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An Open-Label Trial of Alemtuzumab in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)

Phase 4
18 Years
Open (Enrolling)
Chronic Inflammatory Demyelinating Neuropathy

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Trial Information

An Open-Label Trial of Alemtuzumab in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)

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

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.

Inclusion Criteria:

1. Signed informed consent form (ICF).

2. Men or women aged ≥18 years as of the date the ICF is signed.

3. Diagnosis of CIDP made by a consultant neurologist with a special interest in
peripheral neuropathy. CIDP may be diagnosed in the presence of diabetes or
IgG(immunoglobulin- G), IgA and IgM paraproteins without anti- MAG (myelin associated
glycoprotein) antibodies. Documentation of the initial diagnosis including definite
neurophysiological criteria proposed by INCAT (lnflammatory Neuropathy Cause and
Treatment) or EFNS/PNS (European Federation of Neuroscience/Peripheral Nerve Society)
must be available for review.

4. Ongoing treatment(s) for CIDP to include IVIg (Intravenous immune globulin) or
corticosteroids only. Other treatments for CIDP including plasma exchange,
azathioprine, methotrexate and mycophenolate must be washed out for 3 months.
Cyclophosphamide, rituximab and other monoclonal antibodies must be washed out for 12

5. Duration of CIDP > 6 months prior to the date the ICF is signed.

6. Treating neurologist and participant in agreement that alemtuzumab is an appropriate
treatment and documents this is in clinical notes.

Exclusion Criteria:

1. Previous treatment with alemtuzumab.

2. Participation in a controlled trial of an investigational medicinal product within
the past 12 weeks. The duration of required washout will be established based on the
known biological and pharmacokinetic properties of the investigational drug (prior
treatment with herbal medications or nutritional supplements is permitted).

3. Intolerance of pulsed corticosteroids.

4. Alternative cause of peripheral neuropathy such as drug or toxin, hereditary
neuropathy or concomitant diseases such as HIV infection, Lyme disease, chronic
active hepatitis, systemic lupus erythematosus, IgM paraprotein with anti-MAG (myelin
associated glycoprotein) antibodies, vasculitis, thyroid dysfunction, hematological
and non- hematological malignancies.

5. Presence of neurogenic sphincter disturbance.

6. Multifocal motor neuropathy (fulfilling EFNS/PNS (European Federation of
Neuroscience/Peripheral Nerve Society) criteria).

7. Atypical CIDP with pure sensory or persistent uni-focal impairment or significant CNS

8. Active infection, e.g., deep-tissue infection that the Investigator considers
sufficiently serious to preclude study participation.

9. In the Investigator's opinion, is at high risk for infection (e.g., in-dwelling
catheter, dysphagia, decubitus ulcer, history of prior aspiration pneumonia or
recurrent urinary tract infection).

10. Known infection with or seropositivity for human immunodeficiency virus (HIV).

11. Previous or present infection with hepatitis C virus.

12. Previous or present infection with hepatitis B (positive hepatitis B serology).

13. Prior history of invasive fungal infections.

14. Latent tuberculosis, unless effective anti-tuberculosis therapy has been completed,
or active tuberculosis

15. Cervical high risk human papillomavirus (HPV) positivity or abnormal cervical
cytology other than abnormal squamous cells of undetermined significance (ASCUS). The
participant may be eligible after the condition has resolved (e.g., follow-up HPV
(human papilloma virus) test is negative or cervical abnormality has been effectively

16. CD4+ (cluster of differentiation - 4), CD8+ , or CD19+ cell count (i.e., absolute
CD3+CD4+, CD3+CD8+, or CD19+/mm3) cell count Screening. If abnormal cell counts return to within normal limits, eligibility may be

17. Absolute neutrophil count below the LLN at screening. If abnormal cell counts return
to with in normal limits, eligibility may be reassessed.

18. Confirmed platelet count at <100,000/μL within the past year on a sample without platelet clumping.

19. Known bleeding disorder (e.g.,dysfibrinogenemia, factor IX deficiency, hemophilia,
vonWillebrand's disease, disseminated intravascular coagulation (DIC), fibrinogen
deficiency, clotting factor deficiency) or chronic use of anticoagulant treatment.

20. Significant other active autoimmune disease, besides CIDP (e.g., immune cytopenias,
rheumatoid arthritis, systemic lupus erythematosus, other connective tissue
disorders, vasculitis, inflammatory bowel disease, severe psoriasis, thyroiditis).

21. Presence of anti-thyroid stimulating hormone (TSH) receptor (TSHR) antibodies (i.e.,
above LLN).

22. History of malignancy (exception for basal cell skin carcinoma).

23. Major psychiatric disorder not adequately controlled by treatment.

24. History of substance abuse within the last 2 years.

25. Epileptic seizures not adequately controlled by treatment.

26. Of child bearing potential with a positive serum pregnancy test,pregnant,or

27. Unwilling to agree to use a reliable and acceptable contraceptive method throughout
the study period (all participants of reproductive potential). Reliable and effective
contraceptive method(s) include: intrauterine device (IUD), hormonal-based
contraception, surgical sterilization, abstinence, or double- barrier contraception
(condom and occlusive cap [diaphragm or cervical cap with spermicide]).

28. Any hepatorenal function value grade 2 or higher at screening (see Table below, drawn
from the National Cancer Institute (NCI) Common Terminology Criteria for Adverse
Events v3.0 (CTCAE), released 12 December 2003), with the exception of
hyperbilirubinemia due to Gilbert's syndrome:

Hepatic Bilirubin >1.5x ULN (upper limit of normal) SGOT/AST
>2.5x ULN SGPT/ALT >2.5x ULN Alkaline phosphatase
>2.5x ULN

Renal Creatinine >1.5x ULN

29. Other conditions that,in the Investigator's opinion,compromise the participant's
ability to understand the participant information, to give informed consent, to
comply with the trial protocol, or to complete the study.

30. Immunocompromise of any type which would in the view of the investigator make the
risk of alemtuzumab treatment unacceptable.

31. Previous stem cell transplantation.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Change from baseline in disability at 36 months measured by GBS/CIDP Rasch-built Overall Disability Scale (GBS/CIDP-RODS).

Outcome Description:

The RODS CIDP scale is a validated measure of disability in CIDP/GBS.

Outcome Time Frame:

Every six months up to 36 months

Safety Issue:



United States: Institutional Review Board

Study ID:




Start Date:

November 2012

Completion Date:

December 2016

Related Keywords:

  • Chronic Inflammatory Demyelinating Neuropathy
  • CIDP- chronic inflammatory demyelinating neuropathy
  • demyelinating neuropathy
  • alemtuzumab
  • Polyradiculoneuropathy
  • Polyradiculoneuropathy, Chronic Inflammatory Demyelinating



The Johns Hopkins HospitalBaltimore, Maryland  21205