A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
Study Purpose
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following subjects:
- - Subjects receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit in the opinion of the investigator from continuing treatment with luspatercept.
- - Placebo arm subjects from parent protocol (at the time of unblinding or in follow-up) crossing over to luspatercept treatment (provided subjects have met all requirements for entering the rollover study as per the parent protocol).
- - Subjects in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met (unless they meet requirements as per parent protocol to cross-over to luspatercept treatment).
- - Transition Phase (Screening): up to 21 days prior to enrollment.
- - Treatment Phase: For subjects in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study.
- - Follow-up Phase: - 42 Day Safety Follow-up Phase: subjects will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting.
- - Long-term Post-treatment Follow-up (LTPTFU) Phase: All subjects who are continuing in the LTPTFU Phase, will continue to be followed for 5 years from Dose 1 of the parent protocol, or 3 years of post-treatment from last dose of the parent protocol, whichever occurs later.
Recruitment Criteria
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms |
No |
Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies. |
Interventional |
Eligible Ages | 18 Years and Over |
Gender | All |
Inclusion Criteria:
Subjects must meet all the following criteria to be enrolled in this study: 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: 1. Assigned to luspatercept treatment, continues to receive clinical benefit in the opinion of the investigator and should continue to receive luspatercept treatment, OR. 2. 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. 3. 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: 1. 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. 2. 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.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.Trial Details
Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries. |
NCT04064060 |
Phase
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use. |
Phase 3 |
Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data. |
Celgene |
Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study. |
Rodrigo Ito, M.D. |
Principal Investigator Affiliation | Celgene Corporation |
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial. |
Industry |
Overall Status | Recruiting |
Countries | Australia, Belgium, Bulgaria, Canada, France, Germany, Greece, Israel, Italy, Lebanon, Malaysia, Netherlands, Spain, Sweden, Taiwan, Thailand, Tunisia, Turkey, United Kingdom, United States |
Conditions
The disease, disorder, syndrome, illness, or injury that is being studied. |
Myelodysplastic Syndromes (MDS), Beta-thalassemia, Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis |
Arms
Experimental: ACE-536
Luspatercept will be administered as a subcutaneous (SC) injection to subjects by the study staff at the clinical site and administration will be documented in the subject's source record.
Interventions
Drug: - Luspatercept
Luspatercept (ACE-536), an erythroid maturation agent, is a recombinant fusion protein consisting of a modified form of the extracellular domain (ECD) of the human activin receptor type IIB (ActRIIB) linked to the human immunoglobin G 1 (IgG1) Fc domain. ActRIIB receptor and its ligands are members of the transforming growth factor-β (TGF-β) superfamily. Members of the TGF-β superfamily ligands, through their binding to activin receptors, are involved in modulating the differentiation of late-stage erythrocyte precursors (normoblasts) in the bone marrow. Luspatercept for injection is formulated as a sterile, preservative-free, lyophilized cake/powder. Luspatercept for injection is available in 25 mg and 75 mg vials and when reconstituted with water for injection, each consists of 50 mg/mL luspatercept in a 10 mM citrate buffer-based solution
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Status
Recruiting
Address
Childrens Hospital Los Angeles RHU
Los Angeles, California, 90027-6062
Status
Recruiting
Address
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Status
Recruiting
Address
Stanford Cancer Center
Stanford, California, 94305
Status
Recruiting
Address
Moffitt Cancer Center
Tampa, Florida, 33612
Status
Recruiting
Address
Moffitt Cancer Center
Tampa, Florida, 33612
Status
Recruiting
Address
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Status
Recruiting
Address
Boston Children's Hospital
Boston, Massachusetts, 02115
Status
Recruiting
Address
Karmanos Cancer Institute
Detroit, Michigan, 48201
Status
Recruiting
Address
Weill Cornell Medical College
New York, New York, 10065
Status
Recruiting
Address
Cleveland Clinic
Cleveland, Ohio, 44195
Status
Recruiting
Address
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Status
Not yet recruiting
Address
Vanderbilt - Ingram Cancer Center
Nashville, Tennessee, 37232-5505
Status
Not yet recruiting
Address
The University of Texas - MD Anderson Cancer Center
Houston, Texas, 77030
International Sites
Status
Recruiting
Address
Mater Hospital Brisbane
South Brisbane, Queensland, 4101
Status
Recruiting
Address
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Status
Recruiting
Address
Monash Medical Centre
Clayton, Victoria, 3168
Status
Recruiting
Address
Royal Prince Alfred Hospital
Camperdown, , 2050
Status
Not yet recruiting
Address
Prince of Wales Hospital
Randwick, , 2031
Status
Recruiting
Address
Algemeen Ziekenhuis Klina
Brasschaat, , 2930
Status
Recruiting
Address
AZ Sint-Jan AV Brugge
Brugge, , 8000
Status
Recruiting
Address
Universitair Ziekenhuis Gent
Ghent, , 9000
Status
Recruiting
Address
UZ Leuven
Leuven, , 3000
Status
Recruiting
Address
University Multiprofile Hospital for Active Treatment Sveti Georgi EAD
Plovdiv, , 4002
Status
Recruiting
Address
Specialized Hospital for Active Treatment of Haematological Diseases - Sofia
Sofia, , 1756
Status
Not yet recruiting
Address
Multiprofile Hospital for Active Treatment Sveta Marina EAD
Varna, , 9010
Status
Recruiting
Address
Local Institution - 262
Toronto, Ontario, M4N 3M5
Status
Recruiting
Address
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
Status
Recruiting
Address
University Health Network
Toronto, Ontario, M5G 2C4
Status
Recruiting
Address
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 2M9
Status
Recruiting
Address
CHU d'Angers
Angers, , 49033
Status
Recruiting
Address
Hopital Henri Mondor
Creteil, , 94010
Status
Recruiting
Address
Centre Hospitalier Universitaire de Grenoble Hopital Albert Michallon
La Tronche, , 38700
Status
Recruiting
Address
CHRU de Lille-Hopital Claude Huriez
Lille, , 59037
Status
Recruiting
Address
Local Institution - 306
Lille, , 59037
Status
Recruiting
Address
Hopitaux de La Timone
Marseille Cedex 9, , 13385
Status
Recruiting
Address
Hopital Saint Louis
Paris, , 75010
Status
Not yet recruiting
Address
Hopital Haut Leveque
Pessac Cedex, , 33604
Status
Recruiting
Address
Centre Hospitalier Lyon Sud
Pierre Benite cedex, , 69495
Status
Recruiting
Address
Hopital civil
Strasbourg, , 67091
Status
Recruiting
Address
Institut Universitaire du Cancer de Toulouse - Oncopole
Toulouse Cedex 9, , 31059
Status
Not yet recruiting
Address
Hopital Bretonneau
Tours, , 37044
Status
Recruiting
Address
Medizinisches Versorgungszentrum (MVZ) Onkologischer Schwerpunkt am Oskar-Helene-Heim
Berlin, , 14195
Status
Recruiting
Address
Local Institution - 348
Dresden, , 01307
Status
Recruiting
Address
Universitatsklinikum Carl Gustav Carus an der TU Dresden
Dresden, , 01307
Status
Recruiting
Address
Universitaetsklinikum Duesseldorf
Duesseldorf, , 40225
Status
Recruiting
Address
Marien Hospital
Dusseldorf, , 40479
Status
Recruiting
Address
Universitatsklinikum Halle Saale
Halle, , 06120
Status
Recruiting
Address
Local Institution - 342
Hamburg, , 22081
Status
Recruiting
Address
OncoResearch Lerchenfeld GmbH
Hamburg, , 22081
Status
Recruiting
Address
Medizinische Hochschule Hannover
Hannover, , 30625
Status
Not yet recruiting
Address
Universitatsklinikum Leipzig
Leipzig, , 04103
Status
Recruiting
Address
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz
Mainz, , 55131
Status
Recruiting
Address
Klinikum rechts der Isar der Technischen Universitaet Muenchen
München, , 81675
Status
Recruiting
Address
Aghia Sophia' Children's General Hospital of Athens
Athens, , 115 27
Status
Recruiting
Address
Laiko General Hospital of Athens - Center of Thalassemia
Athens, , 115 27
Status
Recruiting
Address
General Hospital Georgios Gennimatas of Athens
Athens, , 11527
Status
Recruiting
Address
University General Hospital of Patras
Rio Patras, , 26500
Status
Recruiting
Address
General Hospital of Thessaloniki Hippokration
Thessaloniki, , 54642
Status
Recruiting
Address
HaEmek Medical Center
Afula, , 18341
Status
Recruiting
Address
Rambam Health Corporation
Haifa, , 3109601
Status
Recruiting
Address
Shaare Zedek Medical Center
Jerusalem, , 91031
Status
Recruiting
Address
Hadassah Medical Center
Jerusalem, , 91120
Status
Recruiting
Address
Galilee Medical Center
Nahariya, , 22100
Status
Recruiting
Address
Rabin Medical Center
Petah Tikva, , 49100
Status
Recruiting
Address
Azienda Ospedaliera Santi Antonio Biagio E Cesare Arrigo
Allessandria, , 15100
Status
Recruiting
Address
Azienda Ospedaliero Universitaria Di Bologna - Policlinico S.Orsola Malpighi
Bologna, , 40138
Status
Recruiting
Address
Presidio Ospedaliero Antonio Perrino
Brindisi, , 72100
Status
Recruiting
Address
Azienda Sanitaria Locale (ASL) Cagliari - Ospedale Regionale per le Microcitemie
Cagliari, , 09121
Status
Recruiting
Address
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant'Anna
Ferrara, , 44124
Status
Recruiting
Address
Azienda Ospedaliera Universitaria Careggi
Firenze, , 50134
Status
Not yet recruiting
Address
Azienda Ospedaliera Universitaria Careggi
Firenze, , 50134
Status
Recruiting
Address
Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
Genoa, , 16128
Status
Recruiting
Address
Presidio Ospedaliero Vito Fazzi
Lecce, , 73100
Status
Recruiting
Address
Maggiore Polyclinic Hospital, IRCCS Ca' Granda
Milano, , 20122
Status
Recruiting
Address
Azienda Ospedaliero Universitaria Di Modena Policlinico
Modena, , 41100
Status
Recruiting
Address
AORN A Cardarelli
Napoli, , 80131
Status
Recruiting
Address
AOU dell'Università degli Studi della Campania Luigi Vanvitelli
Napoli, , 80131
Status
Recruiting
Address
Azienda Ospedaliero Universitaria S. Luigi Gonzaga
Orbassano, , 10043
Status
Recruiting
Address
IRCCS Policlinico San Matteo
Pavia, , 27100
Status
Recruiting
Address
Azienda Ospedaliera Bianchi Melacrino Morelli
Reggio Di Calabria, , 89124
Status
Recruiting
Address
Local Institution - 468
Reggio Di Calabria, , 89124
Status
Recruiting
Address
Fondazione PTV Policlinico Tor Vergata
Roma, , 133
Status
Not yet recruiting
Address
Istituto Clinico Humanitas
Rozzano, , 20089
Status
Not yet recruiting
Address
Ospedale di Circolo di Varese
Varese, , 21100
Status
Recruiting
Address
Azienda Ospedaliera Universitaria Integrata Di Verona
Verona, , 37134
Status
Recruiting
Address
Chronic Care Center
Hazmieh, , 1003
Status
Recruiting
Address
Hospital Sultanah Aminah
Johor Bahru, Johor, 80100
Status
Recruiting
Address
Hospital Sultanah Bahiyah
Alor Setar, Kedah, 05460
Status
Recruiting
Address
Hospital Raja Permaisuri Bainun
Ipoh, Perak, 30990
Status
Recruiting
Address
Queen Elizabeth Hospital
Kota Kinabalu, Sabah, 88586
Status
Recruiting
Address
Hospital Umum Sarawak
Kuching, Sarawak, 93586
Status
Recruiting
Address
University Malaya Medical Centre
Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, 59100
Status
Recruiting
Address
Vrije Universiteit Medisch Centrum (VUMC)
Amsterdam, , 1081 HV
Status
Recruiting
Address
Hospital Universitario Cruces
Barakaldo, , 48903
Status
Recruiting
Address
Hospital Universitari Vall d'Hebron
Barcelona, , 08035
Status
Recruiting
Address
Instituto Catalan de Oncologia-Hospital Duran i Reynals
Barcelona, , 08908
Status
Recruiting
Address
Hospital General Universitario Gregorio Marañon
Madrid, , 28007
Status
Recruiting
Address
Hospital Universitario Central de Asturias
Oviedo, , 33011
Status
Recruiting
Address
Universitario de Salamanca - Hospital Clinico
Salamanca, , 37007
Status
Recruiting
Address
Hospital Universitario Virgen del Rocio
Seville, , 41013
Status
Recruiting
Address
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, , 46026
Status
Recruiting
Address
Sahlgrenska Universitetssjukhuset
Goteborg, , 413 45
Status
Recruiting
Address
Skanes Universitetssjukhus Lund
Lund, , SE-221 85
Status
Recruiting
Address
Karolinska Universitetssjukhuset - Huddinge
Stockholm, , 141 86
Status
Recruiting
Address
Kaohsiung Medical University Hospital
Kaohsiung, San Ming Dist., , 807
Status
Recruiting
Address
China Medical University Hospital
Taichung, , 40447
Status
Recruiting
Address
National Taiwan University Hospital
Taipei, Zhongzheng Dist., , 10002
Status
Recruiting
Address
Chulalongkorn University Faculty of Medicine - King Chulalongkorn Memorial Hospital
Bangkok, , 10330
Status
Recruiting
Address
Siriraj Hospital Mahidol University
Bangkok, , 10700
Status
Recruiting
Address
Chiang Mai University - Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, , 50200
Status
Recruiting
Address
University Hospital Farhat Hached
Sousse, , 4031
Status
Recruiting
Address
Bone Marrow Transplant Center
Tunis, , 1006
Status
Recruiting
Address
Aziza Othmana Hospital
Tunis, , 1008
Status
Recruiting
Address
Military Hospital of Tunis
Tunis, , 1008
Status
Recruiting
Address
Acibadem Adana Hospital
Adana, , 01130
Status
Recruiting
Address
Ankara University Medical Faculty Cebeci Hospital
Ankara, , 06590
Status
Recruiting
Address
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, , 34093
Status
Recruiting
Address
Istanbul University Cerrahpasa Medical Faculty Hospital
Istanbul, , 34098
Status
Recruiting
Address
Ege Universitesi Tip Fakultesi Hastanesi
Izmir, , 35100
Status
Recruiting
Address
Mersin University Medical Faculty
Mersin, , 33343
Status
Recruiting
Address
Aberdeen Royal Infirmary
Aberdeen, , AB25 2ZN
Status
Not yet recruiting
Address
The Leeds Teaching Hospitals NHS Trust - St James's University Hospital
Leeds, , LS9 7TF
Status
Recruiting
Address
Barts Health NHS Trust - The Royal London Hospital
London, , E1 1BB
Status
Recruiting
Address
Whittington Hospital
London, , N19 5NF
Status
Recruiting
Address
University College London Hospitals NHS Foundation Trust - University College Hospital
London, , NW1 2BU
Status
Recruiting
Address
Kings College Hospital
London, , SE5 9RS
Status
Not yet recruiting
Address
Oxford University Hospitals NHS Foundation Trust-Churchill Hospital-Cancer and Haematology Centre
Oxford, , OX3 7LE
Status
Not yet recruiting
Address
Kings Mill Hospital
Sutton in Ashfield, , NG17 4JL