Trial Outcomes & Findings for Phase 2, Open-Label, Multi-Dose Study of Panhematin in Patients With MDS (NCT NCT00467610)

Results Overview

Number of patients with no adverse events.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

participants were followed during therapy with panhematin, and up to six months post completion of therapy, average of 8 months.

Results posted on

2014-10-24

Participant Flow

Between 5/2007 and 10/2008, 6 patients were enrolled on trial and were treated at Rush university medical center.

Participant milestones

Participant milestones
Measure	Group 1 Panhematin treatment arm
Overall Study STARTED	6
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Group 1 Panhematin treatment arm
Overall Study Withdrawal by Subject	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group 1 n=6 Participants Panhematin treatment arm
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants
Age, Continuous	73 years FULL_RANGE 0 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: participants were followed during therapy with panhematin, and up to six months post completion of therapy, average of 8 months.

Number of patients with no adverse events.

Outcome measures

Outcome measures
Measure	Group 1 n=6 Participants Panhematin treatment arm
Safety and Tolerability of Panhematin®.	6 participants

PRIMARY outcome

Timeframe: After 8 weeks of therapy with panhematin

Complete response(CR): \<5% blasts in the bone marrow,with normal maturation of all cell lines, Hemoglobin \>11 g/dL, neutrophils\>1500/mm3 platelets\>100,000/mm3. Partial response (PR): \>50% decrease in blasts, or less advanced IPSS than pretreatment value, same hematological parameters as in CR. Stable disease (SD): No evidence of disease progression in bone marrow, stable peripheral blood counts failure: Increase in bone marrow blast percentage, progression to more advanced IPSS than pretreatment and worsening of cytopenias. (Cheson, 2000)

Outcome measures

Outcome measures
Measure	Group 1 n=5 Participants Panhematin treatment arm
Response Rate ( CR+PR) at Week 8, Based on the IWG Criteria for Response Assessment ( 2000 Version)	0 Participants

SECONDARY outcome

Timeframe: 4 weeks after initiation of treatment with Panhematin

Hematological improvement (HI) Major: HI-Erythroid:\>2 g/dL rise in hemoglobin, or transfusion independence HI-Neutrophil: Absolute increase of \>500/mm3, or \>100% increase HI-Platelet: Absolute increase of \>30,000, or transfusion independence Minor: HI-Erythroid:1 to 2 g/dL increase in hemoglobin or 50% decrease in transfusion dependence. HI-P: For patients with pretreatment platelet count \< 100,000/mm3, ≥ 50% increase with a net increase \> 10,000/mm3 but \< 30,000/mm3. HI-N: For patients with pretreatment ANC \< 1500/mm3, ≥ 100% increase, but \< 500/mm3 increase.

Outcome measures

Outcome measures
Measure	Group 1 n=5 Participants Panhematin treatment arm
Number of Patients Demonstrating Hematological Improvement to Panhematin® at Week 4.	0 participants

SECONDARY outcome

Timeframe: At 8 weeks from start of therapy

Outcome measures

Outcome measures
Measure	Group 1 n=5 Participants Panhematin treatment arm
Hematological Improvement Rate at Week 8 as Defined by the IWG 2000 Criteria for Response Assessment, 2000 Version	1 participants

Adverse Events

Group 1

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Jamile shammo

Rush university medical center

Phone: 312-942-5157

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place