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
- Conditions
- Prior participation on a clinical trial of luspatercept (ACE-536) in protocols eligible for participation in this study ACE-536-LTFU-001 with the following medical conditions:- Myelodysplastic Syndrome (MDS)- Beta (ß)-thalassemia (THAL)- Myelofibrosis (MF)In Lebanon, only patients with beta (ß)-thalassemia (THAL) have participated in previuos clinical trial of luspatercept (ACE-536).Thalassemia
- Registration Number
- LBCTR2019100218
- Lead Sponsor
- Celgene Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 742
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:
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.
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 treatment 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: Adverse events (AEs);Timepoints: Enrollment to 42 days post last dose;Measure: Type, frequency, severity of AEs, relationship of treatment emergent adverse events to luspatercept;Name: Development of other malignancies/pre-malignancies;Timepoints: Enrollment to Long-term Post-treatment Follow-up;Measure: Number and percentage of subjects developing other malignancies/premalignancies;Name: Progression to high/very high risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only). Not applicable for Lebanon patient population consists of B-Thal patients only.;Timepoints: Number and percentage of subjects progressing to high/very high risk MDS or AML;Measure: Enrollment to LTPTF
- Secondary Outcome Measures
Name Time Method ame: Overall survival;Timepoints: Enrollment to Long-term Post-treatment Follow-up;Measure: Time from date of randomization until death from any cause