Trial · NCT01571635
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
NCT01571635, Phase 2, Terminated, Celgene; results first posted 2023-06-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia. Trials, record NCT01571635. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01571635
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
- NCT number
- NCT01571635
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 46 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Beta Thalassemia Major, Beta Thalassemia Intermedia
- Interventions
- SOTATERCEPT (ACE-011)
- First posted
- 2012-04-05
- Last updated
- 2023-06-18
- Other registries' IDs
- eudract:2011-005659-15
- Results first posted
- 2023-06-18
- Limitations and caveats
Sotatercept development in β-thalassemia indication was stopped and no further patients were enrolled after 1 mg/kg cohort was completed. Due to the early termination of the study and no enrollment of the Expansion Cohort, the primary analyses to determine the recommended dose (RD) and the secondary analysis of QoL change from baseline were not conducted.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | 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. |
| Secondary | Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | From baseline to the last dose of study treatment (up to approximately 112 months) | 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. |
| Secondary | Number of Participants With Hemoglobin Level Increase From Baseline in Non-Transfusion Dependent B-Thalassemia Intermedia Participants | Measurements were taken in 9 and 12-week intervals, from baseline up to approximately 112 months | 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. |
| Secondary | Number of Participants Experiencing Adverse Events (AEs) | From first dose up to 112 days after the last dose of study treatment (up to 115 months) | 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. |
| Secondary | Concentrations of Sotatercept in Serum | 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 | Sotatercept was administered as a subcutaneous injection every 21 days during the Treatment Period. Pharmacokinetic (PK) samples were collected at the pre-specified timepoints. |
| Secondary | Number of Participants With Anti-Drug Antibody (ADA) | From first dose up to 4 months after last dose (up to approximately 116 months) | 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. |
| Secondary | Number of Participants Experiencing Quality of Life (QOL) Change From Baseline | From pre-dose up to Dose 8 (168 days/6months) and Dose 16 (336 days/12months) only | 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. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | From first dose up to 28 days post the first dose | All treated participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | From baseline to the last dose of study treatment (up to approximately 112 months) | Safety Population Transfusion Dependent-All participants who received at least one dose of sotatercept and are transfusion dependent. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Hemoglobin Level Increase From Baseline in Non-Transfusion Dependent B-Thalassemia Intermedia Participants | Measurements were taken in 9 and 12-week intervals, from baseline up to approximately 112 months | Non-RBC transfusion dependent participants-participants who are in the safety population and are not transfusion dependent. This is a subgroup of the B-thalassemia intermedia population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Experiencing Adverse Events (AEs) | From first dose up to 112 days after the last dose of study treatment (up to 115 months) | All treated participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Concentrations of Sotatercept in Serum | 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 | All treated participants | ng/mL | MEDIAN |
| SECONDARY | Number of Participants With Anti-Drug Antibody (ADA) | From first dose up to 4 months after last dose (up to approximately 116 months) | All treated participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Experiencing Quality of Life (QOL) Change From Baseline | From pre-dose up to Dose 8 (168 days/6months) and Dose 16 (336 days/12months) only | Expansion cohort |
Source ClinicalTrials.gov
Results by group130
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | Dose Level 1a: 0.1 mg/kg | 0 | Participants | ||
| Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | Dose Level 1b: 0.3 mg/kg | 0 | Participants | ||
| Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | Dose Level 2: 0.5 mg/kg | 0 | Participants | ||
| Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | Dose Level 3: 0.75 mg/kg | 0 | Participants | ||
| Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended Dose | Dose Level 4: 1 mg/kg | 0 | Participants | ||
| Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | Dose Level 1a: 0.1 mg/kg | ≥ 25% reduction | 0 | Participants | |
| Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | Dose Level 1b: 0.3 mg/kg | ≥ 25% reduction | 0 | Participants | |
| Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | Dose Level 2: 0.5 mg/kg | ≥ 25% reduction | 2 | Participants | |
| Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | Dose Level 3: 0.75 mg/kg | ≥ 25% reduction | 4 | Participants | |
| Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During Treatment | Dose Level 4: 1 mg/kg | ≥ 25% reduction | 3 | Participants |
1 to 10 of 130Source ClinicalTrials.gov
See all 130 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Dose Level 1a: 0.1 mg/kg | 8 | |
| Overall Study | STARTED | Dose Level 1b: 0.3 mg/kg | 9 | |
| Overall Study | STARTED | Dose Level 2: 0.5 mg/kg | 8 | |
| Overall Study | STARTED | Dose Level 3: 0.75 mg/kg | 12 | |
| Overall Study | STARTED | Dose Level 4: 1 mg/kg | 9 | |
| Overall Study | COMPLETED | Dose Level 1a: 0.1 mg/kg | 2 | |
| Overall Study | COMPLETED | Dose Level 1b: 0.3 mg/kg | 1 | |
| Overall Study | COMPLETED | Dose Level 2: 0.5 mg/kg | 1 | |
| Overall Study | COMPLETED | Dose Level 3: 0.75 mg/kg | 0 | |
| Overall Study | COMPLETED | Dose Level 4: 1 mg/kg | 0 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events1,985
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | ANAEMIA | Blood and lymphatic system disorders | Dose Level 1a: 0.1 mg/kg | 1 | 8 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Dose Level 1b: 0.3 mg/kg | 0 | 9 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Dose Level 2: 0.5 mg/kg | 0 | 8 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Dose Level 3: 0.75 mg/kg | 1 | 12 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Dose Level 4: 1 mg/kg | 0 | 9 | |
| other | BONE MARROW FAILURE | Blood and lymphatic system disorders | Dose Level 1a: 0.1 mg/kg | 0 | 8 | |
| other | BONE MARROW FAILURE | Blood and lymphatic system disorders | Dose Level 1b: 0.3 mg/kg | 1 | 9 | |
| other | BONE MARROW FAILURE | Blood and lymphatic system disorders | Dose Level 2: 0.5 mg/kg | 0 | 8 | |
| other | BONE MARROW FAILURE | Blood and lymphatic system disorders | Dose Level 3: 0.75 mg/kg | 0 | 12 | |
| other | BONE MARROW FAILURE | Blood and lymphatic system disorders | Dose Level 4: 1 mg/kg | 0 | 9 |
1 to 10 of 1,985Source ClinicalTrials.gov
See all 1,985 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Dose Level 1a: 0.1 mg/kg | 0 | 8 |
| Total, all-cause mortality | Dose Level 1b: 0.3 mg/kg | 0 | 9 |
| Total, all-cause mortality | Dose Level 2: 0.5 mg/kg | 0 | 8 |
| Total, all-cause mortality | Dose Level 3: 0.75 mg/kg | 0 | 12 |
| Total, all-cause mortality | Dose Level 4: 1 mg/kg | 0 | 9 |
| Total, other adverse events | Dose Level 1a: 0.1 mg/kg | 8 | 8 |
| Total, other adverse events | Dose Level 1b: 0.3 mg/kg | 9 | 9 |
| Total, other adverse events | Dose Level 2: 0.5 mg/kg | 8 | 8 |
| Total, other adverse events | Dose Level 3: 0.75 mg/kg | 12 | 12 |
| Total, other adverse events | Dose Level 4: 1 mg/kg | 9 | 9 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 30337358 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01571635
GET https://conductscience.org/api/trials/v1/NCT01571635/results?page=2