A SAD/MAD Study of Safety, Tolerability and Pharmacologic... | NCT04103034

A SAD/MAD Study of Safety, Tolerability and Pharmacologic... | NCT04103034 | Trialant

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
BT200 0.18mg	Experimental	Subjects will receive a single subcutaneous dose of BT200 0.18mg	Drug: BT200
BT200 0.6mg	Experimental	Subjects will receive a single subcutaneous dose of BT200 0.6mg	Drug: BT200
BT200 1.8mg	Experimental	Subjects will receive a single subcutaneous dose of BT200 1.8mg	Drug: BT200
BT200 6.0mg	Experimental	Subjects will receive a single subcutaneous dose of BT200 6.0mg	Drug: BT200
BT200 12.0mg	Experimental	Subjects will receive a single subcutaneous dose of BT200 12.0mg	Drug: BT200
BT200 24.0mg	Experimental	Subjects will receive a single subcutaneous dose of BT200 24.0mg

Name	Type	Description	Arm Group Labels	Other Names
BT200	Drug	BT200 is a PEGylated synthetic RNA oligonucleotide	BT200 0.18mg BT200 0.6mg BT200 1.8mg BT200 12.0mg BT200 18.0 mg

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0	Any AE or bleeding event related to study treatment	Baseline through 8 weeks after dosing up to 56 days

Secondary Outcomes

Measure	Description	Time Frame
Measured Area Under the Curve (AUC)	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	Baseline through 8 weeks after dosing up to 56 days
Maximum Plasma Concentration (Cmax)

Contacts/Locations Module

Name	Affiliation	Role
James C Gilbert, MD	Band Therapeutics	Study Director
Ulla Derhaschnig, MD	Medical University of Vienna	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Medical University of Vienna		Vienna		1090	Austria

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
29847840	Background	Buchtele N, Schwameis M, Gilbert JC, Schorgenhofer C, Jilma B. Targeting von Willebrand Factor in Ischaemic Stroke: Focus on Clinical Evidence. Thromb Haemost. 2018 Jun;118(6):959-978. doi: 10.1055/s-0038-1648251. Epub 2018 May 30.
34818873	Result	Kovacevic KD, Grafeneder J, Schorgenhofer C, Gelbenegger G, Gager G, Firbas C, Quehenberger P, Jilma-Stohlawetz P, Bileck A, Zhu S, Gilbert JC, Beliveau M, Jilma B, Derhaschnig U. The von Willebrand factor A-1 domain binding aptamer BT200 elevates plasma levels of von Willebrand factor and factor VIII: a first-in-human trial. Haematologica. 2022 Sep 1;107(9):2121-2132. doi: 10.3324/haematol.2021.279948.

Arm/Group Information

ID	Title	Description
FG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG001	BT200 0.6mg	Subjects will receive a single subcutaneous dose of BT200 0.6mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG002	BT200 1.8mg	Subjects will receive a single subcutaneous dose of BT200 1.8mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG003	BT200 6.0mg	Subjects will receive a single subcutaneous dose of BT200 6.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG004	BT200 12.0mg	Subjects will receive a single subcutaneous dose of BT200 12.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG005	BT200 24.0mg	Subjects will receive a single subcutaneous dose of BT200 24.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG006	BT200 24.0mg Rep	Subjects will receive a single subcutaneous (SC) dose of BT200 24.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG007	Placebo SAD	Subjects will receive a single subcutaneous dose of placebo Placebo: Sterile saline for injection
FG008	BT200 Loading Dose 24.0mg, Maintenance Doses of 12.0 mg	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: BT200 is a PEGylated synthetic RNA oligonucleotide
FG009	BT200 Loading Doses 48.0mg, Maintenance Doses of 24.0 mg	Subjects will receive an initial subcutaneous loading dose of BT200 48mg followed by 4 weekly (every 7 days) maintenance doses of BT200 24mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG010	Placebo MAD	Subjects will receive an initial subcutaneous loading dose of Placebo followed by 4 weekly (every 7 days) maintenance doses of placebo Placebo: Sterile saline for injection
FG011	BT200 48.0mg + Desmopressin Challenge	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 BT200: BT200 is a PEGylated synthetic RNA oligonucleotide Desmopressin: Sterile solution for injection
FG012	Placebo + Desmopressin Challenge Dose	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 BT200: BT200 is a PEGylated synthetic RNA oligonucleotide Desmopressin: Sterile solution for injection
FG013	Placebo Infusion	Subjects will receive a single IV dose of placebo administered over 24 hours Placebo: Sterile saline for injection
FG014	BT200 36.0mg	Subjects will receive a single subcutaneous (SC) dose of BT200 36.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG015	BT200 48.0 mg	Subjects will receive a single subcutaneous dose (SC) of BT200 48.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
FG016	BT200 18.0 mg	Subjects will receive a single subcutaneous dose of BT200 18.o mg
FG017	BT200 24mg IV Infusion	Subjects will receive a single IV dose of BT200 24mg administered over 24 hours

Periods

Title

Milestones

Reasons Not Completed

Overall Study

Type	Comment	Milestone Data
STARTED		FG0006 subjects FG0016 subjects FG0026 subjects FG0036 subjects FG0046 subjects FG0056 subjects FG0066 subjects FG00720 subjects FG0086 subjects FG0096 subjects FG0104 subjects FG0116 subjects FG0122 subjects FG0132 subjects FG0146 subjects FG0156 subjects FG0166 subjects FG0176 subjects
COMPLETED		FG0006 subjects FG0016 subjects FG0026 subjects FG0036 subjects FG004
NOT COMPLETED		FG0000 subjects FG0010 subjects FG0020 subjects FG0030 subjects FG004

Baseline Characteristics Module

Arm/Group Information

ID	Title	Description
BG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
BG001	BT200 0.6mg	Subjects will receive a single subcutaneous dose of BT200 0.6mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide

Units	Counts
Participants	BG000

Baseline Measures

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Categorical			Count of Participants

Outcome Measures Module

Outcome Measures

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0

Any AE or bleeding event related to study treatment

Safety population

Posted

Count of Participants

Participants

Baseline through 8 weeks after dosing up to 56 days

ID	Title	Description
OG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG001	BT200 0.6mg	Subjects will receive a single subcutaneous dose of BT200 0.6mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide

Adverse Events Module

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 23.1	Systematic Assessment	Grade 3 SAE of back pain considered unrelated to study drug.

Term	Organ System	Source Vocabulary	Assessment Type	Notes	Statistical Information
Nasopharyngitis	Infections and infestations	MedDRA 23.1	Systematic Assessment

More Info Module

Certain Agreements

Title	Organization	Phone	Extension	Email
Tia DeFeo-Fraulini	Band Therapeutics	7819186561		tia.defeo-fraulini@bandtherapeutics.com

Uploaded Document Information

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Apr 22, 2020	Oct 6, 2022	Prot_000.pdf
SAP	No	Yes	No	Statistical Analysis Plan	Sep 17, 2020	Oct 6, 2022	SAP_001.pdf

Condition Browse Module

ID	Term
D020521	Stroke
D004617	Embolism
D013927	Thrombosis
D002537	Intracranial Arteriosclerosis

ID	Term
D002561	Cerebrovascular Disorders
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases

Intervention Browse Module

ID	Term
D003894	Deamino Arginine Vasopressin

ID	Term
D001127	Arginine Vasopressin
D014667	Vasopressins
D010909	Pituitary Hormones, Posterior
D010907	Pituitary Hormones

Title

Denominators

Categories

Title	Measurements
<=18 years	BG0000 BG0010 BG0020 BG0030 BG0041 BG0050 BG0060 BG0071 BG0080 BG0091 BG0100 BG0110 BG0120 BG0130 BG0140 BG0150 BG0160 BG0170 BG0183
Between 18 and 65 years	BG0006 BG0016 BG0026 BG0036 BG004
>=65 years	BG0000 BG0010 BG0020 BG0030 BG004

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0001 BG0010 BG0022 BG0030 BG0040 BG0050 BG0062 BG0072 BG0081 BG0090 BG0101 BG0111 BG0120 BG0130 BG0140 BG0151 BG0160 BG0170 BG01811
Male	BG0005 BG0016 BG0024 BG0036 BG004

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0000 BG0010 BG0020 BG0030 BG0040 BG0050 BG0060 BG0070 BG0080 BG0090 BG0100 BG0110 BG0120 BG0130 BG0140 BG0150 BG0160 BG0170 BG0180
Not Hispanic or Latino	BG0006 BG0016 BG0026 BG0036 BG004
Unknown or Not Reported	BG0000 BG0010 BG0020 BG0030 BG004

Race (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
American Indian or Alaska Native	BG0000 BG0010 BG0020 BG0030 BG0040 BG0050 BG0060 BG0070 BG0080 BG0090 BG0100 BG0110 BG0120 BG0130 BG0140 BG0150 BG0160 BG0170 BG0180
Asian	BG0000 BG0010 BG0020 BG0031 BG004
Native Hawaiian or Other Pacific Islander	BG0000 BG0010 BG0020 BG0030 BG004
Black or African American	BG0001 BG0010 BG0020 BG0030 BG004
White	BG0005 BG0016 BG0026 BG0035 BG004
More than one race	BG0000 BG0010 BG0020 BG0030 BG004
Unknown or Not Reported	BG0000 BG0010 BG0020 BG0030 BG004

Region of Enrollment

Number

participants

Title

Denominators

Categories

Austria

Title	Measurements
	BG0006 BG0016 BG0026 BG0036 BG0046 BG0056 BG0066 BG00720 BG0086 BG0096 BG0104 BG0116 BG0122 BG0132 BG0146 BG0156 BG0166 BG0176 BG018112

Secondary

Measured Area Under the Curve (AUC)

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

Per protocol population-(h*ug/mL)

Posted

Mean

Standard Deviation

h*ug/mL

Baseline through 8 weeks after dosing up to 56 days

ID	Title	Description
OG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG001	BT200 0.6mg	Subjects will receive a single subcutaneous dose of BT200 0.6mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG002	BT200 1.8mg	Subjects will receive a single subcutaneous dose of BT200 1.8mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG003	BT200 6.0mg	Subjects will receive a single subcutaneous dose of BT200 6.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG004	BT200 12.0mg	Subjects will receive a single subcutaneous dose of BT200 12.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG005	BT200 24.0mg	Subjects will receive a single subcutaneous dose of BT200 24.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG006	BT200 24.0mg Rep	Subjects will receive a single subcutaneous (SC) dose of BT200 24.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG007	BT200 Loading Dose 24.0mg, Maintenance Doses of 12.0 mg	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: BT200 is a PEGylated synthetic RNA oligonucleotide
OG008	BT200 Loading Doses 48.0mg, Maintenance Doses of 24.0 mg	Subjects will receive an initial subcutaneous loading dose of BT200 48mg followed by 4 weekly (every 7 days) maintenance doses of BT200 24mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG009	BT200 48.0mg + Desmopressin Challenge	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 BT200: BT200 is a PEGylated synthetic RNA oligonucleotide Desmopressin: Sterile solution for injection
OG010	BT200 36.0mg	Subjects will receive a single subcutaneous (SC) dose of BT200 36.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG011	BT200 48.0 mg	Subjects will receive a single subcutaneous dose (SC) of BT200 48.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG012	BT200 18.0 mg	Subjects will receive a single subcutaneous dose of BT200 18.o mg
OG013	BT200 24mg IV Infusion	Subjects will receive a single IV dose of BT200 24mg administered over 24 hours

Units	Counts
Participants	OG0006 OG0016 OG0026 OG003

Title

Denominators

Categories

Title	Measurements
	OG0001.159± 0.72594 OG0015.405± 2.1113 OG00211.50± 6.5663 OG003

Secondary

Maximum Plasma Concentration (Cmax)

Maximum plasma Concentration measured at pre-dose, 0.5h, 1h, 4h, 8h,14h, 24h, 48h, 72h, 96h, 168h, 14d, 21d, 28d, 42d, 56d

Per protocol population

Posted

Mean

Standard Deviation

ug/mL

Baseline through 8 weeks after dosing up to 56 days

ID	Title	Description
OG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG001	BT200 0.6mg	Subjects will receive a single subcutaneous dose of BT200 0.6mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG002	BT200 1.8mg	Subjects will receive a single subcutaneous dose of BT200 1.8mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG003	BT200 6.0mg	Subjects will receive a single subcutaneous dose of BT200 6.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG004	BT200 12.0mg	Subjects will receive a single subcutaneous dose of BT200 12.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG005	BT200 24.0mg	Subjects will receive a single subcutaneous dose of BT200 24.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG006	BT200 24.0mg Rep	Subjects will receive a single subcutaneous (SC) dose of BT200 24.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG007	BT200 Loading Dose 24.0mg, Maintenance Doses of 12.0 mg	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: BT200 is a PEGylated synthetic RNA oligonucleotide
OG008	BT200 Loading Doses 48.0mg, Maintenance Doses of 24.0 mg	Subjects will receive an initial subcutaneous loading dose of BT200 48mg followed by 4 weekly (every 7 days) maintenance doses of BT200 24mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG009	BT200 48.0mg + Desmopressin Challenge	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 BT200: BT200 is a PEGylated synthetic RNA oligonucleotide Desmopressin: Sterile solution for injection
OG010	BT200 36.0mg	Subjects will receive a single subcutaneous (SC) dose of BT200 36.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG011	BT200 48.0 mg	Subjects will receive a single subcutaneous dose (SC) of BT200 48.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG012	BT200 18.0 mg	Subjects will receive a single subcutaneous dose of BT200 18.o mg
OG013	BT200 24mg IV Infusion	Subjects will receive a single IV dose of BT200 24mg administered over 24 hours

Units	Counts
Participants	OG0006 OG0016 OG0026 OG003

Title

Denominators

Categories

Title	Measurements
	OG0000.01025± 0.0055459 OG0010.04688± 0.018522 OG0020.09432± 0.035964 OG003

Secondary

Time to Maximum Plasma Concentration (Tmax)

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

per protocol population

Posted

Mean

Standard Deviation

(h)

Baseline through 8 weeks after dosing up to 56 days

ID	Title	Description
OG000	BT200 0.18mg	Subjects will receive a single subcutaneous dose of BT200 0.18mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG001	BT200 0.6mg	Subjects will receive a single subcutaneous dose of BT200 0.6mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG002	BT200 1.8mg	Subjects will receive a single subcutaneous dose of BT200 1.8mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG003	BT200 6.0mg	Subjects will receive a single subcutaneous dose of BT200 6.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG004	BT200 12.0mg	Subjects will receive a single subcutaneous dose of BT200 12.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG005	BT200 24.0mg	Subjects will receive a single subcutaneous dose of BT200 24.0mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG006	BT200 24.0mg Rep	Subjects will receive a single subcutaneous (SC) dose of BT200 24.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG007	BT200 Loading Dose 24.0mg, Maintenance Doses of 12.0 mg	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: BT200 is a PEGylated synthetic RNA oligonucleotide
OG008	BT200 Loading Doses 48.0mg, Maintenance Doses of 24.0 mg	Subjects will receive an initial subcutaneous loading dose of BT200 48mg followed by 4 weekly (every 7 days) maintenance doses of BT200 24mg BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG009	BT200 48.0mg + Desmopressin Challenge	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 BT200: BT200 is a PEGylated synthetic RNA oligonucleotide Desmopressin: Sterile solution for injection
OG010	BT200 36.0mg	Subjects will receive a single subcutaneous (SC) dose of BT200 36.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG011	BT200 48.0 mg	Subjects will receive a single subcutaneous dose (SC) of BT200 48.0mg by gradual SC infusion BT200: BT200 is a PEGylated synthetic RNA oligonucleotide
OG012	BT200 18.0 mg	Subjects will receive a single subcutaneous dose of BT200 18.o mg
OG013	BT200 24mg IV Infusion	Subjects will receive a single IV dose of BT200 24mg administered over 24 hours

Units	Counts
Participants	OG0006 OG0016 OG0026 OG003

Title

Denominators

Categories

Title	Measurements
	OG000111.867± 111.7018 OG00180.433± 20.2385 OG00272.364± 22.0757 OG003