Back to results

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

Primary Purpose

Stiff-Person Syndrome

Status

Withdrawn

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

Intravenous Immunoglobulins, Human

Placebos

About this trial

This is an interventional treatment trial for Stiff-Person Syndrome focused on measuring IVIG, stiff-person syndrome, SPS, glycine receptor antibody, intravenous immunoglobulin, GlyRα1

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Mayo Clinic in Rochester

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Intravenous immunoglobulin

Placebo

Arm 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).

Outcomes

Primary Outcome Measures

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.

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.

Secondary Outcome Measures

Full Information

NCT ID

NCT03749096

First Posted

November 16, 2018

Last Updated

June 11, 2019

Sponsor

Mayo Clinic

Collaborators

Grifols Biologicals, LLC

1. Study Identification

Unique Protocol Identification Number

NCT03749096

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.

Study Type

Interventional

2. Study Status

Record Verification Date

June 2019

Overall Recruitment Status

Withdrawn

Why Stopped

Recruitment did not meet sponsors timeline

Study Start Date

December 7, 2018 (Actual)

Primary Completion Date

April 26, 2019 (Actual)

Study Completion Date

April 26, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Mayo Clinic

Collaborators

Grifols Biologicals, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Stiff-Person Syndrome

Keywords

IVIG, stiff-person syndrome, SPS, glycine receptor antibody, intravenous immunoglobulin, GlyRα1

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Masking Description

Randomized double-blind

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Intravenous immunoglobulin

Arm Type

Active Comparator

Arm 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).

Arm Title

Placebo

Arm Type

Placebo Comparator

Intervention Type

Drug

Intervention Name(s)

Intravenous Immunoglobulins, Human

Intervention Description

Immunoglobulins are fractionated blood products made from pooled human plasma.

Intervention Type

Drug

Intervention Name(s)

Placebos

Other Intervention Name(s)

Normal Saline

Intervention Description

Normal Saline

Primary Outcome Measure Information:

Title

Change in Distribution-of-stiffness index

Description

Time Frame

Screen, Week 11

Title

Change in Heightened-sensitivity scale

Description

Time Frame

Screen, Week 11

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Andrew McKeon, MD

Organizational Affiliation

Mayo Clinic

Official's Role

Principal Investigator

Facility Information:

Facility Name

Mayo Clinic in Rochester

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs