Clinical Trials Register

Summary
EudraCT Number:	2018-002915-93
Sponsor's Protocol Code Number:	ACE-536-LTFU-001
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:	2019-05-21
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

Spain - AEMPS

A.2

EudraCT number

2018-002915-93

A.3

Full title of the trial

A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other Luspatercept (ACE-536) clinical trials.

Estudio de fase 3b de extensión, abierto, de un solo grupo, para evaluar la seguridad a largo plazo en sujetos que hayan participado en otros ensayos clínicos con luspatercept (ACE-536).

A.3.1

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

A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety of Luspatercept in subjects who have participated in other Luspatercept (ACE-536) clinical trials.

Estudio de fase 3b de extensión, abierto, de un solo grupo, para evaluar la seguridad de luspatercept a largo plazo en sujetos que hayan participado en otros ensayos clínicos con luspatercept (ACE-536).

A.4.1

Sponsor's protocol code number

ACE-536-LTFU-001

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	Celgene Corporation
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	Celgene Corporation
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	PPD
B.5.2	Functional name of contact point	PPD Project Manager
B.5.3	Address:
B.5.3.1	Street Address	Sulzbacher Str.48
B.5.3.2	Town/ city	Nuremberg
B.5.3.3	Post code	90489
B.5.3.4	Country	Germany
B.5.4	Telephone number	+34900834223
B.5.6	E-mail	RegistroEspanolDeEstudiosClinicos@druginfo.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/14/1331; EU/3/14/1300
D.3 Description of the IMP
D.3.1	Product name	Luspatercept
D.3.2	Product code	ACE-536
D.3.4	Pharmaceutical form	Lyophilisate for 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	Luspatercept
D.3.9.1	CAS number	1373715-00-4
D.3.9.2	Current sponsor code	ACE-536
D.3.9.3	Other descriptive name	Luspatercept
D.3.9.4	EV Substance Code	SUB189400
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	37.5
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	Yes
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	EU/3/14/1331;EU/3/14/1300
D.3 Description of the IMP
D.3.1	Product name	Luspatercept
D.3.2	Product code	ACE-536
D.3.4	Pharmaceutical form	Lyophilisate for 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	Luspatercept
D.3.9.1	CAS number	1373715-00-4
D.3.9.2	Current sponsor code	ACE-536
D.3.9.3	Other descriptive name	Luspatercept
D.3.9.4	EV Substance Code	SUB189400
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	87.5
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	Yes
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

Prior participation on a clinical trial of luspatercept (ACE-536) in protocols eligible for participation in this study ACE-536-LTFU-001 with the following medical conditions:
- myelodysplastic syndrome (MDS)
- beta (β)-thalassemia (THAL)
- myelofibrosis (MF)

Participación previa en un ensayo clínico con luspatercept (ACE-536) en protocolos que reúnan las condiciones para participar en el estudio ACE-536-LTFU-001 con las siguientes condiciones médicas:
- Sindrome mielodisplásicos (SMD)
- beta (β)-thalassemia (THAL)
- mielofibrosis (MF)

E.1.1.1

Medical condition in easily understood language

myelodysplastic syndrome (MDS); beta (β)-thalassemia (THAL); myelofibrosis (MF)

Sindrome mielodisplásicos (SMD); beta (β)-thalassemia (THAL); mielofibrosis (MF)

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

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 long-term safety (including progression to acute myeloid leukemia (AML) and/or other malignancies/pre-malignancies) of luspatercept in subjects who have participated in other luspatercept clinical trials.

Evaluar la seguridad a largo plazo (incluida la progresión a leucemia mieloide aguda (LMA) u otras neoplasias malignas o lesiones premalignas) de luspatercept en pacientes que hayan participado en otros ensayos clínicos con luspatercept.

E.2.2

Secondary objectives of the trial

To follow subjects for overall survival

Hacer un seguimiento de la supervivencia global de los sujetos.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject is ≥ 18 years at the time of signing the informed consent form (ICF).
2. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
3. Subject has been participating in a luspatercept trial and continues to fulfill all the requirements of the parent protocol and the subject has been either:
a. Assigned to luspatercept treatment, continues to receive clinical benefit in the opinion of the investigator and should continue to receive luspatercept treatment, OR
b. Assigned to placebo arm in the parent protocol (at the time of unblinding or in follow-up) and should cross over to luspatercept treatment, OR
c. Assigned to the Follow-up Phase of the parent protocol, previously treated with luspatercept or placebo in the parent protocol who shall continue into Long-term Post-treatment Follow-up Phase in the rollover study until the follow-up commitments are met (unless requirements are met as per parent protocol to crossover
to luspatercept treatment).
4. Subject understands and voluntarily signs an informed consent document prior to any study-related assessments or procedures being conducted.
5. Subject demonstrates compliance, as assessed by the investigator, with the parent study protocol requirements.
6. Applies to on treatment subjects only- females of childbearing potential (FCBP) defined as a sexually mature woman who:
1) has achieved menarche at some point,
2) has not undergone a hysterectomy or bilateral oophorectomy, or
3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must:
a. Have two negative pregnancy tests as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the subject practices true abstinence* from heterosexual contact.
b. Either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy.
7. Applies to on treatment subjects only- Male subjects must:
a. Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy.
* True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [eg, calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable methods of contraception).

1. El sujeto tiene ≥ 18 años en el momento de firmar el formulario de consentimiento informado (ICF).
2. El sujeto está dispuesto y es capaz de cumplir con el programa de visitas del estudio y otros requisitos del protocolo.
3. El sujeto ha estado participando en un ensayo de luspatercept y continúa cumpliendo con todos los requisitos del protocolo principal y el sujeto ha sido:
a. Asignado al tratamiento con luspatercept, continúa recibiendo beneficios clínicos según la opinión del investigador y debe continuar recibiendo el tratamiento con luspatercept, O
b. Asignado al brazo de placebo en el protocolo de los padres (en el momento de cegamiento o en el seguimiento) y debe pasar al tratamiento con luspatercept, O
c. Asignado a la Fase de seguimiento del protocolo principal, previamente tratado con luspatercept o placebo en el protocolo principal, que continuará en la Fase de seguimiento posterior al tratamiento a largo plazo en el estudio de seguimiento hasta que se cumplan los criterios de seguimiento (a menos que Los requisitos se cumplen según el protocolo principal para pasar al tratamiento con luspatercept).
4. El sujeto entiende y firma voluntariamente un documento de consentimiento informado antes de realizar cualquier evaluación o procedimiento relacionado con el estudio.
5. El sujeto demuestra cumplimiento, según lo evaluado por el investigador, con los requisitos del protocolo del estudio de los padres.
6. Aplica solo a sujetos en tratamiento: mujeres en edad fértil (FCBP) definidas como mujeres sexualmente maduras que:
1) ha alcanzado la menarquia en algún momento,
2) no se haya sometido a una histerectomía u ooforectomía bilateral, o
3) no ha sido naturalmente posmenopáusica (la amenorrea después de la terapia contra el cáncer no descarta el potencial de la maternidad) durante al menos 24 meses consecutivos (es decir, ha tenido la menstruación en cualquier momento en los 24 meses consecutivos anteriores) y debe:
a. Tener dos pruebas de embarazo negativas según lo verificado por el investigador antes de comenzar la terapia del estudio. Ella debe aceptar las pruebas de embarazo en curso durante el curso del estudio y después del tratamiento de fin de estudio. Esto se aplica incluso si el sujeto practica la verdadera abstinencia * del contacto heterosexual.
b. Comprométase a una verdadera abstinencia * del contacto heterosexual (que debe revisarse mensualmente y la fuente debe estar documentada) o acuerde usar, y podrá cumplir con anticonceptivos altamente efectivos, sin interrupción, 35 días antes de comenzar el producto de investigación (PI) , durante el tratamiento del estudio (incluidas las interrupciones de la dosis), y durante 84 días después de la interrupción de la terapia del estudio.
7. Aplica solo a sujetos en tratamiento- Los sujetos masculinos deben:
a. Practicar la abstinencia verdadera * (que debe revisarse mensualmente) o acepte usar un condón durante el contacto sexual con una mujer embarazada o con potencial de maternidad durante su participación en el estudio, durante las interrupciones de la dosis y por lo menos 84 días después de la investigación Descontinuación del producto incluso si se ha sometido a una vasectomía exitosa.
* La abstinencia verdadera es aceptable cuando está en línea con el estilo de vida preferido y habitual del sujeto. (La abstinencia periódica [por ejemplo, calendario, ovulación, métodos sintotérmicos, post-ovulación] y el retiro no son métodos anticonceptivos aceptables).

E.4

Principal exclusion criteria

The presence of any of the following will exclude a subject from enrollment:
1. Applies to on treatment subjects only- Concomitant use of any medications/procedures that are prohibited in the parent luspatercept protocol.
2. Subject has met one or more criteria for study discontinuation as stipulated in the parent luspatercept protocol.
3. First luspatercept transition visit into rollover study > 21 days after end of study (EOS) visit (last dose/visit in case of no EOS visit) of the parent luspatercept study with the exception of those subjects already in the Post-treatment Follow up Phase from the parent study. Note-Subject with current dose delays from the parent protocol during the Transition Phase, will continue in the rollover protocol regardless of the delay.
4. Applies to on treatment subjects only- Pregnant or breastfeeding females.
5. Subject has any significant medical condition, laboratory abnormality, psychiatric illness, or is considered vulnerable by local regulations (eg, imprisoned or institutionalized) that would prevent the subject from participating in the study.
6. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
7. Subject has any condition that confounds the ability to interpret data from the study.

La presencia de cualquiera de los siguientes excluirá al paciente del reclutamiento:
1. Aplica solo a sujetos de tratamiento: el uso concomitante de cualquier medicamento / procedimiento que esté prohibido en el protocolo original de luspatercept.
2. El sujeto ha cumplido con uno o más criterios para la interrupción del estudio según lo estipulado en el protocolo original de luspatercept.
3. Primera visita de transición de luspatercept al estudio de reinversión> 21 días después de la visita al final del estudio (EOS) (última dosis / visita en caso de que no haya una visita de EOS) del protocolo original de luspatercept, con la excepción de los sujetos que ya se encuentran en el postratamiento. Fase ascendente del estudio original. Nota: el sujeto con retrasos de la dosis actual del protocolo original durante la fase de transición, continuará en el protocolo de reinversión independientemente de la demora.
4. Aplica solo a sujetos en tratamiento-mujeres embarazadas o lactantes.
5. El sujeto tiene una condición médica importante, anomalías de laboratorio, enfermedades psiquiátricas o se considera vulnerable según las regulaciones locales (por ejemplo, encarcelados o institucionalizados) que impiden que el sujeto participe en el estudio.
6. El sujeto tiene alguna afección, incluida la presencia de anomalías de laboratorio, lo que coloca al sujeto en un riesgo inaceptable si él / ella participara en el estudio.
7. El sujeto tiene cualquier condición que confunda la capacidad de interpretar los datos del estudio.

E.5 End points

E.5.1

Primary end point(s)

1) Adverse events (AEs) - Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept.
2) Progression to high/very high risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)
(MDS and myelofibrosis [MF] only) - Number and percentage of subjects progressing to high/very high risk MDS or AML.
3) Development of other malignancies/pre-malignancies - Number and percentage of subjects developing other malignancies/premalignancies.

1) Acontecimientos Adversos (AA): tipo, frecuencia, gravedad de AA, relación de los acontecimientos adversos emergentes del tratamiento con luspatercept.
2) Progresión hacia un síndrome mielodisplásico (SMD) de riesgo alto / muy alto o leucemia mieloide aguda (LMA)
(Solo MDS y mielofibrosis [MF]) - Número y porcentaje de sujetos que avanzan a un SMD o LMA de riesgo alto / muy alto.
3) Desarrollo de otras neoplasias malignas / pre-malignas: número y porcentaje de sujetos que desarrollan otras neoplasias malignas / premalignidades.

E.5.1.1

Timepoint(s) of evaluation of this end point

1) Adverse events (AEs) - Timeframe: Enrollment to 42 days post last dose
2) Progression to high/very high risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)
(MDS and myelofibrosis [MF] only) - Timeframe: Enrollment to Long-term Post-treatment Follow-up
3) Development of other malignancies/pre-malignancies - Timeframe: Enrollment to Long-term Post-treatment Follow-up

1) Acontecimientos Adversos (AA): Plazo: reclutamiento a 42 días después de la última dosis
2) Progresión hacia un síndrome mielodisplásico (SMD) de riesgo alto / muy alto o leucemia mieloide aguda (LMA)
(Solo MDS y mielofibrosis [MF]) -Plazo: reclutamiento de seguimiento a largo plazo después del tratamiento.
3) Desarrollo de otras neoplasias malignas / pre-malignas- Plazo: reclutamiento de seguimiento a largo plazo después del tratamiento

E.5.2

Secondary end point(s)

Overall survival - Time from date of randomization until death from any cause

Supervivencia general: tiempo desde la fecha de aleatorizacion hasta la muerte por cualquier causa

E.5.2.1

Timepoint(s) of evaluation of this end point

Enrollment to Long-term Post-treatment Follow-up

Reclutamiento de seguimiento a largo plazo después del tratamiento

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

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

151

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

Belgium

Bulgaria

Canada

France

Germany

Greece

Israel

Italy

Lebanon

Malaysia

Netherlands

Spain

Sweden

Taiwan

Thailand

Tunisia

Turkey

United Kingdom

United States

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

Rollover study will be terminated, and subjects will discontinue from study when all subjects fulfill 5years from Dose 1 of parent protocol or 3years of post-treatment from last dose of parent protocol, whichever occurs later. End of Trial is defined as either the date of last visit of last subject to complete the post-treat FU, or date of receipt of last data point from last subject needed for primary, secondary and/or exploratory analysis, as per protocol, whichever is the later date.

El estudio finalizará y los sujetos se retirarán del estudio cdo todos cumplan 5 años desde dosis 1 del prot original o 3 años dp tto desde la últ. dosis del prot orig, lo que ocurra + tarde. Final del ensayo se define como fecha de UVUS que complete el seguimiento posterior al tto, o fecha de recepción de los últimos datos correspondientes al último suj. que sean necesarios para los análisis ppales, secundarios o exploratorios, tal como se especifique en el prot, lo que ocurra + tarde.

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:

223

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

519

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

492

F.4.2.2

In the whole clinical trial

742

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)

Subjects who received at least one dose of IP should undergo EOT and 42d Follow-up evaluations after last dose of IP or EOT Visit, whichever occurs later. Subjects will be followed every 6mths thereafter for monitoring&overall survival from date of last dose of IP until death, withdrawal of consent for further follow-up, or until study termination, or until subject is lost to follow-up, or until parent luspatercept protocol requirements are met. Subjects will receive SoC treatment post study.

Pacientes que recibieron al menos 1 dosis de IP deben someterse a evaluaciones de seguimiento EOT y 42d dp de la última dosis de IP o EOT Visit, lo que ocurra + tarde. Los sujetos serán seguidos c 6 m a partir de entonces para seguimiento y supervivencia gral desde la fecha de última dosis de PI hasta la muerte, retirada de CI , hasta finalización del estudio, hasta que se pierda dte el seguimiento, o hasta que el prot original cumpla con los requisitos. Los sujetos recibirán tto post al SoC.

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

2019-04-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-04-15

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

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IMP with orphan designation in the indication
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