Trial Outcomes & Findings for AMD 3100 for Treatment of Myelokathexis (NCT NCT01058993)
NCT ID: NCT01058993
Last Updated: 2012-06-15
Results Overview
Effectiveness of drug based on increases of blood neutrophil counts to greater than 2.0 x 10\^9 per liter
COMPLETED
PHASE1
6 participants
up to 14 days, depending on when subject reached peak response, i.e., the highest count after the stimulus (plerixafor)
2012-06-15
Participant Flow
Subjects were contacted from the Severe Chronic Neutropenia International Registry office by Audrey Anna Bolyard, Manager of the Registry.
Prior to assignment, we performed physician, EKG (electrocardiogram) and screening labs, including CBC (complete blood count), Differential and smear, comprehensive metabolic panel, urinalysis, and pregnancy testing on females subjects
Participant milestones
| Measure |
Single Arm Study, 5 Escalating Doses of AMD3100 (Plerixafor)
Single arm study to examine the hematological effects, pharmacokinetics and safety of plerixafor in patients with myelokathexis attributable to mutations of CXCR4, utilizing serial, escalating doses of plerixafor administered on days 1, 3, 5, 8, and 10. Five intrapatient escalating dose levels, 20 micrograms per kilogram (mcg/kg), 40 micrograms/kilogram(mcg/kg), 80 micrograms/kilogram(mcg/kg), and 240 micrograms/kilogram (mcg/kg)will be examined. The subjects will be patients at the University of Washington General Clinical Research Center for up to 10 days; the study requires subject be available for up to 14 days. Patients will be monitored for hematological effects of plerixafor and observed for adverse effects. If a normal blood neutrophil count is achieved and maintained for at least 24 hours prior to the highest dose, we will stop at that level.
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|---|---|
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Overall Study
STARTED
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6
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Overall Study
COMPLETED
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6
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
AMD 3100 for Treatment of Myelokathexis
Baseline characteristics by cohort
| Measure |
Single Arm Study, 5 Escalating Doses of AMD3100 (Plerixafor)
n=6 Participants
Single arm study to examine the hematological effects, pharmacokinetics and safety of plerixafor in patients with myelokathexis attributable to mutations of CXCR4, utilizing serial, escalating doses of plerixafor administered on days 1, 3, 5, 8, and 10. Five intrapatient escalating dose levels, 20 micrograms per kilogram (mcg/kg), 40 micrograms/kilogram(mcg/kg), 80 micrograms/kilogram(mcg/kg), and 240 micrograms/kilogram (mcg/kg)will be examined. The subjects will be patients at the University of Washington General Clinical Research Center for up to 10 days; the study requires subject be available for up to 14 days. Patients will be monitored for hematological effects of plerixafor and observed for adverse effects. If a normal blood neutrophil count is achieved and maintained for at least 24 hours prior to the highest dose, we will stop at that level.
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|---|---|
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Age, Categorical
<=18 years
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0 Participants
n=99 Participants
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Age, Categorical
Between 18 and 65 years
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6 Participants
n=99 Participants
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Age, Categorical
>=65 years
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0 Participants
n=99 Participants
|
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Age Continuous
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42.7 years
STANDARD_DEVIATION 17.0 • n=99 Participants
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Sex: Female, Male
Female
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4 Participants
n=99 Participants
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Sex: Female, Male
Male
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2 Participants
n=99 Participants
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Region of Enrollment
United States
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6 participants
n=99 Participants
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Blood neutrophil count (in myelokathexis)
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0.7 10^9 per Liter
n=99 Participants
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blood neutrophil levels
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0.7 10^9 per Liter
STANDARD_DEVIATION 0.6 • n=99 Participants
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PRIMARY outcome
Timeframe: up to 14 days, depending on when subject reached peak response, i.e., the highest count after the stimulus (plerixafor)Population: The number of participants (6) was determined by the fact that we were studying a rare form of neutropenia, WHIMS syndrome, and we therefore recruited subjects who have WHIMS who live on the West coast. Analysis was per protocol.
Effectiveness of drug based on increases of blood neutrophil counts to greater than 2.0 x 10\^9 per liter
Outcome measures
| Measure |
SINGLE Arm Study, 5 Escalating Doses of AMD3100 (Plerixafor)
n=6 Participants
Single arm study to examine the hematological effects, pharmacokinetics and safety of plerixafor in patients with myelokathexis attributable to mutations of CXCR4, utilizing serial, escalating doses of plerixafor administered on days 1, 3, 5, 8, and 10. Five intrapatient escalating dose levels, 20 micrograms per kilogram (mcg/kg), 40 micrograms/kilogram(mcg/kg), 80 micrograms/kilogram(mcg/kg), and 240 micrograms/kilogram (mcg/kg)will be examined. The subjects will be patients at the University of Washington General Clinical Research Center for up to 10 days; the study requires subject be available for up to 14 days. Patients will be monitored for hematological effects of plerixafor and observed for adverse effects. If a normal blood neutrophil count is achieved and maintained for at least 24 hours prior to the highest dose, we will stop at that level.
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Blood Neutrophil Counts.
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4.48 10^9 per Liter
Standard Deviation 1.91
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Adverse Events
Single Arm Study, 5 Escalating Doses of AMD3100 (Plerixafor)
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place