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

Stiff-Person Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03829826
• Other study ID #: 18P.420
• Status: Not yet recruiting
• Start date: June 2019
• Est. completion date: May 2020

Study information

• Verified date: May 2019
• Source: Thomas Jefferson University
• Contact: Marinos C Dalakas, MD
• Phone: 2159557865
• Email: marinos.dalakas[@]jefferson.edu
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 22
• Est. completion date: May 2020
• Est. primary completion date: February 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Stiff-Person Syndrome
• Syndrome

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Thomas Jefferson University

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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	24 MONTHS
Secondary	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	24 MONTHS
Secondary	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.	24 MONTHS