Symptoms and General Pathology

All Conditions

Behaviors and Mental Disorders

Neoplasms

Nervous System Diseases

Immune System Diseases

Rare Diseases

Syndrome

Mental Disorders

Lambert-Eaton Myasthenic Syndrome

Problem Behavior

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

Lambert Eaton Myasthenic Syndrome

Channel Blockers

All Drugs and Chemicals

Amifampridine

Potassium Channel Blockers

3,4-diaminopyridine

Donald B. Sanders

OBJECTIVES: I. Evaluate the safety and effectiveness of 3,4-diaminopyridine (DAP) in the treatment of patients with Lambert-Eaton myasthenic syndrome (LEMS).

II. Determine the side-effects and benefits associated with DAP.

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled study. Patients are randomized to receive 3,4-diaminopyridine (DAP) or placebo orally 3 times daily for 5 days, after which treatment is discontinued and patients are observed for at least 24 hours. At the end of the blinded study, patients may then elect to take open label DAP orally 3 times daily for 6 months; those who do so are monitored for clinical effects and side effects for at least 6 months.

Randomized Study of 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome

Duke University

FDA Office of Orphan Products Development

Antibodies

Immunoglobulins

Immunoglobulins, Intravenous

gamma-Globulins

Rho(D) Immune Globulin

Immunologic Factors

immune globulin

Richard J. Barohn

OBJECTIVES:

I. Determine whether intravenous immunoglobulin is an effective therapy for patients with mild or moderate myasthenia gravis.

PROTOCOL OUTLINE: This study is a randomized, blinded, controlled study. Patients are stratified in both groups according to prior thymectomy (yes vs no) and by Quantitative Myasthenia Gravis Score (equal to or less than 10 vs greater than 10) so that equal numbers are assigned to each group. Patients in group 2 are also stratified as to whether they are currently on azathioprine.

Patients are randomized to receive either intravenous immunoglobulin (IVIG) for 2 days or 5% albumin (the placebo) for 2 days. A second infusion of IVIG for 1 day or albumin placebo for 1 day is given on day 22.

At the end of 6 weeks, after the randomized study, patients may choose to receive 3 additional IVIG infusions.

Patients are followed on days 1, 21, 32, and 42 of randomized or open label study.

Completion date provided represents the completion date of the grant per OOPD records

Randomized Study of Intravenous Immunoglobulin in Patients With Mild or Moderate Myasthenia Gravis

University of Texas

Ear, Nose, and Throat Diseases

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Hearing Loss

Deafness

Neurofibromatoses

Neurofibromatosis 1

Neurofibroma

Neuroma

Neuroma, Acoustic

Neurofibromatosis 2

Nerve Sheath Neoplasms

Neoplasms, Nerve Tissue

Neoplasms by Histologic Type

Neoplastic Syndromes, Hereditary

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Genetic Diseases, Inborn

Peripheral Nervous System Diseases

Peripheral Nervous System Neoplasms

Neurilemmoma

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Vestibulocochlear Nerve Diseases

Retrocochlear Diseases

Ear Diseases

Otorhinolaryngologic Diseases

Otorhinolaryngologic Neoplasms

Cranial Nerve Diseases

Neurofibromatosis

Neurofibromatosis Type 1

Acoustic Neuroma

Neurofibromatosis Type 2

Schwannoma

Neuroendocrine Tumor

Neuroepithelioma

Multichannel Auditory Brain Stem Implant

Steven J. Staller

OBJECTIVES: I. Define the tonotopocity of multichannel brain stem stimulation and use this information to better program the auditory brain stem implant for an individual.

II. Optimize device fitting by combining monopolar and bipolar stimulation, and individual psychoacoustic channels for each patient, to increase the number of usable information channels for each patient and reduce or eliminate undesirable side effects.

III. Evaluate performance and learning effects using optimized fitting procedures.

PROTOCOL OUTLINE: Patients undergo surgery to remove the first side or second side tumor, during which the multichannel auditory brain stem implant is implanted. Initial stimulation is conducted 4-6 weeks after surgery.

Patients are followed every 3 months for the first year, then annually thereafter.

Phase II Study of the Multichannel Auditory Brain Stem Implant for Deafness Following Surgery for Neurofibromatosis 2

Cochlear

Eye Diseases

Infections

Sclerosis

Multiple Sclerosis

Neuromyelitis Optica

Encephalomyelitis

Myelitis

Myelitis, Transverse

Demyelinating Diseases

Encephalomyelitis, Acute Disseminated

Diffuse Cerebral Sclerosis of Schilder

Tuberous Sclerosis

Demyelinating Autoimmune Diseases, CNS

Communicable Diseases

Central Nervous System Infections

Central Nervous System Diseases

Neuroinflammatory Diseases

Spinal Cord Diseases

Leukoencephalopathies

Brain Diseases

Post-Infectious Disorders

Chronic Disease

Disease Attributes

Neuritis

Optic Neuritis

Optic Nerve Diseases

Acute Graft Versus Host Disease

Neuromyelitis Optica Spectrum Disorder

Transverse Myelitis

Acute Disseminated Encephalomyelitis

Tumefactive Multiple Sclerosis

Tuberous Sclerosis Complex

Chronic Graft Versus Host Disease

Anti-Inflammatory Agents

Antineoplastic Agents

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Prednisolone

Methylprednisolone

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Plasma exchange

Brian G. Weinshenker

OBJECTIVES:

I. Evaluate the effectiveness of plasma exchange in the treatment of acute severe attacks of inflammatory demyelinating disease in patients who have failed intravenous steroid therapy.

PROTOCOL OUTLINE: This is a randomized, double-blind study. Patients are stratified by disease type; each stratum is randomized separately.

The first group of patients receives a true plasma exchange using continuous-flow centrifugation with serum albumin and crystalloid replacement every 2 days for a total of 7 exchanges.

The second group receives a sham plasma exchange with no centrifugation every 2 days for a total of 7 exchanges.

Patients cross to the alternate therapy if there is less than a moderate improvement by day 14. The treatment decision is based on a blinded neurologic assessment.

Concurrent corticosteroids and other immunosuppressants, and high-dose barbiturates are not allowed.

Patients are followed at 1 and 6 months after the last exchange.

Phase III Randomized, Double-Blind, Sham-Controlled Study of Plasma Exchange for Acute Severe Attacks of Inflammatory Demyelinating Disease Refractory to Intravenous Methylprednisolone

Mayo Clinic

National Institute of Neurological Disorders and Stroke (NINDS)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Randomized Study of 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome	NCT00004832	Lambert-Eaton M...	3,4-diaminopyri...	18 Years -	FDA Office of Orphan Products Development
Randomized Study of Intravenous Immunoglobulin in Patients With Mild or Moderate Myasthenia Gravis	NCT00004682	Myasthenia Grav...	immune globulin	15 Years -	FDA Office of Orphan Products Development
Phase II Study of the Multichannel Auditory Brain Stem Implant for Deafness Following Surgery for Neurofibromatosis 2	NCT00004437	Neurofibromatos...	Multichannel Au...	12 Years -	FDA Office of Orphan Products Development
Phase III Randomized, Double-Blind, Sham-Controlled Study of Plasma Exchange for Acute Severe Attacks of Inflammatory Demyelinating Disease Refractory to Intravenous Methylprednisolone	NCT00004645	Acute Dissemina... Devic's Syndrom... Marburg's Varia... Balo's Concentr... Acute Transvers...	Plasma exchange	18 Years - 60 Years	Office of Rare Diseases (ORD)

Spots Global Cancer Trial Database for neurologic and psychiatric disorders