Intravenous Immunoglobulin (IVIg) for the Treatment of Stiff-Man Syndrome (SMS)

NCT00001550 | Completed Phase 1

• 2 other identifiers: 96006296-N-0062
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Stiff-man Syndrome (SMS) is a chronic, progressive disorder of the nervous system. It is associated with painful muscle spasms and rigidity involving muscles of the limbs, trunk, and neck. The cause of the disease is unknown, but researchers believe it may be a result of an autoimmune process. Patients with Stiff-man Syndrome may produce antibodies that attack enzymes required for the normal function of the nervous system. Steroids, plasmapheresis, and intravenous immunoglobulin (IVIg) have been given to relieve some of the symptoms of Stiff-man Syndrome. However, none of these therapies have proven to be significantly effective. This study will attempt to determine the effectiveness of intravenous immunoglobulin (IVIg) for the treatment of Stiff-mann Syndrome. Patients participating in this study will be divided into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement in muscle function, mobility, and stiffness.

Conditions

Muscle Rigidity; Spasm; Stiff Man Syndrome

Keywords

Immunotherapy; Muscle Stiffness; Rigidity; Anti-GAD Antibodies; Episodic Spasms; Intravenous High-Dose Immunoglobulin; Stiff-man Syndrome

Interventions

IVIg DRUG

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

Men and non-pregnant women, between 18-75 years of age, who meet a defined criteria for the diagnosis of Stiff-man syndrome (SMS) will be screened as inpatients or in the outpatient clinic. If the diagnosis is confirmed, the patients will be enrolled into the protocol, provided their disease remains symptomatic and poorly responsive to benzodiazepines. Only patients with anti-GAD antibodies will be included. Patients who have not received IVIg in the past 6 months may be included. No pregnant or nursing women (confirmed by a pregnancy screening test). No critically ill patients, such as those with severe cardiomyopathy, and respiratory insufficiency and severely incapacitated patients that require help for self care. No patients with severe renal or hepatic disease, COPD or severe coronary artery disease. No patients with serum IgA level less than 11 mg/dl.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS): lead

Study Sites (1)

National Institute of Neurological Disorders and Stroke (NINDS)

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Solimena M, Folli F, Denis-Donini S, Comi GC, Pozza G, De Camilli P, Vicari AM. Autoantibodies to glutamic acid decarboxylase in a patient with stiff-man syndrome, epilepsy, and type I diabetes mellitus. N Engl J Med. 1988 Apr 21;318(16):1012-20. doi: 10.1056/NEJM198804213181602.

  PMID: 3281011 BACKGROUND

• Solimena M, Folli F, Aparisi R, Pozza G, De Camilli P. Autoantibodies to GABA-ergic neurons and pancreatic beta cells in stiff-man syndrome. N Engl J Med. 1990 May 31;322(22):1555-60. doi: 10.1056/NEJM199005313222202.

  PMID: 2135382 BACKGROUND

• Grimaldi LM, Martino G, Braghi S, Quattrini A, Furlan R, Bosi E, Comi G. Heterogeneity of autoantibodies in stiff-man syndrome. Ann Neurol. 1993 Jul;34(1):57-64. doi: 10.1002/ana.410340111.

  PMID: 8517681 BACKGROUND

MeSH Terms

Conditions

Muscle Rigidity; Spasm; Stiff-Person Syndrome

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Autoimmune Diseases of the Nervous System; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: November 3, 1999
• First Posted: November 4, 1999
• Study Start: April 1, 1996
• Study Completion: May 1, 2002
• Last Updated: July 14, 2006

Record last verified: 2002-05

Locations

US (1)