DRUG
BT200
BT200 is a PEGylated synthetic RNA oligonucleotide
Status
Completed
Phase
Phase 1
Enrollment
112
Locations
1
Results
Posted
Publications
3
Study summary
Study BT200-01 is a first in human (FIH) study in male and female normal human volunteers (NHVs) that uses an Integrated Protocol Design. This Phase 1 study will comprise 4 sub-parts: Part A, a single ascending dose (SAD) study; Part B, a multiple ascending dose (MAD) study; Part C, a desmopressin challenge study to explore (i) whether desmopressin could be used as an antidote, and/or (ii) whether desmopressin stimulated vonWillebrand Factor (VWF) release is overcome with increasing BT200 doses; and Part D, a relative bioavailability (BA) study. The primary objective of this study is to assess the safety and tolerability profile of BT200 in NHVs.
Interventions
DRUG
BT200 is a PEGylated synthetic RNA oligonucleotide
DRUG
Sterile solution for injection
DRUG
Sterile saline for injection
Timeline
First posted
Sep 25, 2019
Study start
Oct 7, 2019
Primary completion
Sep 14, 2020
Study completion
Sep 14, 2020
Results posted
Mar 18, 2024
Registry updated
Mar 18, 2024
Outcomes
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
Time frame · Baseline through 8 weeks after dosing up to 56 days
Any AE or bleeding event related to study treatment
Measured Area Under the Curve (AUC)
Time frame · Baseline through 8 weeks after dosing up to 56 days
Measured Area Under the Curve at timepoints pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d post dose
Maximum Plasma Concentration (Cmax)
Time frame · Baseline through 8 weeks after dosing up to 56 days
Maximum plasma Concentration measured at pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d
Time to Maximum Plasma Concentration (Tmax)
Time frame · Baseline through 8 weeks after dosing up to 56 days
Time to maximum plasma concentration measured at pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d
Eligibility
Inclusion Criteria: 1. Healthy male or female volunteers, age ≥ 18 years old at screening 2. If female, must be post-menopausal or status post hysterectomy 3. Able to comprehend and to give informed consent 4. Able to cooperate with the Investigator, to comply with the requirements of the study, and to complete the full sequence of protocol-related procedures Exclusion Criteria: 1. Clinically significant medical history (including von Willebrand Disease, thrombocytopathy, or any type of bleeding diathesis) or ongoing chronic illness that would jeopardize the safety of the subject or compromise the quality of the data derived from his/her participation in this study 2. Clinically relevant abnormal findings on physical examination or clinically relevant laboratory abnormalities 3. History of infusion hypersensitivity reactions, significant drug allergy, or anaphylactic reactions 4. Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the subject to be able to comply fully with study procedures 5. Use of medication during 2 weeks before the start of the study, which in the judgment of the Investigator may adversely affect the subject's welfare or the integrity of the study's results 6. Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 elimination half-lives (whichever is longer) prior to treatment start
Study locations
Austria. Showing up to 24 locations stored in the fast local snapshot.
Medical University of Vienna
Vienna, Austria
Publications
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