Trial | NCT04103034  Report issue

Title

A SAD/MAD Study of Safety, Tolerability and Pharmacologic Activity of BT200 in Normal Volunteers
A Single/Multiple Ascending Dose Phase 1 Study of the Safety, Tolerability and Pharmacologic Activity of BT200 in Normal Human Volunteers

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Study Participants

    112
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.
Study Started
Oct 07
2019
Primary Completion
Sep 14
2020
Study Completion
Sep 14
2020
Last Update
Apr 14
2022

Drug BT200

BT200 is a PEGylated synthetic RNA oligonucleotide

Drug Desmopressin

Sterile solution for injection

  • Other names: MINIRIN® Injection

Drug Placebo

Sterile saline for injection

BT200 0.18mg Experimental

Subjects will receive a single subcutaneous dose of BT200 0.18mg

BT200 0.6mg Experimental

Subjects will receive a single subcutaneous dose of BT200 0.6mg

BT200 1.8mg Experimental

Subjects will receive a single subcutaneous dose of BT200 1.8mg

BT200 6.0mg Experimental

Subjects will receive a single subcutaneous dose of BT200 6.0mg

BT200 12.0mg Experimental

Subjects will receive a single subcutaneous dose of BT200 12.0mg

BT200 24.0mg Experimental

Subjects will receive a single subcutaneous dose of BT200 24.0mg

BT200 24.0mg rep Experimental

Subjects will receive a single subcutaneous (SC) dose of BT200 24.0mg by gradual SC infusion

Placebo SAD Placebo Comparator

Subjects will receive a single subcutaneous dose of placebo

BT200 loading dose 24.0mg, maintenance doses of 12.0 mg Experimental

Subjects will receive an initial subcutaneous loading doses of BT200 24.0mg followed by 4 weekly (every 7 days) maintenance doses of BT200 12.0mg

BT200 loading doses 48.0mg, maintenance doses of 24.0 mg Experimental

Subjects will receive an initial subcutaneous loading dose of BT200 48mg followed by 4 weekly (every 7 days) maintenance doses of BT200 24mg

Placebo MAD Placebo Comparator

Subjects will receive an initial subcutaneous loading dose of Placebo followed by 4 weekly (every 7 days) maintenance doses of placebo

BT200 48.0mg + desmopressin challenge Experimental

Subjects will receive a single subcutaneous dose of BT200 48.0mg followed by IV infusion (over 30 min) of 0.3µg/kg desmopressin administered 24 hours after single dose of BT200

Placebo + desmopressin challenge dose Placebo Comparator

Subjects will receive a single subcutaneous dose of placebo followed by IV infusion (over 30 min of 0.3µg/kg desmopressin administered 24 hours after single dose of placebo

Placebo infusion Placebo Comparator

Subjects will receive a single IV dose of placebo administered over 24 hours

BT200 36.0mg Experimental

Subjects will receive a single subcutaneous (SC) dose of BT200 36.0mg by gradual SC infusion

BT200 48.0 mg Experimental

Subjects will receive a single subcutaneous dose (SC) of BT200 48.0mg by gradual SC infusion

Criteria 

Inclusion Criteria:

Healthy male or female volunteers, age ≥ 18 years old at screening
If female, must be post-menopausal or status post hysterectomy
Able to comprehend and to give informed consent
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:

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
Clinically relevant abnormal findings on physical examination or clinically relevant laboratory abnormalities
History of infusion hypersensitivity reactions, significant drug allergy, or anaphylactic reactions
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
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
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
No Results Posted