| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Patients with Myeloproliferative Neoplasms (MPNs) |
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| E.1.1.1 | Medical condition in easily understood language |
| MPN is a collective term for a number of blood cancers (diseases of the bone marrow) characterized by the overproduction of platelets. Patients have a high-risk of blood clots and bleeding. |
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| E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 20.0 |
| E.1.2 | Level | PT |
| E.1.2 | Classification code | 10077465 |
| E.1.2 | Term | Myeloproliferative neoplasm |
| E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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| E.1.3 | Condition being studied is a rare disease | Yes |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
The objectives of this extension study are to:
• Continue to assess the safety, tolerability and efficacy of bomedemstat in patients who received bomedemstat in a prior study
• Measure the extent and durability of bomedemstat treatment effects on exploratory endpoints including any impact of bomedemstat on the natural history of MF and ET
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| E.2.2 | Secondary objectives of the trial |
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| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
Patients must meet all of criteria to be eligible for study enrollment:
1. Completed at least one Treatment Period (TP) in a prior bomedemstat MPN protocol (such as, but not limited to, IMG-7289-CTP-102 or IMG-7289-CTP-201).
2. In the estimation of the Investigator, the risk-benefit favors continued dosing with bomedemstat.
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| E.4 | Principal exclusion criteria |
Patients will be excluded from the study if they meet any of the following criteria:
1. Ongoing participation in another investigational study (except observational studies).
2. A history of non-compliance in a prior bomedemstat study (excluding dose suspensions that were medically warranted).
3. Current use of a prohibited medication (e.g., romiplostim).
4. Medical, psychiatric, cognitive, or other conditions that, in the Investigator’s opinion, compromise the patient’s safety, ability to give informed consent, or comply with the trial protocol.
5. Females who are pregnant or breastfeeding or plan to become pregnant or breastfeed during the study.
6. Women of childbearing potential (WOCBP) and fertile men (see Section 6.1) unwilling to agree to use an approved method of contraception from time of enrollment until 14 days* after last bomedemstat dose. Methods of contraception include: estrogen and progestogen combined hormonal contraception which inhibits ovulation; progestogen-only hormonal contraception associated with inhibition of ovulation; intrauterine device (IUD); bilateral tubal occlusion; vasectomized partner in a monogamous sexual relationship (vasectomy or tubal ligation at least six months prior to dosing); and, complete sexual abstinence (defined as refraining from heterosexual intercourse). Patients practicing abstinence must agree to use an approved method of contraception if they become sexually active during the study.
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| E.5 End points |
| E.5.1 | Primary end point(s) |
The safety and tolerability of bomedemstat will be assessed by the analysis of adverse events (AEs), as well as changes in physical examinations, vital signs and laboratory values as detailed below.
•Monitoring of Adverse Events (AEs), serious adverse events (SAEs), and AEs. AEs will be assessed from time of entry into study until EoS or ET in terms of onset, duration, seriousness, severity and causality, using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.
•Changes in physical examinations, vital signs and laboratory values will also be assessed until EoS or ET. Information on the timing of these assessments is presented in the schedule of assessments. The following laboratory tests will be conducted:
oComplete blood counts (CBC) and differential
oCoagulation
oChemistry
oUrinalysis
Additionally, in MF patients:
•Spleen volume reduction will be assessed based on spleen volume measured by MRI (or CT where applicable) approximately every 48 weeks.
Additionally, in ET patients:
•Reduction of platelet counts will be evaluated and assessed based on local laboratory measurements of platelet counts collected serially throughout the study.
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| E.5.1.1 | Timepoint(s) of evaluation of this end point |
AEs will be assessed from time of entry into study until EoS or ET in terms of onset, duration, seriousness, severity and causality, using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.
•Changes in physical examinations, vital signs and laboratory values will also be assessed until EoS or ET.
Additionally, in MF patients:
•Spleen volume reduction will be assessed based on spleen volume measured by MRI (or CT where applicable) approximately every 48 weeks.
Additionally, in ET patients:
•Reduction of platelet counts will be evaluated and assessed based on local laboratory measurements of platelet counts collected serially throughout the study.
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| E.5.2 | Secondary end point(s) |
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| E.5.2.1 | Timepoint(s) of evaluation of this end point |
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| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | No |
| E.6.2 | Prophylaxis | No |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | Yes |
| E.6.5 | Efficacy | Yes |
| E.6.6 | Pharmacokinetic | No |
| E.6.7 | Pharmacodynamic | No |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | No |
| E.6.10 | Pharmacogenetic | No |
| E.6.11 | Pharmacogenomic | No |
| E.6.12 | Pharmacoeconomic | No |
| E.6.13 | Others | No |
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | No |
| E.7.1.1 | First administration to humans | No |
| E.7.1.2 | Bioequivalence study | No |
| E.7.1.3 | Other | No |
| E.7.1.3.1 | Other trial type description | |
| E.7.2 | Therapeutic exploratory (Phase II) | Yes |
| E.7.3 | Therapeutic confirmatory (Phase III) | No |
| E.7.4 | Therapeutic use (Phase IV) | No |
| E.8 Design of the trial |
| E.8.1 | Controlled | No |
| E.8.1.1 | Randomised | No |
| E.8.1.2 | Open | Yes |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | No |
| E.8.1.5 | Parallel group | No |
| E.8.1.6 | Cross over | No |
| E.8.1.7 | Other | No |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | No |
| E.8.2.2 | Placebo | No |
| E.8.2.3 | Other | No |
| E.8.2.4 | Number of treatment arms in the trial | 1 |
| E.8.3 |
The trial involves single site in the Member State concerned
| No |
| E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
| E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 7 |
| E.8.6 Trial involving sites outside the EEA |
| E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
| E.8.6.2 | Trial being conducted completely outside of the EEA | No |
| E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
| Australia |
| Hong Kong |
| New Zealand |
| United States |
| Germany |
| Italy |
| United Kingdom |
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| E.8.7 | Trial has a data monitoring committee | Yes |
| E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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| The End-of-Trial date is considered to be the date of Database Lock. The justification is that the trial only ends when all queries are answered and the database is cleaned. Until that point, sites and/or patients may be called upon for follow up information or clarification of data. |
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| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 3 |
| E.8.9.1 | In the Member State concerned months | |
| E.8.9.1 | In the Member State concerned days | |
| E.8.9.2 | In all countries concerned by the trial years | 3 |
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