Clinical Trial Results:
A Phase 2a Multiple Dose “Basket Design” Study Of The Safety, Tolerability, And Pharmacologic Activity Of BT200 In Patients With Hereditary Bleeding Disorders
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Summary
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EudraCT number |
2020-003807-32 |
Trial protocol |
AT |
Global end of trial date |
24 Aug 2021
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Results information
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Results version number |
v2(current) |
This version publication date |
11 May 2022
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First version publication date |
20 Apr 2022
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Other versions |
v1 |
Version creation reason |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
BT200-02
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04677803 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Medical University of Vienna
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Sponsor organisation address |
Währinger Gürtel 18-20, Vienn, Austria, 1090
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Public contact |
Dept. of Clinical Pharmacology, Medical University of Vienna, 0043 140400298190, klin-pharmakologie@meduniwien.ac.at
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Scientific contact |
Dept. of Clinical Pharmacology, Medical University of Vienna, 0043 140400298190, klin-pharmakologie@meduniwien.ac.at
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
31 May 2021
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
31 May 2021
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Global end of trial reached? |
Yes
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Global end of trial date |
24 Aug 2021
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The primary objective of this study is to obtain clinical proof of mechanism for BT200 in one or more hereditary bleeding disorders.
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Protection of trial subjects |
Prior to any trial-related activity, the Investigator or an authorized physician gave the patient oral
and written information about the trial in a form that the patient was able to read and to understand.
A voluntary, signed and dated Informed Consent Form was to be obtained from the patient prior
to any trial-related activity. The patient had to consent to participate after the nature, scope, and
possible consequences of the clinical trial were explained in a form understandable to the patient.
It was also explained to the patient that he/she was free to refuse to enter the study or to withdraw
from it at any time for any reason without incurring any penalty or withholding of treatment on the
part of the Investigator.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
22 Dec 2020
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Austria: 26
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Worldwide total number of subjects |
26
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EEA total number of subjects |
26
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
25
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From 65 to 84 years |
1
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85 years and over |
0
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Recruitment
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Recruitment details |
Hereditary bleeding disorder Congenital hemophilia A w/o inhibitors on a prophylactic treatment regimen Heterozygous carriers of hem. A with subnormal FVIII levels Von Willebrand disease (VWD) Type 1 VWD type IIb (also known as “VWD Type 2b” or “type 2B VWD”) VWD Type 3 under subst. therapy Acquired von Willebrand Syndrome w/o spec inhib. | ||||||||||||||||||||||||||||||
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Pre-assignment
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Screening details |
Patients underwent the Screening Visit within 28 days prior to dosing on Day 0. All patients were to be dosed with a subcutaneous (SC) injection of 3 mg BT200 on Days 0 and 4. Thereafter, doses of the weekly SC injections were to be titrated between 3 and 9 mg based on the patient’s response. | ||||||||||||||||||||||||||||||
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Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||||||||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||||||||||||||||||||||
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Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Hemophilia A Mild/Mod | ||||||||||||||||||||||||||||||
Arm description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. Hemophilia A: titration until largest possible increase in FVIII activity before PFA-100® CADP-CT increased to 150 seconds. No up-titration from 3 mg was foreseen in severe hemophilia patients on on-demand therapy. | ||||||||||||||||||||||||||||||
Arm type |
Active comparator | ||||||||||||||||||||||||||||||
Investigational medicinal product name |
BT200
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Injection
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Dosage and administration details |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days
7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance
below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg
dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant
changes in PD and safety parameters were observed.
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Arm title
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Hemophilia A Severe | ||||||||||||||||||||||||||||||
Arm description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. Hemophilia A: titration until largest possible increase in FVIII activity before PFA-100® CADP-CT increased to 150 seconds. No up-titration from 3 mg was foreseen in severe hemophilia patients on on-demand therapy. | ||||||||||||||||||||||||||||||
Arm type |
Active comparator | ||||||||||||||||||||||||||||||
Investigational medicinal product name |
BT200
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Injection
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Dosage and administration details |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days
7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance
below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg
dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant
changes in PD and safety parameters were observed.
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Arm title
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VWD Type 2b | ||||||||||||||||||||||||||||||
Arm description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. VWD type IIb: titration until normalization of platelet count and/or FVIII activity level | ||||||||||||||||||||||||||||||
Arm type |
Active comparator | ||||||||||||||||||||||||||||||
Investigational medicinal product name |
BT200
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Injection
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Dosage and administration details |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days
7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance
below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg
dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant
changes in PD and safety parameters were observed.
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Arm title
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VWD Type 3 | ||||||||||||||||||||||||||||||
Arm description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. VWD Type 3 under substitution therapy: titration only between 3 and 6 mg | ||||||||||||||||||||||||||||||
Arm type |
Active comparator | ||||||||||||||||||||||||||||||
Investigational medicinal product name |
BT200
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Injection
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Dosage and administration details |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days
7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance
below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg
dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant
changes in PD and safety parameters were observed.
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Arm title
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aVWS | ||||||||||||||||||||||||||||||
Arm description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. aVWS: titration until normalization of FVIII activity | ||||||||||||||||||||||||||||||
Arm type |
Active comparator | ||||||||||||||||||||||||||||||
Investigational medicinal product name |
BT200
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Injection
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Dosage and administration details |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days
7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance
below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg
dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant
changes in PD and safety parameters were observed.
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Baseline characteristics reporting groups
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Reporting group title |
Hemophilia A Mild/Mod
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. Hemophilia A: titration until largest possible increase in FVIII activity before PFA-100® CADP-CT increased to 150 seconds. No up-titration from 3 mg was foreseen in severe hemophilia patients on on-demand therapy. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Hemophilia A Severe
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. Hemophilia A: titration until largest possible increase in FVIII activity before PFA-100® CADP-CT increased to 150 seconds. No up-titration from 3 mg was foreseen in severe hemophilia patients on on-demand therapy. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
VWD Type 2b
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. VWD type IIb: titration until normalization of platelet count and/or FVIII activity level | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
VWD Type 3
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. VWD Type 3 under substitution therapy: titration only between 3 and 6 mg | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
aVWS
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. aVWS: titration until normalization of FVIII activity | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Hemophilia A Overall
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Subject analysis set type |
Full analysis | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Blood samples for the determination of plasma concentrations were collected on Days 0, 4, 7, 14,
21, 28, 35, 42, 49 and 56 (EOS Visit). On dosing days, the sample was collected prior to
administration of IMP.
The concentration of BT200 which is required to produce a 2-fold increase in FVIIIc activity was
calculated to be in the range from 368 to 508 ng/mL, and the concentration of BT200 which would
be required to produce a 2-fold increase in the VWF Ag level was calculated to be in the range
from 459 to 649 ng/mL. Both of these thresholds were readily achieved after 6 doses of BT200 in
all disease entities.Comment: Type 1 (0 subjects), 3 (1 subject) and aVWS (1 subject) were not analyzed.
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Subject analysis set title |
VWD Type IIb Overall
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Subject analysis set type |
Full analysis | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Blood samples for the determination of plasma concentrations were collected on Days 0, 4, 7, 14,
21, 28, 35, 42, 49 and 56 (EOS Visit). On dosing days, the sample was collected prior to
administration of IMP.
The concentration of BT200 which is required to produce a 2-fold increase in FVIIIc activity was
calculated to be in the range from 368 to 508 ng/mL, and the concentration of BT200 which would.
be required to produce a 2-fold increase in the VWF Ag level was calculated to be in the range
from 459 to 649 ng/mL. Both of these thresholds were readily achieved after 6 doses of BT200 in
all disease entities.
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End points reporting groups
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Reporting group title |
Hemophilia A Mild/Mod
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. Hemophilia A: titration until largest possible increase in FVIII activity before PFA-100® CADP-CT increased to 150 seconds. No up-titration from 3 mg was foreseen in severe hemophilia patients on on-demand therapy. | ||
Reporting group title |
Hemophilia A Severe
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. Hemophilia A: titration until largest possible increase in FVIII activity before PFA-100® CADP-CT increased to 150 seconds. No up-titration from 3 mg was foreseen in severe hemophilia patients on on-demand therapy. | ||
Reporting group title |
VWD Type 2b
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. VWD type IIb: titration until normalization of platelet count and/or FVIII activity level | ||
Reporting group title |
VWD Type 3
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. VWD Type 3 under substitution therapy: titration only between 3 and 6 mg | ||
Reporting group title |
aVWS
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Reporting group description |
On Day 0 and Day 4 each patient was to receive a single SC injection of 3 mg of BT200. On Days 7, 14, 21, and 28, the BT200 dose was to be titrated between 3 mg and 9 mg using the guidance below. It was anticipated that dose adjustments were to be performed in 2-3 mg steps. The 9-mg dose was only to be applied on Day 21-28 under exceptional circumstances, e.g. if no relevant changes in PD and safety parameters were observed. aVWS: titration until normalization of FVIII activity | ||
Subject analysis set title |
Hemophilia A Overall
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Blood samples for the determination of plasma concentrations were collected on Days 0, 4, 7, 14,
21, 28, 35, 42, 49 and 56 (EOS Visit). On dosing days, the sample was collected prior to
administration of IMP.
The concentration of BT200 which is required to produce a 2-fold increase in FVIIIc activity was
calculated to be in the range from 368 to 508 ng/mL, and the concentration of BT200 which would
be required to produce a 2-fold increase in the VWF Ag level was calculated to be in the range
from 459 to 649 ng/mL. Both of these thresholds were readily achieved after 6 doses of BT200 in
all disease entities.Comment: Type 1 (0 subjects), 3 (1 subject) and aVWS (1 subject) were not analyzed.
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Subject analysis set title |
VWD Type IIb Overall
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Blood samples for the determination of plasma concentrations were collected on Days 0, 4, 7, 14,
21, 28, 35, 42, 49 and 56 (EOS Visit). On dosing days, the sample was collected prior to
administration of IMP.
The concentration of BT200 which is required to produce a 2-fold increase in FVIIIc activity was
calculated to be in the range from 368 to 508 ng/mL, and the concentration of BT200 which would.
be required to produce a 2-fold increase in the VWF Ag level was calculated to be in the range
from 459 to 649 ng/mL. Both of these thresholds were readily achieved after 6 doses of BT200 in
all disease entities.
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End point title |
FVIIIc activity change from Baseline to Day 35 [1] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Baseline to Day 35
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Either resolve this issue or provide a justification |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
FVIIIc Observed activity level at Day 35 [2] | ||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Baseline to Day 35
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| Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Either resolve this issue or provide a justification |
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| No statistical analyses for this end point | |||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Treatment Period
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
24.0
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Reporting groups
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Reporting group title |
Overall
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
