Trial · NCT02231879
Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
NCT02231879, Phase 2, Phase 3, Completed, National Institute of Allergy and Infectious Diseases (NIAID); results first posted 2023-04-07
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome. Trials, record NCT02231879. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02231879
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
- NCT number
- NCT02231879
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 20 participants, actual
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- Conditions
- Myelokathexis, Infections, Neutropenia, Warts, Hypogammaglobulinemia
- Interventions
- Plerixafor, G-CSF
- First posted
- 2014-09-04
- Last updated
- 2026-06-26
- Results first posted
- 2023-04-07
- Limitations and caveats
1. Highly variable disease phenotypic expression made crossover design optimal but a placebo arm was not included due to infection risk of severe neutropenia~2. Prophylactic supplemental immunoglobulin and antibiotics were allowed~3. Tolerance of G-CSF was insured prior to randomization but plerixafor was never used before randomization in 17/19 patients,~4. Non-inferiority analysis of primary endpoint was not planned because there were no defined equivalence margins
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Severity of Infection | Scores from 12 months treatment on each study drug | The primary outcome is a Total Infection Severity Score (TISS). Each infection during the 12 month period of treatment on each drug was scored for severity on a scale of 1-10 with higher scores representing more severe infections and then these scores were summed (weighted based on time at risk) to create a total infection severity score (TISS) in each period for each participant (range 0 to infinity). Drug failures during a period were pre-defined to count as more extreme than any TISS score. The primary analysis outcome is based on the Difference in Total Infection Severity Score (TISS) in Period 1 minus TISS in Period 2 analysis, with drug failures in only one period assigned the highest absolute value for the difference, with the sign defined according to the treatment order (e.g., negative for period 2 drug failures only). Higher values of the difference represent a worse outcome in the first period than in the second period. Thus, each participant's infection severity |
| Primary | Difference in Total Infection Severity Score | Scores from 12 months treatment on each study drug | The primary outcome is a difference: the Total Infection Severity Score (TISS) in Period 1 minus the TISS in Period 2. Higher values represent a worse outcome in the first period than in the second period. Each infection during the 12 month period of treatment on each drug was scored for severity on a scale of 1-10 with higher scores representing more severe infections and then these individual infection scores were summed (weighted by time at risk) to create a total infection severity score (TISS) in each period. Because there is no pre-determined number of infections, the range is -infinity to +infinity. If a participant has a drug failure in only one period, their difference rank is either the highest rank (if the failure is in Period 1) or the lowest rank (if the failure is in Period 2), failure on both periods gives a difference of 0. 1 subject failed on both treatments, and 3 subjects failed only on plerixafor. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Severity of Infection | Scores from 12 months treatment on each study drug | There are 19 participants that started both periods, 1 participant had drug failure during both periods, 3 participants had drug failure only during the period on Plerixafor. The outcome measure data table only summarizes the periods that were not drug failures, but the primary analysis includes the treatment failure periods by giving them the most extreme score (see Statistical Analysis section). | score on a scale (with no maximum) | MEDIAN |
| PRIMARY | Difference in Total Infection Severity Score | Scores from 12 months treatment on each study drug | The two populations are the two randomization arms in the crossover trial. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Severity of Infection | G-CSF (Both Periods Combined) | 12.5 | Full Range 0 to 36 | score on a scale (with no maximum) | |
| Severity of Infection | Plerixafor (Both Periods Combined) | 9 | Full Range 0 to 24 | score on a scale (with no maximum) | |
| Difference in Total Infection Severity Score | Plerixafor in Period 1 and G-CSF in Period 2 | 10.6 | Full Range 1 to 19 | score on a scale | |
| Difference in Total Infection Severity Score | G-CSF in Period 1 and Plerixafor in Period 2 | 9.33 | Full Range 1 to 19 | score on a scale |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Plerixafor First Then Filgrastim | 10 | |
| Overall Study | STARTED | Filgrastim First Then Plerixafor | 9 | |
| Overall Study | COMPLETED | Plerixafor First Then Filgrastim | 8 | |
| Overall Study | COMPLETED | Filgrastim First Then Plerixafor | 7 | |
| Overall Study | NOT COMPLETED | Plerixafor First Then Filgrastim | 2 | |
| Overall Study | NOT COMPLETED | Filgrastim First Then Plerixafor | 2 |
Source ClinicalTrials.gov
Adverse events124
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Iron Deficiency Anemia | Blood and lymphatic system disorders | G-CSF | 1 | 19 | 3 |
| other | Iron Deficiency Anemia | Blood and lymphatic system disorders | Plerixafor | 1 | 19 | 2 |
| other | Splenomegaly | Blood and lymphatic system disorders | G-CSF | 2 | 19 | 2 |
| other | Splenomegaly | Blood and lymphatic system disorders | Plerixafor | 0 | 19 | 0 |
| other | Anemia | Blood and lymphatic system disorders | G-CSF | 1 | 19 | 1 |
| other | Anemia | Blood and lymphatic system disorders | Plerixafor | 1 | 19 | 1 |
| other | Tinnitus | Ear and labyrinth disorders | G-CSF | 2 | 19 | 2 |
| other | Tinnitus | Ear and labyrinth disorders | Plerixafor | 1 | 19 | 1 |
| other | Nausea | Gastrointestinal disorders | G-CSF | 6 | 19 | 13 |
| other | Nausea | Gastrointestinal disorders | Plerixafor | 1 | 19 | 1 |
1 to 10 of 124Source ClinicalTrials.gov
See all 124 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | G-CSF | 0 | 19 |
| Total, all-cause mortality | Plerixafor | 0 | 19 |
| Total, other adverse events | G-CSF | 19 | 19 |
| Total, other adverse events | Plerixafor | 19 | 19 |
| Total, serious adverse events | G-CSF | 6 | 19 |
| Total, serious adverse events | Plerixafor | 2 | 19 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| G-CSF vs Plerixafor: deaths from any cause | Trial comparisons |
| G-CSF vs Plerixafor: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02231879
GET https://conductscience.org/api/trials/v1/NCT02231879/events?page=2