Clinical Trials Register

Summary
EudraCT Number:	2020-004533-21
Sponsor's Protocol Code Number:	C1071003
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-12-15
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

Spain - AEMPS

A.2

EudraCT number

2020-004533-21

A.3

Full title of the trial

AN OPEN-LABEL, MULTICENTER, NON-RANDOMIZED PHASE 2 STUDY OF PF-06863135 MONOTHERAPY IN PARTICIPANTS WITH MULTIPLE MYELOMA WHO ARE REFRACTORY TO AT LEAST ONE PROTEASOME INHIBITOR, ONE IMMUNOMODULATORY DRUG AND ONE ANTI-CD38 ANTIBODY

ESTUDIO ABIERTO, MULTICÉNTRICO, NO ALEATORIZADO EN FASE II DE PF-06863135 EN MONOTERAPIA EN PARTICIPANTES CON MIELOMA MÚLTIPLE RESISTENTE A AL MENOS UN INHIBIDOR DE PROTEASOMA, UN FÁRMACO INMUNOMODULADOR Y UN ANTICUERPO ANTI-CD38

A.3.1

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

A Phase 2 Study of PF-06863135 Monotherapy in Participants With MM Who Are Refractory to at Least One PI, One IMiD and One Anti-CD38 mAb

Estudio en fase II de PF-06863135 en monoterapia en participantes con MM resistente a al menos un IP, un FIM y un ACm anti-CD38

A.4.1

Sponsor's protocol code number

C1071003

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	Pfizer 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	Pfizer, 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	Pfizer, Inc.
B.5.2	Functional name of contact point	ClinicalTrials.gov Call Center
B.5.3	Address:
B.5.3.1	Street Address	235 East 42nd Street
B.5.3.2	Town/ city	New York
B.5.3.3	Post code	NY 10017
B.5.3.4	Country	United States
B.5.4	Telephone number	+1800 7181021
B.5.6	E-mail	ClinicalTrials.gov_Inquiries@pfizer.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	PF-06863135
D.3.2	Product code	PF-06863135
D.3.4	Pharmaceutical form	Solution for injection
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	PF-06863135
D.3.9.2	Current sponsor code	PF-06863135
D.3.9.4	EV Substance Code	SUB201694
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	40
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	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

MULTIPLE MYELOMA

Mieloma Múltiple

E.1.1.1

Medical condition in easily understood language

A hematological B-cell malignancy characterized by dysregulated proliferation of BM plasma cells.

Cáncer de la sangre que afecta a células B caracterizada por una regulación incorrecta en la proliferación de células plasmáticas en la médula ósea

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	10028228
E.1.2	Term	Multiple myeloma
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

To determine the efficacy of PF-06863135 in patients with no prior BCMA therapy (Cohort A) and prior BCMA therapy (cohort B).

Determinar la eficacia de PF-06863135 en pacientes sin tratamiento previo frente al BCMA (cohorte A) y con tratamiento previo frente al BCMA (cohorte B).

E.2.2

Secondary objectives of the trial

- To determine additional efficacy of PF-06863135 in Cohort A and Cohort B
- To determine the safety and tolerability of PF-06863135
- To evaluate the PK of PF-06863135
- To evaluate the immunogenicity of PF-06863135

- Determinar la eficacia adicional de PF-06863135 en la cohorte A y la cohorte B.
- Evaluar la seguridad y tolerabilidad de PF-06863135.
- Evaluar la FC de PF-06863135.
- Evaluar la inmunogenicidad de PF-06863135.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
Age and Sex:
1. Male or female participants age ≥18 years.
A female participant is eligible to participate if she is not pregnant or
breastfeeding. Refer to Appendix 4 for all reproductive criteria for male
(Section 10.4.1) and female (Section 10.4.2) participants.

Type of Participant and Disease Characteristics:
2. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
3. Prior diagnosis of MM as defined according to IMWG criteria (Rajkumar et al, 2014).
4. Measurable disease based on IMWG criteria as defined by at least 1 of the following:
a. Serum M-protein >0.5 g/dL by SPEP
b. Urinary M-protein excretion >200 mg/24 hours by UPEP
c. Serum immunoglobulin FLC≥10 mg/dL (≥100 mg/L) AND abnormal serum
immunoglobulin kappa to lambda FLC ratio (<0.26 or >1.65)
5. Refractory to at least one IMiD.
6. Refractory to at least one PI.
7. Refractory to at least one anti-CD38 antibody.
8. Relapsed/refractory to last anti-MM regimen.
Note: Refractory is defined as having disease progression while on therapy or within
60 days of last dose in any line, regardless of response.
9. Cohort A: Has not received prior BCMA-directed therapy.
Cohort B: Has received prior BCMA-directed ADC or BCMA-directed CAR T-cell
therapy, either approved or investigational.
10. ECOG performance status ≤2.
11. LVEF ≥40% as determined by a MUGA scan or ECHO.
12. Adequate hepatic function characterized by the following:
a. Total bilirubin ≤2 x ULN (≤3 x ULN if documented Gilbert’s syndrome);
b. AST ≤2.5 x ULN; and
c. ALT ≤2.5 x ULN
13. Adequate renal function defined by an estimated creatinine clearance ≥30 mL/min (according to the Cockcroft Gault formula, by 24-hour urine collection for creatinine clearance, or according to local institutional standard method).
14. Adequate BM function characterized by the following:
a. ANC ≥1.0 × 10^9/L;
b. Platelets ≥25 × 10^9/L; and
c. Hemoglobin ≥8 g/dL
15. Resolved acute effects of any prior therapy to baseline severity or CTCAE Grade ≤1

Informed Consent:
16. Capable of giving signed informed consent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol.

Los participantes serán aptos para ser incluidos en el estudio solo si cumplen todos los criterios siguientes:
Edad y sexo:
1. Hombres o mujeres de ≥18 años de edad.
Las mujeres participantes serán aptas para participar si no están embarazadas ni en periodo de lactancia. Consulte el Anexo 4paratodoslos criterios reproductivos para el hombre (sección 10.4.1) y la mujer (sección 10.4.2) los participantes.

Tipo de participante y características de la enfermedad
2. Los participantes deben estar dispuestos y ser capaces de cumplir con todas las visitas programadas, el plan de tratamiento, las pruebas analíticas, las consideraciones sobre el estilo de vida y otros procedimientos del estudio.
3. Diagnóstico previo de MM según los criterios del IMWG. (Rajkumar et al., 2014).
4. Enfermedad medible según los criterios del IMWG, definida por al menos 1 de los siguientes criterios:
a. Proteína M en suero >0,5 g/dl mediante EPS
b. Excreción de proteína M en orina >200 mg/24 horas mediante UPEP
c. CLL de inmunoglobulina sérica ≥10 mg/dl (≥100 mg/l) Y cociente anormal de las CLL de inmunoglobulina κ a lambda (<0.26 o >1,65)
5. Resistente a al menos un FIM.
6. Resistente a al menos un IP.
7. Resistente a al menos un anticuerpo anti-CD38.
8. Recidivante/resistente al último régimen contra el MM.
Nota: Se define como resistente la aparición de progresión de la enfermedad durante el tratamiento o en los 60 días posteriores a la última dosis en cualquier línea, independientemente de la respuesta.
9. Cohorte A: No haber recibido tratamiento dirigido al BCMA previo. Cohorte B: Haber recibido anteriormente CAF dirigidos al BCMA o tratamiento con linfocitos T CAR dirigida al BCMA, ya sea aprobado o en investigación.
10. Estado funcional del ECOG ≤2.
11. FEVI ≥40 % determinada mediante una MUGA o un ECO.
12. Función hepática adecuada caracterizada por lo siguiente:
a. Bilirrubina total ≤2 x LSN (≤3 x LSN si se documenta síndrome de Gilbert);
b. AST ≤2,5 x LSN y
c. ALT ≤2,5 x LSN
13. Función renal adecuada definida por un aclaramiento de creatinina estimado ≥30 ml/min según la fórmula de Cockcroft Gault o mediante la recogida de orina de 24 horas para el aclaramiento de creatinina, o de acuerdo con el método de referencia institucional local.
14. Función BM caracterizada por lo siguiente:
a. RAN ≥1,0 × 109/l;
b. Plaquetas ≥25 × 109/l y
c. Hemoglobina ≥8 g/dl.
15. Los efectos agudos de cualquier tratamiento anterior resueltos a la gravedad inicial o a grado ≤1 de los criterios comunes de terminología para acontecimientos adversos (CTCAE).

Consentimiento informado:
16. Capacidad de otorgar el consentimiento informado firmado, tal y como se describe en el Apéndice 1 del protocolo, lo que incluye el cumplimiento de los requisitos y las restricciones indicadas en el DCI y en este protocolo.

E.4

Principal exclusion criteria

Participants are excluded from the study if any of the following criteria apply:
Medical Conditions:
1. Smoldering MM.
2. Active Plasma cell leukemia.
3. amyloidosis.
4. Stem cell transplant within 30 days prior to enrollment or active GVHD.
5. Impaired cardiovascular function or clinically significant cardiovascular diseases, defined as any of the following within 6 months prior to enrollment:
a. Acute myocardial infarction or acute coronary syndromes (eg, unstable angina, coronary artery bypass graft, coronary angioplasty or stenting, symptomatic pericardial effusion);
b. Clinically significant cardiac arrhythmias (eg, uncontrolled atrial fibrillation or uncontrolled paroxysmal supraventricular tachycardia);
c. Thromboembolic or cerebrovascular events (eg, transient ischemic attack,
cerebrovascular accident, deep vein thrombosis or pulmonary embolism);
d. Prolonged QT syndrome (or triplicate average QTcF >470 msec at screening).

6. Active HBV, HCV, SARS-CoV2, HIV, or any active, uncontrolled bacterial, fungal, or viral infection.
7. Any other active malignancy within 3 years prior to enrollment, except for adequately treated basal cell or squamous cell skin cancer, or carcinoma in situ.
8.Other surgical (including major surgery within 14 days prior to enrollment), medical or psychiatric conditions including recent (within the past year) or active suicidal ideation/behavior or laboratory
abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.

Prior/Concomitant Therapy:
9. Previous treatment with an anti-BCMA bispecific antibody.

Prior/Concurrent Clinical Study Experience:
10. Previous administration with an investigational drug within 30 days (or as determined by the local requirement) or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer).

Other Exclusions:
11. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.

12. Known or suspected hypersensitivity to the study drug or any of its excipients.

Los participantes serán excluidos del estudio si cumplen alguno de los criterios siguientes:
Enfermedades:
1. MM latente.
2. Leucemia de células plasmáticas activa.
3. Amiloidosis.
4. Trasplante de células madre en los 30días previos a la inscripción o EICH activa.
5. Deterioro de la función cardiovascular o de enfermedades cardiovasculares clínicamente significativas, definidas como cualquiera de los siguientes trastornos en los 6 meses anteriores a la inscripción:
a. Infarto agudo de miocardio o síndromes coronarios agudos (p. ej., angina inestable, injerto de baipás de la arteria coronaria, angioplastia coronaria o colocación de endoprótesis vascular, derrame pericárdico sintomático);
b. Arritmias cardíacas clínicamente significativas (p. ej., fibrilación auricular no controlada o taquicardia paroxística supraventricular no controlada).
c. Acontecimientos tromboembólicos o cerebrovasculares (p. ej., accidente isquémico transitorio, accidente cerebrovascular, trombosis venosa profunda o embolia pulmonar);
d. Síndrome de QT prolongado (o QTcF promedio por triplicado >470 ms en la selección).
6. VHB activo, VHC, SRAG-CoV2, VIH o cualquier infección bacteriana, fúngica o vírica activa no controlada.
7. Cualquier otra neoplasia maligna activa en los 3 años anteriores a la inscripción, excepto carcinoma basocelular o espinocelular, o carcinoma in situ tratados adecuadamente.
8. Otras afecciones quirúrgicas(incluida la cirugía mayor en los 14días previos a la inscripción),médicas o psiquiátricas, incluidos los comportamientos o ideas suicidas recientes (en el último año), o anomalías de laboratorio que puedan aumentar el riesgo de la participación en el estudio o, a juicio del investigador, hagan que el participante no sea apto para el estudio.

Tratamiento previo/concomitante:
9. Tratamiento previo con un anticuerpo biespecífico anti-BCMA.

Experiencia en estudios clínicos previos o simultáneos
10. Administración previa con un fármaco en investigación en los 30 días (o según lo determine el requisito local) o 5 semividas previos a la primera dosis de la intervención utilizada en este estudio (lo que dure más).

Otras exclusiones:
11. Empleados del centro de investigación o de Pfizer directamente implicados en la realización del estudio, empleados del centro supervisados de alguna otra manera por el investigador, y sus respectivos familiares.
12. Hipersensibilidad conocida o sospecha de hipersensibilidad al fármaco del estudio o a alguno de sus excipientes

E.5 End points

E.5.1

Primary end point(s)

ORR by BICR per IMWG

TRO mediante RCIE según el IMWG.

E.5.1.1

Timepoint(s) of evaluation of this end point

In each of Cohort A and Cohort B, BOR will be assessed based on reported overall responses recorded at evaluation time points from the date of first dose until the first documentation of PD, death or start of new anticancer therapy, whichever occurs first.
- OR will encompass confirmed sCR, CR, VGPR and PR.

En cada una de las cohortes A y B, RO será evaluada en base a TRO comunicada en los tiempos desde la fecha de la primera dosis hasta la primera documentación de PE, muerte o inicio de un nuevo tratamiento antineoplásico, lo que ocurra primero.
- RO definida como RCE confirmada, RC, MBRP y RP.

E.5.2

Secondary end point(s)

To determine additional efficacy of PF-06863135 in Cohort A and Cohort B:
- DOR by BICR and investigator per IMWG
- CCRR by BICR and investigator per IMWG
- ORR by investigator per IMWG
- DOCCR by BICR and investigator per IMWG
- PFS by BICR and investigator per IMWG
- OS
- TTR by BICR and investigator per IMWG
- MRD negativity rate (central lab) per IMWG

To determine the safety and tolerability ofPF-06863135
- AEs and laboratory abnormalities as graded by NCI CTCAE v5.0.
- Severity of CRS and ICANS assessed according to ASTCT criteria (Lee et al, 2019).

To evaluate the PK of PF-06863135
- Pre- and postdose concentrations of PF-06863135

To evaluate the immunogenicity of PF-06863135
- ADAs and NAbs against PF-06863135

Determinar la eficacia adicional de PF-06863135 en la cohorte A y la cohorte B:
- DR por RCIE e investigador según el IMWG.
- RCC por RCIE e investigador según el IMWG.
- TRO por el investigador según el IMWG.
- DRCC por RCIE y el investigador según el IMWG.
- SSP por RCIE y el investigador según el IMWG.
- SG.
- TTR por RCIE y el investigador según el IMWG.
- Tasa de negatividad de EMR (laboratorio central) según el IMWG.

Evaluar la seguridad y tolerabilidad de PF-06863135:
- AA y anomalías en el análisis clasificadas según los CTCAE del NCI v5.0.
- Gravedad del SLC y de los ICANS evaluados según los criterios del ASTCT (Lee et al., 2019).

Evaluar la FC de PF-06863135:
- Concentraciones anteriores y posteriores a la dosis de PF-06863135.

Evaluar la inmunogenicidad de PF-06863135:
- ACF y ACN contra PF-06863135.

E.5.2.1

Timepoint(s) of evaluation of this end point

Analyses of secondary efficacy endpoints will use the Safety Analysis Set. Please refer to section 9.4.3 of the protocol for a complete description of each secondary endpoint.

Los análisis de las variables secundarias utilizarán el Conjunto de Análisis de Seguridad. Por favor, ir a la sección 9.4.3 del protocolo para una descripción completa de cada criterio de valoración secundario.

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

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

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

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.8.2.4

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

Australia

Belgium

Canada

Germany

Japan

Poland

Spain

United Kingdom

United States

France

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

LVLS

LVLS - última visita del último paciente

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

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:

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

F.4.2.2

In the whole clinical trial

150

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

Ninguno

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

2021-05-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-04-28

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

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