Open-Label Extension Study to Provide Continued Access to Avutometinib Alone or in Combination with Defactinib in Patients with Recurrent Low-Grade Serous Ovarian Cancer (LGSOC)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Verastem Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Decision date (initial)

2026-06-22

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Verastem Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety

To provide continued treatment with avutometinib alone or in combination with defactinib for participants with recurrent LGSOC who are deriving continued clinical benefit after completion of the RAMP-201 study.

Secondary objectives 1

1. To monitor and document safety and tolerability of avutometinib alone or in combination with defactinib during extended treatment in this study.

Conditions and MedDRA coding

Recurrent Low-Grade Serous Ovarian Cancer (LGSOC)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Completion of the RAMP-201 study while receiving avutometinib alone or in combination with defactinib, meeting all requirements to transition directly into this OLE study without interruption of treatment. Participants must be deriving ongoing clinical benefit at the time of enrollment in this study (e.g., complete response, partial response, or stable disease), as determined by the investigator. Any active dose hold from the RAMP-201 study should be ≤28 days at the time of Screening for the OLE study.
2. The Investigator determines that continued treatment is in the participant’s best interest and that the participant fulfills all protocol-defined criteria for continued treatment. Eligibility confirmation may be reviewed by the Sponsor to ensure consistency with the protocol.
3. Female participants of childbearing potential must agree to use a highly effective method of contraception (Section 7.1) throughout OLE study participation and for at least 30 days after the last dose of study treatment. Note: Female participants of childbearing potential must have a negative serum or urine pregnancy test at Screening/Eligibility prior to the first OLE dose.
4. Ability and willingness to provide written informed consent for participation in the study and for continued data collection.

Exclusion criteria 9

1. Radiographic or clinical disease progression or discontinuation of treatment due to unacceptable toxicity during the RAMP-201 study or at the time of screening in this study.
2. Participants on an active dose hold in the RAMP-201 study may transition to this study only after the underlying issue has been clinically resolved and the investigator, in consultation with the Sponsor, confirms that continued treatment is appropriate.
3. Receipt of new systemic anti-cancer therapy since participation in the RAMP-201 study.
4. Participation in another interventional clinical study or receipt of other investigational therapy since participation in RAMP-201 study.
5. Any active or uncontrolled medical condition that, in the investigator’s judgment, would pose unacceptable risk or interfere with continued treatment, including but not limited to severe cardiac, pulmonary, neurologic, dermatologic, gastrointestinal, hepatic, or active infectious disease.
6. Any active or history of clinically significant ocular disorder that may increase the risk of MEK-inhibitor–related ocular toxicity (e.g., retinal vein occlusion, serous retinopathy, retinal detachment, or clinically significant macular pathology).
7. Concomitant use of medications known to have clinically significant interactions with avutometinib or defactinib that cannot be safely discontinued or substituted prior to study dosing (e.g., strong CYP3A4 inducers or inhibitors, warfarin) (Section 10.6).
8. Positive pregnancy test at Screening, breastfeeding, or unwillingness to use adequate contraception during the study.
9. Any condition or circumstance that, in the investigator’s opinion, may compromise participant safety, compliance or continued benefit from study treatment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Investigator’s determination of ongoing clinical benefit

Secondary endpoints 1

1. Frequency and severity of side effects, including abnormal findings from laboratory tests or physical examinations

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Verastem Inc.

Sponsor organisation

Verastem Inc.

Address

117 Kendrick Street Suite 500

City

Needham

Postcode

02494-2730

Country

United States

Scientific contact point

Organisation

Verastem Inc.

Contact name

Medical Affairs

Public contact point

Organisation

Verastem Inc.

Contact name

Medical Affairs

Third parties 1

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications