Clinical Trials Register

Summary
EudraCT Number:	2021-001369-19
Sponsor's Protocol Code Number:	MK-7075-006
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:	2021-10-18
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

2021-001369-19

A.3

Full title of the trial

A Multicenter, Open-label, Phase 2, Extension Trial to Study the Long-term Safety in Participants With PROS or Proteus Syndrome Who Are Currently Being Treated with Miransertib in Other Studies

Sperimentazione Clinica di Estensione multicentrica, in aperto, di fase 2 per studiare la sicurezza a lungo termine in pazienti con PROS o Sindrome di Proteus attualmente in trattamento con Miransertib in altri studi

A.3.1

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

An extension study for patients with PROS or Proteus Syndrome currently treated with miransertib.

Uno studio di estensione per i pazienti con PROS o sindrome di Proteus attualmente trattati con miransertib.

A.3.2

Name or abbreviated title of the trial where available

Miransertib Extension Study for PROS / PS Patients

Studio di estensione di miransertib per pazienti PROS / PS

A.4.1

Sponsor's protocol code number

MK-7075-006

A.5.4

Other Identifiers

Name:

IND

Number:

130784

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	MERCK SHARP & DOHME CORP. UNA SUSSIDIARIA DI MERCK & CO. 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	Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., 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	MSD Italia S.r.l.
B.5.2	Functional name of contact point	Divisione Ricerca Clinica
B.5.3	Address:
B.5.3.1	Street Address	Via Vitorchiano 151
B.5.3.2	Town/ city	Roma (RM)
B.5.3.3	Post code	00189
B.5.3.4	Country	Italy
B.5.4	Telephone number	00390636191371
B.5.5	Fax number	00390636380371
B.5.6	E-mail	gcto.italy@merck.co

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	15-4971
D.3 Description of the IMP
D.3.1	Product name	Miransertib
D.3.2	Product code	[MK-7075]
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	MIRANSERTIB
D.3.9.1	CAS number	1817727-88-0
D.3.9.2	Current sponsor code	MK-7075
D.3.9.3	Other descriptive name	Miransertib mesylate; ARQ 092
D.3.9.4	EV Substance Code	SUB190710
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
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	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	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.IMP: 2
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	15-4971
D.3 Description of the IMP
D.3.1	Product name	Miransertib
D.3.2	Product code	[MK-7075]
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	MIRANSERTIB
D.3.9.1	CAS number	1817727-88-0
D.3.9.2	Current sponsor code	MK-7075
D.3.9.3	Other descriptive name	Miransertib mesylate; ARQ 092
D.3.9.4	EV Substance Code	SUB190710
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	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	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

PIK3CA-related overgrowth spectrum (PROS) and Proteus Syndrome (PS)

Spettro di crescita eccessiva legata a PIK3CA (PROS) e sindrome di Proteus (PS)

E.1.1.1

Medical condition in easily understood language

(PIK3CA)-related overgrowth spectrum (PROS) and Proteus Syndrome

Spettro di crescita eccessiva relativa a PIK3CA (PROS) e sindrome di Proteus

E.1.1.2

Therapeutic area

Body processes [G] - Genetic Phenomena [G05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10081236
E.1.2	Term	PIK3CA related overgrowth spectrum
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10074067
E.1.2	Term	Proteus syndrome
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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 safety and tolerability of miransertib administered as monotherapy

Valutare la sicurezza e la tollerabilità del miransertib somministrato come monoterapia

E.2.2

Secondary objectives of the trial

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Has PROS or PS and is currently being actively treated with miransertib as part of Study MK-7075-002 (NCT03094832) or ArQule's CU/EAP (NCT03317366)
2. Is male or female, from 2 years to 120 years of age, inclusive
3. The participant (or legally acceptable representative) has provided documented informed consent/assent for the study. There is no future biomedical research planned for this study
4. A WOCBP must have a negative highly sensitive pregnancy test (urine, as required by local regulations) within 72 hours before the first dose of study intervention. If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive

1. Ha PROS o PS ed è attualmente in trattamento attivo con miransertib come parte dello studio MK-7075-002 (NCT03094832) o ArQule CU/EAP (NCT03317366)
2. È maschio o femmina, da 2 anni a 120 anni di età, inclusi
3. Il partecipante (o un rappresentante legalmente accettabile) ha fornito il consenso/assenso informato documentato per lo studio. Non è prevista nessuna ricerca biomedica futura per questo studio
4. Una WOCBP deve avere un test di gravidanza altamente sensibile negativo (urine, come richiesto dai regolamenti locali) entro 72 ore prima della prima dose di farmaco dello studio. Se un test delle urine non può essere confermato come negativo (es, un risultato ambiguo), è necessario un test di gravidanza su siero. In questi casi, la partecipante deve essere esclusa dalla partecipazione se il risultato del test di gravidanza su siero è positivo

E.4

Principal exclusion criteria

1. Has previously discontinued miransertib due to related SAEs or other intolerance of miransertib
2. Other investigational agents, if any, that were administered between leaving Study MK-7075-02 or ArQule's CU/EAP and entering this trial
3. Inhibitors of the mTOR pathway (eg, sirolimus, everolimus)
4. Immunosuppressive therapies
5. Continuous high dose steroids (topical or low dose steroids equivalent to =10 mg prednisone daily are permitted)

1. Ha precedentemente interrotto miransertib a causa di SAE correlati o altra intolleranza al miransertib
2. Altri agenti sperimentali, se presenti, che sono stati somministrati tra l'uscita dallo studio MK-7075-02 o dal CU/EAP di ArQule e l'ingresso in questo studio
3. Inibitori del percorso mTOR (es. sirolimus, everolimus)
4. Terapie immunosoppressive
5. Steroidi continui ad alte dosi (steroidi topici o a basse dosi equivalenti a =10 mg di prednisone al giorno sono permessi)

E.5 End points

E.5.1

Primary end point(s)

1. Number of participants experiencing a Serious Adverse Event (SAE)
2. Number of participants discontinuing study treatment due to an Adverse Event (AE)

1. Numero di partecipanti che hanno sperimentato un evento avverso grave (SAE)
2. Numero di partecipanti che hanno interrotto il trattamento dello studio a causa di un evento avverso (AE)

E.5.1.1

Timepoint(s) of evaluation of this end point

1. Up to approximately 4 years
2. Up to approximately 4 years

1. Fino a circa 4 anni
2. Fino a circa 4 anni

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

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

E.6.6

Pharmacokinetic

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

Yes

E.8.1.7.1

Other trial design description

In aperto

Open

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

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

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

Australia

Brazil

United States

Italy

United Kingdom

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

LVLS

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

Yes

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

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)

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)

After the end of treatment, decisions will be made regarding participant disposition (safety follow-up, continued treatment with miransertib, or alternate treatment) based on the available data as well as treatment options for PROS and PS.

Dopo la fine del trattamento, saranno prese decisioni in merito alla disposizione dei partecipanti (follow-up di sicurezza, trattamento continuato con miransertib o trattamento alternativo) sulla base dei dati disponibili di trattamento per PROS e PS.

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

2021-10-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-09-15

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

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