UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000014918 |
|---|---|
| Receipt number | R000017293 |
| Scientific Title | Gene Therapy using Intramuscular Administration of AMG0001 in Patients with Peripheral Arterial Disease |
| Date of disclosure of the study information | 2014/08/30 |
| Last modified on | 2020/09/23 17:14:10 |
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Basic information
Public title
Gene Therapy using Intramuscular Administration of AMG0001 in Patients with Peripheral Arterial Disease
Acronym
Angiogenic Gene Therapy with HGF Gene
Scientific Title
Gene Therapy using Intramuscular Administration of AMG0001 in Patients with Peripheral Arterial Disease
Scientific Title:Acronym
Angiogenic Gene Therapy with HGF Gene
Region
| Japan |
Condition
Condition
Arteriosclerosis Obliterans (ASO)
Buerger's disease (Thromboangiitis Obliterans: TAO)
Classification by specialty
| Medicine in general | Cardiology | Vascular surgery |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
The effectiveness and safety of AMG0001 for arteriosclerosis obliterans (ASO) and Buerger's disease will be examined.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Improvement of pain at rest and ischemic ulcer
Key secondary outcomes
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 | Gene |
Interventions/Control_1
An AMG0001 formulation (2.5 mg/ml, 2.1 ml/vial) will be prepared before use and administered to 8 sites with ischemia in the target limb, 0.5 mg of AMG0001 per site (total dose: 4.0 mg).
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
| 85 | years-old | > |
Gender
Male and Female
Key inclusion criteria
(1) Patients who personally give informed consent in writing
(2) Patients aged 20 or older but younger than 85
(3) Patients who have a stenosis or occlusion in superficial femoral artery, popliteal artery, or artery below the popliteal of the treated limb confirmed by MRA or CTA.
(4) The mean ankle pressure is less than 70 mmHg or ABI is not more than 0.6 during the observation period. (However, this shall not apply in the case of Buerger's disease.)
(5) Those who have the following clinical symptoms due to stenosis or occlusion.
･ Rest pain(Fontaine III)
VAS is at least 20 mm during the observation period
･ Ulcer(Fontaine IV)
(6) Patients in whom revascularization in the treated limb is difficult, or patients who are determined that the adaptation is not impossible but there are some risks in surgery
(7) Patients showing no symptom of improvement in the treated limb despite the conduct of conventional medical treatment or intervention for more than two weeks in the observation period after obtaining informed consent. The conventional medical treatment or intervention needs to be continued more than two weeks before obtaining the informed consent.
(8) Patients who agree to contraception by a sperm passage blocking method until the end of Week 12 of treatment after obtaining informed consent
(9) Inpatient, outpatient allowed
Key exclusion criteria
(1) Patients with a necrotized ulcer and/or an ulcer with exposed tendon or bone in the treated or non-treated limb
(2) Patients in whom alcohol or drug dependence had been noted 90 days or less before obtaining informed consent
(3) Patient with malignant tumor history or merger. Patients with no recurrence of breast cancer more than 10 years, and with no recurrence of all other tumor types before obtaining informed consent, may be enrolled in the clinical research. Patients must been completed the cancer screening tests as defined in the protocol.
(4) Patient with serious cardiac, renal or hematological disease, but excluding plateau chronic maintenance dialysis patients.
(5) Patients with HIV antigen or antibody positive
(6) Patients who underwent revascularization or amputation in the treated or non-treated limb, excluding small incision, necrotomy or onychectomy etc.
(7) Patients who underwent sympathectomy or sympathetic block 90 days or less before obtaining informed consent
(8) Patients with an invasive infectious disease which is difficult to control with antibiotics
(9) Patients with proliferative diabetic retinopathy of untreated or treatment-resistant, or patients with neovascularization-type age-related macular degeneration
(10) Patients with diabetic neuropathy (diffuse symmetrical neuropathy)
(11) Participation in another clinical trial 30 days or less before obtaining informed consent
(12) Past history of gene therapy excluding AMG0001
(13) Pregnant or breast-feeding women, women with suspected pregnancy, and women who desire to become pregnant during the clinical research
(14) Patients who were judged to be unsuitable for the clinical research by the attending physician
Target sample size
6
Research contact person
Name of lead principal investigator
| 1st name | Hiromi |
| Middle name | |
| Last name | Rakugi |
Organization
Osaka University Hospital
Division name
Geriatrics and Hypertension
Zip code
565-0871
Address
B-6, 2-2 Yamadaoka Suita,Osaka
TEL
06-6879-3852
rakugi@geriat.med.osaka-u.ac.jp
Public contact
Name of contact person
| 1st name | Munehisa |
| Middle name | |
| Last name | Shimamura |
Organization
Osaka University Graduate School of Medicine
Division name
Department of Health Development and Medicine
Zip code
565-0871
Address
2-2 Yamada-oka, Suita, Osaka
TEL
06-6210-8359
Homepage URL
shimamuu@cgt.med.osaka-u.ac.jp
Sponsor or person
Institute
Osaka University Hospital
Institute
Department
Personal name
Funding Source
Organization
None
Organization
Division
Category of Funding Organization
Self funding
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
AnGes MG, Inc.
IRB Contact (For public release)
Organization
Department of Medical Innovation Osaka University Hospital, Osaka University Hospital
Address
Yamada-oka 2-2, Suita, Osaka, 565-0871, Japan
Tel
06-6210-8296
irb-jimu@hp-mctr.med.osaka-u.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
Institutions
Institutions
Other administrative information
Date of disclosure of the study information
| 2014 | Year | 08 | Month | 30 | Day |
Related information
URL releasing protocol
https://jrct.niph.go.jp/latest-detail/jRCTs053180162
Publication of results
Published
Result
URL related to results and publications
https://www.mhlw.go.jp/content/12401000/000350779.pdf
Number of participants that the trial has enrolled
6
Results
The rate of improvement in the primary endpoint "pain at rest or ulcer size at Week 12 (or at discontinuation") after the observation period was 75% (3 of 4 subjects).
Adverse events occurred in 5 of 6 patients (9 events) and adverse reactions occurred in 2 of 6 patients (4 events) during the treatment period to the follow-up period. Serious adverse events occurred in 1 of 6 patients (1 event). One serious adverse event occurred, but none of the AEs required discontinuation of AMG0001.
Results date posted
| 2020 | Year | 09 | Month | 23 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
| 2018 | Year | 05 | Month | 28 | Day |
Baseline Characteristics
Peripheral arterial disease or Buerger's disease (limited to those with a Fontaine classification III or IV that are difficult to use for vascular reconstruction and intravascular treatment)
Participant flow
AMG0001 was administered intramuscularly to 8 sites (4.0 mg in total) at 0.5 mg per site relative to the ischemic site of the target limb. The treatment was given twice, 4 weeks apart. A third dose was allowed if no improvement tendency was observed after 8 weeks of treatment, if the patient's condition before AMG0001 administration reached the improvement criteria described in the "Primary Efficacy Endpoint," but certain symptoms remained and the third dose was considered necessary, or if the patient's condition reached the improvement criteria described in the "Primary Efficacy Endpoint," but the change in symptoms over time was judged to be associated with instability based on the criteria described in the "Primary Efficacy Endpoint." Efficacy and safety assessments were performed 12 weeks after the first dose ofAMG0001.
In the follow-up period (from the end of the follow-up period to 12 months after the first administration) and the prognostic survey period (from the end of the follow-up period to 24 months after the first administration), efficacy analyses were performed on the data at 6 and 12 months of follow-up.
Adverse events
From the treatment period to the follow-up period, 9 adverse events occurred in 5 of 6 patients (83.3%), and 4 adverse drug reactions occurred in 2 of 6 patients (33.3%). One serious adverse event occurred in 1 of 6 patients (16.7%). One serious adverse event occurred, but none of the AEs required discontinuation of AMG0001. Two SOC adverse events were classified as musculoskeletal and connective tissue disorders, while the other adverse events were one occurrence and not clinically significant.
Outcome measures
1. Primary endpoint
Percentage of improvement in rest pain VAS (visual analog scale) or ulcer size at 12 weeks (or at discontinuation) of the follow-up period
2. Secondary endpoint
2.1 Efficacy evaluation
Rest pain; Ulcers; ABI (ankle-brachial systolic pressure index); Fontaine classfication; Frequency of analgesic-free patients
2.2 Safety evaluation
Adverse events, Adverse reactions, and Serious adverse events
For follow-up results, see the following URLs:
https://www.mhlw.go.jp/content/10808000/000584412.pdf
Plan to share IPD
Not applicable
IPD sharing Plan description
Not applicable
Progress
Recruitment status
Completed
Date of protocol fixation
| 2014 | Year | 08 | Month | 07 | Day |
Date of IRB
| 2014 | Year | 02 | Month | 20 | Day |
Anticipated trial start date
| 2014 | Year | 09 | Month | 08 | Day |
Last follow-up date
| 2019 | Year | 04 | Month | 17 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2014 | Year | 08 | Month | 21 | Day |
Last modified on
| 2020 | Year | 09 | Month | 23 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017293
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