Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome (SPS)

Brief Title

Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome (SPS)

Official Title

Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome

Brief 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. 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 Type

Observational


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.

Secondary Outcome

 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)

Condition

Stiff-Person Syndrome

Intervention

HyQvia

Study Arms / Comparison Groups

 Patients receiving IVIg
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

22

Start Date

June 2019

Completion Date

May 2020

Primary Completion Date

February 2020

Eligibility Criteria

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, 2159557865, [email protected]



Administrative Informations


NCT ID

NCT03829826

Organization ID

18P.420


Responsible Party

Sponsor

Study Sponsor

Thomas Jefferson University


Study Sponsor

, , 


Verification Date

May 2019