A Phase 2 Pilot Study to Evaluate the Safety and the Anti-Tumour Activity of the Myc Inhibitor OMO-103 Administered Intravenously in Patients with Advanced High-Grade Osteosarcoma

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vall D Hebron Institute Of Oncology

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

30 Dec 2024 → ongoing

Decision date (initial)

2024-09-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

The Osteosarcoma Institute · PEPTOMYC SL

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Therapy, Efficacy, Safety

To assess the preliminary anti-tumour activity of OMO-103 monotherapy in patients with high-grade osteosarcoma

Secondary objectives 5

1. To further evaluate the anti-tumour activity of OMO-103 monotherapy in patients with high-grade osteosarcoma.
2. To evaluate the safety and tolerability profile of OMO-103 monotherapy in patients with high-grade osteosarcoma.
3. To characterise the pharmacokinetics (PK) of OMO-103 monotherapy in patients with high-grade osteosarcoma (12-15 years of age).
4. To evaluate quality of life (QoL) in patients with high-grade osteosarcoma
5. To assess the benefit/risk ratio associated with OMO-103 using the Q-TWiST approach (Quality-adjusted Time Without Symptoms of disease recurrence or Toxicity of treatment)

Conditions and MedDRA coding

Advanced High-Grade Osteosarcoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 2. Age ≥12 years at time of informed consent
2. 3. Histologically proven, advanced high-grade osteosarcoma not suitable for local treatments with curative intent
3. 4. Confirmed disease progression by radiological report to at least one line of standard chemotherapy containing cisplatin and anthracycline.
4. 5. Measurable disease as per RECIST v1.1 criteria and documented by CT/MRI (Appendix 1 – RECIST Response Criteria).
5. 6. Provision of a newly obtained tumour biopsy (either from the primary tumour or from metastases) during screening and on-treatment from all patients >16 years of age.
6. 7. Documented progression on or following the last line of therapy
7. 12. If not postmenopausal or surgically sterile, female patients and female sexual partners of male patients must be willing to use at least one highly effective method of birth control (hormonal contraception, IUD, abstinence, condom) for at least a menstrual cycle before and for 3 months after last study drug administration.

Exclusion criteria 9

1. 1. Treatment with systemic anti-cancer therapy within three weeks prior to study drug administration for chemotherapy and 5 half-lives for targeted therapies.
2. 2. Radiation therapy within four weeks prior to study entry. Localised palliative radiotherapy to non-target lesions is allowed.
3. 3. Low-grade osteosarcoma, parosteal, or periosteal osteosarcoma.
4. 4. Prior history of other malignancies other than osteosarcoma (except for basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix) unless the patient has been free of the disease for at least 2 years.
5. 5. Non-malignant systemic disease including cerebrovascular accident, unstable angina pectoris, unstable atrial fibrillation, unstable cardiac arrhythmia, myocardial infarction in the last six months, New York Heart Association (NYHA) Class III or IV heart failure
6. 6. Patients with active uncontrolled infection or known to be serologically positive for human immunodeficiency virus (HIV), hepatitis B (except after vaccination) or hepatitis C infection. Investigators may test as per their discretion.
7. 7. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the patient inappropriate for entry into this study.
8. 8. Patients with symptomatic or unstable central nervous system primary tumour or metastases and/or sarcomatous meningitis
9. Patients with allergies or hypersensitivity reactions to the active substance or to any of its excipients

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Progression-free survival rate at 16 weeks (16-week PFS) according to RECIST V1.1 by Investigator assessment.

Secondary endpoints 5

1. • Objective Response Rate (ORR) • Disease Control Rate (DCR) • Time to Progression (TTP) • Time to Response (TTR) • Duration of Response (DOR) • Overall survival (OS)
2. • Incidence and severity of adverse events (AEs), graded by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.• Safety will be assessed through routine monitoring of adverse events, evaluation of laboratory parameters, vital signs, physical examination and ECGs.
3. PK parameters of OMO-103: AUC; Cmax; tmax; t1/2.
4. • Health-related quality of life (HRQoL) measured by the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire – Core 30 items (EORTC-C30) for adult patients, and by the Pediatric Quality of Life Inventory (PedsQL) for patients between 12 and 17.
5. • Q-TWiST approach [time experiencing toxicity (grade 3/4 AEs) before progression, time without toxicity or symptoms of progression, and time after progression]

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vall D Hebron Institute Of Oncology

Sponsor organisation

Vall D Hebron Institute Of Oncology

Address

Calle Natzaret 115

City

Barcelona

Postcode

08035

Country

Spain

Scientific contact point

Organisation

Vall D Hebron Institute Of Oncology

Contact name

Chief Medical Officer

Public contact point

Organisation

Vall D Hebron Institute Of Oncology

Contact name

Chief Medical Officer

Third parties 4

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 1 · Art. 38 CTR

Results and documents

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

Documents 12 files

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

Application history

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