Immune System Diseases

All Conditions

Musculoskeletal Diseases

Substance Related Disorders

Autoimmune Diseases

Arthritis

Drug-Related Side Effects and Adverse Reactions

Chemically-Induced Disorders

All Drugs and Chemicals

Immunomodulating Agents

All patients starting immunotherapy treatment will complete a questionnaire to identify rheumatological side effects, each time they come for treatment.

Patient will have to fill in a form at each perfusion of immunotherapy to tell if they presents rheumatologic clinical signs

Fill in a form

Francoise Debiais, MD PHD

Alice Bel

Debiais Françoise, MD

Bel Alice

In this study, every patient with cancer who is going to be treated by immunotherapy will fill in a form. In this form, we try to determine if he presents rheumatologic complications of his immunotherapy. If the patient answers yes to one question on the form, the patient will be see in rheumatologic consultation to explore his clinical signs.

The number of patients referred in consultation thanks to this questionnaire will be compared to the number of patients who would have been spontaneously referred by the oncologist.

The aim of this questionnaire is to optimise the management of rheumatological effects secondary to immunotherapy.

Rheumatologic Adverse Events and Cancer Immunotherapy

Rheumatologic Adverse Events and Immunotherapy in Oncology

number of patients referred to a rheumatology consultation thanks to the answers to the questionnaire compared to the number of patients referred to a consultation (from questionnaire and oncologist)

Poitiers University Hospital

Symptoms and General Pathology

Blood and Lymph Conditions

Neoplasms

Rare Diseases

Syndrome

Thrombocytopenia

Fibrosis

Preleukemia

Myelodysplastic Syndromes

Primary Myelofibrosis

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

Blood Platelet Disorders

Cytopenia

Eltrombopag will be initiated at 50 mg/day (Asians 25 mg/day) and dose adjusted up to 150mg/day based response and safety in participants with Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

Eltrombopag will be initiated at 50 mg/day (Asians 25 mg/day) and dose adjusted up to 150mg/day based response and safety

Eltrombopag

Neal S Young, M.D.

Background:

* Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a risk of progression to acute leukemia. Standard treatments for MDS have significant relapse rates. MDS patients with thrombocytopenia who fail standard therapies require regular, expensive, and inconvenient platelet transfusions, and are at risk for further serious bleeding complications.
* Eltrombopag is a drug designed to mimic the protein thrombopoietin, which causes the body to make more platelets. Eltrombopag has been able to increase platelet counts in healthy volunteers and in patients with chronic ITP (a disease where patients destroy their own platelets very rapidly and thus develop thrombocytopenia), but researchers do not know if the drug can increase platelet counts in patients with MDS.

Objectives:

* To find out whether eltrombopag can improve platelet counts in patients with MDS.
* To determine whether eltrombopag is safe for patients with MDS.

Eligibility:

* Patients 18 years of age and older who have consistently low blood platelet counts related to MDS that has not responded to conventional treatment.
* Platelet count ≤ 30,000/μL or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); OR hemoglobin less than 9.0 gr/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry) OR ANC≤500

Design:

* Treatment with eltrombopag tablets once per day for 16-20 weeks.
* Participants will be monitored closely throughout the initial treatment, with weekly blood tests and separate evaluations at the National Institutes of Health (NIH) treatment center every 4 weeks. Bone marrow biopsies may be conducted to check for abnormalities in bone marrow.
* If patients show signs of improved platelet counts after 90 days, treatment will continue with additional doses of eltrombopag.
* Patients who discontinue taking eltrombopag will be evaluated at the NIH treatment center 4 weeks after ending treatment, and again 6 months after ending treatment to check for potential side effects.

The myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia. Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a variable risk of progression to acute leukemia. With the exception of stem cell transplant, the standard treatments for MDS are rarely curative, and relapse rates are significant. MDS patients with cytopenias who fail standard therapies require regular blood or platelet transfusions which are expensive and inconvenient, and are at risk for serious bleeding complications.

Thrombopoietin (TPO) is the principal regulator of platelet production by megakaryocytes in the bone marrow. A 2nd generation TPO-agonist, eltrombopag (Promacta ) has been shown to increase platelets in thrombocytopenic patients with chronic immune thrombocytopenic purpura (ITP). Eltrombopag is administered orally, is well-tolerated, and is FDA approved for the treatment of thrombocytopenia in patients with chronic ITP who failed to respond to standard treatment.

Because the management of MDS patients with persistent cytopenias remains unsatisfactory and novel therapeutic approaches are needed, we propose a non-randomized, pilot, phase II study of eltrombopag in low to Int-2 risk MDS subjects with thrombocytopenia and anemia cytopenias who are either untreated or cytopenias that persist despite treatment with standard therapies to assess its utility in these settings.

Subjects will initiate study medication at an oral dose of 50 mg/day (25 mg/day for East Asians), which will be adjusted as clinically indicated to the lowest dose that maintains a stable platelet count greater than or equal to 20,000/microL above baseline while maximizing tolerability. Treatment response will be any increase in a cytopenia, in the lineage that fulfilled eligibility criteria for enrollment and will be defined as: (a) platelet count increases to 20,000/microL above baseline at 16 or 20 weeks , or stable platelet counts with transfusion independence for a minimum of 8 weeks in subjects who were previously transfusion dependent; (b) erythroid response for subjects with a pretreatment hemoglobin of less than 9 g/dL will be defined as an increase in hemoglobin by greater than or equal to 1.5g/dL without packed red blood cell (PRBC) transfusion support, or a reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment compared with the pretreatment transfusion number in the previous 8 weeks; (c) neutrophil response will be defined in those with a pretreatment absolute neutrophil count (ANC) of \< 0.5 times 10(9)/L as at least a 100% increase or an absolute increase \> 0.5 times 10(9)/L. Subjects meeting a response may remain on the extended access until they meet an off study criteria or the study is closed.

Subjects with response at 16 or 20 weeks may be consented for entry into the extended access part of the trial. In the event that a subject is transfused platelets for a count \>10,000/microL without a medical indication during the study period, the subject may continue on study drug and the response assessment may be extended for an additional 4 weeks to week 20, at the discretion of the principal investigator. Subjects with evidence for a clinical response in any lineage at 16 weeks but not yet meeting full primary endpoint response criteria, and who are tolerating investigational treatment, may receive an additional 4 weeks of eltrombopag and be reassessed after 20 weeks. At that time, if they meet primary endpoint response criteria, they will be eligible to enter the extended access part of the study. If they do not meet primary endpoint response criteria, eltrombopag will be discontinued.

Primary objective is to assess the efficacy of eltrombopag in patients with low to Int-2 risk MDS. Safety of eltrombopag in this subject population will be assessed concurrently.

Secondary objectives include the toxicity profile of extended treatment with eltrombopag (treatment longer than 4 months), reduction in incidence and severity of bleeding episodes, and response following extended access to study drug (treatment longer than 4 months).

The primary endpoint will be the portion of drug responders as defined by changes in the platelet count and/or platelet transfusion requirements, or the proportion of subjects who meet erythroid response, or neutrophil response criteria.. Platelet response is defined as platelet count increases to 20,000/microL above baseline at 16 or 20 weeks, or stable platelet counts with transfusion independence for a minimum of 8 weeks. Erythroid response for subjects with a pretreatment hemoglobin of less than 9 g/dL will be defined as an increase in hemoglobin by greater than or equal to 1.5g/dL without packed red blood cell (PRBC) transfusion support, or a reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment compared with the pretreatment transfusion number in the previous 8 weeks. Neutrophil response will be defined in those with a pretreatment absolute neutrophil count (ANC) of \<0.5 times 10(9)/L as at least a 100% increase or an absolute increase \> 0.5 times 10(9)/L. Subjects with an erythroid, and/or neutrophil response at 16 weeks may continue study medication (extended access) until they meet an off study criteria. Subjects with erythroid, or neutrophil response at 16 weeks may continue study medication for an additional 4 weeks (to ensure eligibility) prior to being consented for entry into the extended access part of the trial. Patients may remain on the extended access until they met an off study criteria.

The toxicity profile will be measured using the CTCAE Version 4.0 criteria.

Secondary endpoints will include incidence of grade 2 or higher bleeding events as measured by CTCAE v. 4.0; changes in serum thrombopoietin level, measured at 4 months; and progression to higher risk MDS as measured by IWG criteria.

A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

The primary endpoint was hematologic response at 16 or 20 weeks, defined as either: (1) an increase in platelet counts =20.000/uL or transfusion independence for a minimum of 8 weeks; (2) hemoglobin (Hb) increase of =1.5g/dL from baseline, or a reduction in red blood cells (RBC) transfusion of at least 50%; or (3) an increase in absolute neutrophil counts (ANC) of =0.5x109/L or by at least 100% in patients with a baseline ANC \<0.5x109/L.

Changes in serum thrombopoietin level at 16 or 20 weeks

Number of participants with progression to higher risk Myelodysplastic syndrome (MDS) as measured by International Working Group (IWG) 2006 criteria in participants With Low to Int-2 Risk Myelodysplastic Syndrome (MDS) following Eltrombopag.

Progression of disease per modified IWG criteria 2006 were considered: if patients with baseline bone marrow blasts of \< 5% increase by ≥ 50% to \>5% blasts; and either at least 50% decrease in granulocytes or platelets from maximum response, decreasing in Hb by 2g/dL or transfusion dependence. The acquisition of new cytogenetic abnormality without meeting other IWG criteria for progression was not considered disease progression.

Number of participants who achieved a robust response and discontinued Eltrombopag in extended access

A robust response is defined as: as platelets \>50,000/μL, hemoglobin \> 10 g/dL in the absence of transfusions, and neutrophils \> 1,000 for more than 8 weeks.

Number of Participants with Grade 2 or Higher Bleeding Events defined by Common Terminology Criteria for Adverse Events (CTCAE) v. 4.0.

The NCI CTCAE is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. The CTCAE displays Grades 1 through 5 with unique clinical descriptions of severity for each AE based on this general guideline. Grade 1 Mild is asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 is moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL)\*. Grade 3 is severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL\*\*. Grade 4 if life-threatening consequences; urgent intervention indicated. Grade 5 is death related to AE.

National Heart, Lung, and Blood Institute (NHLBI)

Any observed or volunteered adverse events that are as listed in the Eltrombopag Package Insert and/or Investigator's Brochure will not be reported unless (1) the adverse event was not present at baseline exam; (2) the adverse event is previously unknown (not on the label); (3) the adverse event is more severe than on the label; (4) the frequency of the adverse events increases above the listed frequency; or (5) meets the criteria for a serious adverse event.

Eltrombopag (EPAG), a thrombopoietin receptor agonist, was started at 50 mg/day, up to a maximal dose of 150 mg/day, increasing the dose by 25mg every 2 weeks.

Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

Eltrombopag in Low to Int-2 Risk Myelodysplastic Syndromes (MDS)

Age, Categorical

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Low Risk Myelodysplastic Syndromes (MDS) are defined as having a low risk or a score of zero by the International Prognostic Scoring System (IPSS). A zero score is defined as: bone marrow blasts less than 5%, a good karyotype, and 1 of the following cytopenia: Hemoglobin \< 10g/dL, Absolute neutrophil count \< 1800/uL, Platelet count \< 100,000/uL.

IPSS score may range from zero which is the lowest score and is associated with better survival to a high score which is 3.5 and is associated with poorer survival when treatment is not provided.

Low Risk Myelodysplastic Syndromes (MDS)

Intermediate-1 Risk Myelodysplastic Syndromes are defined as having low risk with score from 0.5 to 1 by the International Prognostic Scoring System (IPSS). A 0.5 to 1 score is defined as: bone marrow blasts 5 to 10%, intermediate to poor karyotype and 2 or 3 of the following: Hemoglobin \< 10g/dL, Absolute neutrophil count \< 1800/uL, Platelet count \< 100,000/uL.

IPSS score may range from zero which is the lowest score and is associated with better survival to a high score which is 3.5 and is associated with poorer survival when treatment is not provided.

Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

Intermediate-2 Risk Myelodysplastic Syndromes are defined as having a higher risk with score from 1.5 to 2 by the International Prognostic Scoring System (IPSS). A score of 1.5 to 2 is defined as: bone marrow blasts 11 to 30%, poor karyotype, and 2 or 3 of the following: Hemoglobin \< 10g/dL, Absolute neutrophil count \< 1800/uL, Platelet count \< 100,000/uL.

IPSS score may range from zero which is the lowest score and is associated with better survival to a high score which is 3.5 and is associated with poorer survival when treatment is not provided.

Intermediate-2 Risk Myelodysplastic Syndromes (MDS)

The first five participants enrolled were entered when eligibility criteria included only thrombocytopenia. They were not included in the efficacy analysis set, as requested by the Institutional Review Board, but were included for secondary endpoint and sensitivity analyses.

Bhavisha Patel, MD

Eltrombopag in Low to Int-1 Risk Myelodysplastic Syndrome (MDS)

The first 5 subjects enrolled were not counted toward the accrual target due to changes in protocol.

Number of Participants With Response Between Weeks 16 and 20

Changes in Serum Thrombopoietin Level

Number of Participants With Progression to Higher Risk Myelodysplastic Syndrome as Measured by International Working Group Criteria

Analysis included those participants that participated in extended access portion of study

Number of Participants Who Achieved a Robust Response and Discontinued Eltrombopag

Number of Participants With Grade 2 or Higher Bleeding Events Defined by Common Terminology Criteria for Adverse Events v. 4.0

Initial Treatment (Weeks 1 - 20)

Extended Access: Week 20 to Up to Year 5

Nervous System Diseases

Myasthenia Gravis

Muscle Weakness

Neuromuscular Diseases

Paresis

Asthenia

Muscular Diseases

Neuromuscular Manifestations

Neurologic Manifestations

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

Amino Acids

Antibodies

Autoantibodies

Immunoglobulins

Tyrosine

Cohort A: Infusion of MuSK-CAART at various dose levels with or without pre-treatment (6 groups planned).

Cohort B: Infusion of MuSK-CAART at the dose regimen selected from Part A.

Intravenous infusion of MuSK-CAART at different doses. Subjects may also receive MuSK-CAART following pre-treatment with CY, or CY plus FLU.

MuSK-CAART

Cabaletta Bio

Mani Nistor

Ali Habib, MD

Joshua Valdez

David Richman, MD

Andrew J Heim

Mazen Dimachkie, MD

Katie Lewis

Nizar Chahin, MD

Medical Director

Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction. This phase 1 study is being conducted to evaluate the safety of various dosing regimens of an investigational cell therapy, MuSK-CAART, that can be given to patients with anti-MuSK antibody positive Myasthenia Gravis (MuSK MG), who have active disease. Various dosing regimens of MuSK-CAART alone, in combination with cyclophosphamide (CY), and in combination with CY and fludarabine (FLU) will be evaluated. Treatment with MuSK-CAART may potentially lead to complete and durable remission of disease.

Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis

A Phase 1, Open-label, Safety and Dose-finding Study of Autologous Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART) in Subjects With Anti-MuSK-antibody-positive Myasthenia Gravis

Frequency and dose of concomitant therapies.

Measurement of clinical symptoms using the Myasthenia Gravis Activities of Daily Living (MG-ADL) assessment.

Measurement of clinical symptoms using the Quantitative Myasthenia Gravis (QMG) assessment.

Measurement of clinical symptoms using the Myasthenia Gravis Composite (MGC) assessment.

Measurement of Quality of Life using the MG-QOL-15r (Myasthenia Gravis Qualify of Life 15-item scale, revised) questionnaire.

Incidence of adverse events (AEs), including dose-limiting toxicities (DLTs) and AEs that are related to MuSK-CAART.

Total MuSK-CAART positive cells for each manufacturing run.

Percent of total cells for infusion that are CAAR (Chimeric Autoantibody Receptor)-transduced cells.

Cellular kinetics profile of MuSK-CAART after infusion.

Change in MuSK autoantibody titer compared to pre-infusion visit by clinically validated assay.

Adult patients with a diagnosis of cancer receiving checkpoint inhibitor therapy at the University of Pennsylvania's Abramson Cancer Center (ACC).

Kyra J Sacksith

Terri M Laufer, MD

The purpose of this study is to better understand how the treatment of cancer with immune checkpoint inhibitors (ICI) leads to the development of autoimmunity. Specifically, we wish to understand the genetics and immune system features that cause a subset of cancer patients treated with checkpoint inhibitor therapy to develop an immune-related adverse event (irAE).

The goal of this study is to understand how the treatment of cancer with checkpoint inhibitors leads to the development of autoimmunity. Specifically, to understand the genetics and immune system features that cause a subset of cancer patients treated with checkpoint inhibitors to develop autoimmunity. At least 300 patients will be enrolled when they are initially prescribed any checkpoint inhibitor. Peripheral blood and serum from patients when they enroll and examine the genetics, serum factors, and phenotype of the immune system. There is no planned intervention. Subjects will be asked to provide peripheral blood and serum at established time points when they are getting therapeutic infusions and routine clinical labs, as well as, at the time they develop any autoimmune symptoms. Urine may also be collected at the time of enrollment, standard study visits, and/or at the time of an autoimmune complication. Inclusion criteria include a diagnosis of cancer, prescription for a checkpoint inhibitor, and fluency in English. Exclusion criteria include any subjects not willing or able to give consent, children under the age of 18, and history of transplant. There will not be any interventions. Clinical data will be extracted from electronic medical records. Coded data will be stored in a REDCap database. PHI and study key stored at UPenn on a password-protected database on an encrypted drive that is institutionally secured and managed by the University of Pennsylvania.

50 mls of peripheral blood will be drawn at the time that patients are prescribed an immune checkpoint inhibitor (ICI) which will be banked for serum and PBMCs. No more than 50 mls of blood will be drawn at the following established points: at the time of the second drug infusion, at 6 months, at 12 months, and between 18 and 24 months. Additionally, no more than 50 mls of blood will be drawn at any time that a participant develops an autoimmune complication.

An assessment tool that queries symptoms suggestive of a number of common immunotherapy-related adverse events (PRO-CTCAE) will be administered to consented patients at each visit in concert with the standard-of-care patient reported outcomes tool currently in use in Cancer Center clinics.

Autoimmunity After Checkpoint Blockade

Mechanisms of Immunotoxicology in Cancer Patients

The phenotypic analysis will include multiple surface markers that define populations of cells, including activated, memory, and regulatory populations. Cells will also be activated to determine which cytokines are produced.

This is a pilot analysis to better understand how the treatment of cancer with immune checkpoint inhibitors leads to the development of autoimmunity in a subset of cancer patients.

To facilitate collaborations between research groups from diverse disciplines that enables rapid translation of ongoing and future basic research findings that leads to individualized, or personalized care for patients receiving immunotherapies.

University of Pennsylvania

Skin and Connective Tissue Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Gland and Hormone Related Diseases

Diseases and Abnormalities at or Before Birth

Breast Neoplasms

Endometriosis

Ovarian Neoplasms

Carcinoma, Ovarian Epithelial

Hereditary Breast and Ovarian Cancer Syndrome

Ovarian Cancer

Ovarian Epithelial Cancer

For individuals identified with the KRAS-variant. Patients will be prospectively followed to determine the impact of lifestyle on disease risk.

Women with breast cancer who are BRCA negative will be tested for the KRAS-variant, to determine the associations as well as the prevalence.

Women with multiple primary breast cancer will be tested for the KRAS-variant and compared between those with this mutation and those without.

We have shown that the KRAS-variant and other members of this genetic class of mutations associate with altered immunity, leading to immunosuppression as well as autoimmunity.

Participant in these studies will be tested for the KRAS-variant

KRAS-variant and microRNA binding site mutation testing

Joanne Weidhaas, MDPhD

Joanne Weidhaas

The investigators will recruit and enroll individuals that may have the KRAS-variant or other microRNA binding site mutations to join registry studies. The investigators will allow individuals to obtain their results through a physician at the completion of the studies. The investigators current focus is cancer and autoimmunity.

The investigators have identified germ-line microRNA binding site mutations that predict an increased risk of cancer, endometriosis and associated infertility, and unique tumor biology and response to treatment. The goal of this protocol is to further determine the mechanisms of these mutations, such as the KRAS-variant, and their associations with human health, such as cancer. The investigators will collect saliva samples from individual patients who are eligible and choose to enroll in these studies, to test for the KRAS-variant and/or other mutations under study. With specific permission, the investigators will keep excess DNA to further investigate and discover additional similar mutations. The investigators purpose is to have participants answer questionnaires about lifestyle factors in an ongoing manner, to understand the impact of different factors on cancer risk for patients with these mutations.

DNA will be isolated from the submitted sample and kept with permission for additional studies.

Clinical Validation of the Role of microRNA Binding Site Mutations in Cancer Risk, Prevention and Treatment

The Prevalence of the KRAS-variant will be determined in specific populations, such as women with drug resistant endometriosis, or BRCA negative breast cancer. The prevalence will be compared to extensive data on the expected and known prevalence of the KRAS-variant in non-diseased populations. Statistical significance will be determined by Chi-squared analysis.

We will compare the impact of specific treatment approaches for example in women with the KRAS-variant and double primary breast cancer, versus the interventions used in non-KRAS-variant double primary breast cancer patients.

Individuals with the KRAS-variant will be prospectively followed, and lifestyle factors will be associated with changes in health, including cancer development. Our goal is to compare baseline characteristics between individuals with the KRAS-variant who do, versus do not, develop cancer, for example.

MiraKind

Multiple Sclerosis

Multiple Sclerosis, Chronic Progressive

Sclerosis

Neoplasm Metastasis

Demyelinating Autoimmune Diseases, CNS

Demyelinating Diseases

Neoplastic Processes

Chronic Disease

Disease Attributes

Chronic Graft Versus Host Disease

Siponimod

Patients with secondary progressive multiple sclerosis (SPMS) randomized to receive BAF312 (siponimod). Refer to ClinicalTrials.gov record NCT01665144 for more information.

Patients with secondary progressive multiple sclerosis (SPMS) randomized to receive placebo. Refer to ClinicalTrials.gov record NCT01665144 for more information.

Blood draws (65 mLs \[\~4 tablespoons\] per blood draw) at 4 time points: Prior to study medication initiation, and at +6, +12 and+24 months post treatment initiation.

Blood Draw

For participants who volunteer to donate CSF samples: up to 25 mLs (\<2 tablespoons): prior to study medication initiation, and at month 24 post treatment initiation.

CSF collection by lumbar puncture (Optional)

Yang Mao-Draayer, MD, PhD

David Fox, MD

The primary goal of this study is to evaluate the effects of BAF312 (siponimod) on select immune and neuronal (nerve) cells by examining laboratory specimens (blood and/or spinal fluid) at multiple time points, prior to, and following the initiation of BAF312 or placebo treatment, in patients with Secondary Progressive Multiple Sclerosis (SPMS) who are enrolled in a clinical trial (NCT01665144) to evaluate the effectiveness and safety of BAF312.

This study is complementary to a multi-center, randomized, double-blind,parallel-group, placebo-controlled, variable treatment duration study comparing the efficacy and safety of BAF312 to placebo in patients with SPMS (NCT01665144). Investigators will explore both immunological and neuroprotective mechanisms of BAF312 (siponimod), a novel agent in the setting of a SPMS clinical trial.

This study is part of a multi-center study, with the University of Michigan serving as the central site.

Mechanistic Studies of Phase III Trial With BAF312 in Secondary Progressive Multiple Sclerosis

Mechanistic Studies of Phase III Trial With BAF312 in Secondary Progressive Multiple Sclerosis (AMS04)

Evaluation (BAF312 versus placebo) of dominant cytokines produced by myelin basic protein (MBP)-stimulated peripheral blood mononuclear cells (PBMCs), measured by ELISpot.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Rheumatologic Adverse Events and Cancer Immunotherapy	NCT04791150	Oncology Autoimmunity Arthritis Adverse Drug Ev...	Fill in a form	18 Years -	Poitiers University Hospital
Rheumatologic Adverse Events and Cancer Immunotherapy	NCT04791150	Oncology Autoimmunity Arthritis Adverse Drug Ev...	Fill in a form	18 Years -	Poitiers University Hospital
A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)	NCT00961064	Myelodysplastic... Thrombocytopeni...	Eltrombopag	18 Years - 100 Years	National Institutes of Health Clinical Center (CC)
Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis	NCT05451212	MuSK Myasthenia...	MuSK-CAART	18 Years -	Cabaletta Bio
Autoimmunity After Checkpoint Blockade	NCT04119713	Autoimmunity Cancer Immunotoxicity Oncology		18 Years -	University of Pennsylvania
Clinical Validation of the Role of microRNA Binding Site Mutations in Cancer Risk, Prevention and Treatment	NCT02253251	Cancer	KRAS-variant an...	18 Years -	MiraKind
Mechanistic Studies of Phase III Trial With BAF312 in Secondary Progressive Multiple Sclerosis	NCT02330965	Secondary Progr...	Blood Draw CSF collection ...	18 Years - 60 Years	National Institute of Allergy and Infectious Diseases (NIAID)
How Microbes and Metabolism May Predict Skin Cancer Immunotherapy Outcomes	NCT03370861	Autoimmunity Melanoma Merkel Cell Car... Squamous Cell C... Basal Cell Carc... Skin Cancer	Immunotherapy	18 Years -	Washington University School of Medicine
Rheumatologic Adverse Events and Cancer Immunotherapy	NCT04791150	Oncology Autoimmunity Arthritis Adverse Drug Ev...	Fill in a form	18 Years -	Poitiers University Hospital
A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)	NCT00961064	Myelodysplastic... Thrombocytopeni...	Eltrombopag	18 Years - 100 Years	National Institutes of Health Clinical Center (CC)

Spots Global Cancer Trial Database for autoimmunity

Every month we try and update this database with for autoimmunity 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.