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 Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc) (NCT00684255), a non-randomized phase 1 study. The primary outcome is Toxicity.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00684255 |
| Phase | Phase 1 |
| Condition | Systemic Lupus Erythematosus, Systemic Sclerosis |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | New York Medical College |
| Results posted | Yes |
2. Clinical Question
Population
People with medically refractory systemic lupus erythematosus (SLE) or systemic sclerosis (SSc) were the intended population. The record describes selected patients in disease-specific groups. It does not report additional eligibility criteria or details about the condition or prior treatment history beyond the term medically refractory.
Intervention
The study evaluated a reduced-intensity, non-myeloablative regimen of fludarabine, busulfan and Campath, followed by allogeneic stem cell transplantation (AlloSCT). The arm descriptions name Alemtuzumab as part of the regimen and refer to selected patients with SLE or SSc. The summary describes matched family donors and matched unrelated cord blood donors.
Comparator
No like-for-like control comparison is reported. The study was non-randomized, and its disease-specific groups are not described as randomized treatment and control arms. The registry record therefore does not support a causal comparison of the transplant regimen against another treatment or against no transplant.
Primary question
The primary question was whether toxicity associated with reduced-intensity conditioning followed by allogeneic stem cell transplantation could be measured in people with medically refractory SLE or SSc over 1 year. The stated purpose emphasizes whether the approach would be well tolerated. The record does not provide a numerical primary toxicity result or an analysis of that question.
This is a safety-focused early-phase question, rather than a question about comparative effectiveness. The record lists progression-free and overall survival among secondary outcomes, along with chimerism and immune reconstitution. Those outcomes may describe disease course and biological response, but the registry record does not report analyses for them. It also does not define which toxicities count toward the primary outcome, how severity would be graded, or what observation schedule was used. Without those details and without outcome analyses, the primary question can be described from the stated objective, but the results cannot establish how often toxicity occurred or how precisely it was estimated.
3. Trial Design
The study is listed as Phase 1, non-randomized, unmasked and parallel. The registry record identifies medically refractory SLE and SSc as the conditions under study and describes disease-specific reduced-intensity regimen groups. In each description, fludarabine, busulfan and Alemtuzumab are followed by allogeneic stem cell transplantation. The summary describes the donor sources as matched family donors and matched unrelated cord blood donors. The record does not describe a randomized allocation process or a concurrent control group; the disease-specific groups should not be read as a controlled comparison.
Enrollment is recorded as 1. Study start is listed as 2007-08, primary completion as 2008-07, and status as terminated. The reason for termination is not reported in the registry record. These record entries do not establish how much treatment was delivered, whether the intended transplant procedure was completed, or why the study ended. Those details are not reported in the registry record.
The primary outcome is toxicity associated with the conditioning regimen and transplant, with a timeframe of 1 year. Secondary outcomes are chimerism, immune reconstitution, and progression-free and overall survival. The record lists 4 outcome measures, but it does not provide analyses for them. It also does not describe planned methods for assigning participants to the disease-specific groups, monitoring outcomes, handling deviations from treatment, or defining the analysis population. The study design therefore identifies the intervention and intended safety question, but leaves important operational and analytic details unspecified. In particular, the parallel structure does not by itself imply that the groups are comparable: SLE and SSc are different conditions, and the record does not state that they were intended to serve as mutual controls.
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 | Reduced Intensity Regimen SSc |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
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 | Systemic Sclerosis (SSc) |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 1 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Toxicity, measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is toxicity associated with reduced-intensity fludarabine, busulfan and Campath followed by allogeneic stem cell transplantation in people with medically refractory SLE or SSc. Its stated timeframe is 1 year. The purpose of a toxicity endpoint in an early-phase study is generally to characterize harms and tolerability around the intervention, rather than to establish comparative benefit. This is a general explanation of the role of safety endpoints, not a description of additional procedures in this study.
The registry record does not report an operational definition of toxicity, a grading system, specific events included in the endpoint, or rules for attributing an event to the regimen. It also does not state how events would be collected or whether the endpoint is a count, a proportion, a severity measure, or a composite. These omissions matter because the meaning of a toxicity result depends on what events were sought, how they were classified, and when they were assessed. The record gives the endpoint label and timeframe but does not report a primary toxicity analysis or a result that can be interpreted against a prespecified threshold. Consequently, the stated endpoint indicates what the study aimed to assess, not what the treatment's toxicity was found to be.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a very small, early-phase study: enrollment is listed as 1, and the study is non-randomized and unmasked. The record reports no outcome analyses. As a result, there is no reported estimate of the primary toxicity outcome, no estimate of its uncertainty, and no reported analysis of the secondary outcomes. The stated aim and outcome list should not be mistaken for findings.
The adverse-event entry lists the Reduced Intensity Regimen SSc group with 1 at risk and 0 affected for serious events, and 1 at risk and 0 affected for other events. These entries describe the posted counts; they do not establish that the regimen has no risk. With such sparse information, a zero count cannot rule out events that were not observed, not recorded, or not represented by the available record. The deaths entry is null, which is not equivalent to a reported count of zero. The registry record does not state whether the SLE group has a separate safety entry or explain the scope of the listed group.
Precision is central to interpreting safety estimates. In general, a confidence interval conveys the range of values compatible with the observed data under the analysis assumptions. When data are sparse, estimates can be highly uncertain, and absence of an event in the recorded observations does not establish absence of risk. No confidence interval or other uncertainty estimate for toxicity is reported in the registry record, so the precision of the primary outcome cannot be assessed from the reported results.
The study cannot support a treatment-versus-control conclusion. Non-randomized disease-specific groups are not a like-for-like comparison, and the record does not describe a control group. Differences between people with SLE and SSc, or between their clinical courses, could not be separated from an intervention effect using the information reported. The record also gives no outcome analysis for survival, chimerism or immune reconstitution. The defensible reading is therefore limited: the study was designed to assess toxicity and listed some safety counts, but it does not report analyzed results sufficient to quantify toxicity or evaluate the other outcomes.
9. Statistical Methods Explained
The design and reported data place strong limits on what statistical interpretation is possible. The study is labeled Phase 1 and non-randomized, with no masking. Its model is parallel, but the descriptions identify separate groups for SLE and SSc rather than a treatment group and a comparable control group. Enrollment is listed as 1. The registry record reports 0 analyses. These design and reporting features mean that the main statistical task is to distinguish the stated objective and posted counts from estimates that the record does not actually report.
What does non-randomized mean for interpretation?
In a randomized study, allocation by chance is intended to make groups comparable on average, including characteristics that may affect outcomes. That principle is a general feature of randomization. Here allocation is explicitly non-randomized, and no control comparison is identified. Therefore, the record does not provide the basis for attributing differences in outcomes to the intervention. Even if outcomes had been listed separately by disease group, the groups would not automatically function as controls for each other. SLE and SSc define different clinical populations, and the record does not report a design that makes them exchangeable for a causal comparison.
What can a safety count tell a reader?
A count of affected participants can describe observed events within a defined group when the event definition, observation period and denominator are clear. General statistical interpretation also depends on complete event ascertainment and consistent follow-up. The registry record lists at-risk and affected counts for serious and other events in the SSc group, but does not provide event definitions, grading details, collection procedures, or an explanation of how the denominator was determined. The small listed at-risk count also makes the descriptive information particularly limited. A count of 0 affected is an observation in this record, not proof that the underlying event risk is zero.
Uncertainty is usually conveyed with an interval estimate or another appropriate measure. In general, a confidence interval shows the range of parameter values reasonably compatible with data under a statistical model and its assumptions. Sparse observations tend to yield imprecise estimates. No interval estimate, standard error, or model-based toxicity estimate is reported, so there is no reported precision measure to interpret. A reader should not infer one from the count.
Why does the endpoint definition matter?
The primary outcome is named Toxicity and has a timeframe of 1 year, but the record does not specify how toxicity is defined or summarized. A binary outcome, event count, time-to-first-event outcome, and severity score answer different questions and require different analysis methods. This is a general methodological point. Without an operational definition and analysis method, it is not possible to determine which statistical estimand the study intended to report. The record also does not specify censoring rules, follow-up completeness, or how competing events would be handled.
For time-to-event outcomes, general methods account for both when an event occurs and how long participants are followed; they also rely on assumptions about censoring. The record lists progression-free and overall survival as secondary outcomes, but it reports no analyses or follow-up results for them. No survival estimate, event count, or time-to-event comparison should therefore be inferred. Similarly, chimerism and immune reconstitution are listed without reported analytic results or measurement details.
What is not reported about the analysis?
The registry record does not report a statistical analysis plan, the sample-size justification, interim analyses, or procedures for missing data. It does not report an adjusted or unadjusted outcome analysis, a treatment-effect estimate, or a test of a hypothesis. Without an analysis plan, it is not possible to assess whether outcome definitions, analysis populations, and statistical methods were specified before data review. The absence of those details does not establish that no methods were used; it means they are not reported in the registry record.
Any sample-size calculations associated with the record are planning illustrations based on an observed effect, not observed power or evidence that the study had a particular ability to detect an effect. The record’s stated illustration assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; for time-to-event illustrations, the stated quantity is total events, while other illustrations are expressed as participants per arm. These assumptions describe planning illustrations, not the design or results of this non-randomized study. Because the registry record reports no outcome analyses, such illustrations cannot be treated as evidence of a measured treatment effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary toxicity outcome and secondary outcomes of chimerism, immune reconstitution, and progression-free and overall survival. It does not report an analysis for any of them. Nor does it report a statistical plan for prioritizing outcomes, adjusting for multiple testing, or distinguishing confirmatory from exploratory analyses. These details are not reported in the registry record, so no claim can be made about how multiplicity was handled.
Multiplicity is a general concern when several outcomes or comparisons are examined. Testing many questions can increase the chance that at least one result appears unusual by chance. Methods such as prespecifying a primary outcome, controlling an error rate, or describing secondary findings as exploratory can clarify interpretation. Those are general approaches, not methods documented for this study. Given the absence of reported analyses, there are no reported p-values or outcome estimates for which a multiplicity adjustment can be assessed.
Missing-data methods and analysis populations are also not described. The record does not state how incomplete follow-up, unavailable measurements, treatment discontinuation, or participants without outcome data would be handled. It does not identify an intention-to-treat, treated, or evaluable population. The adverse-event listing includes a null deaths entry; that entry does not establish that no deaths occurred, nor does it explain missingness or follow-up status. The record’s enrollment and group counts alone do not resolve these uncertainties.
These gaps matter because analysis choices can change the population represented by an estimate and the interpretation of uncertainty. In general, missing outcomes can bias a result when their absence is related to health status or treatment experience. That is a methodological consideration, not evidence that such bias occurred in this study. With no reported analysis or missing-data approach, the impact cannot be evaluated.
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 |
|---|---|---|---|
| Reduced Intensity Regimen SSc | 0 | 1 | 0.0% |
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 |
|---|---|
| Reduced Intensity Regimen SSc | 0 of 1 |
The serious-adverse-event listing names the Reduced Intensity Regimen SSc group. It records 1 at risk and 0 affected for serious events, and 1 at risk and 0 affected for other events. These are the counts listed in the registry record. They should be read as recorded observations for that group, not as estimates that risk is absent. The record does not describe event definitions, severity grading, collection procedures, follow-up completeness, or the criteria used to count people at risk.
The deaths field is null, not a reported zero. No serious-event terms are listed, but an empty term list does not establish that no serious events occurred outside the recorded entry. The record does not provide a separate safety listing for the SLE group or explain whether the reported listing covers all enrolled participants. With enrollment recorded as 1 and the available safety counts sparse, the figures do not support a precise estimate of toxicity. They also do not permit comparisons with another treatment, because no like-for-like control group is reported.
12. What the Result Does, and Does Not, Mean
The record establishes the study’s stated purpose and design: a Phase 1, non-randomized evaluation of reduced-intensity conditioning followed by allogeneic stem cell transplantation for medically refractory SLE or SSc, with toxicity as the primary outcome over 1 year. It also records enrollment as 1, lists the study as terminated, and identifies a small set of safety counts for the SSc group. These details describe what was planned and what was entered into the registry record.
The record does not establish whether the regimen was well tolerated. The primary toxicity outcome has no reported analysis, and the safety counts are too limited to characterize the range or frequency of harms. A zero affected count in the listed group does not prove that the underlying risk is zero. The null deaths entry should not be converted into a zero-death finding. The reason for termination is not reported in the registry record, so termination cannot be interpreted as a response to safety, feasibility, or any other cause.
The record also does not establish benefit or lack of benefit. Progression-free and overall survival are named as secondary outcomes, but there are no reported analyses for them. Chimerism and immune reconstitution are likewise listed without results. There is no randomized control group or reported comparison capable of separating an intervention effect from differences in patient characteristics or disease course.
General statistical interpretation requires both an estimate and an understanding of its uncertainty, alongside clarity about the population and outcome definition. Those elements are not available for the primary endpoint in the registry record. The appropriate conclusion is narrow: the study was intended to assess tolerability, but the posted results do not provide an analyzed estimate of toxicity, a comparative treatment effect, or evidence that can resolve the study’s clinical question.
13. Why This Trial Matters Statistically
This study illustrates why early-phase safety questions require careful separation of design intent from evidence. The stated primary aim concerns toxicity after reduced-intensity conditioning and allogeneic transplantation. Yet an objective is not a result: without a reported endpoint analysis, readers cannot quantify harms or assess the precision of an estimate. A small enrollment record and sparse event counts make it especially important not to interpret a zero as evidence of no risk.
It also illustrates the limits of reading disease-specific groups as comparison arms. The SLE and SSc groups are defined by different conditions, and the record does not describe a randomized or otherwise controlled comparison. In general, a comparative design needs a credible basis for distinguishing treatment effects from differences among the people receiving each approach. That basis is not reported. This is relevant when interpreting parallel designs, which describe group structure but do not by themselves establish comparability.
Finally, the record shows how missing analytic detail constrains interpretation. It lists a primary outcome, secondary outcomes, and a timeframe, but does not report outcome definitions, statistical methods, missing-data handling, or analyses. Those details are essential for judging what was measured and how reliably. The statistical lesson is not that the intervention succeeded or failed; the record does not support either conclusion. Rather, registry entries need analyzable outcomes, transparent methods, and sufficient reporting before readers can distinguish a study’s research question from evidence answering it.
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 most important trial-specific limitation is the very limited enrollment recorded: 1. The registry record reports no outcome analyses, so it is not possible to estimate the primary toxicity outcome or evaluate the listed secondary outcomes. The posted safety counts identify the SSc group only, and the record does not explain the scope of that listing or provide a corresponding SLE safety entry. The deaths field is null rather than a reported count.
The study is non-randomized and has no reported like-for-like control comparison. Because the disease-specific groups are not described as controls for one another, they cannot support a comparative estimate of treatment effect. The record also states that masking was absent. The implications of open-label assessment depend on how outcomes were defined and collected, details that are not reported in the registry record.
The primary endpoint is labeled Toxicity and assigned a 1 year timeframe, but its operational definition, event grading, ascertainment method, and summary measure are not reported. The record does not explain follow-up completeness, missing-data handling, analysis populations, or methods for the listed secondary outcomes. It provides no statistical analysis plan, sample-size justification, or explanation of interim analyses. The reason the study was terminated is also not reported.
These limitations restrict interpretation of what the record can show; they do not establish that events were missed or that a particular analytic procedure was used. The available information supports a description of the study objective and design, but not a precise safety estimate or a conclusion about comparative clinical outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc) — NCT00684255.
- Registry record last updated 2014-04-01; status last verified 2014-02.
- Results first posted 2009-12-02.
- 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
NCT00684255 is a Phase 1, non-randomized, unmasked parallel study of reduced-intensity conditioning and allogeneic stem cell transplantation in medically refractory SLE or SSc. The regimen is described as fludarabine, busulfan and Alemtuzumab followed by AlloSCT. The primary outcome is toxicity over 1 year; secondary outcomes include chimerism, immune reconstitution, and progression-free and overall survival.
Enrollment is recorded as 1. The listed dates are study start 2007-08 and primary completion 2008-07, and the status is terminated. No outcome analyses are reported. The available SSc safety entry lists 1 at risk and 0 affected for serious events and 1 at risk and 0 affected for other events; deaths are null. The record does not report a control comparison, a primary toxicity estimate, or the reason for termination.