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

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ClinicalTrials.gov Identifier: NCT03829826
Recruitment Status : Not yet recruiting
First Posted : February 4, 2019
Last Update Posted : May 14, 2019
Sponsor:
Information provided by (Responsible Party):
Thomas Jefferson University

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.

Condition or disease Intervention/treatment
Stiff-Person Syndrome Biological: HyQvia

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.


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Study Type : Observational
Estimated Enrollment : 22 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome
Estimated Study Start Date : June 2019
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : May 2020

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
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.
Biological: HyQvia
Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase

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.
Biological: HyQvia
Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase




Primary Outcome Measures :
  1. 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

  2. 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


Secondary Outcome Measures :
  1. 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 ]
    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

  2. If SCIg reduces the anti-GAD antibody titers measured in the patient's blood samples by more than 30% [ Time Frame: 24 MONTHS ]
    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

  3. If >50% of patients prefer either SCIg or IVIg after 12 weeks of treatment based on a YES/NO questionnaire . [ Time Frame: 24 MONTHS ]
    Proving patients' preference is important because SCIg is self-administered at home and more convenient, without the need for hospitalizations.


Biospecimen Retention:   Samples Without DNA
Blood drawn for humoral (immunological) studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03829826


Contacts
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Contact: Marinos C Dalakas, MD 2159557865 marinos.dalakas@jefferson.edu
Contact: Vikram Puvenna 2159554672 vikram.puvenna@jefferson.edu

Sponsors and Collaborators
Thomas Jefferson University

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Responsible Party: Thomas Jefferson University
ClinicalTrials.gov Identifier: NCT03829826     History of Changes
Other Study ID Numbers: 18P.420
First Posted: February 4, 2019    Key Record Dates
Last Update Posted: May 14, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Stiff-Person Syndrome
Syndrome
Disease
Pathologic Processes
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases