UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000022050
Receipt number R000025102
Scientific Title A Phase II, Randomised, Double-Blind, Placebo-Controlled Study Followed by a Continuing Open Label Study, to Verify the Efficacy and Safty of Intrathecal Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)
Date of disclosure of the study information 2016/05/13
Last modified on 2022/05/09 13:39:58

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Basic information

Public title

A Phase II, Randomised, Double-Blind, Placebo-Controlled Study Followed by a Continuing Open Label Study, to Verify the Efficacy and Safty of Intrathecal Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

Acronym

A Phase II, Randomised, Double-Blind, Placebo-Controlled Study Followed by a Continuing Open Label Study, to Verify the Efficacy and Safty of Intrathecal Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

Scientific Title

A Phase II, Randomised, Double-Blind, Placebo-Controlled Study Followed by a Continuing Open Label Study, to Verify the Efficacy and Safty of Intrathecal Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

Scientific Title:Acronym

A Phase II, Randomised, Double-Blind, Placebo-Controlled Study Followed by a Continuing Open Label Study, to Verify the Efficacy and Safty of Intrathecal Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

Region

Japan


Condition

Condition

Amyotrophic Lateral Sclerosis (ALS)

Classification by specialty

Neurology

Classification by malignancy

Others

Genomic information

NO


Objectives

Narrative objectives1

To Verify the Efficacy to Delay Disease Progression of ALS by Intrathecal Repeated Administration of KP-100IT using NP022 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

Basic objectives2

Safety

Basic objectives -Others


Trial characteristics_1

Confirmatory

Trial characteristics_2


Developmental phase

Phase II


Assessment

Primary outcomes

Difference in the change of ALSFRS-R score (delta ALSFRS-R) during the 24 weeks-double blind period, starting from the time point before initial administration (implantation period) to the point of 24 weeks observation between Placebo and KP-100IT groups

Key secondary outcomes

Comparison of changing rate in ALSFRS-R score per 30 days between before and after the treatment

Difference in the following items between at the time before initial administration (implantation period) and at the point of 24 weeks in administration/observation period
ALSFRS-R sub score of each domain (bulbar function, extremities function, respiratory function), %FVC, MMT Score (extremities, neck muscles), Modified Norris Scale (extremities symptom score, bulbar symptom score, total scores), weight, grip strength, ALSAQ40 score, ALS severity classification

Time to death or to a defined event (death, inability of independent ambulation, loss of unilateral upper limb function, tracheostomy, ventilated, tube feeding, loss of vocal conversation)

Time reaching to the state of %FVC<50% (time length until %FVC decreases to <50%)

Time length until ALSFRS-R score decreases by 6 points or more [score change (delta ALSFRS-R) from initial administration (implantation period) is -6 or less]

Discontinuance rate after initial administration

Safety of repeated administration of KP-100IT for a long period

Safety and defects of NP022 when KP-100IT is intrathecally administered repeatedly for a long period


Base

Study type

Interventional


Study design

Basic design

Parallel

Randomization

Randomized

Randomization unit

Individual

Blinding

Double blind -all involved are blinded

Control

Placebo

Stratification

YES

Dynamic allocation

NO

Institution consideration

Institution is not considered as adjustment factor.

Blocking

NO

Concealment

Central registration


Intervention

No. of arms

3

Purpose of intervention

Treatment

Type of intervention

Medicine Device,equipment

Interventions/Control_1

24 weeks repeated intrathecal administration of KP-100IT at 2 mg (once per 2 weeks, double blind period)

Interventions/Control_2

24 weeks repeated intrathecal administration of placebo (once per 2 weeks, double blind period)

Interventions/Control_3

24 weeks repeated-intrathecal administration of KP-100IT at 2 mg (once per 2 weeks, continuing open label period)

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

70 years-old >=

Gender

Male and Female

Key inclusion criteria

[Interim Registration]
Subjects who are
- Diagnosed as "Clinically Definite ALS", "Clinically Probable ALS" or "Clinically Possible-Laboratory-supported ALS" according to the El Escorial revised Criteria, and within 30 months after onset of the disease (at the time of obtaining informed consent (IC))

- Classified as class 1 or 2 in ALS severity classification

- Aged 20 or older and 70 or younger at the time of obtaining informed consent (IC)

- With 2 points or more in every question of ALSFRS-R (in writing and eating action (1), both sides should be 2 points or more)

- %FVC is not less than 70

- Two points or more in every question of bulbar symptom scale in the modified Norris Scale

- Agreed by written IC for entry in the trial

[Registration]
Subjects who
- meet the following criteria in addition to the inclusion criteria for the interim registration


- Change in ALSFRS-R score during 12 weeks-preobsevation period is within the range between -1 and -3

- Can come into hospital by the day before the day of NP022 implantation and can stay hospital until the next day of the first administration

Key exclusion criteria

Subjects who
- Have serious hepatic disorder, renal disorder, cardiovascular disease, pulmonary disorder, hematological disorder or metabolism disorder, and are judged by the investigators to be improper for entry in the trial


- With HbA1c (NGSP scale) of 6.5% or higher at the check of screening period

- Participated in clinical trials of INDs or clinical studies receiving treatment within a month before interim registration (30 days including interim registration date)

- With the following diseases or symptoms which might affect safety: malignant tumor, intrathecal infection, intrathecal tumor, proliferative retinopathy, depressed respiratory function (less than 70 of %FVC)





- Have a medical history of cancer

- Have a drug allergy

- Newly started taking of Riluzole after obtaining IC

- Participated in the Phase I Trial (protocol KP-100-ND001)

- With allergy to antibiotics or intolerance for devices implantation

- With allergy to the material of NP022, and including suspected above



- Have an anatomical difficulty in intrathecal catheterization

- Have an abnormality other than ALS in the spinal cord, the spine, the subarachnoidal cavity, or cerebrospinal fluid circulation, and are judged to be inappropriate for this trial

- With infectious diseases requiring a systemic therapy, meningitis, sepsis, bacteremia, peritonitis, or cutaneous infection, and including suspected above

- With bleeding diathesis

- With impaired coagulation-fibrinolysis, or taking anticoagulant or antiplatelet drugs


- Diagnosed as dementia

- Mentally impaired or have psychiatric disorders, and are judged to be inappropriate for the trial


- Pregnant (including suspected), nursing, wishing to become pregnant, or unable to prevent conception during the trial period using contraceptive methods


- Judged by the investigators to be inappropriate for any reason

Target sample size

48


Research contact person

Name of lead principal investigator

1st name Masashi
Middle name
Last name Aoki, MD, PhD

Organization

Tohoku University Graduate School of Medicine

Division name

Department of Neurology

Zip code

980-8574

Address

1-1 Seiryo-machi, Aoba-ku, Sendai, 980-8574, Japan

TEL

022-717-7000

Email

admin@neurol.med.tohoku.ac.jp


Public contact

Name of contact person

1st name Hitoshi
Middle name
Last name Warita, MD, PhD

Organization

Tohoku University Hospital

Division name

Department of Neurology

Zip code

9808574

Address

1-1 Seiryo-machi, Aoba-ku, Sendai, Japan

TEL

022-717-7000

Homepage URL

http://www.neurol.med.tohoku.ac.jp/

Email

warita@tohoku.ac.jp


Sponsor or person

Institute

Tohoku University

Institute

Department

Personal name



Funding Source

Organization

Japan Agency for Medical Research and Development (AMED)

Organization

Division

Category of Funding Organization

Japanese Governmental office

Nationality of Funding Organization

Japan


Other related organizations

Co-sponsor

Kringle Pharma, Inc.

Name of secondary funder(s)



IRB Contact (For public release)

Organization

Tohoku University Hospital Institutional Review Board

Address

1-1 Sseiryo, Aoba-ku, Sendai, Miyagi

Tel

022-717-7056

Email

chiken@grp.tohoku.ac.jp


Secondary IDs

Secondary IDs

NO

Study ID_1


Org. issuing International ID_1


Study ID_2


Org. issuing International ID_2


IND to MHLW

2016.4.28., 3回


Institutions

Institutions

東北大学病院(宮城県)、大阪大学医学部附属病院(大阪府)


Other administrative information

Date of disclosure of the study information

2016 Year 05 Month 13 Day


Related information

URL releasing protocol

http://www.neurol.med.tohoku.ac.jp/cgi-bin/dayori/webdir/99.html

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 02 Month 26 Day

Date of IRB

2016 Year 03 Month 28 Day

Anticipated trial start date

2016 Year 06 Month 01 Day

Last follow-up date

2021 Year 12 Month 08 Day

Date of closure to data entry

2022 Year 04 Month 15 Day

Date trial data considered complete

2022 Year 05 Month 23 Day

Date analysis concluded

2022 Year 08 Month 10 Day


Other

Other related information



Management information

Registered date

2016 Year 04 Month 25 Day

Last modified on

2022 Year 05 Month 09 Day



Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000025102


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name