UMIN-CTR Clinical Trial

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

steroid treatment-resistant pemphigus

Classification by specialty

Dermatology

Classification by malignancy

Others

Genomic information

NO

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

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

Study type

Interventional

Basic design

Single arm

Randomization

Non-randomized

Randomization unit

Blinding

Open -no one is blinded

Control

Uncontrolled

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

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

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

Institute

Department of Dermatology,
Keio University School of Medicine

Institute

Department

Personal name

Organization

Japan Agency Medical Research and Development

Organization

Division

Category of Funding Organization

Other

Nationality of Funding Organization

Co-sponsor

HOKKAIDO UNIVERSITY HOSPITAL
OKAYAMA UNIVERSITY HOSPITAL
KURUME UNIVERSITY HOSPITAL

Name of secondary funder(s)

ZENYAKU KOGYO CO.,LTD.

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2016

Year

10

Month

04

Day

URL releasing protocol

Publication of results

Unpublished

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

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 related information

Registered date

2016

Year

10

Month

03

Day

Last modified on

2019

Year

12

Month

23

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027938