Symptoms and General Pathology

All Conditions

Neoplasms

Nervous System Diseases

Immune System Diseases

Diseases and Abnormalities at or Before Birth

Eye Diseases

Rare Diseases

Syndrome

Lambert-Eaton Myasthenic Syndrome

Myasthenic Syndromes, Congenital

Nystagmus, Pathologic

Myasthenia Gravis

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

Neuromuscular Diseases

Genetic Diseases, Inborn

Lambert Eaton Myasthenic Syndrome

Congenital Myasthenic Syndromes

Channel Blockers

All Drugs and Chemicals

Amifampridine

Potassium Channel Blockers

Dosage form: tablets containing the equivalent of 10 mg amifampridine per tablet.

Amifampridine Phosphate given based on Investigator assessment of optimal neuromuscular benefit. In adult patients \> 16 years of age, Doses ranged from 30 mg to 80 mg, divided into doses taken 3 to 4 times per day and in pediatric patients age \< 16 years the maximum daily dose was 60 mg/day. No single dose to exceed 20 mg.

Amifampridine Phosphate

Perry Shieh, MD, PhD

Primary:

The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus until the product became commercially available.

Secondary:

The secondary objective of this study under the original protocol was to provide additional long-term safety data on amifampridine phosphate in patients.

Primary The primary objective of this study after its fifth amendment was to provide access to amifampridine phosphate therapy to pediatric patients with LEMS, and pediatric and adult patients with CMS until the product became commercially available for these indications or development of the product for the indication was terminated.

Secondary:

The secondary objective of this study after its fifth amendment was to assess the long-term safety of amifampridine phosphate in pediatric patients with LEMS, and pediatric and adult patients with CMS.

This multicenter, expanded access study was designed to allow EAP-001 Investigators, neuromuscular specialists, and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS, or downbeat nystagmus until such time the product became commercially available for these specific indications or development of the product for the indication was terminated. Given that these are chronic conditions, this study obtained some long-term safety data by evaluating Adverse Events, physical exams, vital signs, standard safety laboratory tests (chemistry, hematology, and urinalysis), pregnancy testing (if applicable) and abnormal ECG findings, where each of these data/measures were performed as part of a routine standard of care treatment provided by the Principal Investigator or the patient's personal physician(s) (i.e., could be performed by a different physician not involved in the treatment of neuromuscular disease).

Planned -100 sites in US and up to 200 patients. Actual- 50 sites activated and 200 patients enrolled.

Patients were seen at screening/baseline, Day 1 and at least once per year at the PIs discretion. With the COVID-19 Pandemic, telehealth visits were allowed and assessments that could be completed via remote telehealth visits were performed via phone or videoconference. Follow-up visits included Physical exams, vital signs, standard safety laboratory tests (chemistry, hematology, and urinalysis), pregnancy testing (if applicable) and ECGs.

Patients were titrated to an optimal individualized dose of amifampridine phosphate based on Principal Investigator assessment of optimal neuromuscular benefit. In patients already taking amifampridine base, amifampridine phosphate was started at an equivalent or lower dose of amifampridine phosphate, at the Principal Investigator's discretion. Therapeutic doses were to range from 10 to 80 mg, divided into doses taken two (2) to four (4) times per day, with a maximum single dose of 20 mg. For upward titration, amifampridine phosphate could be increased by 10 mg increments every four (4) to five (5) days to a maximum of 80 mg per day (ages \>16 years), and to a maximum of 60 mg (ages 2 to 16 years) based on optimal neuromuscular benefit and at the discretion of the Principal Investigator. If needed, titration could occur in 5 mg/dose and per day increments. Drug was shipped to the clinic for the first dose ( Day 1) and subsequent shipments were sent to the patients home by a specialty pharmacy.

In addition to amifampridine phosphate, patients received best supportive care (BSC) treatment as determined by the Principal Investigator using concomitant medications permitted by protocol, including selected oral immunosuppressant's (i.e., prednisone or other corticosteroids, azathioprine, mycophenolate) and peripherally acting cholinesterase inhibitors (e.g., pyridostigmine). Changes to BSC could be made at the Principal Investigator's discretion, as long as prohibited concomitant medications were not used.

Firdapse® was approved for adult patients with LEMS in November 2018 and approved for pediatric patients with LEMS ages 6 years and older in September 2022. The indications for CMS and DBN were not pursued.

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)

Catalyst Pharmaceuticals, Inc.

Blood and Lymph Conditions

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Leukemia, Lymphocytic, Chronic, B-Cell

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Blast Crisis

Neoplasms by Histologic Type

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Chronic Graft Versus Host Disease

Chronic Lymphocytic Leukemia

Chronic Myeloid Leukemia

Acute Graft Versus Host Disease

OPB-31121 200 mg twice daily for 21 days followed by 7 days of rest

Starting dose of 200 mg (two 100 mg tablets) twice a day for 21 days followed by 7 days of rest.

OPB 31121

Gautam Borthakur, MBBS

The goal of this clinical research study is to find the highest tolerable dose of OPB-31121 that can be given to patients with leukemia or myelodysplastic syndrome (MDS).

The Study Drug:

In studies that have been performed in animals, OPB-31121 has been shown to stop certain changes to proteins in the body. Stopping these changes may have an effect on tumor cells. How OPB-31121 works is not fully known.

Study Groups:

The first 3 participants enrolled in the study will receive the lowest level of OPB-31121. Each additional group of 3 or 4 participants will receive a dose level dependent upon how the previous group responded to the study drug.

You may have up to 13 study "cycles" (about 273 days). During each 28 day study cycle, you will receive the study drug for 21 days and then "rest" (no study drug) for 7 days. The cycle is 28 days, 21 days of drug, 7 days of rest.

Study Drug Administration:

The study drug is a pill that should be taken at the same time each day. You should drink 8 ounces (1 cup) of room temperature, non-carbonated water when you take the study drug.

You will eat a snack or meal within 30 minutes after taking the study drug. You should remain sitting upright or standing for at least 30 minutes after taking the drug.

Study Visits for AML, ALL, or MDS participants:

On Day 1 of Cycle 1, you will have the following tests and procedures performed:

* You will have a physical exam, including measurement of your vital signs.
* You will have an ECG.
* Urine will be collected for routine tests.
* Women who are able to have children will have a urine pregnancy test.
* Blood (about 6 tablespoons total) will be drawn about 10 times. These blood draws will be for routine tests, heart, thyroid testing, pharmacokinetic (PK) testing, and genotyping. PK testing measures the amount of study drug in the body at different time points. Genotyping tests are tests that look at pieces of your DNA called genes. These tests will look at some specific genes that may predict how your body may break down certain types of drugs. A small needle called a "heparin lock" may be inserted into a vein to make these multiple blood draws easier.

On Day 2 of Cycle 1, blood (about 2 tablespoons) will be drawn for heart, PK, and biomarker testing.

On Days 8, 15 and 21 of Cycle 1:

* You will have a physical exam, including measurement of your vital signs.
* You will have an ECG.
* Urine will be collected for routine tests.
* Blood (about 2 tablespoons) will be drawn for routine, heart, and thyroid testing.
* Blood (about 6 tablespoons total) will also be drawn for PK testing. Blood will be drawn a total of 10 times, with the last blood draw about 10 hours after you receive the study drug.
* Blood (about 1/2 tablespoon) will be drawn for biomarker testing (Day 21 only).

On Day 22 of Cycle 1, blood (about 1 tablespoon each time) will be drawn 2 times for PK testing.

On Day 23 of Cycle 1, blood (about 1 tablespoon) will be drawn 1 time for PK testing.

On Day 28 of Cycle 1, you will have the following procedures performed:

* You will have an ECG.
* You will have a physical exam, including measurement of your vital signs.
* You will have a bone marrow aspiration sample taken to check the status of the disease. This sample will be taken after you have taken the study drug and only at the end of each odd cycle.

On Day 1 of Cycles 2-13, you will have the following tests and procedures will be performed:

* Blood (about 1-1/2 tablespoons) and urine will be collected for routine, heart, and thyroid tests.
* You will have a physical exam, including measurement of your vital signs.
* You will have an ECG.
* Women who are able to have children will have a urine pregnancy test.
* On Day 1 of Cycles 2 and 3 only, blood (less than 1 tablespoon) will be drawn for biomarker tests.
* You will have a MUGA scan.
* If your doctor thinks it is needed, you will have a bone marrow aspiration/biopsy.

Study Visits for CLL or CML participants:

On Day 1 of Cycle 1, you will have the following tests and procedures performed:

* You will have a physical exam, including measurement of your vital signs.
* You will have an ECG.
* Urine will be collected for routine tests.
* Blood (about 8 tablespoons total) will also be drawn a total of 10 times for routine, heart, thyroid, biomarker, genotyping, and PK testing. The last blood draw will be 24 hours after you receive the study drug (Day 2). A small needle called a "heparin lock" may be inserted into a vein to make these multiple blood draws easier.
* Women who are able to have children will have a urine pregnancy test.
* If your doctor thinks it is needed, you will have a bone marrow biopsy.

On Day 2 of Cycle 1, blood (about 2 tablespoons) will be drawn 24 hours after your study drug dose for heart and PK testing.

On Days 8 and 15 of Cycle 1, blood (about 7 tablespoons) will be drawn for routine and PK testing.

On Days 21 of Cycle 1, you will have an ECG. Blood (about 8 tablespoons in total) will be drawn about 8 times for PK, routine, heart, thyroid, and biomarker testing.

On Days 22 of Cycle 1, blood (about 1 tablespoon each time) will be drawn 2 times for PK testing.

On Day 23 of Cycle 1, blood (about 1 tablespoon) will be drawn 1 time for PK testing.

During Cycles 2-13, blood (about 1 tablespoon) will be drawn every 2 weeks for routine, heart, and thyroid testing.

On Day 1 of Cycles 2-13, you will have the following tests and procedures performed:

* You will have a physical exam, including measurement of your vital signs.
* You will have an ECG and MUGA scan.
* Blood (about 2 tablespoons) will be drawn for routine, heart, thyroid, and biomarker tests.
* Women who are able to have children will have a urine pregnancy test.
* During Cycles 2-3 only, blood (about 1 tablespoon) will be drawn for biomarker testing.

End-of-Study Visit:

Once you are off study, you will have an end-of-study visit. At this visit, the following tests and procedures will be performed:

* You will have a physical exam, including measurement of your vital signs.
* You will have an ECG.
* Blood (about 7 tablespoons) will be drawn for routine, heart, thyroid, and PK tests.
* Urine will be collected for routine tests.
* If you have AML, ALL or MDS, blood (about 1-1/2 tablespoons) will be drawn for biomarker testing.

Follow Up:

Your doctor or other study staff will call you about 30 days after your final visit to see how you have been feeling. The phone call will take about 5-10 minutes.

This is an investigational study. OPB-31121 is not FDA approved or commercially available. At this time, OPB-31121 is only being used in research. Up to 48 patients will take part in this multi-center study.

Study to Assess OPB-31121 in Advanced Leukemias or Myelodysplastic Syndromes

An Open-label, Non-randomized Study to Assess the Pharmacokinetics, Dose Limiting Toxicity and Maximum Tolerated Dose of OPB-31121 in Subjects With Advanced Leukemias or Myelodysplastic Syndromes

MTD defined as highest dose level at which \< 2 of 6 subjects experience dose limiting toxicity (DLT) during the first cycle.

Otsuka Pharmaceutical Co., Ltd.

M.D. Anderson Cancer Center

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Skin and Connective Tissue Diseases

Respiratory Tract (Lung and Bronchial) Diseases

Digestive System Diseases

Uterine Cervical Neoplasms

Breast Neoplasms

Prostatic Neoplasms

Lung Neoplasms

Colorectal Neoplasms

Genital Neoplasms, Male

Urogenital Neoplasms

Genital Diseases

Genital Diseases, Male

Urogenital Diseases

Prostatic Diseases

Male Urogenital Diseases

Intestinal Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Colonic Diseases

Intestinal Diseases

Rectal Diseases

Uterine Neoplasms

Genital Neoplasms, Female

Uterine Cervical Diseases

Uterine Diseases

Genital Diseases, Female

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Patient Navigator (PN) + Educational Materials + Annual Questionnaires

Educational Materials + Exit Questionnaire

Intervention Group:

Cancer Screening Assessment (CSA) questionnaire 45-60 minutes annually for 4 years + Exit Questionnaire at end of study.

Comparison Group:

Exit Questionnaire only.

Questionnaire

Staff member, called patient navigator (PN), who assist with medical care and/or screenings, questionnaires.

Patient Navigator (PN)

Isabel Torres, DRPH,MPH

The goal of this behavioral research study is to improve the use of cancer prevention services, increase early detection, and treatment of cancer. A secondary outcome of this demonstration project proposes that a structured patient navigator (PN) will reduce the cost of Medicare services.

Screening Questionnaire:

Before you can start on this study, you will answer a 20 minute Triage questionnaire. The questionnaire will ask you if you have had cancer, what type of cancer, and when you were last treated.

Cancer-Screening Groups and Treatment Groups:

Once you answer the Triage questionnaire and if you are found to be eligible to take part in this study, you will be placed in the Cancer Screening Group or the Treatment Group based on your answers to the Triage questionnaire.

If you have not been treated for cancer in the past or if it has been more than 5 years since you were last treated, you will be in the Cancer Screening Group and you will complete the screening Cancer Screening Assessment (CSA). The CSA will ask you about cancer screenings, lifestyle, and other health related items. The questionnaire should take about a total of 45-60 minutes to complete.

If you have been treated for cancer in the past 5 years, you will be in the Treatment Group. The CSA will ask you about cancer screenings, cancer treatment, and other health related items. The questionnaire should take about a total of 45-60 minutes to complete.

After completion of the Cancer Screening Assessment (CSA) for either the Cancer Screening Group or the Treatment Group, you will complete a CSA addendum questionnaire. This CSA addendum questionnaire will ask you about barriers and factors that may influence your health related decisions. The questionnaire should take 15-20 minutes to complete.

Intervention and Comparison Groups:

Participants in either the cancer-screening group or the treatment group will be randomly assigned (as in the flip of a coin) to one of 2 additional groups, the "Intervention group" or the "Comparison" group. You will have an equal chance of being in either group.

If you are in the Intervention Group, you will work with a staff member, called a patient navigator (PN), who will assist with medical care and/or screenings (intervention group). For example, the staff member may help schedule screening appointments, medical care appointments, or any other necessary assistance to meet health care needs. The PN will be responsible for recording information about your care and any cancer treatment, should that be necessary.

You will complete a CSA questionnaire 1 time a year for up to 4 years. A staff member will contact you by phone to set up an appointment to give you the CSA questionnaires. All questionnaires will be completed at a time and place that you and the study staff agree upon. For example, the study staff may meet you at a community center, independent-living center, or another agreed upon location. The questionnaire should take about 45-60 minutes to complete.

If you are in the Comparison Group, you will not work with a staff member and you will not complete any additional questionnaires during the 4 years on study.

Both the intervention group and the comparison group will receive educational materials.

Exit Questionnaire:

About 4 years after you join the study, you will be contacted by a staff member and you will set up an exit questionnaire meeting. This questionnaire will be completed at a time and place that you and the staff member agree upon. This questionnaire will contain similar questions to those you have been answering since your participation in the study. The questionnaire will take about 45-60 minutes to complete.

Length of Study:

You will be considered off-study after 4 years.

This is an investigational study. Up to 11,964 participants will take part in this study. A total of 2,812 will be enrolled at M. D. Anderson.

Cancer Prevention and Treatment Demonstration for Ethnic and Racial Minorities

Cancer Prevention and Treatment Demonstration for Hispanic Medicare Eligible Beneficiaries - CMS

Centers for Medicare and Medicaid Services

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome	NCT02189720	Congenital Myas... Lambert-Eaton M... Nystagmus, Acqu...	Amifampridine P...	2 Years -	Catalyst Pharmaceuticals, Inc.
Study to Assess OPB-31121 in Advanced Leukemias or Myelodysplastic Syndromes	NCT01029509	Leukemia	OPB 31121	18 Years -	M.D. Anderson Cancer Center
Cancer Prevention and Treatment Demonstration for Ethnic and Racial Minorities	NCT00453661	Breast Cancer Cervical Cancer Prostate Cancer Colorectal Canc... Lung Cancer	Questionnaire Patient Navigat...	40 Years -	M.D. Anderson Cancer Center
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome	NCT02189720	Congenital Myas... Lambert-Eaton M... Nystagmus, Acqu...	Amifampridine P...	2 Years -	Catalyst Pharmaceuticals, Inc.
Cancer Prevention and Treatment Demonstration for Ethnic and Racial Minorities	NCT00453661	Breast Cancer Cervical Cancer Prostate Cancer Colorectal Canc... Lung Cancer	Questionnaire Patient Navigat...	40 Years -	M.D. Anderson Cancer Center

Spots Global Cancer Trial Database for cms

Every month we try and update this database with for cms 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 cms

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