marrow/and platelet \<100/and ANC \>1) or CR with insufficient hematological recovery (CRi: \< 5% blast in a bone marrow/and platelet \>100/and ANC \<1) post transplant.

Number of participants died from treatment-related death within 4 months post initial treatment/3 months post transplant.

Number of participants alive and disease free in 1 year post transplant.

M.D. Anderson Cancer Center

AML patients with primary induction failure refractory to HD Cytarabine based chemotherapy received matched sibling donor stem cell transplant

Matched Sibling Donor Group

AML patients with primary induction failure refractory to HD Cytarabine based chemotherapy received haplo-identical related donor stem cell transplant

Haploidentical Donor Group

AML patients with primary induction failure refractory to HD Cytarabine based chemotherapy received matched unrelated donor stem cell transplant

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis	NCT00424489	Myasthenia Grav...	Hematopoietic S... Cyclophosphamid... ATG (rabbit) Mesna Methylprednisol... G-CSF	15 Years - 65 Years	Northwestern University
Allogeneic Hematopoietic Stem Cell Transplantation (AlloSCT) Initial Salvage Therapy for Induction Failure Acute Myeloid Leukemia (AML)	NCT02441803	Leukemia	Busulfan Fludarabine Clofarabine Total Body Irra... Thymoglobulin Stem Cell Infus... Cyclophosphamid... Tacrolimus Mycophenolate m... Decitabine Cytarabine Idarubicin	18 Years - 60 Years	M.D. Anderson Cancer Center
Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis	NCT00424489	Myasthenia Grav...	Hematopoietic S... Cyclophosphamid... ATG (rabbit) Mesna Methylprednisol... G-CSF	15 Years - 65 Years	Northwestern University
Allogeneic Hematopoietic Stem Cell Transplantation (AlloSCT) Initial Salvage Therapy for Induction Failure Acute Myeloid Leukemia (AML)	NCT02441803	Leukemia	Busulfan Fludarabine Clofarabine Total Body Irra... Thymoglobulin Stem Cell Infus... Cyclophosphamid... Tacrolimus Mycophenolate m... Decitabine Cytarabine Idarubicin	18 Years - 60 Years	M.D. Anderson Cancer Center
Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis	NCT00424489	Myasthenia Grav...	Hematopoietic S... Cyclophosphamid... ATG (rabbit) Mesna Methylprednisol... G-CSF	15 Years - 65 Years	Northwestern University

Spots Global Cancer Trial Database for atg (rabbit)

Every month we try and update this database with for atg (rabbit) 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.