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

NCT03749096 | Withdrawn Phase 3 FDA Drug

• 1 other identifier: 18-007041
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Stiff-Person Syndrome

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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

• 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

Study Arms (2)

Intravenous immunoglobulin

ACTIVE COMPARATOR

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).

Drug: Intravenous Immunoglobulins, Human

Placebo

PLACEBO COMPARATOR

Drug: Placebos

Interventions

Intravenous Immunoglobulins, Human DRUG

Immunoglobulins are fractionated blood products made from pooled human plasma.

Intravenous immunoglobulin

Placebos DRUG

Normal Saline

Also known as: Normal Saline

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mayo Clinic: lead
• Grifols Biologicals, LLC: collaborator

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Stiff-Person Syndrome

Interventions

Immunoglobulins, Intravenous; Saline Solution

Condition Hierarchy (Ancestors)

Autoimmune Diseases of the Nervous System; Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Andrew McKeon, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Randomized double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prinicple Investigator

Study Record Dates

• First Submitted: November 16, 2018
• First Posted: November 21, 2018
• Study Start: December 7, 2018
• Primary Completion: April 26, 2019
• Study Completion: April 26, 2019
• Last Updated: June 13, 2019

Record last verified: 2019-06

Locations

US (1)