UMIN-CTR Clinical Trial

Basic information
Public title	Open-label, single-arm, multicenter, investigators initiated phase II clinical trial to evaluate the efficacy and safety of IDEC-C2B8 in patients with steroid treatment-resistant pemphigus
Acronym	Open-label, single-arm, multicenter, investigators initiated phase II clinical trial to evaluate the efficacy and safety of IDEC-C2B8 in patients with steroid treatment-resistant pemphigus
Scientific Title	Open-label, single-arm, multicenter, investigators initiated phase II clinical trial to evaluate the efficacy and safety of IDEC-C2B8 in patients with steroid treatment-resistant pemphigus
Scientific Title:Acronym	Open-label, single-arm, multicenter, investigators initiated phase II clinical trial to evaluate the efficacy and safety of IDEC-C2B8 in patients with steroid treatment-resistant pemphigus
Region	Japan

Condition
Condition	steroid treatment-resistant pemphigus
Classification by specialty	Dermatology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy and safety of IDEC-C2B8 in patients with pemphigus who are refractory to steroid therapy (except for paraneoplastic pemphigus)
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	The percentage of patients who achieve remission (complete remission + partial remission) at Week 25
Key secondary outcomes	>Pemphigus Disease Area Index (PDAI) values at Week 25 >Changes from baseline in PDAI >Changes from baseline in pemphigus autoantibody values (anti-Dsg1,anti-Dsg3 autoantibodies) >Changes from baseline in CD19+ B-cell counts, CD20+ B-cell counts, and CD3+ T-cell counts Safety endpoints: >Type, frequency, and severity of adverse events >Type, frequency, and severity of adverse drug reactions Other endpoints: >Pharmacokinetic analysis (changes in peripheral blood concentration of IDEC-C2B8) >Expression rate of human anti-IDEC-C2B8 antibody (HACA) >Changes from baseline in immunoglobulin values

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Rituximab (genetic recombination)
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	80 years-old >=
Gender	Male and Female
Key inclusion criteria	1. Patients who were definitively diagnosed of pemphigus during receive PSL with/without "minimal immunosuppressive adjuvant as necessary" (refer to Table 6 1) and experienced disease relapse and a re-increase in the PDAI score during the course of PSL tapering after remission induction. (However, this is limited to the PDAI score at the time of obtaining consent was 1 point or higher and less than 50 points). 2. Patients who receive standard-of-care PSL consisting of 15-30 mg/day. 3. Patients who have persistent or worse PDAI score at two consecutive points at an interval of at least 7 days during a screening period (allowable variation range: PDAI score within 3 times and a difference of +20 points from the time of tentative registration). 4. Male or female aged from 20 to 80 years 5. Patients who are able to be hospitalized for 3 days (2 night) from the date of the initial administration of IDEC-C2B8 (inpatient or outpatient treatment is possible for second administration). 6. Patients in whom the stable dose of PSL for at least 30 days prior to screening. 7. Patients who signed informed consent form.
Key exclusion criteria	1. Women who are nursing, pregnant, intending to be pregnant. 2. Patients with a history of hypersensitivity or shock to humanized or mouse monoclonal antibodies or mouse-derived components. 3. Patients with severe and uncontrollable organ failure. 4. Patients with any concomitant condition that required treatment. 5. Patients with an active infection. 6. Patients who received an infusion of antibiotics or patients with an infectious disease that requires hospitalization. 7. Patients who received an oral administration of antibiotics. 8. Patients with a history of a bacteremia caused by Staphylococcus aureus or Pseudomonas aeruginosa. 9. Patients who experienced bone soft tissue infections or any type of organ abscess. 10. Patients with a history of severe recurrent infection or chronic infection. 11. Patients with a malignant tumor or a history of a malignant tumor. 12. Patients with a history of addiction to alcohol or drugs. 13. Patients with a severe psychiatric disorder. 14. Patients in whom an operative treatment has been performed. 15. Patients with a history of severe infection after starting immunosuppressive agent administration. 16. Patients who previously received administration of anti-CD20 antibody. 17. Patients who have used a live vaccine or attenuated vaccine. 18. Patients in whom the following clinical laboratory abnormalities are noted during the SCR period. 19. Patients who have begun or increased the dose of an oral immunosuppressive agent. 20. Patients who received IVIG. 21. Patients who received intravenous steroid pulse therapy. 22. Patients who received plasmapheresis. 23. Patients who received intravenous cyclophosphamide pulse therapy . 24. Patients treated with other investigational drugs. 25. Patients who are determined as unfit for the enrollment in the study after an investigation.
Target sample size	10

Research contact person
Name of lead principal investigator	1st name Middle name Last name Masayuki Amagai
Organization	Department of Dermatology, Keio University School of Medicine
Division name	Dermatology
Zip code
Address	35 Shinanomachi, Shinjuku-ku, Tokyo, 160-8582
TEL	03-3353-1211
Email	amagai@keio.jp

Public contact
Name of contact person	1st name Middle name Last name Yasuko Saito
Organization	Keio University Hospital
Division name	Clinical and Translational Research Center
Zip code
Address	35 Shinanomachi, Shinjuku-ku, Tokyo, 160-8582
TEL	03-5315-4278
Homepage URL
Email	pmo@ccr.med.keio.ac.jp

Sponsor
Institute	Department of Dermatology, Keio University School of Medicine
Institute
Department

Funding Source
Organization	Japan Agency Medical Research and Development
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor	HOKKAIDO UNIVERSITY HOSPITAL OKAYAMA UNIVERSITY HOSPITAL KURUME UNIVERSITY HOSPITAL
Name of secondary funder(s)	ZENYAKU KOGYO CO.,LTD.

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2016 Year 10 Month 04 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2016 Year 08 Month 02 Day
Date of IRB	2016 Year 09 Month 01 Day
Anticipated trial start date	2016 Year 10 Month 04 Day
Last follow-up date	2019 Year 03 Month 31 Day
Date of closure to data entry	2019 Year 11 Month 12 Day
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2016 Year 10 Month 03 Day
Last modified on	2019 Year 12 Month 23 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027938