A Phase I Study of OCV-501 in Acute Myeloid Leukemia Patients

• Sponsor: Otsuka Pharmaceutical Co., Ltd. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01440920
• Collaborator: (none)
• Enrollment: 9
• Location: 1
• Arms: 3
• Duration (Months): 22
• Patients Per Site Per Month: 0.4

Study Details

Study Description

Brief Summary

The purpose of this study is to assess the safety, tolerability of OCV-501 in patients with acute myeloid leukemia (AML) who achieved complete remission after induction regimen and who completed a standard consolidation therapy.

Condition or Disease	Intervention/Treatment	Phase
Acute Myeloid Leukemia	Drug: OCV-501; Drug: OCV-501; Drug: OCV-501	Phase 1

Study Design

• Study Type: Interventional
• Actual Enrollment: 9 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase I Study of OCV-501 in the Treatment of Patients With Acute Myeloid Leukemia
• Study Start Date: Sep 1, 2011
• Actual Primary Completion Date: Mar 1, 2013
• Actual Study Completion Date: Jul 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1 0.3 mg	Drug: OCV-501; subcutaneously administered once a week, 4 times at the dose of 0.3 mg
Experimental: Cohort 2 1 mg	Drug: OCV-501; subcutaneously administered once a week, 4 times at the dose of 1 mg
Experimental: Cohort 3 3 mg	Drug: OCV-501; subcutaneously administered once a week, 4 times at the dose of 3 mg

Outcome Measures

Primary Outcome Measures

1. Occurrence of Dose Limiting Toxicities [4 Weeks]

   Dose limiting toxicity (DLT) was defined as any of the following adverse events occurring by Day 29 (7 days after the last investigational medicinal product [IMP] administration) of this trial for which a causal relationship to the IMP could not be ruled out. Severity of the adverse events was evaluated in accordance with the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) ver. 4.0. Blood toxicity did not include hematology parameters of laboratory tests. Non-blood toxicities ≥ Grade 3, excluding cases of anorexia, nausea, vomiting, diarrhea, and constipation where it is possible to continue the clinical trial by use of supportive therapy Blood toxicities ≥ Grade 4, although febrile neutropenia ≥ Grade 3 will be counted as DLT.

Secondary Outcome Measures

1. Recurrence Based on the Response Evaluation Criteria by the International Working Group [4 weeks]

   A case will be designated as relapse if any of the following occur. Reappearance of leukemic blast cells in the peripheral blood or ≥5% blast cells in the bone marrow after complete remission (morphologic relapse).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 60 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Key Inclusion Criteria:

• Patients with acute myeloid leukemia including patients with secondary leukemia. However, the patients with MDS apparently evolved itno AML and patients with AML accompanied by t(15;17)(q22;q12),(PML/RARalpha) , should be excluded.

• Patients who achieved the first complete remission after the induction regimen and finished a standard consolidation therapy.

• Age: ≥ 60years of age(at the time of signature of the informed consent form)

• Sex: Male and Female

• Patients who are capable of giving informed consent

• Patient's blasts cells show expression of WT1mRNA, detected by quantitative RT-PCR.

• Patients must be one of the following HLA DRB1 types: HLA-DRB1*01:01, *04:05, *15:01, *15:02, *08:03 and *09:01.

Key Exclusion Criteria:

• Patients who are scheduled for a bone marrow transplantation

• Patients who were administered exceeded acceptable therapeutic dose of immunosuppressants and adrenal cortical steroids.

• Patients with uncontrollable active infectious diseases

• Patients with autoimmune diseases (including Hashimoto's disease, idiopathic thrombocytopenic purpura, and autoimmune hepatitis) or with a medical history of active autoimmune diseases

• Immunocompetent patients

• Patients with a complication of interstitial pneumonia or with a medical history of interstitial pneumonia

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	National Cancer Center	Tokyo		Japan

Sponsors and Collaborators

• Otsuka Pharmaceutical Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Otsuka Pharmaceutical Co., Ltd.
• ClinicalTrials.gov Identifier: NCT01440920
• Other Study ID Numbers: 311-10-001; JapicCTI-111623
• First Posted: Sep 27, 2011
• Last Update Posted: Mar 8, 2021
• Last Verified: Feb 1, 2021

• Keywords provided by Otsuka Pharmaceutical Co., Ltd.: Acute myeloid leukemia; WT1
• Additional relevant MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute

Study Results

Participant Flow

Arm/Group Title	Cohort 1	Cohort 2	Cohort 3
Arm/Group Description	subcutaneously administered once a week, 4 times at the dose of 0.3 mg	subcutaneously administered once a week, 4 times at the dose of 1 mg	subcutaneously administered once a week, 4 times at the dose of 3 mg
Period Title: Overall Study
STARTED	3	3	3
COMPLETED	3	3	3
NOT COMPLETED	0	0	0

Baseline Characteristics

Arm/Group Title	Cohort 1	Cohort 2	Cohort 3	Total
Arm/Group Description	subcutaneously administered once a week, 4 times at the dose of 0.3 mg	subcutaneously administered once a week, 4 times at the dose of 1 mg	subcutaneously administered once a week, 4 times at the dose of 3 mg	Total of all reporting groups
Overall Participants	3	3	3	9
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%	0 0%
Between 18 and 65 years	1 33.3%	0 0%	1 33.3%	2 22.2%
>=65 years	2 66.7%	3 100%	2 66.7%	7 77.8%
Sex: Female, Male (Count of Participants)
Female	2 66.7%	2 66.7%	0 0%	4 44.4%
Male	1 33.3%	1 33.3%	3 100%	5 55.6%
Race/Ethnicity, Customized (Count of Participants)
Japanese	3 100%	3 100%	3 100%	9 100%
Region of Enrollment (Count of Participants)
Japan	3 100%	3 100%	3 100%	9 100%

Outcome Measures

1. Primary Outcome

Title	Occurrence of Dose Limiting Toxicities
Description	Dose limiting toxicity (DLT) was defined as any of the following adverse events occurring by Day 29 (7 days after the last investigational medicinal product [IMP] administration) of this trial for which a causal relationship to the IMP could not be ruled out. Severity of the adverse events was evaluated in accordance with the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) ver. 4.0. Blood toxicity did not include hematology parameters of laboratory tests. Non-blood toxicities ≥ Grade 3, excluding cases of anorexia, nausea, vomiting, diarrhea, and constipation where it is possible to continue the clinical trial by use of supportive therapy Blood toxicities ≥ Grade 4, although febrile neutropenia ≥ Grade 3 will be counted as DLT.
Time Frame	4 Weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Cohort 1	Cohort 2	Cohort 3
Arm/Group Description	subcutaneously administered once a week, 4 times at the dose of 0.3 mg	subcutaneously administered once a week, 4 times at the dose of 1 mg	subcutaneously administered once a week, 4 times at the dose of 3 mg
Measure Participants	3	3	3
Number [participants]	0 0%	0 0%	0 0%

2. Secondary Outcome

Title	Recurrence Based on the Response Evaluation Criteria by the International Working Group
Description	A case will be designated as relapse if any of the following occur. Reappearance of leukemic blast cells in the peripheral blood or ≥5% blast cells in the bone marrow after complete remission (morphologic relapse).
Time Frame	4 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Cohort 1	Cohort 2	Cohort 3
Arm/Group Description	subcutaneously administered once a week, 4 times at the dose of 0.3 mg	subcutaneously administered once a week, 4 times at the dose of 1 mg	subcutaneously administered once a week, 4 times at the dose of 3 mg
Measure Participants	3	3	3
Number [participants]	0 0%	0 0%	0 0%

Adverse Events

Time Frame	Treatment-emergent adverse events were collected from start of the first IMP administration (Day 1) to Post-treatment observation (Day 50).
Arm/Group Title	Cohort 1		Cohort 2		Cohort 3
Arm/Group Description	subcutaneously administered once a week, 4 times at the dose of 0.3 mg		subcutaneously administered once a week, 4 times at the dose of 1 mg		subcutaneously administered once a week, 4 times at the dose of 3 mg
All Cause Mortality
	Cohort 1		Cohort 2		Cohort 3
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0%)		0/3 (0%)		0/3 (0%)
Serious Adverse Events
	Cohort 1		Cohort 2		Cohort 3
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0%)		0/3 (0%)		0/3 (0%)
Other (Not Including Serious) Adverse Events
	Cohort 1		Cohort 2		Cohort 3
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	3/3 (100%)		3/3 (100%)		3/3 (100%)
Blood and lymphatic system disorders
Anaemia	0/3 (0%)		0/3 (0%)		1/3 (33.3%)
Leukopenia	0/3 (0%)		0/3 (0%)		1/3 (33.3%)
Lymphopenia	0/3 (0%)		0/3 (0%)		1/3 (33.3%)
Neutropenia	0/3 (0%)		0/3 (0%)		1/3 (33.3%)
Thrombocytopenia	0/3 (0%)		0/3 (0%)		1/3 (33.3%)
Eye disorders
Cataract	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Vitreous floaters	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Gastrointestinal disorders
Stomatitis	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
General disorders
Injection site erythema	3/3 (100%)		3/3 (100%)		3/3 (100%)
Injection site induration	2/3 (66.7%)		2/3 (66.7%)		3/3 (100%)
Injection site mass	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Injection site pain	0/3 (0%)		1/3 (33.3%)		0/3 (0%)
Injection site pruritus	1/3 (33.3%)		2/3 (66.7%)		0/3 (0%)
Infections and infestations
Upper respiratory tract infection	1/3 (33.3%)		1/3 (33.3%)		0/3 (0%)
Investigations
Eosinophil count increased	0/3 (0%)		1/3 (33.3%)		0/3 (0%)
Lymphocyte count decreased	1/3 (33.3%)		1/3 (33.3%)		1/3 (33.3%)
Neutrophil count decreased	0/3 (0%)		1/3 (33.3%)		1/3 (33.3%)
Platelet count decreased	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
White blood cell count decreased	1/3 (33.3%)		0/3 (0%)		1/3 (33.3%)
Protein urine present	0/3 (0%)		0/3 (0%)		1/3 (33.3%)
Metabolism and nutrition disorders
Hyperlipidaemia	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Musculoskeletal and connective tissue disorders
Joint swelling	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Nervous system disorders
Dizziness	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Headache	0/3 (0%)		1/3 (33.3%)		0/3 (0%)
Hypoaesthesia	1/3 (33.3%)		0/3 (0%)		0/3 (0%)
Skin and subcutaneous tissue disorders
Dermatitis	0/3 (0%)		1/3 (33.3%)		0/3 (0%)
Erythema	1/3 (33.3%)		0/3 (0%)		0/3 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.