Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenic purpura (ITP) patients
- Conditions
- Immune thrombocytopenic purpuraMedDRA version: 20.1Level: PTClassification code 10074667Term: Immune thrombocytopenic purpuraSystem Organ Class: 10005329 - Blood and lymphatic system disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2018-003534-32-ES
- Lead Sponsor
- Momenta Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 70
1. Participant must be = 18 and = 55 years of age inclusive, at the time of signing the informed consent form (ICF).
2. Good health as indicated by medical history (without hemolysis or thrombosis that may impact current study), physical examination, vital signs (with systolic blood pressure below 140 mmHg), clinical laboratory tests, and 12-lead electrocardiogram, and all abnormal findings are assessed as not clinically significant by the Investigator.
3. Body weight must be between 50 and 110 kg, inclusive, and body mass index (BMI) between 18.5 and 30 kg/m2, inclusive, at screening.
4. Healthy male and females are eligible.
a.Male participants:
• If male, surgically or biologically sterile. If not sterile, agreement to use an acceptable form of birth control with sexual partner or abstain from sexual relations for 100 days following the last treatment.
b. Female participants:
• A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies:
? Not a woman of childbearing potential (WOCBP) (Surgically sterilized via hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or of non-childbearing potential (i.e., postmenopausal for at least 1 year).
OR
? A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 90 days after the last dose of study intervention.
• Of child-bearing potential, with a fertile male sexual partner, willing to use 2 adequate methods of contraception from 30 days prior to dosing until 90 days after the last dose of study treatment. Adequate contraception is defined as using hormonal contraceptives or an intrauterine device combined with at least 1 of the following forms of contraception: a diaphragm or cervical cap, or a condom. Also, total abstinence, in accordance with the lifestyle of the subject, is acceptable.
5. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
6. Ability to speak, read, and understand primary medical care language(s) at the site.
7. If female, a negative serum pregnancy test at screening and a negative urine or serum pregnancy test at check-in to the clinic for the baseline visit, and not nursing or planning a pregnancy through 90 days following the last dose of study medication.
Patients:
1. Participant must be 18 to 64 years of age, inclusive, at the time of signing the ICF.
2. Diagnosed with ITP (with or without splenectomy) according to the Hematology Guidelines for at least 3 months prior to screening.
3. Platelet count = 15 × 10^9/L and < 50 × 10^9/L within 96 hours prior to start of study drug infusion on Day 1
4. Body weight must be < 110 kg
5. Maintenance immunosuppressive therapy, steroid therapy, cyclosporine A, mycophenolate mofetil, azathioprine, thrombopoietin receptor agonists, or danazol are allowed, but the dosages of all these medications must be stable for at least 4 weeks prior to Visit 1 (Day 1).
6. No history of clotting disorder other than ITP including no myocardial infarction within the last 6 months. No arrhythmia known to increase the risk of thrombotic events (eg, atrial fibrillation).
For inclusion criteria patients nr. 07 - 10 please see protocol; no more possibility here.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 E
1. Previously received M254.
2. History of any drug allergy, hypersensitivity, or intolerance to any drug product that in the opinion of the Investigator would place the subject at particular risk and compromise the safety of the subject in the
study.
3. History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, oncologic, psychiatric disease, or any other condition, that in the opinion of the Investigator would jeopardize the safety of the subject or the validity of the study results.
4. History of splenectomy, asthma (with the exception of childhood asthma that has resolved), chronic obstructive pulmonary disease, or recurrent or current gastrointestinal or respiratory infections.
5. Any illness within 5 days, or clinically significant airway infections within 30 days, prior to first study drug dosing.
6. On fluid restriction.
7. Any prescription medication(s) within 14 days of dose administration (or 5 half-lives, whichever is longer) or any non-prescribed systemic or topical medication (including any herbal product) within 7 days prior to dose administration.
8. Plans to participate in another clinical trial while enrolled in this study and/or received an investigational drug and/or device within 60 days prior to dose administration.
9. Positive urine drug screen at screening.
10. Positivity for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) at screening.
11. History or current diagnosis of substance dependence (except nicotine and caffeine) or alcohol abuse over the past 2 years, according to the criteria of the Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision (DSM-IV-TR).
12. Smokes or has smoked more than 5 cigarettes per day in the last 90 days and is unable to stop smoking during in-patient observation period in clinic.
13. Unwilling to abstain from alcohol for at least 24 hours prior to dosing with study medication until the time of discharge from the study unit and at least 24 hours prior to each ambulatory visit.
14. Donation or significant loss of whole blood (480 mL or more) within 30 days or plasma within 14 days prior to admission.
15. Vaccination within 1 month before dosing, or plans to receive vaccination within 3 months after the last dose.
16. Veins unsuitable for cannulation or multiple venipunctures on either arm.
17. Patients with any prior history of arterial or venous thrombosis, AND = 2 of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (eg, Factor V Leiden, antithrombin III deficiency, antiphospholipid syndrome, etc), hyperviscosity (cryoglobulines and chylomicronemia)
18. Patients with selective IgA deficiency with known anti-IgA antibodies.
Patients:
1. Patients with any prior history of arterial or venous thrombosis, AND = 2 of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (eg, Factor V Leiden, antithrombin III deficiency, antiphospholipid syndrome, etc), hyperviscosity (cryoglobulines and chylomicronemia)
2. Any clinically relevant abnormality, other than ITP, which in the opini
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method