Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
3
Countries
1
Sites
1
Hematological Disorders
To evaluate the long-term safety of CC-486 in participants who have received CC-486 as monotherapy in the parent CC-486 studies and whom the Investigators feel may derive clinical benefit from continuing treatment with CC 486.
Key facts
- Sponsor
- Celgene Corp.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- completed 15 Apr 2025
- Decision date (initial)
- 2023-09-07
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- Celgene Corporation
External identifiers
- EU CT number
- 2023-503272-25-00
- WHO UTN
- U1111-1292-3813
- ClinicalTrials.gov
- NCT02494258
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Others
To evaluate the long-term safety of CC-486 in participants who have received CC-486 as monotherapy in the parent CC-486 studies and whom the Investigators feel may derive clinical benefit from continuing treatment with CC 486.
Secondary objectives 1
- To follow participants who were treated with CC-486 or placebo in the parent CC486 study for survival if required by the parent CC-486 study protocol
Conditions and MedDRA coding
Hematological Disorders
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10065553 | Bone marrow failure | 100000004851 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Previously participated in, received CC-486, and continues to fulfill the eligibility criteria in one of the parent CC-486 studies
- Continue to derive clinical benefit from treatment with CC-486 as per Investigator’s opinion.
- In order to be enrolled for the survival follow up in the Follow up Phase, participants must have been in a parent CC-486 study where monitoring for survival was required.
- Both women of childbearing potential (WOCBP) and men who are sexually active with WOCBP must use an efficient method of contraception after the last dose of CC-486 in the parent CC-486 study and prior to the 1st dose of CC-486 in this study without interruption; throughout participation in this study, and for a period after treatment completion as specified in the protocol.
Exclusion criteria 5
- Concomitant use of drugs that are prohibited.
- Prior chemotherapy (including injectable azacitidine), radiotherapy or any investigational agent after the last dose of CC-486 received in the parent CC 486 study.
- Received CC-486 in combination with another compound during the parent CC-486 study.
- Transition into rollover study ≥ 45 days after End of Study visit of the parent CC-486 study.
- Pregnant or lactating females. There are no exclusion criteria to prevent entry or remaining on the follow-up phase of this study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Safety: type, frequency, severity, and relationship of treatment emergent adverse events (AEs) to investigational product (CC-486).
Secondary endpoints 1
- Survival: time from randomization until death from any cause.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 4
PRD9836719 · Product
- Active substance
- Azacitidine
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 300 mg milligram(s)
- Max total dose
- 6300 mg milligram(s)
- Max treatment duration
- 124 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD9836740 · Product
- Active substance
- Azacitidine
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 300 mg milligram(s)
- Max total dose
- 6300 mg milligram(s)
- Max treatment duration
- 124 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD9836736 · Product
- Active substance
- Azacitidine
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 300 mg milligram(s)
- Max total dose
- 6300 mg milligram(s)
- Max treatment duration
- 124 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD9836742 · Product
- Active substance
- Azacitidine
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 300 mg milligram(s)
- Max total dose
- 6300 mg milligram(s)
- Max treatment duration
- 124 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Celgene Corp.
- Sponsor organisation
- Celgene Corp.
- Address
- Route 206 And Province Line Road
- City
- Princeton
- Postcode
- 08543-4000
- Country
- United States
Scientific contact point
- Organisation
- Celgene Corp.
- Contact name
- GSM-CT
Public contact point
- Organisation
- Celgene Corp.
- Contact name
- GSM-CT
Third parties 3
| Organisation | City, country | Duties |
|---|---|---|
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| WCG Clinical Inc. ORG-100040730
|
Plymouth Meeting, United States | Code 8 |
| Labcorp Endpoint Clinical Inc. ORG-100040567
|
Wakefield, United States | Interactive response technologies (IRT) |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Finland | Ended | 1 | 1 |
| Rest of world
United Kingdom, United States
|
— | 2 | — |
Investigational sites
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Summary of results Art. 37(4) CTR
| Title | Submission date | Status | Type |
|---|---|---|---|
| 2023-503272-25-00_Final Summary of Results SUM-123342
|
2026-03-13T13:11:59 | Submitted | Summary of Results |
Layperson summary Annex V
| Title | Submission date | Status | Type |
|---|---|---|---|
| 2023-503272-25-00_Lay Person Summary of Results | 2026-04-08T09:08:06 | Submitted | Laypersons Summary of Results |
Documents 4 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Laypersons summary of results (for publication) | 2023-503272-25-00_Lay Person Summary of Results | N/A |
| Protocol (for publication) | D1_Celgene_CC-486-GEN-001_Protocol_ForPub | 1.0 EU |
| Summary of results (for publication) | 2023-503272-25-00_Final Summary of Results | N/A |
| Synopsis of the protocol (for publication) | D1_Celgene_CC-486-GEN-001_Synopsis of the Protocol_For Pub | 2.0 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-05-16 | Finland | Acceptable 2023-09-04
|
2023-09-07 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-04-12 | Finland | Acceptable 2024-05-27
|
2024-05-28 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-08-13 | Acceptable 2024-05-27
|