Stiff-Person SyndromeNCT03829826
Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome (SPS)
An observational study of Stiff-Person Syndrome, run by Thomas Jefferson University.
Trial summary
This is a pilot, proof-of concept investigator-initiated trial planned for 22 patients with the diagnosis of Stiff Person Syndrome (SPS). The study will compare efficacy of treatment using subcutaneous immunoglobulin therapy (SCIg) compared to intravenous immunoglobulin (IVIg) therapy. 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?
- HyQvia
- Type of treatment
- Injection or infusion
- How many people
- 22 participants
- Where
- No sites listed yet
- Study dates
- Jun 2019to May 2020
- Phase
- Observational
- 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
Study population: Yes No Individuals with impaired decision-making capacity X Women of reproductive potential X Pregnant women/fetuses/neonates X Men of reproductive potential X Minorities X Prisoners X Economically or educationally disadvantaged persons X Students/employees X
Who may be able to join
- Men or women aged >18 years
- Diagnosis of SPS based on standard criteria
- IVIg Group: Receiving the equivalent of 1-2 g/kg IVIg every 4 weeks with dependence* on IVIg to maintain clinical response *Dependence is clinically determined either by symptomatic worsening of condition at the end of the inter-dose interval or by worsening after dose reduction or discontinuation within the previous 3 months.
- IVIg-Naïve Group: Patients with symptomatic SPS and never treated with IVIg (IVIg-naïve group), poorly controlled with standard therapy
Who cannot join
- Pregnancy, planned pregnancy, breast-feeding or unwillingness to practice contraception
- Severe concurrent medical conditions, which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
- Initiation of immunomodulatory treatment other than IVIg in the past 3 months
- Participation in a trial of an investigational medicinal product in the past 12 weeks
- Presence of any medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome
This is a pilot, proof-of concept investigator-initiated trial planned for 22 patients with the diagnosis of Stiff Person Syndrome (SPS). The study will compare efficacy of treatment using subcutaneous immunoglobulin therapy (SCIg) compared to intravenous immunoglobulin (IVIg) therapy. The majority of IVIg naïve subjects (those not already receiving IVIg) are typically managed with non-immunotherapy mostly Gamma Aminobutyric Acid (GABA) -enhancing drugs such as Baclofen or Diazepam.
Read the detailed description
Study Design:This is a proof of concept observational prospective, open label, study on the safety, efficacy and convenience of treatment with SCIg study of 22 patients at Thomas Jefferson University Hospital. Two cohorts of patients within the total of 22 will be included; half of them (11 patients) currently receiving and responding to IVIg and the other half starting de novo on SCIg. Patients diagnosed with SPS according to defined sets of symptoms will be eligible to enroll.
The primary clinical outcome will be based on clinical efficacy measures, as used before for the IVIg trial, based on changes in the Stiffness Index and Heightened Sensitivity scores, using the validated scales that the investigators have had previously utilized and validated (Dalakas et al 2001; see attached at the end of the protocol). These same measurements will be applied while on IVIg (weeks 0, 4, 8, 12) and will be compared to the measurements obtained during SCIg (weeks 16, 20, 24, 28). The secondary outcome will be Quality of Life (QoL) responses and patient preference for each treatment.
- Conditions
- Stiff-Person Syndrome
- Being tested
- HyQvia
- 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.
HyQviaBiological
Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase
| Study group | Role | What they receive |
|---|---|---|
| Patients receiving IVIg | Patients are currently receiving IVIg regularly for at least every 6 weeks and exhibit a favorable response will be recruited into the study (11 patients). They will be observed for 12 weeks under their existing IVIg regimen and will undergo measurements of their impairment using the previously validated Stiffness and Sensitivity scales and quality of life questionnaire (QoL) at weeks 0, 4, 8, 12. At week 12, prior to the first SCIg infusion, blood will be drawn for humoral (immunological) studies. One week following the last dose of IVIg (at week 13), the participants will be started on SCIg at a total dose equivalent to the monthly dose of IVIg they have been receiving. Receives: Biological: HyQvia | |
| de novo SCIg patients/IVIg-Naive Group | This other arm of the trial will include 11 patients naïve to IVIg who do not receive other immunotherapies while being symptomatic. These patients after a 12-week observation period will start directly on SCIg drug (HYQVIA), following the same schedule as described above for the previous group. Receives: Biological: HyQvia |
What is being measured
The primary outcome is the main question the study is designed to answer.
A >50% change from baseline on the Stiffness Index scores ( scale from 0-6; each item adds one) after 12 weeks of treatment.
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
Measured: 24 MONTHS
A >50% change from baseline on the Heightened Sensitivity scores (scales from 1-7, each item adds one) after 12 weeks of treatment.
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
Measured: 24 months
Secondary outcomes (3)
A meaningful change on Quality of Life (QoL) measures after 12 weeks of SCIg based on 6 sets of QoL Questionnaires (mobility, self-care, usual activities, pain, anxiety/depression, health state)
Proving that SCIg affects Quality of life, as IVIg does, will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
Measured: 24 MONTHS
If SCIg reduces the anti-GAD antibody titers measured in the patient's blood samples by more than 30%
An effect on the circulating anti-Glutamic Acid Decarboxylase (GAD) antibodies will elucidate the mechanism of action of SCIg and any correlation between efficacy and antibody titers
Measured: 24 MONTHS
If >50% of patients prefer either SCIg or IVIg after 12 weeks of treatment based on a YES/NO questionnaire .
Proving patients' preference is important because SCIg is self-administered at home and more convenient, without the need for hospitalizations.
Measured: 24 MONTHS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2019
- Study startJun 2019 (estimated)
- Primary completionFeb 2020 (estimated)
- Study completionMay 2020 (estimated)
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 Thomas Jefferson UniversityThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 22 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03829826
- Sponsor's protocol ID
- 18P.420
- Status last verified
- May 2019
- First submitted
- 7 Jan 2019
- Last updated
- 14 May 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome
- ClinicalTrials.gov ID
- NCT03829826
- Acronym / sponsor ID
- 18P.420
- Conditions
- Stiff-Person Syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 22 participants (estimated)
- Lead sponsor
- Thomas Jefferson UniversityUniversity, hospital or other
- Study start
- Jun 2019 (estimated)
- Primary completion
- Feb 2020 (estimated)
- Study completion
- May 2020 (estimated)
- First posted
- 4 Feb 2019 (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 pilot, proof-of concept investigator-initiated trial planned for 22 patients with the diagnosis of Stiff Person Syndrome (SPS). The study will compare efficacy of treatment using subcutaneous immunoglobulin therapy (SCIg) compared to intravenous immunoglobulin (IVIg) therapy. The majority of IVIg naïve subjects (those not already receiving IVIg) are typically managed with non-immunotherapy mostly Gamma Aminobutyric Acid (GABA) -enhancing drugs such as Baclofen or Diazepam.
Detailed description
Study Design:This is a proof of concept observational prospective, open label, study on the safety, efficacy and convenience of treatment with SCIg study of 22 patients at Thomas Jefferson University Hospital. Two cohorts of patients within the total of 22 will be included; half of them (11 patients) currently receiving and responding to IVIg and the other half starting de novo on SCIg. Patients diagnosed with SPS according to defined sets of symptoms will be eligible to enroll.
The primary clinical outcome will be based on clinical efficacy measures, as used before for the IVIg trial, based on changes in the Stiffness Index and Heightened Sensitivity scores, using the validated scales that the investigators have had previously utilized and validated (Dalakas et al 2001; see attached at the end of the protocol). These same measurements will be applied while on IVIg (weeks 0, 4, 8, 12) and will be compared to the measurements obtained during SCIg (weeks 16, 20, 24, 28). The secondary outcome will be Quality of Life (QoL) responses and patient preference for each treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients receiving IVIg | – | Biological: HyQviaPatients are currently receiving IVIg regularly for at least every 6 weeks and exhibit a favorable response will be recruited into the study (11 patients). They will be observed for 12 weeks under their existing IVIg regimen and will undergo measurements of their impairment using the previously validated Stiffness and Sensitivity scales and quality of life questionnaire (QoL) at weeks 0, 4, 8, 12. At week 12, prior to the first SCIg infusion, blood will be drawn for humoral (immunological) studies. One week following the last dose of IVIg (at week 13), the participants will be started on SCIg at a total dose equivalent to the monthly dose of IVIg they have been receiving. |
| de novo SCIg patients/IVIg-Naive Group | – | Biological: HyQviaThis other arm of the trial will include 11 patients naïve to IVIg who do not receive other immunotherapies while being symptomatic. These patients after a 12-week observation period will start directly on SCIg drug (HYQVIA), following the same schedule as described above for the previous group. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Yes No Individuals with impaired decision-making capacity X Women of reproductive potential X Pregnant women/fetuses/neonates X Men of reproductive potential X Minorities X Prisoners X Economically or educationally disadvantaged persons X Students/employees X
Key inclusion criteria (4 listed)
- Men or women aged >18 years
- Diagnosis of SPS based on standard criteria
- IVIg Group: Receiving the equivalent of 1-2 g/kg IVIg every 4 weeks with dependence* on IVIg to maintain clinical response *Dependence is clinically determined either by symptomatic worsening of condition at the end of the inter-dose interval or by worsening after dose reduction or discontinuation within the previous 3 months.
- IVIg-Naïve Group: Patients with symptomatic SPS and never treated with IVIg (IVIg-naïve group), poorly controlled with standard therapy
Key exclusion criteria (5 listed)
- Pregnancy, planned pregnancy, breast-feeding or unwillingness to practice contraception
- Severe concurrent medical conditions, which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
- Initiation of immunomodulatory treatment other than IVIg in the past 3 months
- Participation in a trial of an investigational medicinal product in the past 12 weeks
- Presence of any medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study.
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 |
|---|---|---|
| HyQviaImmune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase | Biological | Patients receiving IVIg, de novo SCIg patients/IVIg-Naive Group |
Primary outcome
A >50% change from baseline on the Stiffness Index scores ( scale from 0-6; each item adds one) after 12 weeks of treatment.
Time frame: 24 MONTHS
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
A >50% change from baseline on the Heightened Sensitivity scores (scales from 1-7, each item adds one) after 12 weeks of treatment.
Time frame: 24 months
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
3 secondary outcomes
A meaningful change on Quality of Life (QoL) measures after 12 weeks of SCIg based on 6 sets of QoL Questionnaires (mobility, self-care, usual activities, pain, anxiety/depression, health state)
Time frame: 24 MONTHS
If SCIg reduces the anti-GAD antibody titers measured in the patient's blood samples by more than 30%
Time frame: 24 MONTHS
If >50% of patients prefer either SCIg or IVIg after 12 weeks of treatment based on a YES/NO questionnaire .
Time frame: 24 MONTHS
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 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.
Questions about this trial
What is this clinical trial studying?
An observational study of Stiff-Person Syndrome, run by Thomas Jefferson University.
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 5 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 14 May 2019. Status can change between updates, so confirm with the study contact before making plans.
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.
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 Marinos C Dalakas, MD (2159557865, marinos.dalakas@jefferson.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
A >50% change from baseline on the Stiffness Index scores ( scale from 0-6; each item adds one) after 12 weeks of treatment. (time frame: 24 MONTHS). 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 14 May 2019.