Clinical Trials Register

Summary
EudraCT Number:	2020-004162-18
Sponsor's Protocol Code Number:	PDY16894
National Competent Authority:	Germany - PEI
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-02-11
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Germany - PEI

A.2

EudraCT number

2020-004162-18

A.3

Full title of the trial

A multicenter, Phase 2a, open-label, non-randomized study evaluating the efficacy, safety, and tolerability of BIVV020 in adults with persistent/chronic immune thrombocytopenia (ITP)

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A Phase 2a study evaluating BIVV020 in adults with persistent/chronic immune thrombocytopenia (ITP)

A.4.1

Sponsor's protocol code number

PDY16894

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1253-2343

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Sanofi-Aventis Recherche & Développement
B.1.3.4	Country	France
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Sanofi-Aventis Recherche & Développement
B.4.2	Country	France
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Sanofi-Aventis Deutschland GmbH
B.5.2	Functional name of contact point
B.5.3.4	Country	Germany
B.5.6	E-mail	medinfo.de@sanofi.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.2	Product code	BIVV020
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	BIVV020
D.3.9.2	Current sponsor code	BIVV020
D.3.9.3	Other descriptive name	BIVV020
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	150
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

immune thrombocytopenia (ITP)

E.1.1.1

Medical condition in easily understood language

Blood and lymphatic diseases

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	23.0
E.1.2	Level	PT
E.1.2	Classification code	10083842
E.1.2	Term	Immune thrombocytopenia
E.1.2	System Organ Class	10005329 - Blood and lymphatic system disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

• To evaluate the effect of BIVV020 on the durability of platelet

E.2.2

Secondary objectives of the trial

• To assess the safety and tolerability of BIVV020
• To assess the pharmacokinetics of BIVV020
• To assess the response rate of treatment with BIVV020
• To assess the time to response
• To assess the effect of treatment with BIVV020 on the requirement for rescue ITP therapy
• To assess the immunogenicity of BIVV020

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Male and female participants ≥18 years of age at the time of signing the
informed consent
- Confirmed diagnosis of primary ITP; for participants who previously
received sutimlimab in study TDR16218 (NCT03275454), a response to
sutimlimab must have been obtained, as defined by platelet count ≥30 ×
10^9/L on 2 visits at least 7 days apart
- For participants who have not previously received sutimlimab:
persistent/chronic ITP (ITP lasting for ≥6 months) and all the following conditions:
a) Platelet count ≤30 × 10^9/L on 2 occasions at least 5 days apart during
the Screening Period;
b) Lack of an adequate platelet count response (as defined by
maintenance of sustained platelet count ≥30 × 109/L in the absence of bleeding) to at least 2 ITP treatments, 1 of which was a thrombopoietin receptor agonist. Other ITP treatments include: IVIg, anti-D immunoglobulin, corticosteroids, splenectomy, rituximab, cyclophosphamide, azathioprine, danazol, cyclosporin A, mycophenolate mofetil, or fostamatinib.
c) If receiving weekly thrombopoietin receptor agonist dosing, the last
dose must have been administered ≥7 days before the first dose of
BIVV020. If receiving daily thrombopoietin receptor agonist dosing, the
last dose must have been administered ≥24 hours before the first dose of
BIVV020
d) If applicable, concurrent administration of ITP medications (eg.
corticosteroids, IVIg, azathioprine, danazol, cyclosporin A, mycophenolate
mofetil, or thrombopoietin receptor agonists) is acceptable provided the
patient has been on a stable dose for at least 1 month.
e) If previously dosed with rituximab, the last dose of rituximab must have
been administered at least 12 weeks before the first dose of BIVV020
- Documented vaccinations against encapsulated bacterial pathogens
(Neisseria meningitidis, including serogroup B where available,
Haemophilus influenzae, and Streptococcus pneumoniae) within 5 years
of enrollment
- Contraceptive use for women of childbearing potential and men who are
sexually active with a female partner of childbearing potential

E.4

Principal exclusion criteria

Participants are excluded from the study if any of the following criteria apply:
- Clinically significant medical history or ongoing chronic illness that
would jeopardize the safety of the participant or compromise the quality of
the data derived from his/her participation in the study
- Clinical diagnosis of SLE
- Clinically relevant infection within the month prior to enrollment
- History of venous or arterial thrombosis within the year prior to
enrollment
- Secondary ITP from any cause including lymphoma, chronic
lymphocytic leukemia, and drug-induced thrombocytopenia
- Positive hepatitis B surface antigen (HBsAg) or active HCV
infection
- HIV infection
- Pregnant or lactating women
- Hemoglobin level <10 g/dL

E.5 End points

E.5.1

Primary end point(s)

-Proportion of participants with a durable platelet Response.
Durable platelet response is defined for naïve participants as the proportion
of participants with a platelet count ≥50 × 109/L at ≥50% of scheduled visits,
or for participants with baseline platelet count <15 × 109/L, a ≥20 × 109/L
increase in platelet count from baseline at ≥50% of scheduled visits, without
receiving rescue ITP therapy, as assessed from Week 3 to Week 24.
Durable platelet response is defined for participants who previously received
sutimlimab as maintenance of platelet count ≥30 × 109/L at ≥50% of
scheduled visits, without receiving rescue ITP therapy, as assessed from
Week 3 to Week 24.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 3 to Week 24

E.5.2

Secondary end point(s)

1) Number of participants with treatment emergent adverse Events
2) Plasma concentrations of BIVV020
3) Response rate of treatment with BIVV020:Response rate at Weeks 24 and 52, defined as a platelet count ≥50 × 109/L
and a greater than 2-fold increase from baseline, measured on 2 occasions
at least 7 days apart, with the absence of bleeding (bleeding is defined as
bleeding with a score ≥2 on the WHO bleeding scale), and the lack of combination ITP therapy during this period.
4)Time to first platelet Response:
Time from baseline to first platelet response, defined as greater than or
equal to each of the following values: 50 × 109/L and 100 × 109/L (confirmed by 2 measurements at least 7 days apart)
5) Proportion of patients who did not require
rescue therapy for an acute episode of
thrombocytopenia after Week 3
6) Number of participants with incidence and titer (if relevant) of anti-BIVV020 antibodies

E.5.2.1

Timepoint(s) of evaluation of this end point

1)Week 52
2)Week 52
3)Weeks 24 and 52
4)Baseline to Week 52
5)From Week 3 to Week 52
6)Week 24, Week 52

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Austria

Belgium

Germany

Netherlands

Spain

United Kingdom

Czechia

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LSLV

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2021-02-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-03-16

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2023-02-07

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