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

NCT ID: NCT03749096

Last Updated: 2019-06-13

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-12-07
• Study Completion Date: 2019-04-26

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.

Conditions

Stiff-Person Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Intravenous immunoglobulin

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).

Group Type: ACTIVE_COMPARATOR

Intravenous Immunoglobulins, Human

Intervention Type: DRUG

Immunoglobulins are fractionated blood products made from pooled human plasma.

Placebo

Group Type: PLACEBO_COMPARATOR

Placebos

Intervention Type: DRUG

Normal Saline

Interventions

Intravenous Immunoglobulins, Human

Immunoglobulins are fractionated blood products made from pooled human plasma.

Intervention Type: DRUG

Placebos

Normal Saline

Intervention Type: DRUG

Other Intervention Names

Normal Saline

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Grifols Biologicals, LLC

INDUSTRY

Sponsor Role: collaborator

Mayo Clinic

OTHER

Sponsor Role: lead

Responsible Party

Andrew McKeon

Prinicple Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Andrew McKeon, MD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Countries

United States

Other Identifiers

18-007041

Identifier Type: -

Identifier Source: org_study_id