Clinical Trials List
Protocol NumberACE-536-LTFU-001
NCT Number(ClinicalTrials.gov Identfier)NCT04064060
2019-08-01 - 2027-08-31
Phase III
Recruiting2
Terminated1
ICD-10D56.9
Thalassemia, unspecified
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
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Trial Applicant
PPD DEVELOPMENT (HK) LIMITED
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Sponsor
Celgene Corporation
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- 陳君明 Division of Radiology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Hui-Hua Hsiao Division of Hematology & Oncology
- 蔡郁棻 Division of Hematology & Oncology
- 唐世豪 Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- 張修豪 Division of Pediatrics
- YUNG-LI YANG Division of Pediatrics
- 林凱信 Division of Pediatrics
- SHIANN-TANG JOU Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Condition/Disease
Thalassemia
Objectives
Primary Objective
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.
Secondary Objective
To follow subjects for overall survival
Test Drug
Luspatercept (ACE-536)
Active Ingredient
Luspatercept (ACE-536)
Dosage Form
injection
Dosage
25 mg/vial; 75 mg/vial
Endpoints
Primary Outcome Measures :
Adverse Events (AEs) [ Time Frame: From enrollment until at least 42 Day Safety Follow-up Phase or EOS (Approximately 5 years). ]
Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept
Number of subjects progressing to high/very high risk MDS or AML. [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only).
Percentage of subjects progressing to high/very high risk MDS or AML [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only)
Number of subjects developing other malignancies/pre-malignancies [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Development of other malignancies/pre-malignancies
Percentage of subjects developing other malignancies/pre-malignancies [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Development of other malignancies/pre-malignancies
Secondary Outcome Measures :
Overall Survival [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Time from date of randomization until death from any cause
Adverse Events (AEs) [ Time Frame: From enrollment until at least 42 Day Safety Follow-up Phase or EOS (Approximately 5 years). ]
Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept
Number of subjects progressing to high/very high risk MDS or AML. [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only).
Percentage of subjects progressing to high/very high risk MDS or AML [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Progression to high/very high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only)
Number of subjects developing other malignancies/pre-malignancies [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Development of other malignancies/pre-malignancies
Percentage of subjects developing other malignancies/pre-malignancies [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Development of other malignancies/pre-malignancies
Secondary Outcome Measures :
Overall Survival [ Time Frame: Enrollment to Long-term post-treatment follow-up (Approximately, 5 years) ]
Time from date of randomization until death from any cause
Inclution 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.
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.
Exclusion Criteria
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.
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.
The Estimated Number of Participants
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Taiwan
10 participants
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Global
700 participants
