Trial · NCT03342404
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
NCT03342404, Phase 2, Completed, Celgene; results first posted 2021-11-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia. Trials, record NCT03342404. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03342404
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
- NCT number
- NCT03342404
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 145 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Thalassemia
- Interventions
- Luspatercept, Placebo, Best Supportive Care (BSC)
- First posted
- 2017-11-17
- Last updated
- 2023-12-20
- Other registries' IDs
- eudract:2015-003225-33
- Results first posted
- 2021-11-18
Source ClinicalTrials.gov
Registered outcomes30
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Achieving Erythroid Response (Week 13 to Week 24) | From Week 13 to Week 24 of study treatment | Erythroid Response is defined as an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from Weeks 13 to 24 of treatment in the absence of transfusions. Baseline hemoglobin (Hb) is the average of 2 or more Hb measurements at least 1 week apart within 4 weeks prior to Dose 1. |
| Secondary | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Tiredness and Weakness (T/W) Domain Score (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | The NTDT-PRO assesses the severity of anemia-related symptoms with a daily recall of symptoms composed of 6 items: 1. Tiredness (lack of energy) when not doing physical activity 2. Tiredness when doing physical activity 3. Weakness (lack of strength) when not doing physical activity 4. Weakness when doing physical activity 5. Shortness of breath when not doing physical activity 6. Shortness of breath when doing physical activity. The Tiredness/Weakness (T/W) domain score is the average score of items 1 through 4 above. T/W domain score ranges from 0 (best outcome, no tiredness/weakness) to 10 (worst outcome, extreme tiredness/weakness). Weekly T/W Scores are the average of daily scores for that week. The mean of weekly scores over a continuous 12-week period (from Week 13 to Week 24) are compared to the T/W Domain Score at baseline. Baseline is defined as the last value taken on or before the first dose of study drug administered in Week 13. |
| Secondary | Mean Change From Baseline in Hemoglobin Values in the Absence of Transfusion (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | Mean change from baseline in mean of hemoglobin (Hb) values over a continuous 12-week interval from Week 13 to Week 24 in the absence of transfusions. Baseline was defined as the average of 2 or more Hb measurements at least 1 week apart within 4 weeks before Dose 1 Day 1. |
| Secondary | Percentage of Participants Achieving Erythroid Response (Week 37 to Week 48) | Assessed over a continuous 12 week period (from week 37 through week 48) | Erythroid Response is defined as an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from Weeks 37 to 48 of treatment in the absence of transfusions. Baseline hemoglobin (Hb) is the average of 2 or more Hb measurements at least 1 week apart within 4 weeks prior to Dose 1. |
| Secondary | Mean Change From Baseline in Mean Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Fatigue Subscale Score (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | The FACIT-F is a multidimensional, self-report quality of life instrument which includes the Functional Assessment of Cancer Therapy - General (FACT-G) questionnaire (27 items over 4 different domains), and the Fatigue subscale (FS) component (13 items). FACIT-F version 4 has been used for this study. For the Fatigue subscale, each of the 13 items is scored from 0 ("not at all") to 4 ("very much"). The scores from individual items are summed to generated the final FS score, which ranges from 0 (best outcome) to 52 (worst outcome). The questionnaire is completed every other dose, and the mean of FS scores from Week 13 to Week 24 is compared to the FS score at baseline (last score available before start of study treatment). Baseline is defined as the last value taken on or before the first dose of study drug administered in Week 13. |
| Secondary | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Shortness of Breath (SoB) Domain Score (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | The NTDT-PRO V2.1 assess the severity of anemia-related symptoms associated with NTD β-thalassemia. It is a daily electronic diary with recall of symptoms during the past 24 hours, composed of 6 items: 1. Tiredness (lack of energy) when not doing physical activity 2. Tiredness (lack of energy) when doing physical activity 3. Weakness (lack of strength) when not doing physical activity 4. Weakness (lack of strength) when doing physical activity 5. Shortness of breath when not doing physical activity 6. Shortness of breath when doing physical activity. The Shortness of Breath (SoB) domain score represents the average score of items 5 and 6 above. SoB domain score ranges from 0 (best outcome, no shortness of breath) to 10 (worst outcome, extreme shortness of breath). Weekly SoB Scores represent the average of daily scores for that week. The mean of weekly scores over a continuous 12 week period (from Week 13 to Week 24) are compared to the SoB Domain Score at baseline. |
| Secondary | Mean Change From Baseline in Hemoglobin Values in the Absence of Transfusion (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | Mean change from baseline in mean of hemoglobin (Hb) values over a continuous 12-week interval from Week 37 to Week 48 in the absence of transfusions. Baseline was defined as the average of 2 or more Hb measurements at least 1 week apart within 4 weeks before Dose 1 Day 1. |
| Secondary | Mean Change From Baseline in Mean Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Fatigue Subscale Score (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | The FACIT-F is a multidimensional, self-report quality of life instrument which includes the Functional Assessment of Cancer Therapy - General (FACT-G) questionnaire (27 items over 4 different domains), and the Fatigue subscale (FS) component (13 items). FACIT-F version 4 has been used for this study. For the Fatigue subscale, each of the 13 items is scored from 0 ("not at all") to 4 ("very much"). The scores from individual items are summed to generated the final FS score, which ranges from 0 (best outcome) to 52 (worst outcome). The questionnaire is completed every other dose, and the mean of FS scores from Week 37 to Week 48 is compared to the FS score at baseline (last score available before start of study treatment). Baseline is defined as the last value taken on or before the first dose of study drug administered in Week 37. |
| Secondary | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Tiredness and Weakness (T/W) Domain Score (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | NTDT-PRO V2.1 assess the severity of anemia-related symptoms. It's a daily recall of symptoms during the past 24 hours, composed of 6 items: 1. Tiredness (lack of energy) when not doing physical activity 2. Tiredness when doing physical activity 3. Weakness (lack of strength) when not doing physical activity 4. Weakness when doing physical activity 5. Shortness of breath when not doing physical activity 6. Shortness of breath when doing physical activity. The Tiredness/Weakness (T/W) domain score represents the average score of items 1 through 4 above. T/W domain score ranges from 0 (best outcome, no tiredness/weakness) to 10 (worst outcome, extreme tiredness/weakness). Weekly T/W Scores represent the average of daily scores for that week. The mean of weekly scores over a continuous 12-week period (from Week 37 to Week 48) are compared to the T/W Domain Score at baseline. Baseline is defined as the last value taken on or before the first dose of study drug administered in Week 37. |
| Secondary | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Shortness of Breath (SoB) Domain Score (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | NTDT-PRO V2.1 assess the severity of anemia-related symptoms. It is a daily recall of symptoms during the past 24 hours, composed of 6 items: 1. Tiredness (lack of energy) when not doing physical activity 2. Tiredness when doing physical activity 3. Weakness (lack of strength) when not doing physical activity 4. Weakness when doing physical activity 5. Shortness of breath when not doing physical activity 6. Shortness of breath when doing physical activity. The Shortness of Breath (SoB) domain score represents the average score of items 5 and 6 above. SoB domain score ranges from 0 (best outcome, no shortness of breath) to 10 (worst outcome, extreme shortness of breath). Weekly SoB Scores represent the average of daily scores for that week. The mean of weekly scores over a continuous 12-week period (from Week 37 to Week 48) are compared to the SoB Domain Score at baseline. Baseline is defined as the last value taken on or before the first dose of study drug administered in Week 37. |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 registered outcomesOutcomes30
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Achieving Erythroid Response (Week 13 to Week 24) | From Week 13 to Week 24 of study treatment | All treated participants. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Percent of Participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Tiredness and Weakness (T/W) Domain Score (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | All treated participants with available measurements at Baseline and from Week 13 to Week 24. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in Hemoglobin Values in the Absence of Transfusion (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | All treated participants with available measurements at Baseline and From Week 13 to Week 24. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | g/dL | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants Achieving Erythroid Response (Week 37 to Week 48) | Assessed over a continuous 12 week period (from week 37 through week 48) | All treated participants. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Percentage of Participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Mean Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Fatigue Subscale Score (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | All treated participants with available measurements at Baseline and at least one post-baseline FACIT-F questionnaire completed. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Shortness of Breath (SoB) Domain Score (Week 13 to Week 24) | Baseline and over a continuous 12 week period (from week 13 through week 24) | All treated participants with available measurements at Baseline and From Week 13 to Week 24. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in Hemoglobin Values in the Absence of Transfusion (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | All treated participants with available measurements at Baseline and From Week 37 to Week 48. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | g/dL | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in Mean Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Fatigue Subscale Score (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | All treated participants with available measurements at Baseline and at least one post-baseline FACIT-F questionnaire completed. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Tiredness and Weakness (T/W) Domain Score (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | All treated participants with available measurements at Baseline and at Week 37 to Week 48. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Shortness of Breath (SoB) Domain Score (Week 37 to Week 48) | Baseline and over a continuous 12 week period (from week 37 through week 48) | All treated participants with available measurements at Baseline and From Week 37 to Week 48. Placebo participants are assessed up to crossing over to luspatercept. The luspatercept group does not include placebo participants who crossed over to luspatercept. | Score on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 outcomesResults by group92
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Achieving Erythroid Response (Week 13 to Week 24) | Luspatercept | 77.1 | 95% Confidence Interval 67.4 to 85.0 | Percent of Participants | |
| Percentage of Participants Achieving Erythroid Response (Week 13 to Week 24) | Placebo | 0.0 | 95% Confidence Interval 0.0 to 7.3 | Percent of Participants | |
| Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Tiredness and Weakness (T/W) Domain Score (Week 13 to Week 24) | Luspatercept | -0.68 | Standard Error 0.176 | Score on a scale | |
| Mean Change From Baseline in Non-Transfusion Dependent β-thalassemia-Patient Reported Outcome (NTDT-PRO) Tiredness and Weakness (T/W) Domain Score (Week 13 to Week 24) | Placebo | -0.20 | Standard Error 0.240 | Score on a scale | |
| Mean Change From Baseline in Hemoglobin Values in the Absence of Transfusion (Week 13 to Week 24) | Luspatercept | 1.48 | Standard Error 0.078 | g/dL | |
| Mean Change From Baseline in Hemoglobin Values in the Absence of Transfusion (Week 13 to Week 24) | Placebo | 0.07 | Standard Error 0.108 | g/dL | |
| Percentage of Participants Achieving Erythroid Response (Week 37 to Week 48) | Luspatercept | 70.8 | 95% Confidence Interval 60.7 to 79.7 | Percentage of Participants | |
| Percentage of Participants Achieving Erythroid Response (Week 37 to Week 48) | Placebo | 2.0 | 95% Confidence Interval 0.1 to 10.9 | Percentage of Participants | |
| Mean Change From Baseline in Mean Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Fatigue Subscale Score (Week 13 to Week 24) | Luspatercept | 1.64 | Standard Error 0.774 | Score on a scale | |
| Mean Change From Baseline in Mean Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Fatigue Subscale Score (Week 13 to Week 24) | Placebo | 0.26 | Standard Error 1.066 | Score on a scale |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Blinded Treatment Phase | STARTED | Luspatercept | 96 | |
| Blinded Treatment Phase | STARTED | Placebo | 49 | |
| Blinded Treatment Phase | COMPLETED | Luspatercept | 68 | |
| Blinded Treatment Phase | COMPLETED | Placebo | 18 | |
| Blinded Treatment Phase | NOT COMPLETED | Luspatercept | 28 | |
| Blinded Treatment Phase | NOT COMPLETED | Placebo | 31 | |
| Open-Label Phase | STARTED | Luspatercept | 0 | |
| Open-Label Phase | STARTED | Placebo | 38 | |
| Open-Label Phase | COMPLETED | Luspatercept | 0 | |
| Open-Label Phase | COMPLETED | Placebo | 27 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events408
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 | Extramedullary haemopoiesis | Blood and lymphatic system disorders | Luspatercept | 9 | 96 | |
| other | Extramedullary haemopoiesis | Blood and lymphatic system disorders | Placebo | 2 | 49 | |
| other | Extramedullary haemopoiesis | Blood and lymphatic system disorders | Open-Label Placebo-Luspatercept | 2 | 38 | |
| other | Palpitations | Cardiac disorders | Luspatercept | 9 | 96 | |
| other | Palpitations | Cardiac disorders | Placebo | 6 | 49 | |
| other | Palpitations | Cardiac disorders | Open-Label Placebo-Luspatercept | 4 | 38 | |
| other | Tachycardia | Cardiac disorders | Luspatercept | 6 | 96 | |
| other | Tachycardia | Cardiac disorders | Placebo | 2 | 49 | |
| other | Tachycardia | Cardiac disorders | Open-Label Placebo-Luspatercept | 2 | 38 | |
| other | Ear pain | Ear and labyrinth disorders | Luspatercept | 3 | 96 |
1 to 10 of 408Source ClinicalTrials.gov
See all 408 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Luspatercept | 1 | 96 |
| Total, all-cause mortality | Placebo | 0 | 49 |
| Total, all-cause mortality | Open-Label Placebo-Luspatercept | 0 | 38 |
| Total, other adverse events | Luspatercept | 96 | 96 |
| Total, other adverse events | Placebo | 48 | 49 |
| Total, other adverse events | Open-Label Placebo-Luspatercept | 38 | 38 |
| Total, serious adverse events | Luspatercept | 23 | 96 |
| Total, serious adverse events | Placebo | 13 | 49 |
| Total, serious adverse events | Open-Label Placebo-Luspatercept | 3 | 38 |
Source ClinicalTrials.gov
Papers6
The papers the trial lists on ClinicalTrials.gov.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Luspatercept vs Placebo: Number of Participants With Anti-drug Antibody (ADA) Positive Test for Luspatercept |
Source Trial comparisons
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/NCT03342404
GET https://conductscience.org/api/trials/v1/NCT03342404/registered?page=2