Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)Phase 2RECIPENCT03864185
The Evaluation of Efficacy and Safety of Rituximab in Refractory CIDP Patients With IgG4 Autoantibodies
A Phase 2 study testing Rituximab (genetical recombination) in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), run by Nagoya University at 4 sites in Japan.
Trial summary
To evaluate the efficacy and safety of rituximab (genetical recombination) intravenously administered to CIDP patients with positive or negative IgG4 autoantibody. Read more about this study
- Who is it for?
- Ages 12 years and olderHealthy volunteers can take part
- Conditions the study is for
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
- What is being tested?
- Rituximab (genetical recombination)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 4 sitesJapan
- Study dates
- 28 Mar 2019to 27 May 2021
- Phase
- Phase 2
- 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 12 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Patients with definite CIDP diagnosed according to the modified diagnostic criteria of European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) (2010) by the time of enrollment in the study
- Patients meeting one of the following conditions:
- Patients with positive serum IgG4 autoantibody (CNTN-1 or NF-155) confirmed by the time of enrollment in the study
- Patients with negative serum IgG4 autoantibody (CNTN-1 and NF-155) confirmed by the time of enrollment in the study
- Patients with refractory CIDP not responding adequately to treatment with corticosteroid for 12 weeks, and intravenous immunoglobulin therapy (IVIg) for 8 weeks by the time of enrollment in the study, or those who are unable to administer or continue corticosteroid and IVIg
- Patients with total adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale scores of 2 to 8 at both preliminary enrollment and enrollment, and with the total score at enrollment equal to or worse than that at preliminary enrollment
- Patients aged 12 years or older at informed consent
- Patients who give their voluntary written consent after having received adequate information on this study (legally acceptable representatives should also give consent for underage patients, and informed assent should be obtained from children aged 12 to 15)
Who cannot join
- Patients with disease meeting one of the following exclusion criteria defined in the modified EFNS/PNS diagnostic criteria (2010).
- Borrelia burgdorferi infection (Lyme disease), diphtheria, drug or toxin exposure probably to have caused the neuropathy Hereditary demyelinating neuropathy
- Prominent sphincter disturbance
- Diagnosis of multifocal motor neuropathy
- IgM monoclonal gammopathy with high titre antibodies to myelin-associated glycoprotein
- Other causes for a demyelinating neuropathy including POEMS syndrome, osteosclerotic myeloma, diabetic and non-diabetic lumbosacral radiculoplexus neuropathy PNS lymphoma and amyloidosis may occasionally have demyelinating features
Show all 21Show fewer
- Patients who have started or have increased the dose of corticosteroid for CIDP within 12 weeks prior to the enrollment
- Patients who have started or have increased the dose of IVIg within 8 weeks prior to the enrollment
- Patients who have underwent plasmapheresis within 8 weeks prior to the enrollment or patients with refractory disease not responding adequately to 8 weeks of plasmapheresis (plasma exchange or double-filtration plasmapheresis)
- Patients who have started or have increased the dose of an immunosuppressant (azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, interferon alpha, interferon beta, etanercept, methotrexate, mitoxantrone, alemtuzumab, cladribine, tacrolimus, fingolimod) within 12 weeks prior to the enrollment
- Patients who have underwent hematopoietic stem cell transplant prior to the enrollment
- Patients who have used rituximab (genetical recombination) prior to the enrollment
- Patients who have participated in another clinical study within 3 months prior to the enrollment (enrollment is allowed for those participating in a clinical study in the range of Indications or Dosage and Administration in Japan) or patients who are participating in another study
- Patients with poorly controlled diabetes (HbA1c of 7 % or higher)
- Patients who have or are suspected to have active infection (infection requiring treatment with systemic antimicrobial, antifungal, or antiviral agents) at the time of the enrollment
- Patients tested positive for HBs antigen, HBs antibody, HBc antibody, and/or HCV antibody (patients with positive HBs antibody or HBc antibody can be enrolled when a hepatitis B virus-DNA test is negative [below the limit of detection], and hepatitis B virus-DNA and aspartate/alanine transaminase levels are monitored at fixed intervals), or patients with positive HIV antibody or HTLV-1 antibody at the time of the enrollment
- Patients with leukopenia (less than 2,000 /mm3), neutropenia (less than 1,000 /mm3), or lymphopenia (less than 500 /mm3) at the time of the enrollment
- Patients with history of serious hypersensitivity or anaphylactic reaction to one of the ingredients in the investigational drug or murine protein-containing products
- Patients with serious comorbidity (e.g., hepatic, renal, cardiac, lung, hematologic, or brain disease)
- Female patients who are pregnant, lactating, or potentially pregnant, or patients who are not willing to use contraceptive measures during the study period
- Patients who are judged to be unsuitable by the investigator or a sub-investigator
Trial locations and contacts
4 sites in 1 country.
Japan4 sites
Chiba University Hospital
Chiba
Kyushu University Hospital
Fukuoka
Nagoya University Hospital
Nagoya, Aich, 466-8560
Yamaguchi University Hospital
Ube
About this study
Official title: The Evaluation of Efficacy and Safety of Rituximab (Genetical Recombination) in Refractory Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Patients With Immunoglobulin G4 (IgG4) Autoantibodies in the Exploratory Clinical Trial
To evaluate the efficacy and safety of rituximab (genetical recombination) intravenously administered to CIDP patients with positive or negative IgG4 autoantibody.
- Being tested
- Rituximab (genetical recombination), Placebo
- Keywords
- IgG4 autoantibody, Neurofascin-155, Contactin-1, Rituximab
- Medical subject headings
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rituximab (genetical recombination)Biological
Administer 375 mg/m2 of rituximab (genetical recombination) IV infusion once weekly for 4 doses.
PlaceboOther
Administer placebo IV infusion once weekly for 4 doses.
| Study group | Role | What they receive |
|---|---|---|
| Rituximab group (IgG4 autoantibody positive) | Active comparator | Not described Receives: Biological: Rituximab (genetical recombination) |
| Placebo group (IgG4 autoantibody positive) | Placebo comparator | Not described Receives: Other: Placebo |
| Rituximab group (IgG4 autoantibody negative) | Active comparator | Not described Receives: Biological: Rituximab (genetical recombination) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of patients with improvement in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale
Primary analysis will compare scores of adjusted INCAT Disability Scale evaluated prior to treatment (at the time of enrollment) and scores at each timepoint after week 26 to calculate the proportion of patients who achieve an improvement of one and more from the baseline.
The INCAT Disability Scale is an index to evaluate disorders in lower (gait) and upper (elevation of the upper arms and fine movement of the fingertips) extremities. The INCAT score is a 10-point scale and ranges from 0 (normal) to 10 (worst). For the "adjusted" INCAT score, a change in upper extremity score from 0 to 1 or 1 to 0 will not be considered meaningful in this evaluation.
Measured: Up to 52 weeks
Secondary outcomes (19)
Change in grip strength (kPa)
The differences of Grip strength (kPa) between prior to treatment and at each timepoint are summarized.
Measured: Up to 52 weeks
Change in Rasch-built Overall Disability Scale (R-ODS) score
The differences of R-ODS score between prior to treatment and at each timepoint are summarized.
R-ODS is consist of a 24-item questionnaire about daily living task with 3 response options: (0) "impossible to perform," (1) "performed with difficulty," and (2) "easily performed. The R-ODS score is a 48-point scale (range: 0-48), and is converted into a centile metric score with values ranging from 0 (most severe activity and social participation limitations) to 100 (no activity and social participation limitations).
Measured: Up to 52 weeks
Change in Medical Research Council (MRC) Sum Score
The differences of MRC Sum Score between prior to treatment and at each timepoint are summarized.
The MRC Sum Score is a scale to assess for 8 muscle groups (right and left side) as follow: Shoulder abduction, Elbow flexion, Wrist extension, Index finger abduction, Hip flexion, Knee extension, Foot dorsiflexion, Great toe dorsiflexion.The MRC Sum Score is a 80-point scale and ranges from 0 (paralysis) to 80 (normal strength).
Measured: Up to 52 weeks
Change in motor nerve distal latency
The differences of distal latency between prior to treatment and at each timepoint are summarized.
Measured: Up to 52 weeks
Change in motor nerve proximal latency
The differences of proximal latency between prior to treatment and at each timepoint are summarized.
Measured: Up to 52 weeks
Change in motor nerve compound muscle action potential (CMAP)
The differences of CMAP between prior to treatment and at each timepoint are summarized.
Measured: Up to 52 weeks
Change in motor nerve conduction velocity
The differences of motor nerve conduction velocity between prior to treatment and at each timepoint are summarized.
Measured: Up to 52 weeks
Cerebrospinal fluid protein level
Cerebrospinal fluid protein level at each timepoint are summarized.
Measured: Up to 52 weeks
B cell counts (CD19 positive and CD20 positive cell counts) and T cell counts (CD3 positive, CD4 positive, and CD8 positive cell counts)
B cell counts (CD19 positive and CD20 positive cell counts) and T cell counts (CD3 positive, CD4 positive, and CD8 positive cell counts) at each timepoint are summarized.
Measured: Up to 52 weeks
Expression of HACA
The number of patients expressing HACA, and the proportion and its 95% confidence interval of these patients at each timepoint are summarized.
Measured: Up to 52 weeks
Serum rituximab (genetical recombination) level
Serum rituximab (genetical recombination) level at each timepoint are summarized.
Measured: Up to 52 weeks
Maximum serum concentration (Cmax) of rituximab (genetical recombination)
Cmax of rituximab (genetical recombination) is summarized.
Measured: Up to 52 weeks
Area under the curve (AUC) of blood concentration of rituximab (genetical recombination)
AUC of blood concentration of rituximab (genetical recombination) is summarized.
Measured: Up to 52 weeks
Half-life (t1/2) of rituximab (genetical recombination)
t1/2 of rituximab (genetical recombination) is summarized.
Measured: Up to 52 weeks
Clearance (CL) of rituximab (genetical recombination)
CL of rituximab (genetical recombination) is summarized.
Measured: Up to 52 weeks
Mean residence time (MRT) of rituximab (genetical recombination)
MRT of rituximab (genetical recombination) is summarized.
Measured: Up to 52 weeks
Volume of distribution (Vds) of rituximab (genetical recombination) level
Vds of rituximab (genetical recombination) is summarized.
Measured: Up to 52 weeks
Serum antibody titers of IgG4 (CNTN-1 and NF-155) and these IgG subclass
Serum titers (CNTN-1 and NF-155, and these IgG subclasses 1 to 4) at each timepoint are summarized.
Measured: Up to 52 weeks
Serum neurofilament
Serum neurofilament level at each timepoint is summarized.
Measured: Up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2019
- Study start28 Mar 2019
- Primary completion27 May 2021
- Study completion27 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)Studies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nagoya University
- Sponsor type
- University, hospital or other
- Collaborators
- Japan Agency for Medical Research and Development, Zenyaku Kogyo Co., Ltd.
- Responsible party
- Principal investigator, Masahiro Iijima, Nagoya University
- Investigators
- Masahiro Iijima, Ph. D, Principal investigator, Nagoya University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03864185
- Sponsor's protocol ID
- CAMCR-011
- Other IDs
- jRCT2041180037, UMIN000035753
- Acronym
- RECIPE
- Status last verified
- Aug 2021
- First submitted
- 19 Feb 2019
- Last updated
- 20 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Shimizu S, Iijima M, Fukami Y, Tamura N, Nakatochi M, Ando M, Nishi R, Koike H, Kaida K, Koga M, Kanda T, Ogata H, Kira JI, Mori M, Kuwabara S, Katsuno M. Efficacy and Safety of Rituximab in Refractory CIDP With or Without IgG4 Autoantibodies (RECIPE): Protocol for a Double-Blind, Randomized, Placebo-Controlled Clinical Trial. JMIR Res Protoc. 2020 Apr 1;9(4):e17117. doi: 10.2196/17117.
PubMed 32234705
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Evaluation of Efficacy and Safety of Rituximab (Genetical Recombination) in Refractory Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Patients With Immunoglobulin G4 (IgG4) Autoantibodies in the Exploratory Clinical Trial
- ClinicalTrials.gov ID
- NCT03864185
- Acronym / sponsor ID
- RECIPE / CAMCR-011
- Keywords
- IgG4 autoantibody, Neurofascin-155, Contactin-1, Rituximab
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Nagoya UniversityUniversity, hospital or other
- Collaborators
- Japan Agency for Medical Research and Development, Zenyaku Kogyo Co., Ltd.
- Study start
- 28 Mar 2019 (actual)
- Primary completion
- 27 May 2021 (actual)
- Study completion
- 27 May 2021 (actual)
- First posted
- 6 Mar 2019 (actual)
- 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.
To evaluate the efficacy and safety of rituximab (genetical recombination) intravenously administered to CIDP patients with positive or negative IgG4 autoantibody.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Rituximab group (IgG4 autoantibody positive) | Active comparator | Biological: Rituximab (genetical recombination) |
| Placebo group (IgG4 autoantibody positive) | Placebo comparator | Other: Placebo |
| Rituximab group (IgG4 autoantibody negative) | Active comparator | Biological: Rituximab (genetical recombination) |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patients with definite CIDP diagnosed according to the modified diagnostic criteria of European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) (2010) by the time of enrollment in the study
- Patients meeting one of the following conditions:
- Patients with refractory CIDP not responding adequately to treatment with corticosteroid for 12 weeks, and intravenous immunoglobulin therapy (IVIg) for 8 weeks by the time of enrollment in the study, or those who are unable to administer or continue corticosteroid and IVIg
- Patients with total adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Scale scores of 2 to 8 at both preliminary enrollment and enrollment, and with the total score at enrollment equal to or worse than that at preliminary enrollment
- Patients aged 12 years or older at informed consent
Key exclusion criteria (21 listed)
- Patients with disease meeting one of the following exclusion criteria defined in the modified EFNS/PNS diagnostic criteria (2010).
- Patients who have started or have increased the dose of corticosteroid for CIDP within 12 weeks prior to the enrollment
- Patients who have started or have increased the dose of IVIg within 8 weeks prior to the enrollment
- Patients who have underwent plasmapheresis within 8 weeks prior to the enrollment or patients with refractory disease not responding adequately to 8 weeks of plasmapheresis (plasma exchange or double-filtration plasmapheresis)
- Patients who have started or have increased the dose of an immunosuppressant (azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, interferon alpha, interferon beta, etanercept, methotrexate, mitoxantrone, alemtuzumab, cladribine, tacrolimus, fingolimod) within 12 weeks prior to the enrollment
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 |
|---|---|---|
| Rituximab (genetical recombination)Administer 375 mg/m2 of rituximab (genetical recombination) IV infusion once weekly for 4 doses. | Biological | Rituximab group (IgG4 autoantibody negative), Rituximab group (IgG4 autoantibody positive) |
| PlaceboAdminister placebo IV infusion once weekly for 4 doses. | Other | Placebo group (IgG4 autoantibody positive) |
Primary outcome
Rate of patients with improvement in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale
Time frame: Up to 52 weeks
Primary analysis will compare scores of adjusted INCAT Disability Scale evaluated prior to treatment (at the time of enrollment) and scores at each timepoint after week 26 to calculate the proportion of patients who achieve an improvement of one and more from the baseline. The INCAT Disability Scale is an index to evaluate disorders in lower (gait) and upper (elevation of the upper arms and fine movement of the fingertips) extremities. The INCAT score is a 10-point scale and ranges from 0 (normal) to 10 (worst). For the "adjusted" INCAT score, a change in upper extremity score from 0 to 1 or 1 to 0 will not be considered meaningful in this evaluation.
19 secondary outcomes
Change in grip strength (kPa)
Time frame: Up to 52 weeks
Change in Rasch-built Overall Disability Scale (R-ODS) score
Time frame: Up to 52 weeks
Change in Medical Research Council (MRC) Sum Score
Time frame: Up to 52 weeks
Change in motor nerve distal latency
Time frame: Up to 52 weeks
Change in motor nerve proximal latency
Time frame: Up to 52 weeks
Change in motor nerve compound muscle action potential (CMAP)
Time frame: Up to 52 weeks
Change in motor nerve conduction velocity
Time frame: Up to 52 weeks
Cerebrospinal fluid protein level
Time frame: Up to 52 weeks
B cell counts (CD19 positive and CD20 positive cell counts) and T cell counts (CD3 positive, CD4 positive, and CD8 positive cell counts)
Time frame: Up to 52 weeks
Expression of HACA
Time frame: Up to 52 weeks
Serum rituximab (genetical recombination) level
Time frame: Up to 52 weeks
Maximum serum concentration (Cmax) of rituximab (genetical recombination)
Time frame: Up to 52 weeks
Area under the curve (AUC) of blood concentration of rituximab (genetical recombination)
Time frame: Up to 52 weeks
Half-life (t1/2) of rituximab (genetical recombination)
Time frame: Up to 52 weeks
Clearance (CL) of rituximab (genetical recombination)
Time frame: Up to 52 weeks
Mean residence time (MRT) of rituximab (genetical recombination)
Time frame: Up to 52 weeks
Volume of distribution (Vds) of rituximab (genetical recombination) level
Time frame: Up to 52 weeks
Serum antibody titers of IgG4 (CNTN-1 and NF-155) and these IgG subclass
Time frame: Up to 52 weeks
Serum neurofilament
Time frame: Up to 52 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2021.
- Background: PubMed 32234705Shimizu S, Iijima M, Fukami Y, Tamura N, Nakatochi M, Ando M, Nishi R, Koike H, Kaida K, Koga M, Kanda T, Ogata H, Kira JI, Mori M, Kuwabara S, Katsuno M. Efficacy and Safety of Rituximab in Refractory CIDP With or Without IgG4 Autoantibodies (RECIPE): Protocol for a Double-Blind, Randomized, Placebo-Controlled Clinical Trial. JMIR Res Protoc. 2020 Apr 1;9(4):e17117. doi: 10.2196/17117.
- Individual participant dataSharing planned: No
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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.
Masahiro Iijima, Ph. D principal investigator
Nagoya University Hospital
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rituximab (genetical recombination) in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), run by Nagoya University at 4 sites in Japan.
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 8 inclusion and 21 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: finished, as of the sponsor's last update on 20 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Japan. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Rate of patients with improvement in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale (time frame: Up to 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03864185, which also lists 1 linked publication.
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 20 Aug 2021.