Clinical Trials Register

Summary
EudraCT Number:	2016-004390-41
Sponsor's Protocol Code Number:	BO42863
National Competent Authority:	Netherlands - Competent Authority
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-10-01
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Netherlands - Competent Authority

A.2

EudraCT number

2016-004390-41

A.3

Full title of the trial

A Phase 1/2 Study of the Highly-selective RET Inhibitor, BLU-667, in Patients with Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors

A.3.1

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

A study of BLU-667 in patients with lung cancer, and thyroid cancer, and other solid tumors

A.3.2

Name or abbreviated title of the trial where available

ARROW

A.4.1

Sponsor's protocol code number

BO42863

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03037385

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	F. Hoffmann-La Roche Ltd
B.1.3.4	Country	Switzerland
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	F. Hoffmann-La Roche Ltd
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	F. Hoffmann-La Roche Ltd
B.5.2	Functional name of contact point	Trial Information Support Line-TISL
B.5.3	Address:
B.5.3.1	Street Address	Grenzacherstrasse 124
B.5.3.2	Town/ city	Basel
B.5.3.3	Post code	CH-4070
B.5.3.4	Country	Switzerland
B.5.6	E-mail	global.rochegenentechtrials@roche.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	Pralsetinib
D.3.2	Product code	RO7499790
D.3.4	Pharmaceutical form	Capsule
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	Pralsetinib
D.3.9.1	CAS number	2097132-94-8
D.3.9.2	Current sponsor code	RO7499790
D.3.9.3	Other descriptive name	BLU-667
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	100
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	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

Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other Advanced Solid Tumors

E.1.1.1

Medical condition in easily understood language

Thyroid Cancer, Non-Small Cell Lung Cancer (NSCLC) and Other
Advanced Solid Tumors

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10066914
E.1.2	Term	Thyroid tumor
E.1.2	System Organ Class	100000004864

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	10025044
E.1.2	Term	Lung cancer
E.1.2	System Organ Class	100000004864

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10065252
E.1.2	Term	Solid tumor
E.1.2	System Organ Class	100000004864

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10029514
E.1.2	Term	Non-small cell lung cancer NOS
E.1.2	System Organ Class	100000004864

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Phase 1:
• To determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of pralsetinib.
• To determine the safety and tolerability of pralsetinib.

Phase 2
• To determine the ORR by RECIST v1.1 (or RANO, if appropriate for
tumor type) by disease type, and/or RET-altered status (including
patients treated at MTD/RP2D in Phase 1), and/or prior treatment status
specified in enrollment group definition, if appropriate.
• To further define the safety and tolerability of pralsetinib

E.2.2

Secondary objectives of the trial

Phase 1
-ORR by RECIST or RANO for all patients treated with IMP according to disease type, RET-altered, prior treatment status, if appropriate. Patients initially treated with MTD/RP2D will be pooled with Phase 2 patients
-BL RET gene status in plasma +/or tumor tissue + correlate with ORR, CBR, DCR + DOR
-IMP PK profile + correlate drug exposure with safety
-IMP PDs, incl changes in blood calcitonin + CEA in MTC patients only + changes in tumor DUSP6 and SPRY4 levels in all patients
Phase 2
-Additional measures DOR, CBR, DCR, PFS + OS in all patients by disease type, RET-altered status, prior treatment status. Patients treated at MTD/RP2D in Phase 1 will be pooled with Phase 2 patients
-BL RET gene status in plasma +/or tumor tissue + correlate with ORR, CBR, DOR, + DCR
-IMP PK profile + correlate drug exposure with safety assessments, incl changes in ECG + efficacy
-IMP PDs, incl changes in blood calcitonin + CEA in MTC patients only
-Brain activity in NSCLC patients

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Patient is ≥ 18 years of age.
2. Diagnosis during dose escalation (Phase 1) – Pathologically documented, definitively diagnosed non-resectable advanced solid tumor.
• All patients treated at doses > 120 mg per day must have MTC, or a RET-altered solid tumor per local assessment of tumor tissue and/or blood.
• Phase 1 enrichment patients must have MTC or a RET-altered solid tumor per local assessment of tumor tissue and/or blood.
3. Diagnosis during dose expansion (Phase 2) – All patients (with the exception of patients with MTC enrolled in Groups 3 and 4) must have an oncogenic RET fusion or mutation (excluding synonymous, frameshift, and nonsense mutations) solid tumor, as determined by local or central testing of tumor or circulating tumor nucleic acid in blood; as detailed below.
• Group 1 – patients must have pathologically documented, definitively diagnosed locally advanced or metastatic NSCLC with a RET fusion previously treated with a platinum-based chemotherapy.
• Group 2 – patients must have pathologically documented, definitively
diagnosed locally advanced or metastatic NSCLC with a RET fusion not
previously treated with a platinum-based chemotherapy, including those
who have not had any systemic therapy. Prior platinum chemotherapy in
the neoadjuvant and adjuvant setting is permitted if the last dose of
platinum was 4 months or more before the first dose of study drug.
• Group 3 – patients must have pathologically documented, definitively diagnosed advanced MTC that has progressed within 14 months prior to the Screening Visit and was previously treated with cabozantinib and/or vandetanib.
• Group 4 – patients must have pathologically documented, definitively diagnosed advanced MTC that has progressed within 14 months prior to the Screening Visit and was not previously treated with cabozantinib or vandetanib.
• Group 5 – patients must have a pathologically documented, definitively diagnosed advanced solid tumor with an oncogenic RET fusion, have previously received SOC appropriate for their tumor type (unless there is no accepted standard therapy for the tumor type or the investigator has determined that treatment with standard therapy is not appropriate), and must not eligible for any of the other groups.
• Group 6 – patients must have a pathologically documented, definitively diagnosed advanced solid tumor with an oncogenic RET fusion or mutation, previously treated with a selective TKI that inhibits RET, such as selpercatinib.
• Group 7: patients must have a pathologically documented, definitively diagnosed advanced solid tumor with an oncogenic RET mutation previously treated with SOC appropriate for the tumor type and not eligible for any of the other groups.
4. Patient must have non-resectable disease. Prior to Protocol Amendment 9, patients must have progressed following standard therapy or has not adequately responded to standard therapy, or the patient must be intolerant to, or the Investigator has determined that treatment with standard therapy is not appropriate, or there must be no accepted standard therapy for their disease.
5. Dose expansion (Phase 2) patients in all groups (except group 7) must have measurable disease per RECIST v1.1 (or RANO, if appropriate for tumor type).
6. Patient agrees to provide tumor tissue (archived, if available or a fresh biopsy) for RET status confirmation and is willing to consider an on-treatment tumor biopsy, if considered safe and medically feasible by the treating Investigator. For Phase 2, Group 6, patients are required to undergo a pretreatment biopsy to define baseline RET status in tumor
tissue.
7. Patient has Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-1.
8. Patient or legal guardian provides informed consent to participate in the study.

E.4

Principal exclusion criteria

1. Patient's cancer has a known primary driver alteration other than RET Investigators should discuss enrollment with Sponsor regarding comutations
2. Patient has any of the following within 14 d prior to the first dose of IMP:
a. Platelet count < 75 × 109/L
b. Absolute neutrophil count < 1.0 × 109/L
c. Hemoglobin < 9.0 g/dL red blood cell transfusion and erythropoietin may be used to reach at least 9.0 g/dL, but must have been administered at least 2 weeks prior to 1st IMP dose
d. AST or ALT > 3 × ULN if no hepatic metastases are present; > 5 × ULN if hepatic metastases are present
e. Total bilirubin > 1.5 × ULN; > 3 × ULN with direct bilirubin > 1.5 × ULN in presence of Gilbert's disease
f. Estimated (Cockroft-Gault formula) or measured creatinine clearance < 40 mL/min
g. Total serum phosphorous > 5.5 mg/dL
3. Patient has a QTcF > 470 msec. Patient has a history of prolonged QT syndrome or Torsades de pointes. Patient has a familial history of prolonged QT syndrome
4. Patient has clinically significant uncontrolled, cardiovascular disease including congestive heart failure Grade III or IV according to the NY Heart Association classification; myocardial infarction or unstable angina within the previous 6 months, uncontrolled hypertension, or clinically significant, uncontrolled arrhythmias, including bradyarrhythmias that may cause QT prolongation
5. Patient has CNS metastases or a primary CNS tumor associated with progressive neurological symptoms or requires increasing doses of corticosteroids to control the CNS disease. If a patient requires corticosteroids for management of CNS disease, the dose must have been stable for the 2 weeks preceding C1D1
6. Presence of clinically symptomatic interstitial lung disease or interstitial pneumonitis, including radiation pneumonitis
7. Patient received the following anti-cancer therapy:
a. Any systemic anticancer therapy (except for immunotherapy or other antibody therapies) and all forms of radiotherapy within 14 d or 5 halflives prior to first IMP dose. IMP may be started within these washout periods if considered by the Investigator to be safe and within the best interest of the patient prior Sponsor approval
b. Any immunotherapy or other antibody therapy within 28 d prior to the 1st dose of IMP (immune related toxicities must have resolved to < Grade 2 prior to starting IMP)
8. Dose expansion patients in Groups 1-5 and 7 (Phase 2): patient has previously received treatment with a selective RET inhibitor such as selpercatinib
9. Patient received neutrophil growth factor support within 14 d of 1st IMP dose
10. Patient requires treatment with a prohibited medication or herbal remedy that cannot be discontinued at least 2 weeks before start of IMP administration. IMP may be started within 14 d or 5 half-lives of prior therapy if considered by the Investigator to be safe and within the best
interest of the patient, with prior Sponsor approval
11. Patient has had a major surgical procedure within 14 d of the first IMP dose
12. Patient has a history of another primary malignancy that has been diagnosed or required therapy (except maintenance anti-hormonal therapy) within the past year. The following prior malignancies are notNot exclusionary are: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, curatively treated localized thyroid cancer, and completely resected carcinoma in situ of any site
13. Patient is unwilling or unable to comply with scheduled visits, drug administration plan, laboratory tests, or other study procedures and study restrictions
14. Women who are unwilling, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ highly effective contraception during IMP administration period and for at least 14 days after the last IMP dose. Men who are unwilling, if not surgically sterile, to abstain from sexual intercourse or employ highly effective contraception during the IMP administration period and for at least 7 days after the last IMP dose
15. Pregnant females, as documented by a serum β-hCG pregnancy test consistent with pregnancy, obtained within 7 days prior to the first dose of study drug. Females with β-hCG values that are within the range for pregnancy but are not pregnant (false-positives) may be enrolled with written consent of the Sponsor, after pregnancy has been ruled out. Females of non-childbearing potential (postmenopausal for more than 1 year; bilateral tubal ligation; bilateral oophorectomy; hysterectomy) do not require a serum β-hCG test
16. If female, patient is breastfeeding
17.Patient has prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, or laboratory abnormality that, in the Investigators/Sponsors opinion, could affect the safety of the patient; alter the absorption, distribution, metabolism, or excretion of the study drug; or impair the assessment of study results

E.5 End points

E.5.1

Primary end point(s)

Phase 1
• MTD and RP2D (recommended Phase 2 dose) of pralsetinib.
• Overall safety profile of pralsetinib, as assessed by the type, frequency,
severity, timing, and relationship to study drug of any AEs, SAEs,
changes in vital signs, ECGs, and safety laboratory tests.
Phase 2
• ORR by patients' disease type, and/or RET-altered status if applicable,
and/or prior treatment status if appropriate.
• Overall safety profile of BLU-667, as assessed by the type, frequency,
severity, timing, and relationship to study drug of any AEs, SAEs,
changes in vital signs, ECGs, and safety laboratory tests.

E.5.1.1

Timepoint(s) of evaluation of this end point

- MTD and RP2D of pralsetinib– End of Cycle 1 in Phase 1
- Overall safety profile of pralsetinib– Safety assessments will be ongoing
throughout all cycles.

E.5.2

Secondary end point(s)

Phase 1
• ORR for all pralsetinib treated patients according to patients' disease
type and/or RET-altered status if applicable, and/or prior treatment
status if appropriate. Patients initially treated with MTD/RP2D in Phase
1 will be pooled with Phase 2 patients accordingly.
• RET gene status and correlation between RET gene status and ORR,
CBR, DOR, PFS, OS, and DCR, and other measures of antineoplastic
activity (in plasma and/or tissue) in all patients according to their
disease type and/or RET-altered status, if applicable, and/or prior
treatment status, if appropriate.
• PK parameters of pralsetinib: Pharmacokinetic parameters of interest
will include, as appropriate, maximum plasma drug concentration
(Cmax), time to maximum plasma drug concentration (Tmax), time of
last quantifiable plasma drug concentration (Tlast), area under the
plasma concentration versus time curve from time 0 to 24 hours
postdose (AUC0-24), plasma drug concentration at 24 hours postdose
(C24); apparent
volume of distribution (Vz/F), terminal elimination half-life (t½),
apparent oral clearance (CL/F), and accumulation ratio (R).
• Pharmacodynamic parameters of pralsetinib: changes in tumor/blood
including, but not limited to, changes in blood calcitonin and CEA (MTC
patients); and changes in tumor biomarker levels (DUSP6 and SPRY4)
levels (all patients)
Phase 2
• DOR, CBR, DCR, PFS, and OS in all patients by disease type and/or
RET-altered status, if applicable, and/or prior treatment status, if
appropriate.
• RET gene status and correlation between RET gene status and ORR,
CBR, DOR, DCR, and other measures of antineoplastic activity (in plasma
and/or tissue) in all patients according to their disease type and/or RETaltered
status, if applicable, and/or prior treatment status, if
appropriate.
• PK parameters of pralsetinib: Pharmacokinetic parameters of interest
will include, as appropriate, maximum plasma drug concentration
(Cmax), time to maximum plasma drug concentration (Tmax), time of
last quantifiable plasma drug concentration (Tlast), area under the
plasma concentration versus time curve from time 0 to 24 hours
postdose (AUC0-24), plasma drug concentration at 24 hours postdose
(C24); apparent
volume of distribution (Vz/F), terminal elimination half-life (t½),
apparent oral clearance (CL/F), and accumulation ratio (R).
• ECG Assessment: In Phase 2, the effects of BLU-667 on ECG
parameters will be evaluated for approximately 20 patients using 12-
lead ECGs extracted from continuous recordings (12-lead Holter) on
C1D1 and C1D15. Individual ECGs will be extracted in replicate from the
12-lead Holter recordings at specified time points
and will be evaluated by a central laboratory. QT intervals will be
measured from lead II and will be corrected for heart rate (QTc) using
Fridericia's correction factors (QTcF). The primary QTc parameter will be
QTcF. Secondary parameters (heart rate, PR, and QRS, and T-wave
morphology) will also be evaluated. Potential effects of pralsetinib will be
evaluated as change from predose baseline heart rate, PR, QRS, and
QTcF by postdose time point. For the purpose of QT assessment,
exposure-response analysis will be performed on the relationship
between pralsetinib systemic levels and change in QTcF.
• Pharmacodynamic parameters of BLU-667: changes in tumor/blood
including, but not limited to, changes in blood calcitonin and CEA (MTC
patients only).

E.5.2.1

Timepoint(s) of evaluation of this end point

– Blood samples collected on day 1 of Cycle 1 (through 24 hours post-dose of C1D1), C1D15 (through 24 hours post-dose of C1D15), and pre-dose on D 1 of C2-C4, and EOT.
– Blood Samples collected at pre-dose of C1D1, C1D15, C2D1, C3D1 and every odd cycle thereafter, and EOT. Tumor sample collected at screening, C2D1, and EOT.
- Blood Samples collected at pre-dose of C1D1, C1D15, C2D1, C3D1 and every odd cycle thereafter, and EOT. Tumor sample collected at screening, C2D1, and EOT.
- MRI or CT scans for disease response assessment (per RECIST, version 1.1) at Screening, on D1 of every odd-numbered cycle starting with C3, and EOT.

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

Yes

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)

Yes

E.7.1.1

First administration to humans

Yes

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

Yes

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.8.3

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

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

Hong Kong

Korea, Republic of

Singapore

Taiwan

United States

Belgium

France

Germany

Italy

Netherlands

Spain

United Kingdom

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 time that the last patient completes his/her last visit (LPLV), including assessments performed as part of PFS follow-up, if the patient enters the PFS follow-up part of the study

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:

453

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

194

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

252

F.4.2.2

In the whole clinical trial

647

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)

Normal standard of care.

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

2018-10-01

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-12-10

P. End of Trial
P.	End of Trial Status	Completed

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Select Trial Status:	Select Trial Phase:
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Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
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