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

Phase 3

Withdrawn

• Conditions: Stiff-Person Syndrome
• Interventions: Drug: Intravenous Immunoglobulins, Human; Drug: Placebos

• Registration Number: NCT03749096
• Lead Sponsor: Mayo Clinic

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 Timeline

• Study First Submitted: 2018-11-16
• Study First Submitted QC: 2018-11-19
• Study First Posted: 2018-11-21
• Study Start: 2018-12-07
• Primary Completion: 2019-04-26
• Study Completion: 2019-04-26
• Status Verified: 2019-06
• Last Update Submitted: 2019-06-11
• Last Update Posted: 2019-06-13

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Intravenous immunoglobulin	Intravenous Immunoglobulins, Human	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).
Placebo	Placebos	-

Primary Outcome Measures

Name	Time	Method
Change in Distribution-of-stiffness index	Screen, Week 11	Scores on this index range from 0 to 6 and reflect the extent of stiffness. with One point being given for stiffness in each of the following areas: lower trunk, upper trunk, legs, arms, face, and abdomen. Lower scores indicate less stiffness.
Change in Heightened-sensitivity scale	Screen, Week 11	Scores range from 1 to 7 based on response; one point being given for each source of or type of spasm, as follows: unexpected noises, visual stimuli, somatosensory stimuli, voluntary activities, emotional upset or stress, no specific stimuli, and nocturnal spasms. Lower scores indicate less frequent spasms.

Trial Locations

Locations (1)

Mayo Clinic in Rochester; 🇺🇸 Rochester, Minnesota, United States