Myelofibrosis Clinical Trials

⁃ Pre-Transplant Inclusion Criteria (Step 1)

• Male or female subject aged ≥ 18 years.

• Diagnosis of primary or secondary myelofibrosis.

• Eligible to undergo a myeloablative or reduced intensity conditioning regimen (MAC or RIC)

• Eligible to undergo a standard of care bone marrow biopsy with aspirate as part of his or her routine pre-transplant work-up.

• Peripheral blood stem cell (PBSC) graft

• 10/10 HLA matched related or matched unrelated donor

• ECOG performance status ≤ 2.

• For female subjects: Negative pregnancy test or evidence of post-menopausal status. The post-menopausal status will be defined as having been amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:

‣ Women \< 50 years of age:

• Amenorrheic for ≥ 12 months following cessation of exogenous hormonal treatments; and

∙ Luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution; or

∙ Underwent surgical sterilization (bilateral oophorectomy or hysterectomy).

⁃ Women ≥ 50 years of age:

• Amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments; or

∙ Had radiation-induced menopause with last menses \>1 year ago; or

∙ Had chemotherapy-induced menopause with last menses \>1 year ago; or

∙ Underwent surgical sterilization (bilateral oophorectomy, bilateral salpingectomy, or hysterectomy).

• Female subjects of childbearing potential and male subjects with a sexual partner of childbearing potential must agree to use a highly effective method of contraception as described in Section 5.4.1.

⁃ Treatment Inclusion Criteria (Step 2)

• Male or female subject aged ≥ 18 years.

• Diagnosis of primary or secondary myelofibrosis.

• Have undergone a myeloablative or reduced-intensity conditioning regimen (MAC or RIC) and be 50-80 days from Day 0 of transplant at initiation of study therapy.

• Peripheral blood stem cell (PBSC) graft

• 10/10 HLA matched related or matched unrelated donor

• ECOG Performance Status ≤ 2.

• Adequate organ function as defined as:

‣ Hepatic:

• Total Bilirubin ≤ 1.5x institutional upper limit of normal (ULN)

∙ AST(SGOT)/ALT(SGPT) ≤ 3 × institutional ULN

⁃ Renal:

• Estimated creatinine clearance ≥ 30 mL/min by Cockcroft-Gault formula:

∙ TSH and T4 within normal limits or adequately controlled thyroid function.

• For female subjects: Negative pregnancy test or evidence of post-menopausal status. The post-menopausal status will be defined as having been amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:

‣ Women \< 50 years of age:

• Amenorrheic for ≥ 12 months following cessation of exogenous hormonal treatments; and

∙ Luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution; or

∙ Underwent surgical sterilization (bilateral oophorectomy or hysterectomy).

⁃ Women ≥ 50 years of age:

• Amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments; or

∙ Had radiation-induced menopause with last menses \>1 year ago; or

∙ Had chemotherapy-induced menopause with last menses \>1 year ago; or

∙ Underwent surgical sterilization (bilateral oophorectomy, bilateral salpingectomy, or hysterectomy).

• Female subjects of childbearing potential and male subjects with a sexual partner of childbearing potential must agree to use a highly effective method of contraception as described in Section 5.4.1.

• Male subjects must agree to use a condom during intercourse for the duration of study therapy as described in Section 5.4.1.

• Recovery to baseline or ≤ Grade 1 CTCAE v5.0 from toxicities related to any prior cancer therapy, unless considered clinically not significant by the treating investigator.

• Able to provide informed consent and willing to sign an approved consent form that conforms to federal and institutional guidelines.