July 10, 2014
Institutional Review Board, Hospital for Special Surgery
The safety of our participants is our top priority. The trial/study is approved and periodically reviewed by the Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.
Before enrolling in a clinical trial or research study, the investigator will explain the purpose of the trial/study, its expected benefits, any possible risks or side effects, and what your role will be. If you want to join the trial/study, you must sign informed consent documents. You can leave a trial/study at any time without penalty.
For further information, see Understanding Clinical Trials/Research Studies.
Lindsay Kaplan
Nicole Kassenbaum
Lauren Landford
Dora K. Leung, MD
Erin Manning, MD
Janki Panchal
Leah Pilossoph, RN
Rahul Remanan
Mona Shabazi
Alexander Shtilbans, MD, PhD, MS
A total of 16 subjects will complete this study. Subjects that meet entry criteria will be randomized to receive 5 successive days of intravenous immunoglobulin (IVIG) in a dose of 2 grams per kg, or an equivalent volume of saline. The brand will be identical for all subjects. Time between screen and the first infusion will be up to 4 weeks. A second infusion will be administered approximately 6 weeks (+/- 1 week) after the end of the first infusion. This infusion will be a crossover of the first infusion, either to IVIG if they received saline initially, or to saline, if they received IVIG on first infusion. A return visit will occur approximately 2 (9-14 days) and 6 weeks after the first infusion and approximately 2 weeks (9-14 days) after the second infusion to assess clinical effect. Electrophysiological evaluations will be performed at the time of the clinical evaluation 9-14 days after each infusion.
Inclusion Criteria
1. Motor nerve conduction studies have demonstrated the presence of abnormal A-waves
2. Capable of providing informed consent and complying with trial procedures
3. Age 18 or older
4. Patients with predominantly lower motor neuron syndromes defined as weakness at least one extremity.
5. Able to tolerate extensive electrodiagnostic studies in three successive visits (screen, 2 weeks after each infusion)
6. IgA immunoglobulin levels are within normal limits
7. Has never received IVIG or has not received IVIG during the month immediately before screening
8. Absence of exclusion criteria
Exclusion Criteria
1. Motor nerve conduction studies show evidence of conduction block (loss of compound muscle potential amplitude exceeding 50% between two consecutive stimulation sites outside of common sites of compression)
2. Motor nerve conduction studies do not show severe (>50% of the lower limits of normal, LLN) segmental slowing
3. IgA immunoglobulin levels are lower than normal range for lab performing measurements
4. History of previous allergic reaction to intravenous immunoglobulin infusions
5. Exposure to any experimental agent within 30 days prior to screening
6. Women who are pregnant or planning to become pregnant
7. Women of childbearing potential not practicing contraception
8. Women who are breastfeeding
9. Enrollment in another research study within 30 days prior to screening
10. Alcoholism
11. Dementia (MMSE <22)*
12. Active congestive heart failure
13. Impaired kidney function, defined as creatinine levels of 2.5 x ULN
14. Impaired liver function, defined as AST or ALT of 3 X ULN
15. Patients with a known cause of peripheral neuropathy
Nicole Kassebaum, MS
646.797.8592
KassebaumN@hss.edu