Phase 2, Open-Label, Multi-Dose Study of Panhematin in Patients With MDS
Condition: Myelodysplastic Syndrome
MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome
Intervention: Panhematin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 6.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: myelodysplastic; MDS; Panhematin; Hemin

Summary:

      This is a Phase II, open-label clinical trial examining the role of Panhematin® in patients
      with MDS. The objective of this study is to evaluate the safety and efficacy of Panhematin®
      (hematin for injection) in the treatment of adult patients (≥ 18 years of age) with low-risk
      MDS.

      The study will be conducted on an outpatient basis and will consist of the following:

        -  A Screening Period (within 28 days of the Day 1)

        -  Screening bone marrow aspiration and biopsy up to 60 days prior to receiving study
           medication

        -  An 8-week Treatment Period (Days 1 through 4 of Week 1, and weekly visits during Weeks 2
           through 8); partial and complete responders in any of the three cell lines may continue
           treatment for an additional 4 weeks

        -  A 6-month Post treatment Follow-up Period (monthly clinic visits during Weeks 12 40)
    

Eligibility criteria:

        Inclusion Criteria:

        A patient will be eligible for study participation if all of the following criteria are
        met:

          1. The patient must sign and date the IRB/IEC approved Informed Consent Form/HIPAA
             Authorization prior to study participation.

          2. Patient is at least 18 years of age.

          3. If female:

               1. Patient, either male or female, is either not of childbearing potential, defined
                  as postmenopausal for at least 1 year or surgically sterile (bilateral tubal
                  ligation, bilateral oophorectomy or hysterectomy), or if of childbearing
                  potential, must comply with an effective method of birth control acceptable to
                  the Investigator during the study (oral contraceptives, Depo-Provera,
                  intra-uterine device), for at least 1 month prior to enrollment and for 1 month
                  following the completion of the study.

               2. Patient is not breastfeeding.

               3. Patient of childbearing potential must have a negative urine or serum pregnancy
                  test during the screening period.

          4. Patient has a diagnosis of low- or intermediate-1 risk MDS, as determined by the
             International Prognostic Scoring (IPSS) (score of 0-1).

          5. Patient must be transfusion dependent (i.e., received ≥ 2 units over an 8-week period
             prior to registration) or have a hemoglobin value ≤ 10 g/dL on the screening
             laboratories.

          6. Patients must have ≤ 10% blasts in the bone marrow and peripheral blood.

          7. Patient must have a platelet counts > 50,000/microliters and absolute neutrophil
             counts (ANC) >500/microliters.

          8. Patient must have adequate hepatic and renal functions, defined as serum bilirubin,
             serum glutamic-oxaloacetic transaminase (SGOT), and serum glutamate pyruvate
             transaminase (SGPT) ≤ 2 times the upper limit of normal (ULN), and creatinine ≤ 1.5
             times the ULN.

          9. Patient must have an ECOG score of ≤ 2.

         10. The patient has a negative human immunodeficiency virus antibody (HIV) test result.

        Exclusion Criteria:

        A patient will be ineligible for study participation if any of the following criteria are
        met:

          1. The patient has a history of an allergic reaction or significant sensitivity to
             Panhematin®.

          2. The patient has taken or used any investigational drug or device in the 30 days prior
             to screening.

          3. The patient has chronic myelomonocytic leukemia (CMML).

          4. The patient has a history of deep vein thrombosis or known hypercoagulable state.

          5. The patient has a history of a pre-existing medical condition that, in the opinion of
             the investigator, will interfere with the participation in the study.

          6. The patient has poor peripheral venous access, if central venous access is not
             available.

          7. The patient has an uncontrolled active infection.

          8. The patient has positive test results for hepatitis B surface antigen, and hepatitis C
             virus antibody.

          9. The patient has any other condition or prior therapy that, in the opinion of the
             Investigator, would make the patient unsuitable for the study.