Chronic Inflammatory Demyelinating NeuropathyPhase 4NCT01757574
Alemtuzumab in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)
A Phase 4 study testing Alemtuzumab infusion in people with Chronic Inflammatory Demyelinating Neuropathy, run by Johns Hopkins University at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Inflammatory Demyelinating Neuropathy
- What is being tested?
- Alemtuzumab infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2012to Aug 2016
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent form (ICF).
- Men or women aged ≥18 years as of the date the ICF is signed.
- 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.
- 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 months.
- Duration of CIDP > 6 months prior to the date the ICF is signed.
- Treating neurologist and participant in agreement that alemtuzumab is an appropriate treatment and documents this is in clinical notes.
Who cannot join
- Previous treatment with alemtuzumab.
- 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).
- Intolerance of pulsed corticosteroids.
- 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.
- Presence of neurogenic sphincter disturbance.
- Multifocal motor neuropathy (fulfilling EFNS/PNS (European Federation of Neuroscience/Peripheral Nerve Society) criteria).
Show all 33Show fewer
- Atypical CIDP with pure sensory or persistent uni-focal impairment or significant CNS involvement.
- Active infection, e.g., deep-tissue infection that the Investigator considers sufficiently serious to preclude study participation.
- 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).
- Known infection with or seropositivity for human immunodeficiency virus (HIV).
- Previous or present infection with hepatitis C virus.
- Previous or present infection with hepatitis B (positive hepatitis B serology).
- Prior history of invasive fungal infections.
- Latent tuberculosis, unless effective anti-tuberculosis therapy has been completed, or active tuberculosis
- 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 treated).
- CD4+ (cluster of differentiation - 4), CD8+ , or CD19+ cell count (i.e., absolute CD3+CD4+, CD3+CD8+, or CD19+/mm3) cell count <lower limit of normal (LLN) at Screening. If abnormal cell counts return to within normal limits, eligibility may be reassessed.
- Absolute neutrophil count below the LLN at screening. If abnormal cell counts return to with in normal limits, eligibility may be reassessed.
- Confirmed platelet count <LLN of the evaluating laboratory at Screening or documented at <100,000/μL within the past year on a sample without platelet clumping.
- 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.
- 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).
- Presence of anti-thyroid stimulating hormone (TSH) receptor (TSHR) antibodies (i.e., above LLN).
- History of malignancy (exception for basal cell skin carcinoma).
- Major psychiatric disorder not adequately controlled by treatment.
- History of substance abuse within the last 2 years.
- Epileptic seizures not adequately controlled by treatment.
- Of child bearing potential with a positive serum pregnancy test,pregnant,or lactating.
- 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]).
- 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
- 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.
- Immunocompromise of any type which would in the view of the investigator make the risk of alemtuzumab treatment unacceptable.
- Previous stem cell transplantation.
Trial locations and contacts
1 site in 1 country.
United States1 site
The Johns Hopkins Hospital
Baltimore, Maryland, 21231
About this study
Official title: An Open-Label Trial of 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.
Read the detailed description
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.
- Being tested
- Alemtuzumab infusion
- Keywords
- CIDP- chronic inflammatory demyelinating neuropathy, demyelinating neuropathy, alemtuzumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alemtuzumab infusionDrug
| Study group | Role | What they receive |
|---|---|---|
| Alemtuzumab | Experimental | Open label study of alemtuzumab Receives: Drug: Alemtuzumab infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in disability at 36 months measured by GBS/CIDP Rasch-built Overall Disability Scale (GBS/CIDP-RODS).
The RODS CIDP scale is a validated measure of disability in CIDP/GBS.
Measured: Every six months up to 36 months
Secondary outcomes (1)
Change from baseline in disability measured with Overall Neuropathy Limitations Scale (ONLS) at 36 months.
ONLS is a validated measure of neurological disfunction.
Measured: Every six months up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2012 (estimated)
- Study startNov 2012
- Primary completionAug 2016
- Study completionAug 2016
Why it stopped: At the request of the global principal investigator, no subjects were enrolled in this study at any site.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Inflammatory Demyelinating NeuropathyStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01757574
- Sponsor's protocol ID
- CAMIMD01108IST
- Status last verified
- May 2017
- First submitted
- 6 Dec 2012
- Last updated
- 18 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Trial of Alemtuzumab in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIPD)
- ClinicalTrials.gov ID
- NCT01757574
- Acronym / sponsor ID
- CAMIMD01108IST
- Keywords
- CIDP- chronic inflammatory demyelinating neuropathy, demyelinating neuropathy, alemtuzumab
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: At the request of the global principal investigator, no subjects were enrolled in this study at any site.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- Nov 2012 (actual)
- Primary completion
- Aug 2016 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 31 Dec 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alemtuzumab | Experimental | Drug: Alemtuzumab infusionOpen label study of alemtuzumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Signed informed consent form (ICF).
- Men or women aged ≥18 years as of the date the ICF is signed.
- 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.
- 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 months.
- Duration of CIDP > 6 months prior to the date the ICF is signed.
Key exclusion criteria (33 listed)
- Previous treatment with alemtuzumab.
- 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).
- Intolerance of pulsed corticosteroids.
- 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.
- Presence of neurogenic sphincter disturbance.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Alemtuzumab infusion | Drug | Alemtuzumab |
Primary outcome
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
The RODS CIDP scale is a validated measure of disability in CIDP/GBS.
1 secondary outcome
Change from baseline in disability measured with Overall Neuropathy Limitations Scale (ONLS) at 36 months.
Time frame: Every six months up to 36 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Alemtuzumab infusion in people with Chronic Inflammatory Demyelinating Neuropathy, run by Johns Hopkins University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 18 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2017.