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NCT01842646 · OUTCOME

Overall International Working Group (IWG) 2006 Response Rate

Phase II Hedgehog Inhibitor for Myelodysplastic Syndrome (MDS) · Source last updated 2021-06-14

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
COUNT_OF_PARTICIPANTS
Unit
Participants
Interval / dispersion
Not reported
Time frame
Up to 2 years, 4 months

What was measured

Response recorded from the start of the treatment until disease progression/recurrence. All responses must last for at least 8 weeks. Complete Remission (CR): Bone marrow: ≤ 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia will be noted, Peripheral blood: Hemoglobin ≥ 11 g/dL, Platelets ≥ 100 x 10\^9/L, Neutrophils ≥ 1.0 x 10\^9/L, Blasts 0% ; Partial Remission (PR): All CR criteria if abnormal before treatment, except: Bone marrow blasts decreased by ≥ 50% over pretreatment but still \> 5%, Cellularity and morphology not relevant; Marrow CR or Hematological Improvement (HI): Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment Peripheral blood: if HI responses, they will be noted in addition to marrow CR. Further investigation of PF-04449913 would not be warranted if it produced an overall response rate (CR + PR + marrow CR+HI) of 10% or less (p0), and would be warranted if it produced an overall response rate of 30% or more (p1).

Analysis population: All participants

Groups in this outcome

Experimental: PF-04449913 Treatment

Treatment to be administered on an outpatient basis. All participants to be treated with an oral dose PF-04449913 at 100 mg daily in 4-week cycles for a total of 4 cycles.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Experimental: PF-04449913 Treatment35

Reported measurements

Source class 1

Complete Remission (CR)

Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Experimental: PF-04449913 Treatment0Not reportedNot reportedNot reportedNot reported

Hematological Improvement (HI)

Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Experimental: PF-04449913 Treatment2Not reportedNot reportedNot reportedNot reported

Stable Disease (SD)

Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Experimental: PF-04449913 Treatment19Not reportedNot reportedNot reportedNot reported

Progressive Disease (PD)

Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Experimental: PF-04449913 Treatment14Not reportedNot reportedNot reportedNot reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        0
      title
      Complete Remission (CR)
    2. measurements
      1. group Id
        OG000
        value
        2
      title
      Hematological Improvement (HI)
    3. measurements
      1. group Id
        OG000
        value
        19
      title
      Stable Disease (SD)
    4. measurements
      1. group Id
        OG000
        value
        14
      title
      Progressive Disease (PD)
denoms
  1. counts
    1. group Id
      OG000
      value
      35
    units
    Participants
description
Response recorded from the start of the treatment until disease progression/recurrence. All responses must last for at least 8 weeks. Complete Remission (CR): Bone marrow: ≤ 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia will be noted, Peripheral blood: Hemoglobin ≥ 11 g/dL, Platelets ≥ 100 x 10\^9/L, Neutrophils ≥ 1.0 x 10\^9/L, Blasts 0% ; Partial Remission (PR): All CR criteria if abnormal before treatment, except: Bone marrow blasts decreased by ≥ 50% over pretreatment but still \> 5%, Cellularity and morphology not relevant; Marrow CR or Hematological Improvement (HI): Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment Peripheral blood: if HI responses, they will be noted in addition to marrow CR. Further investigation of PF-04449913 would not be warranted if it produced an overall response rate (CR + PR + marrow CR+HI) of 10% or less (p0), and would be warranted if it produced an overall response rate of 30% or more (p1).
groups
  1. description
    Treatment to be administered on an outpatient basis. All participants to be treated with an oral dose PF-04449913 at 100 mg daily in 4-week cycles for a total of 4 cycles.
    id
    OG000
    title
    Experimental: PF-04449913 Treatment
param Type
COUNT_OF_PARTICIPANTS
population Description
All participants
reporting Status
POSTED
time Frame
Up to 2 years, 4 months
title
Overall International Working Group (IWG) 2006 Response Rate
type
PRIMARY
unit Of Measure
Participants

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice