This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for PD and Safety of TG-0054 Combined With G-CSF in Multiple Myeloma, Non-Hodgkin Lymphoma and Hodgkin Disease Patients (NCT02104427), a na phase 2 study. The primary outcome is Proportion of Patients From Whom a Total Number of CD34+ Cells ≥5.0 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02104427 |
| Phase | Phase 2 |
| Condition | Multiple Myeloma, Non-Hodgkin Lymphoma, Hodgkin Disease |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | GPCR Therapeutics, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Patients with multiple myeloma, non-Hodgkin lymphoma or Hodgkin disease undergoing hematopoietic stem-cell mobilization. The registry record reports enrollment of 12. Its endpoint description states that there were 9 patients with multiple myeloma and 3 with non-Hodgkin lymphoma or Hodgkin disease; further detail about the analyzed population is not reported in the registry record.
Intervention
TG-0054 combined with G-CSF. The described regimen uses G-CSF at 10 μg/kg/day by subcutaneous injection from Day 1 to Day 8, and TG-0054 at 3.14 mg/kg by 15-minute intravenous infusion from Day 5 to Day 9 as needed to reach the target collection goal.
Comparator
The efficacy study is listed as single-group, with allocation recorded as not applicable and masking as none. The registry record does not report a concurrent randomized comparator. Although safety reporting includes a G-CSF-alone group label, the record explicitly cautions that the arms are not a like-for-like control comparison.
Primary question
What proportion of patients had a cumulative CD34+ cell collection of at least 5.0 x 10^6 cells/kg within the first 4 leukapheresis sessions, during the Day 5 to Day 8 timeframe? The registry record describes this endpoint but does not report the observed proportion or its 95% confidence interval.
3. Trial Design
This was a Phase II, open-label study of TG-0054 combined with G-CSF in patients with multiple myeloma, non-Hodgkin lymphoma or Hodgkin disease. The registry record lists the study as completed, with a start date of 2015-02 and primary completion in 2015-11. Enrollment was 12.
The trial is characterized as single-group, with allocation recorded as not applicable and masking recorded as none. The listed intervention is TG-0054 combined with G-CSF. The regimen description specifies G-CSF at 10 μg/kg/day by subcutaneous injection from Day 1 to Day 8, and TG-0054 at 3.14 mg/kg by 15-minute intravenous infusion from Day 5 to Day 9 as needed to reach the target collection goal.
The registry record describes the endpoint population as 9 patients with multiple myeloma and 3 patients with non-Hodgkin lymphoma or Hodgkin disease. The record does not give further detail about the enrollment flow or the final analysis population. Its primary endpoint is a collection threshold based on cumulative CD34+ cells obtained within the first 4 leukapheresis sessions.
The adverse-event information uses separate labels for G-CSF alone and G-CSF plus TG-0054. The registry record cautions that these are not a like-for-like control comparison and does not describe a randomized allocation between them. The record does not report why these safety categories are presented separately or how they relate to treatment sequence. They should therefore be read as safety-reporting categories, not as evidence of a controlled efficacy comparison.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | TG-0054 Combined With G-CSF |
|---|---|
| STARTED | 12 |
| COMPLETED | 12 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: There were total 9 MM patients and 3 NHL or HD patients . A MM patient was treated with Revlimid, Following 2 leukapheresis sessions, a cumulative total of 0.18 x 10\^6 cells/kg were collected. PI and Medical Monitor didn't feel that further leukapheresis sessions would achieve more cells, so the patient didn't have additional sessions.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | TG-0054 Combined With G-CSF |
|---|---|---|
| Participants analysed | 12 | |
| Age, Continuous | years | 58 (41 to 69) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 8 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 1 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 8 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Weight | kg | 82.4 (54.6 to 114.0) |
| BMI | kg/m^2 | 29.3 (21.9 to 37.1) |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Patients From Whom a Total Number of CD34+ Cells ≥5.0 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions, measured over Day 5 to Day 8.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Proportion of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the proportion of patients from whom a cumulative total of at least 5.0 x 10^6 CD34+ cells/kg was collected within the first 4 leukapheresis sessions. The stated timeframe is Day 5 to Day 8. The record explains that each patient’s endpoint value was calculated by summing the CD34+ cell numbers from up to the first 4 sessions, then determining whether that total met the specified threshold.
This endpoint expresses whether a collection goal was reached within a defined number of collection sessions. A proportion is useful for describing how commonly a threshold was achieved in the studied group; it does not show the distribution of individual cell counts or how far values fell above or below the threshold. The threshold and collection window also make the endpoint specific to the study’s stated mobilization and collection question.
The registry record lists a 95% confidence interval as the endpoint’s dispersion measure, but it does not report the observed proportion or the interval limits. Consequently, the endpoint definition is available, but its estimated result and precision cannot be assessed from the registry record. Secondary outcomes include other CD34+ collection thresholds and pharmacodynamics, but their results are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record gives the study question, treatment regimen, primary endpoint definition and selected safety counts. It does not give the primary endpoint result. In particular, it does not report the proportion reaching at least 5.0 x 10^6 CD34+ cells/kg within the first 4 leukapheresis sessions, nor the limits of the listed 95% confidence interval. Without that estimate, the degree to which the observed group met the endpoint cannot be described from the record.
The endpoint is a threshold-based proportion. Its interpretation would depend on both the observed proportion and its uncertainty. A confidence interval conveys the range of values reasonably compatible with the data under the method used; a wide interval would indicate limited precision, while a narrower interval would indicate greater precision. The numerical interval is not reported in the registry record, so the precision of the primary estimate cannot be evaluated.
The trial’s single-group design is important when interpreting any observed outcome. Without a randomized concurrent comparator, an observed collection proportion would describe outcomes among patients receiving the regimen, not establish what would have happened under another regimen. The registry record specifically cautions that the G-CSF-alone and combination labels in the safety information are not a like-for-like control comparison. No causal efficacy comparison should be inferred from those labels.
Enrollment was 12, and the endpoint description reports 9 patients with multiple myeloma and 3 with non-Hodgkin lymphoma or Hodgkin disease. These details establish the small scale and diagnostic mix described in the record, but do not substitute for the missing endpoint estimate. The record also notes a patient with multiple myeloma treated with Revlimid who had a cumulative collection of 0.18 x 10^6 cells/kg after 2 leukapheresis sessions; the description is truncated, so further information about that patient’s endpoint handling is not reported in the registry record.
The safety counts show 0 serious adverse events among 12 at risk in each displayed category and 0 deaths in each category. Other adverse events affected 2 in the G-CSF-alone category, 6 in the combination category and 7 overall. These figures describe the reported safety counts; the registry record does not provide event terms for serious events or enough context to interpret the categories as comparative risks. The overall count should not be treated as the sum of the category counts.
In short, the record supports a description of the study design and reported safety counts, but not a conclusion about the primary endpoint’s observed performance or comparative effects. Key results and analysis details are not reported in the registry record.
9. Statistical Methods Explained
What does a proportion measure?
In general, a proportion is the number of people meeting a defined condition relative to the people assessed for that condition. Here, the condition is reaching the stated cumulative CD34+ cell threshold within the specified collection sessions. A proportion summarizes how often that threshold was met in the analyzed group; it does not describe each patient’s exact collection total. The registry record does not report the observed proportion, so no result can be interpreted from the endpoint definition alone.
How should the confidence interval be understood?
The registry record specifies a 95% confidence interval for the primary endpoint. In general, a confidence interval expresses uncertainty around an estimate under the statistical method used. It is not a guarantee that the true value lies within the particular interval, nor does it replace the estimate itself. Interpretation requires both the point estimate and interval limits. Neither the numerical estimate nor the interval limits are reported in the registry record.
The record does not identify the procedure used to construct the interval. For a proportion, different interval methods can behave differently, especially with small samples or estimates near the boundaries. Since the method and numerical result are not reported, the interval’s construction and precision cannot be independently assessed.
What does the single-group design allow?
In general, a single-group study can describe outcomes observed under the treatment regimen in its enrolled participants. It does not by itself distinguish treatment effects from changes that might have occurred without the treatment or under a different regimen. A randomized comparator can help address that question by making treatment groups comparable in expectation; this study is listed as single-group, with allocation not applicable and masking none.
The safety information separately lists G-CSF alone and G-CSF plus TG-0054, but the registry record says the arms are not a like-for-like control comparison. Those labels do not establish randomization or comparability. Differences in the counts across those categories should not be treated as estimates of a treatment effect.
What does the endpoint’s collection rule imply?
The endpoint uses a cumulative total from up to the first 4 leukapheresis sessions and asks whether it meets the specified threshold. In general, a threshold endpoint simplifies a continuous measurement into a yes-or-no classification. This can make a target-based question easier to communicate, but it discards information about the size of the collection and about how close patients were to the cutoff. The registry record does not report individual collection values or the number meeting the primary threshold.
The endpoint description says that a patient’s value is based on the sum from up to the first 4 sessions. The record also includes a narrative about a patient whose collection stopped after 2 sessions, but the description is truncated. The endpoint handling for that patient and any broader rules for incomplete collection are not reported in the registry record.
What can be inferred from the planning assumptions?
The record describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between arms and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. It also distinguishes participant-per-arm illustrations from total-event illustrations for time-to-event outcomes. These are planning assumptions, not observed power, and they do not establish the trial’s original design rationale. No resulting sample-size values or observed effect are reported in the registry record, and the study itself is listed as single-group rather than a randomized two-arm efficacy comparison.
In general, sample-size planning asks how much information a future study would need under specified assumptions. Such an illustration depends on the assumed effect, variability, significance level, allocation and analysis method. It is not evidence that the completed study had a particular probability of detecting an effect. The record does not report the protocol’s sample-size justification or a statistical analysis plan.
What remains unknown about the analysis?
The registry record lists 4 outcome measures and 0 analysis entries. It does not report the primary estimate, its confidence interval limits, a statistical test, or methods for handling missing observations. It also does not define analysis populations beyond the limited description of the endpoint population. These omissions prevent evaluation of the analysis choices and of how the reported results, if any, were derived.
In general, interpretation of a clinical endpoint depends on a clear definition of who was analyzed, how missing data were handled, and whether the analysis followed a prespecified plan. These details matter because exclusions or incomplete measurements can change the estimate and its uncertainty. They are not reported in the registry record, so the endpoint definition should not be mistaken for a complete statistical analysis description.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 4 outcome measures, including the primary CD34+ collection threshold, other collection targets and a pharmacodynamic outcome. It records 0 analysis entries. The primary endpoint is defined, but its observed result and 95% confidence interval limits are not reported in the registry record. Results for the secondary outcomes are also not reported in the registry record.
When a study assesses multiple outcomes, multiplicity can arise because several statistical questions are examined. In general, conducting many tests can increase the chance of observing an apparently notable result by chance. A prespecified hierarchy or adjustment can address some multiplicity concerns, but the registry record does not report a multiplicity strategy or statistical analysis plan. It is therefore not possible to determine how outcome testing was planned or interpreted.
The record does not report missing-data counts, missing-data handling, or an imputation method. It also does not describe a complete analysis population or state how participants who did not complete the collection process were handled. The primary endpoint description mentions a patient whose collection stopped after 2 sessions, but the accompanying text is truncated and does not establish the analysis treatment of that case.
These omissions limit assessment of potential bias from incomplete observations, endpoint selection or analysis choices. They do not establish that data were missing or that an inappropriate method was used; those details are simply not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| 10 µg/kg/Day G-CSF Alone | 0 | 12 | 0.0% |
| 10 µg/kg/Day G-CSF + 3.14 mg/kg TG-0054 | 0 | 12 | 0.0% |
| Overall | 0 | 12 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| 10 µg/kg/Day G-CSF Alone | 0 of 12 |
| 10 µg/kg/Day G-CSF + 3.14 mg/kg TG-0054 | 0 of 12 |
| Overall | 0 of 12 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| 10 µg/kg/Day G-CSF Alone | 2 of 12 |
| 10 µg/kg/Day G-CSF + 3.14 mg/kg TG-0054 | 6 of 12 |
| Overall | 7 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 10 µg/kg/Day G-CSF Alone | 10 µg/kg/Day G-CSF + 3.14 mg/kg TG-0054 | Overall |
|---|---|---|---|---|
| Bone pain | Musculoskeletal and connective tissue disorders | 2 of 12 | 0 of 12 | 2 of 12 |
| Hypocalcaemia | Metabolism and nutrition disorders | 1 of 12 | 0 of 12 | 1 of 12 |
| Diarrhoea | Gastrointestinal disorders | 0 of 12 | 1 of 12 | 1 of 12 |
| Dry mouth | Gastrointestinal disorders | 0 of 12 | 1 of 12 | 1 of 12 |
| Nausea | Gastrointestinal disorders | 0 of 12 | 1 of 12 | 1 of 12 |
| Hypokalaemia | Metabolism and nutrition disorders | 0 of 12 | 1 of 12 | 1 of 12 |
| Oedema peripheral | General disorders | 0 of 12 | 1 of 12 | 1 of 12 |
| Platelet count decreased | Investigations | 0 of 12 | 1 of 12 | 1 of 12 |
| Paraesthesia | Nervous system disorders | 0 of 12 | 1 of 12 | 1 of 12 |
The registry record reports 0 serious adverse events among 12 at risk in the G-CSF-alone category and 0 among 12 at risk in the G-CSF plus TG-0054 category. The overall category also reports 0 serious adverse events among 12 at risk. Deaths are reported as 0 in each category. No serious adverse-event terms are listed.
For other adverse events, the record reports 2 affected in the G-CSF-alone category, 6 in the combination category and 7 overall, with 12 at risk in each displayed category. These are reported affected-participant counts, not a description of event severity or duration. The overall count should not be calculated by adding the category counts; the record presents it separately.
The categories do not establish a controlled safety comparison. The registry record explicitly cautions that the arms are not a like-for-like control comparison and does not explain their relationship to treatment exposure. Thus, the counts describe what the record reports, but cannot support a causal claim about differences between regimens. Further detail about event definitions, timing and ascertainment is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The study addresses whether patients receiving TG-0054 combined with G-CSF reached a prespecified CD34+ cell collection threshold within a defined number of leukapheresis sessions. The registry record gives the endpoint rule, treatment regimen and enrollment information. It does not report the primary endpoint proportion or its confidence interval, so the central efficacy result cannot be characterized from the registry record.
Even if an observed proportion were available, a single-group result would describe outcomes in the enrolled study group rather than establish what the outcome would have been under a different treatment. The separate G-CSF-alone safety label does not supply a valid efficacy comparator; the registry record warns that the categories are not a like-for-like control comparison. The design therefore does not support a randomized treatment-effect interpretation.
The safety information records no serious adverse events and no deaths in the displayed categories, while it reports other adverse events affecting participants in the G-CSF-alone, combination and overall categories. These counts are descriptive. They do not by themselves establish comparative safety, and the registry record does not provide serious-event terms or detailed event descriptions.
The endpoint is also a threshold summary. It indicates whether the collection total met a target, not the full pattern of cell yields or the amount by which individual patients did or did not meet that target. That distinction matters when interpreting any proportion. The missing endpoint result and interval leave both the observed frequency and its precision unknown from the registry record.
Accordingly, the record documents a completed Phase II study and its stated mobilization question, but does not provide enough result information to determine how often the primary target was reached or how precisely that frequency was estimated. It also does not support claims about superiority, comparative effectiveness or treatment benefit.
13. Why This Trial Matters Statistically
This study is statistically notable for the contrast between a clearly defined threshold endpoint and the absence of its reported result in the registry record. The primary question is concrete: whether cumulative CD34+ collection met a specified target within a specified collection window. Yet the proportion and its 95% confidence interval are not reported. This leaves the main efficacy estimate unavailable for interpretation.
The single-group design also defines the scope of what the study can answer. It can be used to describe outcomes observed among participants receiving the regimen, but it does not provide a randomized concurrent comparison for estimating a causal difference. The safety categories labeled G-CSF alone and combination do not resolve that limitation, because the registry record says they are not a like-for-like control comparison.
The record illustrates why endpoint definitions, analysis populations and results all matter. A threshold endpoint needs an observed estimate and a measure of uncertainty to show both the frequency of reaching the goal and the precision of that estimate. A description of how the endpoint is calculated cannot substitute for those results. Likewise, enrollment and subgroup descriptions do not reveal the outcome distribution.
Finally, the record’s limited analysis detail constrains reproducibility and interpretation. It lists outcome measures but no analysis entries, and it does not report a multiplicity plan, missing-data approach or full statistical analysis plan. These are not evidence of a particular flaw; they are unanswered questions in the registry record. The study is therefore informative about the intended clinical question and design, while its reported statistical evidence remains incomplete.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The primary limitation for interpreting efficacy is that the registry record does not report the observed primary endpoint proportion or the limits of its stated 95% confidence interval. It therefore does not show how many participants reached the collection threshold or how precise the estimate was. Results for the listed secondary outcomes are not reported in the registry record either.
The study is listed as single-group, open-label, with allocation not applicable and masking none. The record does not describe a randomized concurrent efficacy comparator. Its safety categories include a G-CSF-alone label, but the record warns that the arms are not a like-for-like control comparison. This limits comparative interpretation.
The enrollment was 12, and the endpoint description reports 9 patients with multiple myeloma and 3 with non-Hodgkin lymphoma or Hodgkin disease. The record does not report further detail about the analysis population, subgroup-specific outcomes or how broadly the results would apply beyond the enrolled group.
The endpoint narrative includes a patient with multiple myeloma treated with Revlimid who had a cumulative collection of 0.18 x 10^6 cells/kg after 2 leukapheresis sessions, followed by truncated text. The reason for stopping is described, but the remaining explanation and the analytic handling are not reported in the registry record.
Other limitations of the available record are the absence of a reported analysis plan, multiplicity approach, missing-data method and detailed adverse-event descriptions. These gaps prevent a fuller appraisal of analysis conduct and safety context. They should be understood as limits of what is reported, not evidence that a particular procedure was or was not used.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: PD and Safety of TG-0054 Combined With G-CSF in Multiple Myeloma, Non-Hodgkin Lymphoma and Hodgkin Disease Patients — NCT02104427.
- Registry record last updated 2021-04-19; status last verified 2021-04.
- Results first posted 2017-12-13.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT02104427 is a completed Phase II, open-label, single-group study of TG-0054 combined with G-CSF for hematopoietic stem-cell mobilization in patients with multiple myeloma, non-Hodgkin lymphoma or Hodgkin disease. Enrollment was 12; the study started in 2015-02 and primary completion was in 2015-11.
The primary endpoint is the proportion reaching at least 5.0 x 10^6 CD34+ cells/kg within the first 4 leukapheresis sessions, during Day 5 to Day 8. The registry record describes the endpoint and lists a 95% confidence interval, but does not report the result or interval limits. It lists 4 outcome measures and 0 analysis entries.
Safety reporting lists 0 serious adverse events and 0 deaths in the displayed categories; other adverse events affected 2 in the G-CSF-alone category, 6 in the combination category and 7 overall. The record cautions that the categories are not a like-for-like control comparison. The primary efficacy result and further analysis details are not reported in the registry record.