Alemtuzumab in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)
The objectives of this study are to determine the safety, tolerability and preliminary efficacy of alemtuzumab for infusion for the treatment of CIDP. Eligible subjects will be treated with alemtuzumab at the beginning of the study and then followed for three years. During the three year period, subjects will under go monthly safety evaluations consisting of blood and urine testing, symptom surveys and examination. Detailed neurological testing including nerve conduction testing, Rasch-built Overall Disability Scale (CIDP/RODS) and Overall Neuropathy Limitations Scale (ONLS) assessments will be performed every six months for three years.
The study will also investigate and compare the responsiveness of the outcome measures being used.
Chronic Inflammatory Demyelinating Neuropathy
Drug: Alemtuzumab infusion
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open-Label Trial of Alemtuzumab in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)|
- Change from baseline in disability at 36 months measured by GBS/CIDP Rasch-built Overall Disability Scale (GBS/CIDP-RODS). [ Time Frame: Every six months up to 36 months ] [ Designated as safety issue: No ]The RODS CIDP scale is a validated measure of disability in CIDP/GBS.
- Change from baseline in disability measured with Overall Neuropathy Limitations Scale (ONLS) at 36 months. [ Time Frame: Every six months up to 36 months ] [ Designated as safety issue: No ]ONLS is a validated measure of neurological disfunction.
- Questionnaire survey [ Time Frame: Monthly up to 36 months ] [ Designated as safety issue: Yes ]At monthly intervals, participants will undergo routine blood monitoring and complete a questionnaire survey. All physicians, study staff and participants will be given ITP and Goodpasture's disease education.
|Study Start Date:||November 2012|
|Estimated Study Completion Date:||December 2016|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
Open label study of alemtuzumab
|Drug: Alemtuzumab infusion|
This is an open-label multi-center trial of alemtuzumab in the treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP). The study will have 4 phases.
Approximately 16 eligible participants will receive at least one cycle of alemtuzumab (5 days of drug infusion). Additional cycles of alemtuzumab (3 days of drug infusion) may be provided at the discretion of the participants' treating physician such as if clinical worsening occurs.
Phase 1: Screening Participants meeting the inclusion and exclusion criteria and having signed the informed consent document will enter the screening phase and undergo baseline evaluations.
Phase 2: Drug Infusion Participants will receive alemtuzumab by infusion using a standardized protocol. Participants will be maintained on their prior CIDP therapy during the drug infusions and then followed at regular intervals.
Phase 3. Alteration of CIDP therapy CIDP therapy may be altered at the discretion of the treating physician - either taper, discontinuation, or increase of current or additional medications. For those participants on chronic corticosteroid therapy, following pulse IV methylprednisolone and alemtuzumab cycle, corticosteroids will be tapered as rapidly and as far as possible according to according to the investigator's discretion. While the aim will be to discontinue corticosteroids, it is recognized that some participants will have a suppressed pituitary-adrenal axis and complete discontinuation may not be possible.
Phase 4: Extended Follow-up Each participant will be followed per protocol for a minimum of 36 months. All participants will undergo safety assessments and monitoring for at least 36 months after the last cycle of alemtuzumab. Additional cycles of alemtuzumab may be provided at the discretion of the participants' treating physician with at least a 12 month interval between cycles.
NUMBER OF PARTICIPANTS: Approximately 16 participants will join this study.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01757574
|Contact: Michael Polydefkis, MD MHSfirstname.lastname@example.org|
|Contact: Kathy Burks, CRNPemail@example.com|
|United States, Maryland|
|The Johns Hopkins Hospital||Recruiting|
|Baltimore, Maryland, United States, 21231|
|Contact: Michael Polydefkis, MD 410-502-2909 firstname.lastname@example.org|
|Contact: David Cornblath, MD 410-955-2229|
|Principal Investigator: Michael Polydefkis, MD MHS|
|Sub-Investigator: David Cornblath, MD|