Clinical Trials Register

Summary
EudraCT Number:	2016-003498-16
Sponsor's Protocol Code Number:	20290
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2017-02-01
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

Germany - BfArM

A.2

EudraCT number

2016-003498-16

A.3

Full title of the trial

A Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients with Advanced Solid or Primary Central Nervous System Tumors

A.3.1

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

A study to test the safety and efficacy of the drug larotrectinib for the treatment of tumors with NTRK-fusion in children

A.3.2

Name or abbreviated title of the trial where available

SCOUT

A.4.1

Sponsor's protocol code number

20290

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02637687

A.5.4

Other Identifiers

Name:

previous sponsor: Loxo Oncology Inc.

Number:

LOXO-TRK-15003

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/179/2017

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Bayer Consumer Care AG
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	Bayer Consumer Care AG
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	Bayer AG
B.5.2	Functional name of contact point	Bayer Clinical Trials Contact
B.5.3	Address:
B.5.3.1	Street Address	N/A
B.5.3.2	Town/ city	Berlin
B.5.3.3	Post code	13342
B.5.3.4	Country	Germany
B.5.4	Telephone number	+4930300139 003
B.5.6	E-mail	clinical-trials-contact@bayer.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	larotrectinib
D.3.2	Product code	BAY 2757556
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	larotrectinib
D.3.9.2	Current sponsor code	BAY 2757556
D.3.9.3	Other descriptive name	LOXO-101
D.3.9.4	EV Substance Code	SUB179762
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	25
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.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	larotrectinib
D.3.2	Product code	BAY 2757556
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	larotrectinib
D.3.9.2	Current sponsor code	BAY 2757556
D.3.9.3	Other descriptive name	LOXO-101
D.3.9.4	EV Substance Code	SUB179762
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.IMP: 3
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	larotrectinib
D.3.2	Product code	BAY 2757556
D.3.4	Pharmaceutical form	Oral solution
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	larotrectinib
D.3.9.2	Current sponsor code	BAY 2757556
D.3.9.3	Other descriptive name	LOXO-101
D.3.9.4	EV Substance Code	SUB179762
D.3.10	Strength
D.3.10.1	Concentration unit	% percent
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	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

Solid tumors harboring NTRK fusion

E.1.1.1

Medical condition in easily understood language

Solid cancers harboring an NTRK gene fusion, rare cancers caused by
specific changes in the gene

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	10007959
E.1.2	Term	Central nervous system neoplasm NOS
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	PT
E.1.2	Classification code	10007958
E.1.2	Term	Central nervous system neoplasm
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	HLT
E.1.2	Classification code	10007960
E.1.2	Term	Central nervous system neoplasms malignant NEC
E.1.2	System Organ Class	100000004852

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	10065147
E.1.2	Term	Malignant solid tumor
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	23.0
E.1.2	Level	PT
E.1.2	Classification code	10081769
E.1.2	Term	NTRK gene fusion overexpression
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

Phase 1: The primary objective of the study is to determine the safety of
oral larotrectinib, including DLT, in pediatric patients with advanced
solid or primary CNS tumors.
Phase 2: To determine the overall response rate (ORR) as determined by
an independent radiology review committee and measured by the
proportion of patients with best overall confirmed response of complete
response (CR) or partial response (PR) according to the appropriate
tumor response criteria following treatment with larotrectinib in
pediatric patients with an advanced cancer harboring a fusion involving
NTRK1, NTRK2, or NTRK3 (collectively referred to as NTRK fusions)
fusions.

E.2.2

Secondary objectives of the trial

Phase 1: - To characterize the PK properties of larotrectinib, describe the antitumor activity of larotrectinib and describe pain and health related quality of life (HRQoL) in pediatric patients with advanced solid or primary CNS tumors.
- To identify the MTD and/or the recommended dose of larotrectinib for further clinical investigation in this patient population.
Phase 2: - To determine the ORR.
- To evaluate the duration of response (DOR), duration of progression free survival (PFS) and duration of OS.
- To estimate the proportion of patients that has any tumor regression as a best response.
- To assess the safety profile and tolerability of larotrectinib.
- To evaluate the clinical benefit rate (CBR) and the concordance of prior molecular profiling.
- To characterize post-operative staging and surgical margin status.
- To describe the putative pretreatment surgical plan and capture the
post treatment actual approach with an emphasis on the functional and
cosmetic outcome.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Phase 1 (Closed):
-- Dose escalation: Birth through 21 years of age at C1D1 with a locally
advanced or metastatic solid tumor or primary CNS tumor that has
relapsed, progressed or was nonresponsive to available therapies and
for which no standard or available systemic curative therapy exists; OR
Infants from birth and older with a diagnosis of malignancy and with a
documented NTRK fusion that has progressed or was nonresponsive to
available therapies, and for which no standard or available curative
therapy exists; OR Patients with locally advanced infantile fibrosarcoma
who would require, in the opinion of the investigator, disfiguring surgery
or limb amputation to achieve a complete surgical resection. Phase I
dose escalation cohorts are closed to enrollment.
-- Dose expansion: In addition to the above stated inclusion criteria,
patients must have a malignancy with a documented NTRK gene fusion
with the exception of patients with infantile fibrosarcoma, congenital
mesoblastic nephroma or secretory breast cancer. Patients with infantile
fibrosarcoma, congenital mesoblastic nephroma or secretory breast
cancer may enroll into this cohort with documentation of an ETV6
rearrangement by FISH or RT-PCR or a documented NTRK fusion by next
generation sequencing.
- Phase 2:
-- Infants from birth and older at C1D1 with a locally advanced or
metastatic infantile fibrosarcoma, patients with locally advanced
infantile fibrosarcoma who would require, in the opinion of the
investigator, disfiguring surgery or limb amputation to achieve a
complete surgical resection; OR Birth through 21 years of age at C1D1
with a locally advanced or metastatic solid tumor or primary CNS tumor
that has relapsed, progressed or was nonresponsive to available
therapies and for which no standard or available systemic curative
therapy exists with a documented NTRK gene fusion (or in the case of
infantile fibrosarcoma, congenital mesoblastic nephroma or secretory
breast cancer with documented ETV6 rearrangement (or NTRK3
rearrangement after discussion with the sponsor) by FISH or RT-PCR or
a documented NTRK fusion by next generation sequencing) (identified
through molecular assays as routinely performed at CLIA or other
similarly certified laboratories). Patients with NTRK-fusion positive
benign tumors are also eligible; OR Potential patients older than 21
years of age with a tumor diagnosis with histology typical of a pediatric
patient and an NTRK fusion may be considered for enrollment following
discussion between the local site Investigator and the Sponsor.
- Patients with primary CNS tumors or cerebral metastasis
- Karnofsky (those 16 years and older) or Lansky (those younger than 16 years) performance score of at least 50.
- Adequate hematologic function
- Adequate hepatic and renal function

E.4

Principal exclusion criteria

Patients meeting any of the following criteria are to be excluded from study participation:
1. Major surgery within 14 days (2 weeks) prior to C1D1.
2. Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to C1D1; ongoing cardiomyopathy; or current prolonged QTc interval > 480 milliseconds.
3. Active uncontrolled systemic bacterial, viral, or fungal infection.
4. Malabsorption syndrome or other condition affecting oral absorption.
5. Current treatment with a strong CYP3A4 inhibitor or inducer other than those allowed per Section 6.3.2 of the study protocol.
6. Pregnancy or lactation.
7. Phase 2 only: Prior progression while receiving approved or investigational tyrosine kinase inhibitors targeting TRK, including entrectinib, crizotinib and lestaurtanib. Patients who received a TRK inhibitor for less than 28 days of treatment and discontinued because of intolerance remain eligible.

E.5 End points

E.5.1

Primary end point(s)

Phase 1:
1. Number of participants in an assigned dose cohort with treatment
emergent adverse events (TEAEs) by grade assessed by National Cancer
Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE)
v 4.03 who experience a Dose-limiting toxicity (DLT).
2. Number of participants with TEAEs
3. Severity of TEAEs
Phase 2:
4. Overall response rate (ORR) by independent radiology review
committee (IRRC)

E.5.1.1

Timepoint(s) of evaluation of this end point

Phase 1:
1. From Day 1 to Day 28 of Cycle 1 (1 Cycle=28 days)
2. From first dose of larotrectinib up to 93 months
3. From first dose of larotrectinib up to 93 months
Phase 2:
4. From first dose of Larotrectinib to disease progression or subsequent
therapy or surgical intervention or death, up to 76 months

E.5.2

Secondary end point(s)

Phase 1:
1. Maximum concentration of larotrectinib in plasma (Cmax)
2. Area under the concentration versus time curve from time 0 to
t(AUC0–t) of larotrectinib in plasma
3. Oral clearance (CL/F)
4. Cerebral spinal fluid/plasma ratio of Larotrectinib
5. Maximum tolerated dose (MTD)
6. Recommended dose for Phase 2
7. Overall Response Rate (ORR)
8. Mean change from baseline in Pain scores as assessed by the Wong-
Baker Faces scale
9. Mean change in Health-related quality of life scores by PedsQL-Core
Phase 2:
10. Best overall response (BOR)
11. Duration of response (DOR)
12. Progression-free survival (PFS)
13. Overall survival (OS)
14. Number of participants with Treatment emergent adverse events
(TEAEs)
15. Severity of adverse events as assessed by NCI-CTCAE grading V 4.03
16. Clinical Benefit Rate (CBR)
17. Concordance coefficient
18. Post-operative tumor staging
19. Post-operative surgical margin assessment
20. Pre-treatment surgical plan to preserve function and cosmetic
outcome
21. Post-treatment plans to conserve function and cosmetic outcome

E.5.2.1

Timepoint(s) of evaluation of this end point

Phase 1:
1-3. Cohort 1 and 2: Cycle 1 Day 1 (C1D1) at 1 and 4 hours post-dose
and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and
Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at
pre-dose, 1 and 4 hours post-dose
4. C1D1 in conjunction with the post-dose 1-hour PK sample
5. Up to 16 months
6. Up to 16 months
7. Up to 93 months
8. Up to 93 months
9. Up to 93 months
Phase 2:
10. Up to 76 months
11. Up to 76 months
12. Up to 76 months
13. Up to 112 months
14. Up to 112 months
15. Up to 112 months
16. Up to 112 months
17. Up to 76 months
18. Up to 112 months
19. Up to 112 months
20. Up to 112 months
21. Up to 112 months

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

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

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

Dose escalation

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

Australia

Canada

China

Israel

Japan

United States

Switzerland

Russian Federation

Turkey

Ukraine

Czechia

Denmark

France

Germany

Ireland

Italy

Netherlands

Poland

Spain

Sweden

Korea, Republic of

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

Last Patient Last Visit

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

Number of subjects for this age range:

155

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)

Yes

F.1.1.3.1

Number of subjects for this age range:

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)

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Patients from 1 year old to their legal majority age, with a documented NTRK fusion
Infants from birth with a diagnosis malignancy and with a documented NTRK fusion and for patients with a locally advanced or metastatic infantile fibrosarcoma

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

155

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)

None

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

2017-05-30

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-04-10

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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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:
Results Status:
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