Clinical Trials Register

Summary
EudraCT Number:	2018-002984-24
Sponsor's Protocol Code Number:	CYT-C2-001
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:	2020-10-21
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

2018-002984-24

A.3

Full title of the trial

Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old

Studio multicentrico, in aperto, a braccio singolo volto a valutare la sicurezza di Cystadrops® in pazienti pediatrici con cistinosi, con età compresa tra 6 mesi a meno di 2 anni

A.3.1

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

Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old

Studio per valutare la sicurezza di Cystadrops® nei pazienti con cistinosi pediatrica di eta' compresa tra 6 mesi e meno di 2 anni

A.3.2

Name or abbreviated title of the trial where available

SCOB2 (Study Cystadrops® Ophthalmic for Patients Below 2 years)

SCOB2 (Studio Cystadrops® Oftalmico per pazienti di età inferiore a 2 anni)

A.4.1

Sponsor's protocol code number

CYT-C2-001

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04125927

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/172/2017

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Recordati Rare Diseases
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	Recordati Rare Diseases
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	Recordati Rare Diseases
B.5.2	Functional name of contact point	Medical department
B.5.3	Address:
B.5.3.1	Street Address	Immeuble Le Wilson, 70 Avenue du General de Gaulle
B.5.3.2	Town/ city	Puteaux
B.5.3.3	Post code	92800
B.5.3.4	Country	France
B.5.4	Telephone number	+33147736458
B.5.6	E-mail	plisson.c@recordati.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	Yes
D.2.1.1.1	Trade name	Cystadrops
D.2.1.1.2	Name of the Marketing Authorisation holder	Recordati Rare Diseases
D.2.1.2	Country which granted the Marketing Authorisation	France
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/3/08/578
D.3 Description of the IMP
D.3.1	Product name	Cystadrops
D.3.2	Product code	[045251016]
D.3.4	Pharmaceutical form	Eye drops, solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Ocular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Mercaptamina
D.3.9.1	CAS number	156-57-0
D.3.9.2	Current sponsor code	Not applicable
D.3.9.3	Other descriptive name	ß-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
D.3.9.4	EV Substance Code	SUB08770MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	up to
D.3.10.3	Concentration number	4
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

Nephropatic Cystinosis patients with corneal cystine crystal deposits

Pazienti con cistinosi nefropatica con depositi di cristalli di cistina corneale

E.1.1.1

Medical condition in easily understood language

Metabolic disease characterized by abnormal accumulation of the amino acid Cystine in various organs of the body, one of whom is the eyes

Malattia metabolica caratterizzata da un accumulo anormale dell'amminoacido Cistina in vari organi del corpo, uno dei quali è gli occhi

E.1.1.2

Therapeutic area

Body processes [G] - Metabolic Phenomena [G03]

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	10071112
E.1.2	Term	Nephropathic cystinosis
E.1.2	System Organ Class	100000004850

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 assess the safety profile of Cystadrops® over a 90-day period as measured by the incidence of:
o Serious ocular Adverse Events (ocular SAEs)
o Serious Adverse Drug Reactions (SADRs) related to Cystadrops®
o All AEs that required temporary discontinuation/ withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visits

o Eventi avversi oculari seri (SAE oculari)
o Reazioni avverse al farmaco serie (SADR) correlate a Cystadrops®
o Tutti gli EA per cui si è reso necessario interrompere/sospendere temporaneamente il trattamento (IMP) o visite oftalmiche non programmate/di emergenza

E.2.2

Secondary objectives of the trial

To assess the efficacy of Cystadrops® by measuring ophthalmologic assessments (Corneal cystine crystal score (CCCS), photophobia and best corrected visual acuity (BCVA)) of both eyes after 90 days of treatment with Cystadrops® when possible considering the age of the patients.

•Valutare l'efficacia di Cystadrops® eseguendo delle valutazioni oftalmologiche (Punteggio relativo ai cristalli di cistina nella cornea (Corneal Cystine Crystal Score, CCCS), fotofobia e Migliore acuità visiva corretta (Best Corrected Visual Acuity, BCVA)) su entrambi gli occhi dopo 90 giorni di trattamento con Cystadrops® quando possibile considerando l'età dei pazienti

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients must meet all inclusion criteria to be eligible for study participation.
1. Patient aged from 6 months to less than 2 years old
2. Cystinosis diagnosed patients confirmed by the physician and with presence of corneal cystine crystal deposits assessed during ophthalmic examination
3. Evidence of a signed and dated informed consent document indicating that parents/ legally acceptable representatives had been informed of all pertinent aspects of the study (if required by regulation)
4. Parents/ legally acceptable representatives who are willing to comply with regular visits and ophthalmic exams

Per essere idonei alla partecipazione allo studio, i pazienti dovranno soddisfare tutti i criteri di inclusione.
1. Pazienti con età compresa tra 6 mesi e meno di 2 anni.
2. Pazienti con diagnosi di cistinosi confermata dal medico e con presenza di depositi di cristalli di cistina nella cornea valutati durante l'esame oftalmico.
3. Sottoscrizione e datazione del Modulo di consenso informato che indichi che i genitori/rappresentanti legalmente autorizzati siano stati informati di tutti gli aspetti pertinenti dello studio (se richiesto dalla normativa).
4. Genitori/rappresentanti legalmente autorizzati che siano disposti a rispettare le visite regolari e gli esami oftalmici.

E.4

Principal exclusion criteria

Patients will not be eligible for study participation if they meet any of the exclusion criteria, or will be discontinued at the discretion of the Investigator in consultation with the medical monitor if they develop any of the exclusion criteria during the study.
1. Contraindications to any of the Cystadrops® components
2. Participation in another ophthalmic investigational study or intent to participate during the course of the study
3. Any medical condition that would, in the opinion of the Investigator, interfere with the evaluation of the study objectives

I pazienti non potranno partecipare allo studio se soddisfano uno qualsiasi dei criteri di esclusione oppure, nel caso in cui sviluppino uno qualsiasi dei criteri di esclusione durante lo studio, la loro partecipazione allo studio verrà interrotta (a discrezione dello Sperimentatore e previa consultazione con il Monitor medico).
1. Controindicazioni a uno qualsiasi dei componenti di Cystadrops®
2. Partecipazione ad un altro studio oftalmico sperimentale o intenzione di parteciparvi durante il corso dello studio
3. Qualsiasi condizione medica che, secondo lo Sperimentatore, interferirebbe con la valutazione degli obiettivi dello studio

E.5 End points

E.5.1

Primary end point(s)

Occurrence of the following safety criteria between signature of the informed consent form and the end of study visit (EOS, Day 90):
o Ocular treatment emergent SAEs,
o SADRs related to Cystadrops®
o All AEs that required temporary discontinuation/ withdrawal of treatment (IMP), or unscheduled/emergency ophthalmic visits

Comparsa dei criteri di sicurezza seguenti nel periodo che intercorre dalla sottoscrizione del Modulo di consenso informato (ICF) alla visita di fine dello studio (EOS, Giorno 90):
o SAE emergenti dal trattamento oculare,
o SADR correlati a Cystadrops®
o Tutti gli EA per cui si è reso necessario interrompere/sospendere temporaneamente il trattamento (IMP) o visite oftalmiche non programmate/di emergenza

E.5.1.1

Timepoint(s) of evaluation of this end point

D90

E.5.2

Secondary end point(s)

Change from Day 1 (Inclusion) to Day 90 (EOS) in CCCS, photophobia, and BCVA

Variazione, dal Giorno 1 (inclusione) al Giorno 90 (EOS), di CCCS, fotofobia e BCVA

E.5.2.1

Timepoint(s) of evaluation of this end point

D90

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

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)

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

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

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

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

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

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

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

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Subjects are aged from 6months to less than 2years old so by definition not able to give consent

I soggetti hanno un'età compresa tra 6 mesi e meno di 2 anni, quindi per definizione non sono in grado di dare il consenso

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)

IMP can be provided by the site to parents and legally acceptable representatives of patients below 2 years of age at the time of EOS visit (Day 90) free of charge until the patient reaches 2 years of age. Decision to propose continuation of treatment with the IMP will be made by Investigators for each patient at EOS visit (Day 90) based on an individual medical benefit/risk assessment for the concerned patient. See protocol for full details

Il farmaco in esame può essere fornito dal centro ai genitori e ai rappresentanti legali dei pazienti di età inferiore ai 2 anni al momento della visita EOS (giorno 90) gratuitamente fino a quando il paziente non raggiunge i 2 anni. La decisione di proporre la prosecuzione del trattamento con il farmaco (IMP) sarà presa dagli investigatori per ciascun paziente alla visita EOS (giorno 90) sulla base di una valutazione del beneficio / rischio medico individuale per il paziente interessato.

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

2020-06-24

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-06-17

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

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