Current partner codePEPTIDESDE
NCT04103034·Phase 1·INTERVENTIONAL

A SAD/MAD Study of Safety, Tolerability and Pharmacologic Activity of BT200 in Normal Volunteers

Status

Completed

Phase

Phase 1

Enrollment

112

Locations

1

Results

Posted

Publications

3

Study summary

What the protocol is testing.

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

Treatment arms and agents.

DRUG

BT200

BT200 is a PEGylated synthetic RNA oligonucleotide

DRUG

Desmopressin

Sterile solution for injection

DRUG

Placebo

Sterile saline for injection

Timeline

From registration to results.

  1. First posted

    Sep 25, 2019

  2. Study start

    Oct 7, 2019

  3. Primary completion

    Sep 14, 2020

  4. Study completion

    Sep 14, 2020

  5. Results posted

    Mar 18, 2024

  6. Registry updated

    Mar 18, 2024

Outcomes

What the study measures.

Primary 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

Secondary outcomes

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

Who can take part.

Minimum age
18 Years
Maximum age
Not reported
Sex
ALL
Healthy volunteers
Yes

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

1 registered sites.

Austria. Showing up to 24 locations stored in the fast local snapshot.

Medical University of Vienna

Vienna, Austria

Related trials

More studies on Desmopressin.

Related PeptideStat pages

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