Beta Thalassemia MajorPhase 2NCT01571635
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
A Phase 2 study testing SOTATERCEPT (ACE-011) in people with Beta Thalassemia Major and Beta Thalassemia Intermedia, run by Celgene at 18 sites in 4 countries.
Trial summary
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta Thalassemia Major, Beta Thalassemia Intermedia
- What is being tested?
- SOTATERCEPT (ACE-011)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 18 sitesin 4 countries
- Study dates
- 10 Oct 2012to 24 May 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women 18 years of age at the time of signing the informed consent document with a diagnosis of β-thalassemia major (including all subtypes) or β-thalassemia intermedia.
- For transfusion dependent subjects: permanent transfusion dependency is defined as requiring packed red blood cells (pRBCs) and iron chelation therapy:
- Average transfusion requirement of at least 2 units/30 days of pRBCs (Gale, 2011) confirmed for a minimum of 168 days (six months) immediately preceding enrollment (study Day 1, first Dose);
- No transfusion-free period of more than 45 consecutive days during the 168 days immediately preceding enrollment (study Day 1, first Dose);
- Prior transfusion hemoglobin levels ≤ 10.5 g/dL.
- For non-transfusion dependent subjects: non-transfusion dependency is defined as a transfusion free for a minimum of 168 days immediately preceding enrollment (study Day 1, first Dose), with the exception of ≤ to one episode of transfusion in the period of a minimum of 168 days immediately preceding enrollment (study Day 1, first Dose) (One episode of transfusion is defined as ≤ 4 transfusion units administered, occurred within 42 days [first transfusion is counted as day 1] due to concurrent illness [e.g. infection], [Guidelines Clin Management of Thalassaemia, 2008]). (This inclusion criteria is not valid for France).
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- Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 to 1
- No concurrent severe hepatic disease:
- Aspartate Aminotransferase (AST) or Alanine Transaminase (ALT) no greater than 3 x upper limit of normal (ULN);
- Albumin ≥ 3 g/dL.
- Serum creatinine ≤ 1.5 x ULN.
- Females of childbearing potential participating in the study are to use highly effective methods of birth control during study participation and for 112 days (approximately five times the mean terminal half-life of sotatercept [23 days] based on multiple-dose PK data) following the last dose of sotatercept. FCBP must have a negative serum beta Human Chorionic Gonadotropin (β-HCG) pregnancy test within three days of Sotatercept dosing (Day 1). Subjects must be counseled concerning measures to be used to prevent pregnancy and potential toxicities prior to the first dose of sotatercept. A FCBP is a sexually mature woman who has not undergone a hysterectomy or bilateral oophorectomy or who has not been postmenopausal for at least 24 consecutive months (i.e., who has had menses at some time in the preceding 24 months).
- Males must agree to use a latex condom during any sexual contact with FCBSs while participating in the study and for 112 days following the last dose of Sotatercept, even if he has undergone a successful vasectomy. Subjects must be counseled concerning measures to be used to prevent pregnancy and potential toxicities prior to the first dose of sotatercept.
- Agreement to adhere to the study visit schedule, understand and comply with all protocol requirements.
- Understand and provide written informed consent.
Who cannot join
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing participating in the study.
- Evidence of active Hepatitis C antibody (HCV), Hepatitis B surface antigen (HBsAg and HB core Ab), or Human Immunodeficiency Virus (HIV) antibody.
- Known history of thromboembolic events ≥ Grade 3 according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 (current active minor version).
- Subjects with insulin dependent diabetes.
- Subjects with major cardiac problems such as:
- Major risk of heart failure, confirmed with myocardiac T2* ≤ 10 ms. Myocardiac T2* performed in the last one and a half years prior to subject enrollment (study Day 1, first Dose) will be considered valid.
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- Cardiac arrhythmia which requires treatment (i.e. atrial fibrillation).
- Treatment with another investigational drug or device < 28 days prior to study entry.
- Use of an Erythropoiesis Stimulating Agent (ESA) within the 28 days prior to enrollment (study Day 1, first Dose).
- Subjects on hydroxyurea treatment for which the dose was changed in the last one year prior to subject enrollment (study Day 1, first Dose).
- Subjects on anticoagulant therapy, such as warfarin.
- Subjects who started bisphosphonates within the last three months prior to subject enrollment (study Day 1, first Dose).
- Pregnant or lactating females.
- Uncontrolled hypertension. Controlled hypertension for this protocol is considered ≤ Grade 1 according to NCI CTCAE version 4.0 (current active minor version) (Appendix B).
- A history of major organ damage including:
- Liver disease with ALT > 3x ULN or histopathological evidence of liver cirrhosis on liver biopsy;
- Heart disease with ejection fraction ≥ Grade 2 according to NCI CTCAE version 4.0 (current active minor version);
- Kidney disease with a calculated creatinine clearance < 40 mL/min (Cockcroft-Gault formula);
- Pulmonary fibrosis or pulmonary hypertension as confirmed by a specialist.
- Adrenal insufficiency.
- Heart failure as classified by the New York Heart Association (NYHA) classification of 3 or higher (Appendix C).
- Major surgery within 30 days prior to study Day 1 (subjects must have completely recovered from any previous surgery prior to study Day 1).
- History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the Investigational Product (see Investigator Brochure).
Trial locations and contacts
18 sites in 4 countries.
18 sites in 4 countries
Italy8 sites
Universita degli Studi di Cagliari - ASL8
Cagliari, 09121
Universita Degli Studi Di Cagliari
Cagliari, 09121
Local Institution - 200
Genoa, 16128
Ospedale Galliera
Genoa, 16128
Ente Ospedaliero Ospedali Galliera
Genova, 16128
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122
Fondazione IRCCS Ospedale Maggiore
Milan, 20122
Local Institution - 201
Milan, 20122
France5 sites
Hopital Henri Mondor
Créteil, 94010
Groupe Hospitalier Henri Mondor
Créteil
Hospital of Necker
Paris, 75015
Local Institution - 001
Paris, 75015
Hôpital Necker-Enfants Malades
Paris
United Kingdom3 sites
Local Institution - 100
London, WC1E 6BT
UCL Cancer Institute
London, WC1E 6BT
UCL Cancer Institue
London, WC1E6BT
Greece2 sites
Local Institution - 300
Ampelokipi - Athens, 115 26
Laiko General Hospital
Ampelokipi - Athens, 11526
About this study
Official title: A Phase 2A, Open-label Dose Finding Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With BETA(b)-THALASSEMIA.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Being tested
- SOTATERCEPT (ACE-011)
- Keywords
- Beta-Thalassaemia
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SOTATERCEPT (ACE-011)Drug
Also called (ACE-011)
0.1 mg/kg to 1.5mg/kg Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period.
| Study group | Role | What they receive |
|---|---|---|
| Sotatercept dose level 0.1mg/kg | Experimental | Experimental 0.1 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period Receives: Drug: SOTATERCEPT (ACE-011) |
| Sotatercept dose level 0.3mg/ kg | Experimental | Experimental 0.3 mg/kg - Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period Receives: Drug: SOTATERCEPT (ACE-011) |
| Sotatercept dose level 0.5mg/kg | Experimental | Experimental 0.5 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period Receives: Drug: SOTATERCEPT (ACE-011) |
| Sotatercept dose level 0.75mg/kg | Experimental | Experimental 0.75 mg/kg - Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period Receives: Drug: SOTATERCEPT (ACE-011) |
| Sotatercept dose level 1.0mg/kg | Experimental | Experimental 1.0 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period Receives: Drug: SOTATERCEPT (ACE-011) |
| Sotatercept dose level 1.5mg/kg | Experimental | Experimental 1.5 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period Receives: Drug: SOTATERCEPT (ACE-011) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose
Number of participants with dose-limiting toxicities (DLT) are used to determine the potential recommended dose (PRD). PRD is defined as the highest dose with up to 1 out of 6 patients experiencing a DLT. DLT is defined as any side effects of the study treatment serious enough to prevent an increase in dose or level of treatment, including at least one of the following: Hypertension ≥ Grade 3 per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0; Hgb > 14 g/dL sustained for four weeks; any NCI CTCAE toxicity ≥ Grade 3. Grade 3 is defined as severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily life.
PRD was identified as 1 mg/kg. Due to study termination, no patients were enrolled after 1 mg/kg cohort or in the Expansion Cohort. Thus, primary analyses to determine recommended dose (RD) were not conducted.
Measured: From first dose up to 28 days post the first dose
Secondary outcomes (6)
Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment
Transfusion burden at baseline is defined as the total number of units of RBC transfusions that participants received within 168 days (24 weeks) prior to the first dose of study therapy. Transfusion burden during treatment is defined as the total number of RBC transfusion units that each participant received during the treatment divided by the treatment duration and multiplied by 168 days. The result is a 168-day transfusion burden average. Baseline measurement includes RBC transfusion history for transfusion dependent and non-transfusion dependent participants, starting at 168 days prior to enrollment.
Measured: From baseline to the last dose of study treatment (up to approximately 112 months)
Number of Participants With Hemoglobin Level Increase From Baseline in Non-Transfusion Dependent B-Thalassemia Intermedia Participants
The Number of participants with a change in Hemoglobin levels will be listed for non-RBC transfusion dependent participants. Baseline assessments are the average of the last two measurements prior to the start of therapy.
Measured: Measurements were taken in 9 and 12-week intervals, from baseline up to approximately 112 months
Number of Participants Experiencing Adverse Events (AEs)
An Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a clinical investigation participant administered study treatment and that does not necessarily have a causal relationship with this treatment. Adverse events are graded on a scale from 1 to 5, with Grade 1 being mild and asymptomatic; Grade 2 is moderate requiring minimal, local or noninvasive intervention; Grade 3 is severe or medically significant but not immediately life-threatening; Grade 4 events are usually severe enough to require hospitalization; Grade 5 events are fatal. Treatment emergent adverse events (TEAE) are defined as an AE that began after the start of trial medication treatment; or if the event was continuous from baseline and was serious, trial medication-related, or resulted in death, discontinuation, or interruption or reduction of trial therapy.
Measured: From first dose up to 112 days after the last dose of study treatment (up to 115 months)
Concentrations of Sotatercept in Serum
Sotatercept was administered as a subcutaneous injection every 21 days during the Treatment Period. Pharmacokinetic (PK) samples were collected at the pre-specified timepoints.
Measured: Dose 1, Day 8; Dose 1, Day 15; Dose 2, Day 1; Dose 2, Day 8; Dose 3, Day 1; Dose 3, Day 8; Dose 4, Day 1; Dose 5, Day 1; Dose 6, Day 1
Number of Participants With Anti-Drug Antibody (ADA)
The number of participants with Anti-Sotatercept Antibody is a summary of antidrug antibody (ADA) status for ADA-evaluated participants. A participant is counted as 'positive' if there is any positive result captured during the study, a participant is counted as 'negative' if there is no positive result captured during the study. ADA data was collected Day 1 in dose schedules 1 through 6. Starting from Dose 7, ADA was measured at Day 1 every 3 Doses, then finally at the post-treatment follow-up visit at Month 2 and Month 4.
Measured: From first dose up to 4 months after last dose (up to approximately 116 months)
Number of Participants Experiencing Quality of Life (QOL) Change From Baseline
The number of participants in the expansion cohort experiencing changes from baseline in Quality of Life. QOL was planned to be assessed at Day 168 (6 months) and Day 336 (12 months), after Dose 1 Day 1, independent of Dose Delay for participants enrolled in the Expansion Cohort only. QOL was planned to be calculated using the SF-36 and the FACT Anemia. The SF-36 is a Medical Outcomes Study (MOS) consisting of 36 questions developed to determine health status. The SF-36 measures eight scales: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. The FACT Anemia measures fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) Measurement System.
Due to early study termination, no participants were enrolled in the expansion cohort and QOL was not assessed.
Measured: From pre-dose up to Dose 8 (168 days/6months) and Dose 16 (336 days/12months) only
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2012 (estimated)
- Study start10 Oct 2012
- Primary completion2 Jul 2015
- Study completion24 May 2022
Why it stopped: Business objectives have changed
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Beta Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by CelgeneThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01571635
- Sponsor's protocol ID
- ACE-011-B-THAL-001
- Other IDs
- 2011-005659-15
- Status last verified
- May 2023
- First submitted
- 3 Apr 2012
- Last updated
- 18 Jun 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Cappellini MD, Porter J, Origa R, Forni GL, Voskaridou E, Galacteros F, Taher AT, Arlet JB, Ribeil JA, Garbowski M, Graziadei G, Brouzes C, Semeraro M, Laadem A, Miteva D, Zou J, Sung V, Zinger T, Attie KM, Hermine O. Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase II, open-label, dose-finding study. Haematologica. 2019 Mar;104(3):477-484. doi: 10.3324/haematol.2018.198887. Epub 2018 Oct 18.
PubMed 30337358Cappellini M, et al. A Phase 2a, Open-Label, Dose-Finding Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta ( )-Thalassemia: Interim Results. Presented at the 55th Annual Meeting of the American Society of Hematology (ASH), December 7-10, 2013, New Orleans, LA. Abstract No. 3448
"Porter J, et al. Interim Results From a Phase 2A, Open-Label, Dose-Finding Study To Determine The Safety, Efficacy, And Tolerability of Sotatercept (ACE-001) In Adults with Beta-Thalassemia. Presented at the 19thCongress of the European Hematology Association, June 12-15, 2015, Milan, Italy. Abstract No. S622 "
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2A, Open-label Dose Finding Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With BETA(b)-THALASSEMIA.
- ClinicalTrials.gov ID
- NCT01571635
- Acronym / sponsor ID
- ACE-011-B-THAL-001
- Keywords
- Beta-Thalassaemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business objectives have changed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 10 Oct 2012 (actual)
- Primary completion
- 2 Jul 2015 (actual)
- Study completion
- 24 May 2022 (actual)
- First posted
- 5 Apr 2012 (estimated)
- Results
- Posted 18 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Sotatercept dose level 0.1mg/kg | Experimental | Drug: SOTATERCEPT (ACE-011)Experimental 0.1 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period |
| Sotatercept dose level 0.3mg/ kg | Experimental | Drug: SOTATERCEPT (ACE-011)Experimental 0.3 mg/kg - Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period |
| Sotatercept dose level 0.5mg/kg | Experimental | Drug: SOTATERCEPT (ACE-011)Experimental 0.5 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period |
| Sotatercept dose level 0.75mg/kg | Experimental | Drug: SOTATERCEPT (ACE-011)Experimental 0.75 mg/kg - Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period |
| Sotatercept dose level 1.0mg/kg | Experimental | Drug: SOTATERCEPT (ACE-011)Experimental 1.0 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period |
| Sotatercept dose level 1.5mg/kg | Experimental | Drug: SOTATERCEPT (ACE-011)Experimental 1.5 mg/kg -Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Men and women 18 years of age at the time of signing the informed consent document with a diagnosis of β-thalassemia major (including all subtypes) or β-thalassemia intermedia.
- For transfusion dependent subjects: permanent transfusion dependency is defined as requiring packed red blood cells (pRBCs) and iron chelation therapy:
- For non-transfusion dependent subjects: non-transfusion dependency is defined as a transfusion free for a minimum of 168 days immediately preceding enrollment (study Day 1, first Dose), with the exception of ≤ to one episode of transfusion in the period of a minimum of 168 days immediately preceding enrollment (study Day 1, first Dose) (One episode of transfusion is defined as ≤ 4 transfusion units administered, occurred within 42 days [first transfusion is counted as day 1] due to concurrent illness [e.g. infection], [Guidelines Clin Management of Thalassaemia, 2008]). (This inclusion criteria is not valid for France).
- Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 to 1
- No concurrent severe hepatic disease:
Key exclusion criteria (23 listed)
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing participating in the study.
- Evidence of active Hepatitis C antibody (HCV), Hepatitis B surface antigen (HBsAg and HB core Ab), or Human Immunodeficiency Virus (HIV) antibody.
- Known history of thromboembolic events ≥ Grade 3 according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 (current active minor version).
- Subjects with insulin dependent diabetes.
- Subjects with major cardiac problems such as:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SOTATERCEPT (ACE-011)Also: (ACE-011)0.1 mg/kg to 1.5mg/kg Sotatercept will be administered as a subcutaneous injection once every 21 days during the treatment period. | Drug | Sotatercept dose level 0.1mg/kg, Sotatercept dose level 0.3mg/ kg, Sotatercept dose level 0.5mg/kg, Sotatercept dose level 0.75mg/kg, Sotatercept dose level 1.0mg/kg, Sotatercept dose level 1.5mg/kg |
Primary outcome
Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose
Time frame: From first dose up to 28 days post the first dose
Number of participants with dose-limiting toxicities (DLT) are used to determine the potential recommended dose (PRD). PRD is defined as the highest dose with up to 1 out of 6 patients experiencing a DLT. DLT is defined as any side effects of the study treatment serious enough to prevent an increase in dose or level of treatment, including at least one of the following: Hypertension ≥ Grade 3 per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0; Hgb > 14 g/dL sustained for four weeks; any NCI CTCAE toxicity ≥ Grade 3. Grade 3 is defined as severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily life. PRD was identified as 1 mg/kg. Due to study termination, no patients were enrolled after 1 mg/kg cohort or in the Expansion Cohort. Thus, primary analyses to determine recommended dose (RD) were not conducted.
6 secondary outcomes
Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment
Time frame: From baseline to the last dose of study treatment (up to approximately 112 months)
Number of Participants With Hemoglobin Level Increase From Baseline in Non-Transfusion Dependent B-Thalassemia Intermedia Participants
Time frame: Measurements were taken in 9 and 12-week intervals, from baseline up to approximately 112 months
Number of Participants Experiencing Adverse Events (AEs)
Time frame: From first dose up to 112 days after the last dose of study treatment (up to 115 months)
Concentrations of Sotatercept in Serum
Time frame: Dose 1, Day 8; Dose 1, Day 15; Dose 2, Day 1; Dose 2, Day 8; Dose 3, Day 1; Dose 3, Day 8; Dose 4, Day 1; Dose 5, Day 1; Dose 6, Day 1
Number of Participants With Anti-Drug Antibody (ADA)
Time frame: From first dose up to 4 months after last dose (up to approximately 116 months)
Number of Participants Experiencing Quality of Life (QOL) Change From Baseline
Time frame: From pre-dose up to Dose 8 (168 days/6months) and Dose 16 (336 days/12months) only
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 30337358Cappellini MD, Porter J, Origa R, Forni GL, Voskaridou E, Galacteros F, Taher AT, Arlet JB, Ribeil JA, Garbowski M, Graziadei G, Brouzes C, Semeraro M, Laadem A, Miteva D, Zou J, Sung V, Zinger T, Attie KM, Hermine O. Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase II, open-label, dose-finding study. Haematologica. 2019 Mar;104(3):477-484. doi: 10.3324/haematol.2018.198887. Epub 2018 Oct 18.
- BackgroundCappellini M, et al. A Phase 2a, Open-Label, Dose-Finding Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta ( )-Thalassemia: Interim Results. Presented at the 55th Annual Meeting of the American Society of Hematology (ASH), December 7-10, 2013, New Orleans, LA. Abstract No. 3448
- Background"Porter J, et al. Interim Results From a Phase 2A, Open-Label, Dose-Finding Study To Determine The Safety, Efficacy, And Tolerability of Sotatercept (ACE-001) In Adults with Beta-Thalassemia. Presented at the 19thCongress of the European Hematology Association, June 12-15, 2015, Milan, Italy. Abstract No. S622 "
- BMS Clinical Trial InformationLink provided by the sponsor.
- BMS Clinical Trial Patient RecruitingLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bristol-Myers Squibb study director
Bristol-Myers Squibb
18 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SOTATERCEPT (ACE-011) in people with Beta Thalassemia Major and Beta Thalassemia Intermedia, run by Celgene at 18 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Italy, France, United Kingdom, Greece. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose (time frame: From first dose up to 28 days post the first dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01571635, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2023.