Spinocerebellar AtaxiasPhase 1NCT02287064
An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias
A Phase 1 study testing Intravenous Immune Globulin (IVIG) in people with Spinocerebellar Ataxias, run by University of South Florida at 1 site in United States.
Trial summary
The purpose of this study is to learn how Intravenous Immune Globulin (IVIG) will affect Spinocerebellar Ataxia (SCA) symptoms and how it will affect motor and nervous system function in participants Subtypes of SCA to be examined will include SCA types 1, 2, 3, 6, 10 and 11. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Spinocerebellar Ataxias
- What is being tested?
- Intravenous Immune Globulin (IVIG)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2015to Dec 2016
- Phase
- Phase 1
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Outpatients with SCA types 1, 2, 3, 6, 10, or 11, diagnosed by a movement disorder specialist.
- Age 18 years to 80 years.
- Able to ambulate with or without assistance for 30 feet.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG do not reveal clinically significant abnormalities (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
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- Diagnosis of peripheral neuropathy. See exclusion criteria 3 for specific types of peripheral neuropathy to be excluded.
- Throughout the study, all possible efforts will be made to maintain subject levels of activity, exercise or physical therapy.
- Subject permission (informed consent).
Who cannot join
- Any unstable illness that in the investigator's opinion precludes participation in this study.
- Use of any investigational product within the past 30 days.
- Presence of diabetes (as determined by blood glucose labs within the past 6 months), nutritional deficiency causing neuropathy (vitamin B1, 3, 6, and 12 or vitamin E), injuries, autoimmune disorders (HIV, lupus, pediatric Guillain-Barre syndrome, neurosarcoidosis, monoclonal gammopathy), tumors, infections (leprosy), exposures to toxins (alcohol, arsenic, mercury), thyroid disease or hereditary causes (cerebral amyloid angiopathy) known to result in the presence of peripheral neuropathy.
- Dementia or other psychiatric illness that prevents the subject from giving informed consent (MMSE less than 25).
- Legal incapacity or limited legal capacity.
- Presence of severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (as evidenced by labs reported within the past 6 months).
- Clinically significantly abnormal white blood cell count, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
- Immunoglobulin A, Vitamin B (1, 3, 6, or 12), vitamin E or folate deficiencies (evidenced by screening lab evaluations).
Trial locations and contacts
1 site in 1 country.
Mary Freeman, LPN contact
United States1 site, 1 recruiting
- Recruiting
University of South Florida
Tampa, Florida, 33612
Mary Freeman, LPN, 813-974-5909
Tanya Aranca, BS, 813-974-5909
About this study
The purpose of this study is to learn how Intravenous Immune Globulin (IVIG) will affect Spinocerebellar Ataxia (SCA) symptoms and how it will affect motor and nervous system function in participants Subtypes of SCA to be examined will include SCA types 1, 2, 3, 6, 10 and 11.
- Conditions
- Spinocerebellar Ataxias
- Being tested
- Intravenous Immune Globulin (IVIG)
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous Immune Globulin (IVIG)Drug
IVIG will be infused over a course of five days in the form of GAMMAGARD LIQUID 10% solution, available from Baxter. For neurological and autoimmune diseases 2 grams per kilogram of body weight is implemented for three months over a five day course once a month.
There is very limited reliable dose ranging data for IVIG in the treatment of any condition, and most dosing has been empiric. In our experience, we have empirically observed a more potent immunomodulatory effect from "induction dose" IVIG (2 g/kg) continued each month, than with the "booster dose" maintenance dose of 1gm/kg. Though there is no category one evidence to support this practice, neither is there such evidence to refute it. Additionally, results from previous trials of IVIG in SCAs show this dosage to be relatively safe and effective at this rate of infusion
| Study group | Role | What they receive |
|---|---|---|
| Intravenous Immune Globulin (IVIG) | Experimental | Not described Receives: Drug: Intravenous Immune Globulin (IVIG) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in Scale for the Assessment and Rating Ataxia (SARA)
The primary outcomes will be the changes in the patient's SARA total score and frequency and severity of adverse events.
Measured: Will be assesed at abseline, day 14, day28 and day 56.
Secondary outcomes (3)
clinician and patient global impression of improvement (CGI and PGI)
Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.
Measured: Will be assesed at abseline, day 14, day28 and day 56.
Neurologic dysfunction as assessed by STAND scores
Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.
Measured: Will be assesed at abseline, day 14, day28 and day 56.
9-hole peg test
Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.
Measured: Will be assesed at abseline, day 14, day28 and day 56.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2014 (estimated)
- Study startApr 2015
- Primary completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar AtaxiasStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Responsible party
- Sponsor
- Investigators
- Theresa Zesiewicz, MD, Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02287064
- Sponsor's protocol ID
- SCA IVIG 2014
- Status last verified
- Jun 2016
- First submitted
- 5 Nov 2014
- Last updated
- 8 Jun 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias
- ClinicalTrials.gov ID
- NCT02287064
- Acronym / sponsor ID
- SCA IVIG 2014
- Conditions
- Spinocerebellar Ataxias
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Study start
- Apr 2015
- Primary completion
- Dec 2016 (estimated)
- First posted
- 10 Nov 2014 (estimated)
- 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.
The purpose of this study is to learn how Intravenous Immune Globulin (IVIG) will affect Spinocerebellar Ataxia (SCA) symptoms and how it will affect motor and nervous system function in participants Subtypes of SCA to be examined will include SCA types 1, 2, 3, 6, 10 and 11.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intravenous Immune Globulin (IVIG) | Experimental | Drug: Intravenous Immune Globulin (IVIG) |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Outpatients with SCA types 1, 2, 3, 6, 10, or 11, diagnosed by a movement disorder specialist.
- Age 18 years to 80 years.
- Able to ambulate with or without assistance for 30 feet.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG do not reveal clinically significant abnormalities (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
Key exclusion criteria (8 listed)
- Any unstable illness that in the investigator's opinion precludes participation in this study.
- Use of any investigational product within the past 30 days.
- Presence of diabetes (as determined by blood glucose labs within the past 6 months), nutritional deficiency causing neuropathy (vitamin B1, 3, 6, and 12 or vitamin E), injuries, autoimmune disorders (HIV, lupus, pediatric Guillain-Barre syndrome, neurosarcoidosis, monoclonal gammopathy), tumors, infections (leprosy), exposures to toxins (alcohol, arsenic, mercury), thyroid disease or hereditary causes (cerebral amyloid angiopathy) known to result in the presence of peripheral neuropathy.
- Dementia or other psychiatric illness that prevents the subject from giving informed consent (MMSE less than 25).
- Legal incapacity or limited legal capacity.
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 Immune Globulin (IVIG)IVIG will be infused over a course of five days in the form of GAMMAGARD LIQUID 10% solution, available from Baxter. For neurological and autoimmune diseases 2 grams per kilogram of body weight is implemented for three months over a five day course once a month. There is very limited reliable dose ranging data for IVIG in the treatment of any condition, and most dosing has been empiric. In our experience, we have empirically observed a more potent immunomodulatory effect from "induction dose" IVIG (2 g/kg) continued each month, than with the "booster dose" maintenance dose of 1gm/kg. Though there is no category one evidence to support this practice, neither is there such evidence to refute it. Additionally, results from previous trials of IVIG in SCAs show this dosage to be relatively safe and effective at this rate of infusion | Drug | Intravenous Immune Globulin (IVIG) |
Primary outcome
Changes in Scale for the Assessment and Rating Ataxia (SARA)
Time frame: Will be assesed at abseline, day 14, day28 and day 56.
The primary outcomes will be the changes in the patient's SARA total score and frequency and severity of adverse events.
3 secondary outcomes
clinician and patient global impression of improvement (CGI and PGI)
Time frame: Will be assesed at abseline, day 14, day28 and day 56.
Neurologic dysfunction as assessed by STAND scores
Time frame: Will be assesed at abseline, day 14, day28 and day 56.
9-hole peg test
Time frame: Will be assesed at abseline, day 14, day28 and day 56.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2016.
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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.
Mary Freeman, LPN contact
Theresa Zesiewicz, MD principal investigator
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intravenous Immune Globulin (IVIG) in people with Spinocerebellar Ataxias, run by University of South Florida 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 9 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 8 Jun 2016. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mary Freeman, LPN (813-974-5909), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in Scale for the Assessment and Rating Ataxia (SARA) (time frame: Will be assesed at abseline, day 14, day28 and day 56.). 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 8 Jun 2016.