A study to assess the safety, tolerability, pharmacokinetic, pharmacodynamic, immunogenicity and antitumour activity of IPN60300 in adults with locally advanced or metastatic solid tumours

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ipsen Pharma

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

10 Feb 2026 → ongoing

Decision date (initial)

2025-12-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Ipsen Pharma SAS

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Pharmacokinetic, Efficacy, Safety, Others

Phase Ia
To evaluate the safety and tolerability of IPN60300 as a single agent and determine the MTD/MAD in participants with tumour type of interest
Phase Ib
To determine the optimal dosing regimen ofIPN60300 in participants with selected tumour(s)

Secondary objectives 1

1. Phase Ia • To characterise the PK of IPN60300 (ADC), the total antibody, and free toxin after single and repeat dose administrations of IPN60300 • To assess the immunogenicity potential of IPN60300 • To assess the preliminary clinical benefit of IPN60300 by evaluation of anti-tumour activity Phase Ib • To assess the clinical benefit of IPN60300 by evaluation of antitumour activity • To further assess the immunogenicity potential of IPN60300

Conditions and MedDRA coding

Advanced or metastatic solid tumours

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. (1) Participant must be ≥18 years of age, at the time of signing the informed consent.
2. (2) Participants with histologically or cytologically documented, locally advanced, or metastatic solid tumours that relapsed or were refractory after being previously treated with standard of care therapy; or for which there is no available established therapy; or standard therapy is contraindicated or not deemed appropriate by the treating investigator.
3. (3) Participants must have measurable disease per Response Evaluation Criteria in Solid Tumours (RECIST) version 1.1.
4. (4) Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
5. (5) Male and female participants: Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
6. (6) Adequate bone marrow function within 7 days before first dose of study intervention
7. (7) Adequate renal function within 7 days before first dose of study intervention
8. (8) Adequate hepatic function or laboratory abnormalities indicating hepatic injury within 7 days before first dose of study intervention
9. (9) Prothrombin time or international normalised ratio (INR) ≤1.5 × ULN
10. (10) At the time of screening, a tumour tissue specimen is required for enrolment into the dose escalation and dose optimisation portions of the study for retrospective central laboratory determination
11. (11) Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF)
12. (12) Have a life expectancy of more than 3 months for disease-related mortality, as evaluated by the investigator

Exclusion criteria 17

1. (1) Known second malignancy either progressing or requiring active treatment within the last 2 years prior to first dose of study intervention within the last 2 years prior to first dose of study intervention.
2. (2) Residual toxicity from prior anticancer therapy that are NCI CTCAE version 5.0 Grade 2 or higher. Stable chronic Grade 2 toxicities from previous treatments may be eligible per the judgement of investigator.
3. (3) History of major surgery within 4 weeks prior to the first dose of study intervention
4. (4) Previous solid organ transplantation
5. (5) Pre-existing, acute or chronic severe corneal disorders, sequelae from severe cornedisorders, or a history of corneal transplantation
6. (6) Active brain metastases or leptomeningeal metastases with exception to asymptomatic and treated brain metastases
7. (7) History of stroke or significant cerebrovascular disease (ie, transient ischemic attack) within 6 months prior to initiation of study intervention.
8. (8) History of clinically significant cardiac disease within 6 months prior to the initiation of study intervention, including but not limited to unstable angina, acute myocardial infarction, endoscopic or open-heart cardiac surgery, or heart failure classified as NYHA Grade 2 or higher
9. (9) History of clinically significant respiratory disease within 6 months prior to the initiation of study intervention, including severe chronic obstructive pulmonary disease or asthma.
10. (10) History of noninfectious interstitial lung disease (ILD)/pneumonitis/radiation pneumonitis that required steroids or has current ILD/pneumonitis.
11. (11) Clinically significant gastrointestinal disorder including bleeding, occlusion, diarrhoea >Grade 1, malabsorption syndrome, ulcerative colitis, inflammatory bowel disease or partial bowel obstruction.
12. (12) Any evidence of severe active infection or inflammatory condition
13. (13) Significant concurrent, uncontrolled medical condition that would put participants at unacceptable risk from study participation or preclude them from complying with study procedures as per investigator assessment
14. (14) Participants with uncontrolled human immunodeficiency virus (HIV). HIV-infected participants are eligible if they meet the criteria described in protocol
15. (15) Known active infection with hepatitis B virus (HBV) OR hepatitis C virus (HCV). Participants are eligible if they meet the criteria described in protocol
16. (16) Prior anticancer therapy, including chemotherapy, targeted therapy, therapeutic anticancer antibodies, or radio-/toxin-immunoconjugates
17. (17) Ongoing immunosuppressive therapy, including systemic corticosteroids.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Phase Ia: - Percentage of participants with dose limiting toxicity (DLT). Cycle1: within 21 days following study drug administration.
2. Phase Ia and Ib: - Percentage of participants experiencing treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TE-SAEs). From the first IPN60300 administration until 30 days after the last dose
3. Phase Ib: Objective response rate (ORR) - ORR is defined as the percentage of participants with best overall response (BOR) of complete response (CR) or partial response (PR), as determined by investigator per RECIST version 1.1. At end of study (up to approximately 3 years)

Secondary endpoints 14

1. Phase Ia and Ib: - Percentage of Treatment-Emergent Anti-Drug Antibodies (ADA), Including Binding and Neutralizing Antibodies. Prior study drug administration until the End of Treatment (EoT) visit (approximately up to 3 years)
2. Phase Ia: Time to maximum observed drug concentration (tmax) of IPN60300 after single and multiple doses of IPN60300. At Cycle 1 and Cycle 2: within 21 days following study drug administration
3. Phase Ia - Maximum observed drug concentration (cmax) of IPN60300 after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration
4. Phase Ia - Area under the plasma concentration time curve over one dosing interval (AUCtau) of IPN60300 after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration
5. Phase Ia - Tmax of Total Antibody after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration.
6. Phase Ia - Cmax of Total Antibody after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration.
7. Phase Ia - AUCtau of Total Antibody after single and multiple doses of IPN6030. At Cycles 1 and 2: Within 21 days following study drug administration.
8. Phase Ia - Tmax of free toxin after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration.
9. Phase Ia - Cmax of free toxin after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration.
10. Phase Ia - AUCtau of free toxin after single and multiple doses of IPN60300. At Cycles 1 and 2: Within 21 days following study drug administration.
11. Phase Ia - Objective Response Rate (ORR). ORR is defined as the percentage of participants with BOR of CR or PR, as determined by investigator per RECIST version 1.1. At end of study (up to approximately 3 years)
12. Ph Ib - DoR is defined as the time from the first documented evidence of CR or PR until progressive disease, as determined by investigator per RECIST version 1.1, or death from any cause, whichever occurs first. At end of study (up to approximately 3 years)
13. Ph Ib - PFS is defined as the time from the date of first IPN60300 administration to the date of the first documented disease progression, as determined by investigator per RECIST version 1.1, or death due to any cause, whichever occurs first. At end of study (up to approximately 3 years)
14. Ph Ib - DCR is defined as the percentage of participants with BOR of CR, PR or stable desease (SD), as determined by investigator per RECIST version 1.1. At end of study (up to approximately 3 years)

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ipsen Pharma

Sponsor organisation

Ipsen Pharma

Address

70 Rue Balard

City

Paris

Postcode

75015

Country

France

Scientific contact point

Organisation

Ipsen Pharma

Contact name

Ipsen Clinical Study Enquiries

Public contact point

Organisation

Ipsen Pharma

Contact name

Ipsen Clinical Study Enquiries

Third parties 13

Locations

2 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 16 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