Stiff-Person SyndromePhase 3NCT03749096
Randomized Placebo Controlled Trial of IVIg in Glycine Receptor Antibody Positive Stiff-person Syndrome
A Phase 3 study testing Intravenous Immunoglobulins, Human in people with Stiff-Person Syndrome, run by Mayo Clinic at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stiff-Person Syndrome
- What is being tested?
- Intravenous Immunoglobulins, Human
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Dec 2018to 26 Apr 2019
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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:
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: IVIg in Glycine Receptor Antibody Positive Stiff-person Syndrome (SPS) Spectrum Disorders.
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.
Read the 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
- Being tested
- Intravenous Immunoglobulins, Human, Placebos
- Keywords
- IVIG, stiff-person syndrome, SPS, glycine receptor antibody, intravenous immunoglobulin, GlyRα1
- Medical subject headings
- Stiff-Person Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous Immunoglobulins, HumanDrug
Immunoglobulins are fractionated blood products made from pooled human plasma.
PlacebosDrug
Also called Normal Saline
Normal Saline
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: Intravenous Immunoglobulins, Human |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Screen, Week 11
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2018
- Study start7 Dec 2018
- Primary completion26 Apr 2019
- Study completion26 Apr 2019
Why it stopped: Recruitment did not meet sponsors timeline
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stiff-Person SyndromeStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Grifols Biologicals, LLC
- Responsible party
- Principal investigator, Andrew McKeon, Mayo Clinic
- Investigators
- Andrew McKeon, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03749096
- Sponsor's protocol ID
- 18-007041
- Status last verified
- Jun 2019
- First submitted
- 16 Nov 2018
- Last updated
- 13 Jun 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IVIg in Glycine Receptor Antibody Positive Stiff-person Syndrome (SPS) Spectrum Disorders.
- ClinicalTrials.gov ID
- NCT03749096
- Acronym / sponsor ID
- 18-007041
- Conditions
- Stiff-Person Syndrome
- Keywords
- IVIG, stiff-person syndrome, SPS, glycine receptor antibody, intravenous immunoglobulin, GlyRα1
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Recruitment did not meet sponsors timeline
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- Grifols Biologicals, LLC
- Study start
- 7 Dec 2018 (actual)
- Primary completion
- 26 Apr 2019 (actual)
- Study completion
- 26 Apr 2019 (actual)
- First posted
- 21 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intravenous immunoglobulin | Active comparator | Drug: Intravenous Immunoglobulins, HumanIVIg 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 | Placebo comparator | Drug: Placebos |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravenous Immunoglobulins, HumanImmunoglobulins are fractionated blood products made from pooled human plasma. | Drug | Intravenous immunoglobulin |
| PlacebosAlso: Normal SalineNormal Saline | Drug | Placebo |
Primary outcome
Change in Distribution-of-stiffness index
Time frame: 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
Time frame: 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.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew McKeon, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Intravenous Immunoglobulins, Human in people with Stiff-Person Syndrome, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 13 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Distribution-of-stiffness index (time frame: Screen, Week 11). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2019.