Randomized Placebo Controlled Trial of IVIg in Glycine Receptor Antibody Positive Stiff-person Syndrome

Brief Title

Randomized Placebo Controlled Trial of IVIg in Glycine Receptor Antibody Positive Stiff-person Syndrome

Official Title

IVIg in Glycine Receptor Antibody Positive Stiff-person Syndrome (SPS) Spectrum Disorders.

Brief Summary

      This is a randomized double-blind controlled trial of intravenous immunoglobulin (IVIg) for
      glycine receptor antibody positive (GlyRα1) antibody Stiff Person Syndrome (SPS) spectrum
      disorders. Adult patients will be enrolled over the course of 36 months. Study duration per
      patient will be 11 weeks. Total study duration will be 39 months. All treatment and study
      visits will occur at Mayo Clinic in Rochester, MN.
    

Detailed Description

      The purpose of this study is to determine whether intravenous immunoglobulin (IVIG) treatment
      reduces stiffness and spasms in patients with SPS spectrum disorders compared to patients who
      do not receive any treatment. In this study, investigators want to compare the effects, good
      and/or bad, of IVIG with placebo on participants with SPS to find out which is better. This
      is a "randomized, double-blind" study. Which treatment participants will receive is randomly
      determined. Participants have an equal chance of receiving IVIG or the placebo.

      SPS spectrum cohort will include any from the SPS spectrum patient (stiff-limbs, stiff-trunk,
      stiff-limbs and trunk, and PERM). Study visits will be considered part of clinical care and
      will occur at: enrollment (will coincide with screening visit), and 11 weeks. IVIg/placebo
      (saline) treatments will be administered on 2 consecutive days in week 1, week 5, week 9 in
      all patients.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Change in Distribution-of-stiffness index


Condition

Stiff-Person Syndrome

Intervention

Intravenous Immunoglobulins, Human

Study Arms / Comparison Groups

 Intravenous immunoglobulin
Description:  IVIg dose will be 2g/kg ideal body weight every 4 weeks (in 2 divided doses on consecutive days) for 12 weeks (3 cycles total).

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

0

Start Date

December 7, 2018

Completion Date

April 26, 2019

Primary Completion Date

April 26, 2019

Eligibility Criteria

        Inclusion Criteria

          -  Patient must be 18 years of age or older

          -  Must have symptoms of SPS for less than 3 years

          -  If taking corticosteroids, the patients must be on a stabile dose for 30 days prior to
             enrolment

          -  Patients will have a diagnosis of SPS spectrum disorder based on both of clinical and
             serological status

        Exclusion Criteria

          -  Patients on immune suppressants initiated/dose increased in the prior 6 months

          -  History of thrombotic episodes within the 2 years prior to enrollment

          -  Known allergic or other severe reactions to blood products including intolerability to
             previous IVIG

          -  Previous adequate trial of IVIG as determined by the Principal Investigator

          -  IgA deficiency

          -  Reproductive status:

               -  Women who are pregnant, breastfeeding

               -  Women and men of childbearing potential who are unwilling or unable to use an
                  acceptable method of birth control to avoid pregnancy for the entire study
                  period, as evaluated by the investigator.

          -  Any surgical procedure within 4 weeks prior to baseline.

          -  Evidence of serious uncontrolled concomitant diseases that may preclude patient
             participation; Other nervous system disease, cardiovascular disease,
             hematologic/hematopoiesis disease, respiratory disease, muscular disease, endocrine
             disease, renal/urologic disease, digestive system disease, congenital or acquired
             severe immunodeficiency

          -  Known active infection within 4 weeks prior to baseline.

          -  Evidence of chronic active hepatitis B or C.

          -  Active ischemic heart disease in the past year prior to baseline.

          -  Patients should not have severe renal or hepatic disease

          -  Severe hypertension
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Andrew McKeon, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03749096

Organization ID

18-007041


Responsible Party

Principal Investigator

Study Sponsor

Mayo Clinic

Collaborators

 Grifols Biologicals, LLC

Study Sponsor

Andrew McKeon, MD, Principal Investigator, Mayo Clinic


Verification Date

June 2019