Phase 2, Open-Label, Multi-Dose Study of Panhematin in Patients With MDS

• Status: Terminated
• Trial end date: 2009-01-01
• Target enrollment: 0
• Participant gender: All

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)

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rush University Medical Center
• Collaborator: H. Lundbeck A/S

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.