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

Brief Title

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

Official Title

The Efficacy of High-Dose Intravenous Immunoglobulin Therapy in Patients With Stiff-Man Syndrome: A Double-Blind, Placebo-Controlled Trial

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.
    

Detailed Description

      Stiff-man Syndrome (SMS) is a chronic, disabling neurological disorder characterized by
      severe and painful axial and limb rigidity enhanced by anxiety, sudden motion or external
      stimuli. Although the cause of SMS is unknown, immunologic mechanisms have been implicated on
      the basis of circulating autoantibodies in the patient's serum and CSF, against GAD (glutamic
      acid decarboxylase), the enzyme involved in the synthesis of GABA (gamma aminobutyric acid).
      Uncontrolled studies have also shown that plasmapheresis, corticosteroids and high dose
      intravenous immunoglobulin (IVIg) are variably effective in improving the clinical symptoms
      of these patients. The purpose of the present study is to demonstrate in a double blind,
      placebo-control design, the efficacy of IVIg in patients with SMS. The effect of IVIg will be
      assessed with a series of objective measurements including muscle function, mobility and
      stiffness. Changes in the circulating anti-GAD antibodies will be also examined and their
      pathogenetic role in the cause of SMS will be determined. If IVIg proves effective, it will
      be a valuable tool in the treatment of these patients who are currently dependent on high
      doses of Valium (up to 60-100 mg daily), or steroids and experience significant side effects.
    

Study Phase

Phase 1

Study Type

Interventional




Condition

Muscle Rigidity

Intervention

IVIg


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

20

Start Date

April 1996

Completion Date

May 2002


Eligibility Criteria

        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.
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

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Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00001550

Organization ID

960062

Secondary IDs

96-N-0062


Study Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)


Study Sponsor

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Verification Date

May 2002