- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02808793
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002
March 12, 2019 updated by: Allakos Inc.
A Phase 1, Single Ascending Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AK002 in Patients With Indolent Systemic Mastocytosis
This is a Phase 1 study to investigate the safety and tolerability of AK002 in patients with indolent systemic mastocytosis (ISM).
Study Overview
Study Type
Interventional
Enrollment (Actual)
25
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Berlin, Germany
- Charite - Universitatsmedizin Berlin
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 65 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Provided written informed consent
- Male or female aged ≥18 and ≤65 years at the time of signing the informed consent form
- Confirmed diagnosis of ISM based on World Health Organization (WHO) criteria (Appendix 1)
Presence of at least 1 of the following SM related symptoms:
- Flushing (at least 1 episode per week)
- Pruritus (minimum MAS2 score of 4) (Appendix 2)
- Diarrhea (minimum MAS2 score of 4) (Appendix 2)
- Anaphylaxis (at least 1 episode [grade 2 or higher] within the last 12 months)
- Serum total tryptase exceeded 15 ng/mL* at 2 or more measurements obtained 1 or more months apart within the last 2 years (*Note: this varies from the minor criterion of "persistently exceeds 20 ng/mL" in the WHO criteria for diagnosis of ISM)
- Willing and able to comply with the study procedures and visit schedule, including follow-up visits
- Able to communicate effectively with the study site personnel
- Negative Screening urine drug tests (alcohol, amphetamines/methamphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine, cotinine, methadone, methaqualone, opiates, phencyclidine)
- Negative Screening ova and parasite test
- Determined by the Investigator to be in good health as documented by the medical history, physical examination (PE), vital sign assessments, 12- lead ECG, clinical laboratory assessments, and by general observations
- Women of child bearing potential, must be using highly effective methods of birth control (failure rate <1% per year when used consistently and correctly) at least 4 weeks prior to Screening until Day 85. Women should be informed of the potential risks associated with becoming pregnant while enrolled. Accepted forms of contraception are implants, injectables, combined oral contraceptives, and some intrauterine devices (IUDs). In addition, a barrier method must always be used concomitantly to the highly effective method. Double-barrier is not considered a highly effective method. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) is not an acceptable means of contraception. Female patients are considered to not be of child-bearing potential when they are post-menopausal for at least 2 years with follicle-stimulating hormone (FSH) levels >40 mIU/mL, are surgically sterilized, or have undergone hysterectomy.
- Male patients with female partners of childbearing potential must agree to use a condom without spermicide during sexual activity with female partners of childbearing potential. Female sexual partners of male patients must be willing to avoid pregnancy according to the above described methods.
Exclusion Criteria:
- Known hypersensitivity to any constituent of the study drug
- Presence of an associated hematologic non-mast-cell lineage disorder or MC leukemia
- Any disease or condition (medical or surgical) which, in the opinion of the Investigator, might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, hepatic, skeletal, or central nervous system; or other conditions that may interfere with the absorption, distribution, metabolism or excretion of AK002, or would place the patient at increased risk
- The presence of abnormal laboratory values considered to be clinically significant by the Investigator
- Participation in a concurrent interventional study with the last intervention occurring within 30 days prior to administration of study drug (90 days or 5 half-lives, whichever is longer, for biologic products)
- Treatment with chemotherapy or radiotherapy in the preceding 6 months
- Treatment for a clinically significant helminthic parasitic infection within 6 months of screening
- Use during the 7 days before Screening (or 5 half-lives, whichever is longer) or expected to require the use of angiotensin converting enzyme (ACE) inhibitors or beta blockers
- Use during the 30 days before Screening (or 5 half lives, whichever is longer) or expected to require the use of omalizumab, immunosuppressive drugs, or systemic corticosteroids with a daily dose >10 mg prednisone or equivalent
- Vaccination with live attenuated vaccines within 30 days prior to initiation of treatment in the study, during the treatment period, or vaccination expected within 5 half-lives (4 months) of the study drug administration
- Donation or loss of >500 mL of blood within 56 days prior to administration of study drug or donation of plasma within 7 days prior to administration of study drug
- Has not refrained from excessive caffeine consumption (>3 cups of coffee per day or equivalent) for 48 hours prior to study drug administration and agreed to this do so throughout the inpatient period
- Positive hepatitis serology results, except for vaccinated patients or patients with past but resolved hepatitis, at Screening
- Positive HIV serology results at Screening
- Any other reason that in the opinion of the Investigator or the Medical Monitor makes the patient unsuitable for enrollment
- Patient is vulnerable (e.g., patient kept in detention)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: AK002
IV dose of AK002
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To evaluate the safety and tolerability by evaluating Clinical laboratory parameters and adverse events assessed using the CTCAE version 4
Time Frame: From study start to Day 85 or early term visit
|
From study start to Day 85 or early term visit
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Evaluate PK of AK002 in patients with ISM
Time Frame: Through out the study from baseline to Day 85 or early term visit
|
Through out the study from baseline to Day 85 or early term visit
|
Evaluate the change from baseline in absolute peripheral counts of eosinophils and basophils.
Time Frame: Through out the study from screening to Day 85 or early term visit
|
Through out the study from screening to Day 85 or early term visit
|
Evaluate the change from baseline in serum tryptase and eosinophil grande protein levels.
Time Frame: Through out the study from screening to Day 29 or early term visit
|
Through out the study from screening to Day 29 or early term visit
|
Measure changes form baseline in the 24-hour urine histamine metabolites.
Time Frame: Starting pre dose on day -1 to days 1, 3 and 4
|
Starting pre dose on day -1 to days 1, 3 and 4
|
Mastocytosis Quality of Life Questionnaire
Time Frame: Through out the study from screening to Day 85 or early term visit
|
Through out the study from screening to Day 85 or early term visit
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Marcus Maurer, MD, Department of Dermatology and Allergy, Charité - Universitätsmedizin Berlin
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 1, 2016
Primary Completion (Actual)
December 1, 2018
Study Completion (Actual)
December 1, 2018
Study Registration Dates
First Submitted
June 16, 2016
First Submitted That Met QC Criteria
June 17, 2016
First Posted (Estimate)
June 22, 2016
Study Record Updates
Last Update Posted (Actual)
March 13, 2019
Last Update Submitted That Met QC Criteria
March 12, 2019
Last Verified
March 1, 2019
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- AK002-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Indolent Systemic Mastocytosis
-
University Hospital, ToulouseRecruitingMastocytosis, Indolent SystemicFrance
-
University Hospital, ToulouseNot yet recruiting
-
Blueprint Medicines CorporationActive, not recruitingIndolent Systemic MastocytosisCanada, United States, Spain, United Kingdom, Germany, Belgium, Switzerland, Italy, Netherlands, Denmark, France, Norway, Sweden
-
AB ScienceRecruitingIndolent Systemic MastocytosisFrance, Netherlands, Germany, United Kingdom, Russian Federation, Poland, Romania, Ukraine
-
Patara PharmaCompletedSystemic Mastocytosis | Mastocytosis | Indolent Systemic MastocytosisFrance, Germany, Spain, Italy, Netherlands
-
University Medical Center GroningenUnknown
-
AB ScienceCompletedIndolent Systemic MastocytosisFrance, United States
-
University Hospital, ToulouseCompletedIndolent Systemic Mastocytosis | Cutaneous MastocytosisFrance
-
National Institute of Allergy and Infectious Diseases...CompletedIndolent Systemic MastocytosisUnited States
-
Cogent Biosciences, Inc.RecruitingMastocytosis | Mastocytosis, Systemic | SSM | Mastocytosis, IndolentUnited States, Italy, Canada, Australia, Spain, Switzerland, Norway, United Kingdom, Belgium, Poland, Netherlands, Germany, France, Czechia, Greece, Ireland
Clinical Trials on AK002
-
Allakos Inc.Active, not recruitingChronic Spontaneous UrticariaUnited States, Germany, Poland
-
Allakos Inc.Completed
-
Allakos Inc.CompletedChronic UrticariaUnited States, Germany
-
Allakos Inc.Active, not recruitingAtopic DermatitisUnited States, Germany
-
Allakos Inc.CompletedEosinophilic Gastritis | Eosinophilic DuodenitisUnited States
-
Allakos Inc.CompletedEosinophilic Gastritis | Eosinophilic GastroenteritisUnited States
-
Allakos Inc.CompletedEosinophilic Gastroenteritis | Eosinophilic DuodenitisUnited States
-
Allakos Inc.WithdrawnEosinophilic Gastritis | Eosinophilic DuodenitisUnited States
-
Allakos Inc.CompletedEosinophilic Gastritis | Eosinophilic Gastroenteritis | Eosinophilic DuodenitisUnited States
-
Allakos Inc.CompletedEosinophilic Gastritis | Eosinophilic DuodenitisUnited States