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

NCT03829826 | Unknown DMC FDA Drug

• 1 other identifier: 18P.420
• Study Type: observational
• Target: 22
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Stiff-Person Syndrome

Outcome Measures

Primary Outcomes (2)

• 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

  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

  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)

  24 MONTHS

• If SCIg reduces the anti-GAD antibody titers measured in the patient's blood samples by more than 30%

  24 MONTHS

• If >50% of patients prefer either SCIg or IVIg after 12 weeks of treatment based on a YES/NO questionnaire .

  24 MONTHS

Study Arms (2)

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

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

Interventions

HyQvia BIOLOGICAL

Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase

Patients receiving IVIg; de novo SCIg patients/IVIg-Naive Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Thomas Jefferson University: lead

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood drawn for humoral (immunological) studies.

MeSH Terms

Conditions

Stiff-Person Syndrome

Condition Hierarchy (Ancestors)

Autoimmune Diseases of the Nervous System; Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Marinos C Dalakas, MD

CONTACT

2159557865; marinos.dalakas@jefferson.edu

Vikram Puvenna

CONTACT

2159554672; vikram.puvenna@jefferson.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2019
• First Posted: February 4, 2019
• Study Start: June 1, 2019
• Primary Completion: February 1, 2020
• Study Completion: May 1, 2020
• Last Updated: May 14, 2019

Record last verified: 2019-05