Clinical Trials Register

Summary
EudraCT Number:	2014-000253-36
Sponsor's Protocol Code Number:	CD-IA-MEDI-551-1155
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2014-11-14
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

Poland - Office for Medicinal Products

A.2

EudraCT number

2014-000253-36

A.3

Full title of the trial

A Double-masked, Placebo-controlled Study with Open-label Period to Evaluate the Efficacy and Safety of MEDI-551 in Adult Subjects with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders.

A.3.1

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

A Clinical Study to test the effect of the drug MEDI-551 in Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders

A.3.2

Name or abbreviated title of the trial where available

A Double-masked, Placebo-controlled Study with Open label Period to Evaluate MEDI-551 in NMO/NMOSD

A.4.1

Sponsor's protocol code number

CD-IA-MEDI-551-1155

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02200770

A.5.3

WHO Universal Trial Reference Number (UTRN)

U1111-1159-8686

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	MedImmune LLC
B.1.3.4	Country	United States
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	MedImmune LLC
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	MedImmune LLC
B.5.2	Functional name of contact point	Clinical Trial Enquiries
B.5.3	Address:
B.5.3.1	Street Address	One MedImmune Way
B.5.3.2	Town/ city	Gaithersburg
B.5.3.3	Post code	MD 20878
B.5.3.4	Country	United States
B.5.4	Telephone number	13013980000
B.5.6	E-mail	clinicaltrialenquiries@medimmune.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/17/1856 - EMA/OD/267/16
D.3 Description of the IMP
D.3.1	Product name	MEDI-551
D.3.2	Product code	MEDI-551
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Inebilizumab
D.3.9.1	CAS number	1299440-37-1
D.3.9.2	Current sponsor code	MEDI-551
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	10
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	Yes
D.3.11.13.1	Other medicinal product type	MEDI-551 is a humanized, affinity-optimized, afucosylated immunoglobulin G1 kappa (IgG1κ) monoclonal antibody

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders (NMO/NMOSD)

E.1.1.1

Medical condition in easily understood language

Neuromyelitis optica (NMO) and NMO Spectrum Disorders, involve inflammation and damage of the optic nerve (optic neuritis), spinal cord (myelitis) and certain areas of the brain.

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10029322
E.1.2	Term	Neuromyelitis optica
E.1.2	System Organ Class	100000004852

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To compare the efficacy of MEDI-551 versus placebo in reducing the risk of an NMO/NMOSD attack

E.2.2

Secondary objectives of the trial

1. To compare the efficacy of MEDI-551 versus placebo on the reduction of EDSS worsening
2. To compare the efficacy of MEDI-551 versus placebo on the change from baseline of low-contrast visual acuity score
3. To compare the efficacy of MEDI-551 versus placebo in reducing the cumulative active MRI lesion count
4. To compare the efficacy of MEDI-551 versus placebo in reducing NMO/NMOSD-related in-patient hospitalizations
5. To characterize the long-term efficacy of MEDI-551 by means of annualized attack rate.
6. To evaluate the safety and tolerability of a single course of MEDI-551 in randomised control phase and repeated dose of MEDI-551 in open label phase
7. To characterize the pharmacokinetic (PK) profile and immunogenicity of MEDI-551

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1) Men and women 18 years or older with diagnosis of NMO/NMOSD.
2) Confirmation of NMO/NMOSD status:
a.) AQP4-IgG sero-positive NMO/NMOSD with at least one attack requiring rescue
therapy in the last year or two attacks requiring rescue therapy in the last 2 years.
b.) AQP4-IgG sero-negative NMO with at least one attack requiring rescue therapy in the
last year or two attacks requiring rescue therapy in the last 2 years.
3) Able and willing to give written informed consent and comply with the requirements of the study protocol.
4) EDSS <= 7.5 (8 in special circumstances).
5) Men and women of reproductive potential must agree to use a highly effective method of birth control from screening to 6 months after final dose of the investigational product.

E.4

Principal exclusion criteria

1) Lactating and pregnant females.
2) Treatment with any investigational agent within 4 weeks of screening.
3) Known history of a severe allergy or reaction to any component of the investigational product formulation or history of anaphylaxis following any biologic therapy.
4) Known active severe bacterial, viral, or other infection or any major episode of infection requiring hospitalization.
5) History of of alcohol, drug, or chemical abuse, or a recent history of such abuse < 1 year prior to randomization.
6) Receipt of the following at any time prior to randomization:
a) Alemtuzumab
b) Total lymphoid irradiation
c) Bone marrow transplant
d) T-cell vaccination therapy
7) Receipt of rituximab or any experimental B-cell depleting agent, unless the subject’s B cells have returned to lower limit of normal (LLN) or greater prior to randomization.
8) Receipt of IVIG within 1 month prior to randomization.
9) Receipt of any of the following within 3 months prior to randomization:
a) Natalizumab (Tysabri®)
b) Cyclosporin
c) Methotrexate
d) Mitoxantrone
e) Cyclophosphamide
f) Tocilizumab
g) Eculizumab
10) History of Hepatitis B and/or Hepatitis C (Hep B/C at screening).
11) Known history of a primary immunodeficiency (congenital or acquired) or an underlying condition such as human immunodeficiency virus (HIV) infection.
12) History of malignancies, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy > 3 months prior to randomization.
13) Any concomitant disease other than NMO/NMOSD that required treatment with oral or intravenous steroids at doses over 20 mg a day for over 21 days.

E.5 End points

E.5.1

Primary end point(s)

Time to an NMO/NMOSD attack determined by Adjudication Committee.

E.5.1.1

Timepoint(s) of evaluation of this end point

on or before Day 197

E.5.2

Secondary end point(s)

1) Attack Rate
2) Number of subjects with Adverse Events
3) PK profile
4) Incidence of anti-drug antibodies (ADAs) directed against MEDI-551

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Worsening from baseline in EDSS at last visit during the RCP.2. Change from baseline in low-contrast visual acuity binocular score measured by low-contrast Landolt C Broken Rings Chart, at last visit during RCP.3.Cumulative total active MRI lesions (new Gd-enhancing or new/enlarging T2) during the RCP.4.Number of NMO/NMOSD-related in-patient hospitalizations. In-patient hospitalization is defined as more than an overnight stay.5.Annualized attack rate (total number of AC-determined NMO/NMOSD attacks normalized by person years) during any exposure to MEDI-551.

6.Treatment-emergent adverse events (TEAEs), including treatment-emergent serious adverse events (TESAEs). Laboratory measurements as well as their changes or shift from baseline over time. 7.Pharmacokinetic profile of MEDI-551.
8.Incidence of anti-drug antibodies (ADAs) directed against MEDI-551 for the duration of the study, both predose and postdose for each subject.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

Yes

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

Yes

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)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Open label extension

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

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

Information not present in EudraCT

E.8.4

The trial involves multiple sites in the Member State concerned

Information not present in EudraCT

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Information not present in EudraCT

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

Australia

Bulgaria

Canada

Colombia

Czech Republic

Estonia

Germany

Greece

Hong Kong

Hungary

Israel

Japan

Korea, Republic of

Mexico

Moldova, Republic of

New Zealand

Peru

Poland

Portugal

Russian Federation

Serbia

South Africa

Spain

Taiwan

Thailand

Turkey

Ukraine

United States

E.8.7

Trial has a data monitoring committee

Yes

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

The end of the study is defined as the date of the last protocol-specified visit/assessment (including telephone contact) for the last subject in the study. This will occur when the last subject completes the 52-week safety follow up.

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:

221

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

231

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)

Revert to standard of care for the condition

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

2014-12-30

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-11-25

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2020-11-06

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