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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01211691




Registration number
NCT01211691
Ethics application status
Date submitted
24/09/2010
Date registered
29/09/2010
Date last updated
17/11/2015

Titles & IDs
Public title
Study of KB004 in Subjects With Hematologic Malignancies (Myelodysplastic Syndrome, MDS, Myelofibrosis, MF)
Scientific title
Study of the Anti-EphA3 Monoclonal Antibody KB004 in Subjects With EphA3-Expressing Hematologic Malignancies
Secondary ID [1] 0 0
KB004-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndrome (MDS) 0 0
Myelofibrosis (MF) 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Blood 0 0 0 0
Other blood disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KB004, Monoclonal Antibody

Experimental: Phase 1 dose levels: KB004 - IV infusion 1x Weekly for a 21 day dosing cycle
Subjects with heme malignancies will be assigned to one of 11 planned KB004 (dose levels (20mg, 40mg, 70mg, 100mg, 140mg, 190mg, 250mg, 330mg)

Experimental: Phase 2 dose levels: KB004 - IV infusion 1x Weekly for a 21 day dosing cycle
Subjects will be assigned to the recommended Phase 2 dose of 250 mg


Treatment: Drugs: KB004, Monoclonal Antibody
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1: Determine a possible maximum tolerated dose (MTD)
Timepoint [1] 0 0
Once weekly for the first three weeks of study treatment
Primary outcome [2] 0 0
Phase 2: To characterize preliminary clinical activity based on the International Working Group (IWG) criteria specific to the hematologic malignancy
Timepoint [2] 0 0
Evaluations at designated timepoints
Secondary outcome [1] 0 0
Phase 1: Examine clinical activity
Timepoint [1] 0 0
Evaluations at designated timepoints
Secondary outcome [2] 0 0
Phase 1/2: Safety and Tolerability
Timepoint [2] 0 0
Duration of study participation
Secondary outcome [3] 0 0
Phase 1/2: Pharmacokinetic profile
Timepoint [3] 0 0
Cycle 1: multiple timepoints. Thereafter, single samples at designated cycles
Secondary outcome [4] 0 0
Phase 1/2: Assess immunogenicity
Timepoint [4] 0 0
Cycle 1: multiple timepoints. Thereafter, single samples at designated cycles

Eligibility
Key inclusion criteria
Key Inclusion Criteria (Phase 1):

- Confirmed hematologic malignancy, including Acute Myeloid Leukemia (AML), Chronic
Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML), Acute Lymphocytic Leukemia
(ALL), Myelodysplastic Syndrome (MDS), Multiple Myeloma (MM), Myelofibrosis (MF),
Myeloproliferative Neoplasms (MPN) or MDS/MPN overlap diseases. (Once Phase 2 has started
subjects with AML will be eligible for inclusion in the Phase 1 portion of the study only
if their malignancy has been shown to have c-Cbl mutation, trisomy 3, trisomy 11, inv(16),
or elevated FLT3. [Other AML and subjects with MDS will no longer be eligible for inclusion
in the Phase 1 portion of the study]).

Key Inclusion Criteria (Phase 2):

- Part A: AML or MDS patients with an acceptable level of EphA3 expression

- Part B: MF patients with an acceptable level of EphA3 expression

Key Inclusion Criteria (Both Phases):

- Confirmed hematologic malignancy refractory to or progressed following standard
treatments, or subjects not considered medically suitable to receive standard of care
treatment or who refuse standard of care treatment

- Acceptable level of EphA3 expression

- Eastern Cooperative Oncology Group (ECOG) =1

- Acceptable laboratory results

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria (Both Phases):

- For subjects with AML, more than 2 prior therapies for AML (induction and
consolidation with or without a hypomethylating agent given in a maintenance setting
are considered 1 therapy)

- History of or current central nervous system (CNS) involvement that may increase risk
of bleeding

- Recent major surgery

- Ongoing surgical or wound healing complications

- Active clinically significant bleeding

- Uncontrolled hypertension

- Significant intercurrent illness

- Known history of prolonged bleeding times or platelet dysfunction

- Active infection requiring IV antibiotics, IV antifungals, or IV antivirals within 2
weeks prior to Cycle 1, Day 1

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Suspended
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/09/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2016
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
- Adelaide
Recruitment hospital [2] 0 0
- Prahran
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3007 - Prahran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Humanigen, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a global, multicenter, open-label, repeat-dose, Phase 1/2 study consisting of a Dose
Escalation Phase (Phase 1) and a Cohort Expansion Phase (Phase 2). In both phases, KB004 will
be administered by IV infusion once weekly as part of a 21-day dosing cycle.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01211691
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Morgan Lam
Address 0 0
Humanigen, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01211691