Clinical Trials Register

Summary
EudraCT Number:	2022-001686-12
Sponsor's Protocol Code Number:	20210210
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-11-09
Trial 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

Italy - Italian Medicines Agency

A.2

EudraCT number

2022-001686-12

A.3

Full title of the trial

A Phase 2 Long-Term Extension (LTE) Study to Evaluate The Safety and Efficacy of Efavaleukin Alfa in Subjects with Moderately to Severely Active
Ulcerative Colitis

Studio di fase II di estensione a lungo termine (LTE) volto a valutare la sicurezza e l’efficacia di efavaleukin alfa nei soggetti con colite ulcerosa da moderatamente a gravemente attiva

A.3.1

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

Long-term Safety and Efficacy of Efavaleukin Alfa in Subjects With Moderately to Severely Active Ulcerative Colitis

Sicurezza ed efficacia a lungo termine di efavaleukin alfa nei soggetti con colite ulcerosa da moderatamente a gravemente attiva

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

20210210

A.5.4

Other Identifiers

Name:

Number:

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	AMGEN INC.
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	Amgen Inc.
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	Amgen S.r.l. a socio unico
B.5.2	Functional name of contact point	Medical Information
B.5.3	Address:
B.5.3.1	Street Address	Via E. Tazzoli 6
B.5.3.2	Town/ city	Milano
B.5.3.3	Post code	20154
B.5.3.4	Country	Italy
B.5.4	Telephone number	0039026241121
B.5.5	Fax number	0039026241121
B.5.6	E-mail	medicalinformationitaly@amgen.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.3.1	Product name	Efavaleukin Alfa
D.3.2	Product code	[AMG 592]
D.3.4	Pharmaceutical form	Solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Efavaleukin alfa
D.3.9.2	Current sponsor code	AMG 592
D.3.9.4	EV Substance Code	SUB195522
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	2
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	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
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	Yes
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Powder for solution for injection/infusion
D.8.4	Route of administration of the placebo	Subcutaneous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Moderate to Severe Active Ulcerative Colitis (UC)

Colite ulcerosa attiva da moderata a grave (CU)

E.1.1.1

Medical condition in easily understood language

Ulcerative Colitis (UC)

Colite ulcerosa (CU)

E.1.1.2

Therapeutic area

Body processes [G] - Immune system processes [G12]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10045365
E.1.2	Term	Ulcerative colitis
E.1.2	System Organ Class	100000004856

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 evaluate the long-term safety and tolerability of efavaleukin alfa in subjects with moderate to severe ulcerative colitis (UC)

Valutare la sicurezza e la tollerabilità a lungo termine di efavaleukina alfa in soggetti con colite ulcerosa (CU) da moderata a grave

E.2.2

Secondary objectives of the trial

- To evaluate the effect of efavaleukin alfa long-term treatment on clinical response
- To evaluate the effect of efavaleukin alfa long-term treatment on clinical remission
- To evaluate the effect of efavaleukin alfa long-term treatment on durable clinical remission
- To evaluate the effect of efavaleukin alfa long-term treatment on endoscopic remission
- To evaluate the effect of efavaleukin alfa long-term treatment on histologic remission
- To evaluate the effect of efavaleukin alfa long-term treatment on corticosteroid-free remission
- To evaluate the effect of efavaleukin alfa long-term treatment on combined endoscopic and histologic remission
- To evaluate the effect of efavaleukin alfa long-term treatment on symptomatic remission
- To evaluate the effect of efavaleukin alfa long-term treatment on change in histological score

- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla risposta clinica
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione clinica.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione clinica duratura.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione endoscopica.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione istologica.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione senza corticosteroidi.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione combinata endoscopica e istologica.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla remissione sintomatica.
- Valutare l'effetto del trattamento a lungo termine con efavaleukina alfa sulla variazione del punteggio istologico.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

101 Subject has provided informed consent prior to initiation of any study specific activities/procedures.
102 Subject has completed the week 52 endoscopy in the phase 2 dosefinding study (20170104) and who in the opinion of the investigator may benefit from continued treatment.

101 Il soggetto ha fornito il consenso informato prima di iniziare qualsiasi attività/procedura specifica dello studio.
102 Soggetto che ha completato l'endoscopia alla settimana 52 nello studio di fase 2 di determinazione della dose (20170104) e che, a giudizio dello sperimentatore, può trarre beneficio dal proseguimento del trattamento.

E.4

Principal exclusion criteria

201 Permanent discontinuation of investigational product during the 52-week phase 2 dose finding study (20170104) for any reason.
Disease Related
202 Adenoma and dysplasia exclusion criteria:
- Any current sporadic adenoma without dysplasia (adenomatous polyps occurring proximal to known areas of colitis) that has not been removed.
- Dysplasia occurring in flat mucosa, sporadic adenomas containing dysplasia, and dysplasia-associated lesions or masses will be managed as follows:
1. Any history or current evidence of high-grade dysplasia.
2. Any history or current evidence of dysplasia occurring in flat mucosa. This includes histopathology reporting indefinite for dysplasia, low-grade dysplasia, and high-grade dysplasia.
3. Any history or current evidence of a nonadenoma like dysplasia associated lesions or masses, with or without evidence of dysplasia.
4. Any current sporadic adenoma containing dysplasia or any current adenoma-like dysplasia-associated lesions or masses that has not been
removed.
Other Medical Conditions
203 Any malignancy diagnosed during Study 20170104, including evidence of cutaneous basal or squamous cell carcinoma or melanoma
204 Active infection (including chronic, acute, recurrent, opportunistic infections) at the time of eligibility evaluation requiring intravenous (IV)
anti-infectives or hospitalization (infections requiring oral and/or topical anti-infective[s] for > 7 days may be allowed in consultation with the
Amgen physician).
205 Required systemic corticosteroid use for any indication other than UC. The only exception is corticosteroids used for the treatment of adrenal insufficiency are allowed.
Prior/Concomitant Therapy
206 Plan to receive a live (attenuated) vaccine during the treatment period and up to 6 weeks after the last dose of investigational product in
the LTE study.
Prior/Concurrent Clinical Study Experience
207 Currently receiving treatment in another investigational device or drug study. Other investigational procedures while participating in this
study are excluded.

Please refer to the protocol for the full list.

201 Interruzione permanente del prodotto in sperimentazione durante lo studio di ricerca della dose di fase 2 di 52 settimane (20170104) per qualsiasi motivo.
Malattia correlata
202 Criteri di esclusione per adenoma e displasia:
- Qualsiasi adenoma sporadico in corso senza displasia (polipi adenomatosi che si verificano in prossimità di aree note di colite) che non sia stato rimosso.
- La displasia che si verifica nella mucosa piatta, gli adenomi sporadici contenenti displasia e le lesioni o le masse associate alla displasia saranno gestite come segue:
1. Qualsiasi storia o evidenza attuale di displasia di alto grado.
2. Qualsiasi storia o evidenza attuale di displasia che si verifica nella mucosa piatta. Questo include il referto istopatologico indefinito per displasia, displasia di basso grado e displasia di alto grado.
3. Qualsiasi anamnesi o evidenza attuale di lesioni o masse associate a displasia non adenoma, con o senza evidenza di displasia.
4. Qualsiasi adenoma sporadico attuale contenente displasia o qualsiasi lesione o massa associata a displasia simile a un adenoma che non sia stata rimossa.
Altre condizioni mediche
203 Qualsiasi neoplasia diagnosticata durante lo Studio 20170104, compresa l'evidenza di un carcinoma cutaneo a cellule basali o squamose o di un melanoma
204 Infezione attiva (incluse infezioni croniche, acute, ricorrenti e opportunistiche) al momento della valutazione di eleggibilità che richieda l'uso di anti-infettivi per via endovenosa (IV) o ricovero ospedaliero (le infezioni che richiedono antinfettivi orali e/o topici per > 7 giorni possono essere consentite in consultazione con il medico di Amgen).
205 Uso obbligatorio di corticosteroidi sistemici per qualsiasi indicazione diversa dall'UC. L'unica eccezione è rappresentata dai corticosteroidi utilizzati per il trattamento dell'insufficienza surrenalica.
Terapia precedente/concomitante
206 Si prevede di ricevere un vaccino vivo (attenuato) durante il periodo di trattamento e fino a 6 settimane dopo l'ultima dose di prodotto in sperimentazione nello studio LTE.
Esperienza di studio clinico precedente/concorrente
207 Attualmente in trattamento con un altro dispositivo o farmaco in sperimentazione. Sono escluse altre procedure sperimentali durante la partecipazione a questo studio.

Si rimanda al protocollo per l'elenco completo

E.5 End points

E.5.1

Primary end point(s)

Treatment-emergent adverse events

Eventi avversi emergenti al trattamento

E.5.1.1

Timepoint(s) of evaluation of this end point

Throughout the study

Durante tutto lo studio

E.5.2

Secondary end point(s)

- Clinical response at week 52 and 104
- Clinical remission at week 52 and 104
- Durable clinical remission at week 52 and 104
- Endoscopic remission at week 52 and 104
- Histologic remission at week 52 and 104
- Corticosteroid-free remission at week 52 and 104 in subjects receiving corticosteroids at randomization of Study 20170104
- Combined endoscopic and histologic remission at week 52 and 104
- Symptomatic remission at week 52 and 104
- Change from baseline of Study 20170104 in histological score (Geboes) at week 52 and 104

- Risposta clinica alla settimana 52 e 104
- Remissione clinica alla settimana 52 e 104
- Remissione clinica duratura alla settimana 52 e 104
- Remissione endoscopica alla settimana 52 e 104
- Remissione istologica alla settimana 52 e 104
- Remissione senza corticosteroidi alla settimana 52 e 104 nei soggetti che ricevevano corticosteroidi alla randomizzazione dello studio 20170104
- Remissione endoscopica e istologica combinata alla settimana 52 e 104
- Remissione sintomatica alla settimana 52 e 104
- Variazione dal basale dello Studio 20170104 nel punteggio istologico (Geboes) alla settimana 52 e 104

E.5.2.1

Timepoint(s) of evaluation of this end point

Weeks 52 and 104

Settimane 52 e 104

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

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

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

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

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

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

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in 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

Information not present in EudraCT

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

Argentina

Canada

Japan

Korea, Republic of

Mexico

Taiwan

United States

Austria

Finland

Poland

Bulgaria

Netherlands

Romania

Spain

Switzerland

Czechia

Germany

Greece

Italy

Belgium

Denmark

Hungary

Russian Federation

Turkey

Ukraine

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

End of week 104 +6 week Safety follow up

Alla fine della settimana 104 + 6 settimane di 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:

150

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

100

F.4.2.2

In the whole clinical trial

200

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)

After last patient last visit an individual subject will enter in a safety follow-up of 6 weeks

Dopo l'ultima visita del paziente, il singolo soggetto sarà sottoposto ad un follow-up di 6 settimane

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

2023-02-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-11-17

P. End of Trial
P.	End of Trial Status	Ongoing

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