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New treatment option for Myelofibrosis (PMF)
Official title Reparixin in Patients With Myelofibrosis Myeloproliferative Neoplasms Research Consortium (MPN-RC 120)
ClinicalTrials.gov ID: NCT05835466
What this study is testing
What is reparixin?
reparixin is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for myelofibrosis (pmf).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is an open label, phase II study to assess the efficacy, safety, and tolerability of Reparixin in patients with DIPSS intermediate-2, or high-risk primary myelofibrosis (PMF), post essential thrombocythemia/polycythemia vera related MF (Post ET/PV MF) after prior treatment, and those who are ineligible or refuse treatment, with a Janus kinase inhibitor (JAKi). 26 patients will be enrolled.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Be ≥ 18 years of age at time of signing the informed consent form (ICF)
- Willing to voluntarily sign the ICF
- Have a pathologically confirmed diagnosis of PMF, post-ET-MF, or post-PV-MF as per the World Health Organization (WHO) diagnostic criteria with...
- Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Willing to undergo a bone marrow biopsy at screening o A bone marrow biopsy obtained within 90 days of screening without intervening treatments and...
You likely can't join if
- History of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within the last 6...
- Other invasive malignancies within the last 3 years, except non-melanoma skin cancer and localized cured prostate and cervical cancer
- Moderate or severe cardiovascular disease meeting one or both of the below criteria:
- Presence of cardiac disease, including a myocardial infarction within 6 months prior to study entry, unstable angina pectoris, New York Heart...
- Documented major electrocardiogram (ECG) abnormalities (not responding to medical treatments)
- Presence of active serious infection
See the full eligibility criteria
- Be ≥ 18 years of age at time of signing the informed consent form (ICF)
- Willing to voluntarily sign the ICF
- Have a pathologically confirmed diagnosis of PMF, post-ET-MF, or post-PV-MF as per the World Health Organization (WHO) diagnostic criteria with intermediate-2 or higher risk disease by DIPSS
- Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Willing to undergo a bone marrow biopsy at screening o A bone marrow biopsy obtained within 90 days of screening without intervening treatments and approved by the study chair may suffice.
- Be refractory/resistant to or intolerant of/inappropriate for JAKi therapy as defined by at least one of the following:
- Treatment for ≥ 3 months with inadequate how well it works as demonstrated by persistent palpable splenomegaly ≥ 5cm or symptoms related to splenomegaly,
- Treatment for ≥ 28 days complicated by either:
- Development of a red blood cell transfusion requirement (at least 2 units/month for 2 months)
- CTCAE grade ≥ 3 AEs of thrombocytopenia, anemia, hematoma, or hemorrhage while being treated with a JAKi
- Development of non-hematological toxicity that makes patient intolerant of JAKi therapy
- In the Investigator's judgment, are not candidates for available approved JAKi
- Recovery to ≤ Grade 1 or baseline of any toxicities due to prior systemic treatments, excluding alopecia
- At least two weeks must have elapsed between the last dose of any MF-directed drug treatments or other investigational therapies and start of reparixin o Participants may continue hydroxyurea until the day prior to C1D1...
- Have adequate organ function as demonstrated by the following:
- ALT (SGPT) and/or AST (SGOT) ≤ 3x upper limit of normal (ULN), or ≤ 4 x ULN (if upon judgment of the treating physician, it is believed to be due to MF-related EMH);
- Direct bilirubin ≤ 1.5 x ULN; or ≤ 2x ULN (if upon judgment of the treating physician, it is believed to be due to MF-related EMH or documented Gilbert's syndrome);
- Creatinine clearance ≥ 40 mL/min;
- Platelet count ≥ 25 x 109/L;
- Bone marrow and peripheral blood blast count \< 10%;
- ANC ≥ 1000 mm3.
- Life expectancy of at least six months
- Women of childbearing potential (WCBP) and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 120 days following completion of therapy. WCBP must also...
- Ability to adhere to the study visit schedule and all protocol requirements
- History of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within the last 6 months
- Other invasive malignancies within the last 3 years, except non-melanoma skin cancer and localized cured prostate and cervical cancer
- Moderate or severe cardiovascular disease meeting one or both of the below criteria:
- Presence of cardiac disease, including a myocardial infarction within 6 months prior to study entry, unstable angina pectoris, New York Heart Association Class III/IV congestive heart failure, or uncontrolled...
- Documented major electrocardiogram (ECG) abnormalities (not responding to medical treatments)
- Presence of active serious infection
- Any serious, unstable medical or psychiatric condition that would prevent (as judged by the Investigator) the participant from signing the ICF or any condition, including the presence of laboratory abnormalities, which...
- Participants who have undergone a hematopoietic cell transplant (HCT) within 100 days of the first dose of study therapy, participants on immunosuppressive therapy post-HCT at screening, use of calcineurin inhibitors...
- Known history of human immunodeficiency virus (HIV), or known active hepatitis A, B, or C infection
- Impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of reparixin, including any unresolved nausea, vomiting, or diarrhea \> CTCAE grade 1
- Is or has an immediate family member (e.g., spouse, parent/legal guardian, sibling, or child) who is investigational site or sponsor staff directly involved with this trial, unless prospective institutional review board...
- Organ transplant recipients other than bone marrow transplant
- Women who are pregnant or lactating
- History of splenectomy
- Known hypersensitivity to sulfonamides o Hypersensitivity to sulphanilamide antibiotics alone (e.g. sulfamethoxazole) does not qualify for exclusion
- Known hypersensitivity to non-steroidal anti-inflammatory drugs (NSAID), including ibuprofen
The study team makes the final eligibility decision.
Where it's taking place
- Tampa, Florida, United States
- Atlanta, Georgia, United States
- Buffalo, New York, United States
- New York, New York, United States
- Winston-Salem, North Carolina, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Tampa, Florida, United States; Atlanta, Georgia, United States; Buffalo, New York, United States; New York, New York, United States; Winston-Salem, North Carolina, United States; Cleveland, Ohio, United States and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.