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
- Myelodysplastic Syndrome (MDS)bone marrow failure disorders10002086
- Registration Number
- NL-OMON56073
- Lead Sponsor
- Celgene Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 5
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 cross-over 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 or amenorrhea due to other medical reasons 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. A medically supervised serum pregnancy
test (conducted locally) is to be obtained and verified negative in all female
subjects of childbearing potential at enrollment (for details refer
to Section 6.1.7). 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. 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.
* True abstinence is acceptable when this is in line with the preferred and
usual lifestyle of the subject. (Periodic abstinence [eg, calendar, ovulation,
symptothermal, post-ovulation methods] and withdrawal are not acceptable
methods of contraception).
1. 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. Applies to on treatment subjects only- More than 26 days between last
luspatercept dose in the parent protocol and first dose into ACE- 536LTFU-001
protocol unless dose delay or dose discontinuation criteria
met.
4. Applies to on treatment subjects only- Pregnant or breastfeeding females. If
breastfeeding, agree to stop breastfeeding prior to the participation in the
study and not to resume breastfeeding during treatment with luspatercept and
until 3 months after the last dose.
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 <p>1- Adverse events (AEs): Type, frequency, severity of AEs, relationship of<br /><br>treatment emergent adverse events to luspatercept. Timeframe: Enrollment to 42<br /><br>days post last dose.<br /><br><br /><br>2- Progression to high/very high risk myelodysplastic syndrome (MDS) or<br /><br>Progression to acute myeloid leukemia (AML) (MDS and myelofibrosis [MF] only).<br /><br>Number and percentage of subjects progressing to high/very high risk MDS or AML<br /><br>. Timeframe; Enrollment to LTPTFU<br /><br><br /><br>3- Development of other malignancies/pre-malignancies. Number and percentage<br /><br>of subjects developing other malignancies/premalignancies. Timeframe;<br /><br>Enrollment to LTPTFU </p><br>
- Secondary Outcome Measures
Name Time Method <p>Overall survival: Time from date of randomization until death from any cause.<br /><br>Timeframe: Enrollment to LTPTFU<br /><br>Treatment-emergent EMH masses - Number and percentage of subjects developing<br /><br>treatment emergent EMH masses</p><br>