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

Stiff-Person Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03749096
• Other study ID #: 18-007041
• Status: Withdrawn
• Phase: Phase 3
• Start date: December 7, 2018
• Est. completion date: April 26, 2019

Study information

• Verified date: June 2019
• Source: Mayo Clinic
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: April 26, 2019
• Est. primary completion date: April 26, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Stiff-Person Syndrome
• Syndrome

Intervention

Drug:
• Intravenous Immunoglobulins, Human
Immunoglobulins are fractionated blood products made from pooled human plasma.
• Placebos
Normal Saline

Locations

Country	Name	City	State
United States	Mayo Clinic in Rochester	Rochester	Minnesota

Sponsors (2)

Lead Sponsor	Collaborator
Mayo Clinic	Grifols Biologicals, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Distribution-of-stiffness index	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.	Screen, Week 11
Primary	Change in Heightened-sensitivity scale	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.	Screen, Week 11