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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.
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.
Not yet recruiting
Thomas Jefferson University
Published on BioPortfolio: 2019-02-10T19:56:21-0500
This is a randomized double-blind controlled trial of intravenous immunoglobulin (IVIg) for glycine receptor antibody positive (GlyRα1) antibody Stiff Person Syndrome (SPS) spectrum disor...
The purpose of the study is to acquire additional data on safety, tolerability and immunogenicity of HyQvia in pediatric (age two to
The purpose of this study is to assess the long-term safety, tolerability, and immunogenicity of the subcutaneous (SC) treatment with Immune Globulin Subcutaneous (IGSC) facilitated with r...
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatme...
Most immunodeficiencies are related to severe immunoglobulin deficiencies which require lifelong replacement therapy with immunoglobulin G (IgG) to reduce the incidence and severity of inf...
Stiff person syndrome (SPS) is usually characterized by truncal muscle rigidity and episodic painful spasms, but it sometimes appears with ocular symptoms called "stiff eyes". We recorded saccade move...
A 57-year-old man with a known case of stiff person syndrome (SPS) presented with an insidious-onset 3-month history of right groin pain and inability to bear weight on the right lower extremity. Radi...
A rare neuromuscular disorder with onset usually in late childhood or early adulthood, characterized by intermittent or continuous widespread involuntary muscle contractions; FASCICULATION; hyporeflexia; MUSCLE CRAMP; MUSCLE WEAKNESS; HYPERHIDROSIS; TACHYCARDIA; and MYOKYMIA. Involvement of pharyngeal or laryngeal muscles may interfere with speech and breathing. The continuous motor activity persists during sleep and general anesthesia (distinguishing this condition from STIFF-PERSON SYNDROME). Familial and acquired (primarily autoimmune) forms have been reported. (From Ann NY Acad Sci 1998 May 13;841:482-496; Adams et al., Principles of Neurology, 6th ed, p1491)
A condition characterized by persistent spasms (SPASM) involving multiple muscles, primarily in the lower limbs and trunk. The illness tends to occur in the fourth to sixth decade of life, presenting with intermittent spasms that become continuous. Minor sensory stimuli, such as noise and light touch, precipitate severe spasms. Spasms do not occur during sleep and only rarely involve cranial muscles. Respiration may become impaired in advanced cases. (Adams et al., Principles of Neurology, 6th ed, p1492; Neurology 1998 Jul;51(1):85-93)
Interference with the FREEDOM or PERSONAL AUTONOMY of another person, with justifications referring to the promotion of the person's good or the prevention of harm to the person. (from Cambridge Dictionary of Philosophy, 1995); more generally, not allowing a person to make decisions on his or her own behalf.
A person authorized to decide or act for another person, for example, a person having durable power of attorney.
An infection of the SMALL INTESTINE caused by the flagellated protozoan GIARDIA LAMBLIA. It is spread via contaminated food and water and by direct person-to-person contact.
An antibody is a protein produced by the body's immune system when it detects harmful substances, called antigens. Examples of antigens include microorganisms (such as bacteria, fungi, parasites, and viruses) and chemicals. Antibodies may be produc...