This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record.
1. Trial at a Glance
ABC-01 was a phase 1 treatment study evaluating hydroxychloroquine in combination with hormonal therapy for metastatic estrogen receptor-positive breast cancer progressing on hormonal therapy.
| Feature | ABC-01 |
|---|---|
| ClinicalTrials.gov identifier | NCT02414776 |
| Status | TERMINATED |
| Phase | Phase 1 |
| Condition | Estrogen Receptor Positive Breast Cancer |
| Lead sponsor | Western Regional Medical Center |
| Design model | Single group |
| Masking | None |
| Intervention | Hydroxychloroquine (drug) |
2. Clinical Question
Population
Patients with estrogen receptor-positive breast cancer with metastatic disease progressing on hormonal therapy.
Intervention
Orally administered hydroxychloroquine with hormonal therapy.
Comparator
No comparator arm; the study used a single-group design.
Primary question
What is the safety profile of hydroxychloroquine with hormonal therapy at the study dose?
3. Trial Design
The registry describes ABC-01 as a single-group, open-label phase 1 treatment study. The study included one intervention arm and enrolled 3 participants.
| Design feature | Description |
|---|---|
| Allocation | NA |
| Design model | SINGLE_GROUP |
| Masking | NONE |
| Primary purpose | TREATMENT |
| Number of arms | 1 |
4. Endpoints
| Endpoint | Definition | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events as a Measure of the Safety Profile of Orally Administered Hydroxychloroquine with Hormonal Therapy | Assess dose-limiting side effects such as neutropenia, anemia, or thrombocytopenia, and non-hematologic side effects including nausea, vomiting, diarrhea, or vision problems at the study dose. | 18 months |
5. Planned Analysis
The registry identifies safety as the primary endpoint. For a phase 1 safety study, adverse events are typically summarized descriptively, including the number of participants experiencing adverse events, the type of events observed, severity, and relationship to study treatment.
The ClinicalTrials.gov record does not report posted statistical analyses or trial results for the primary endpoint.
6. Statistical Methodology
Descriptive safety analysis
Early-phase safety studies generally focus on characterizing tolerability rather than estimating comparative treatment effects. With a single-group design, analyses are centered on observed participant-level outcomes rather than between-group hypothesis testing.
Without a comparator arm, the study design cannot estimate a randomized treatment effect. Safety findings are interpreted within the context of observed events among treated participants.
7. Statistical Methods Explained
Why was a single-group design used?
Phase 1 studies frequently use single-group designs when the primary objective is initial assessment of safety and tolerability rather than confirmation of efficacy compared with another treatment.
Why are adverse events the primary endpoint?
Safety endpoints are central in early development because they characterize whether further evaluation of a treatment approach is appropriate.
How are adverse events usually summarized?
Safety analyses commonly use counts and percentages of participants experiencing events, along with descriptions of severity and timing.
Why is there no treatment-effect estimate?
A treatment-effect estimate requires a comparison framework. A single-arm study does not contain a randomized control group for such a comparison.
8. Limitations
- No comparator arm: The single-group design limits comparative interpretation.
- Small enrollment: The study enrolled 3 participants, limiting precision of safety estimates.
- Phase 1 objective: The study was designed around safety assessment rather than confirmatory efficacy testing.
- Registry reporting: The ClinicalTrials.gov record does not report posted statistical analyses or outcome results.
9. Why This Trial Matters Statistically
ABC-01 illustrates important statistical principles in early-phase clinical research.
| Concept | Application |
|---|---|
| Single-arm design | Safety assessment without randomized comparison |
| Descriptive statistics | Primary framework for adverse event summaries |
| Sample size | Important determinant of precision in early studies |
| Endpoint selection | Safety profile as the primary measurement objective |
10. Limitations and Interpretation
Statistical interpretation: ABC-01 is structured as a safety-focused phase 1 study. The available registry information describes the design and endpoint but does not provide posted statistical analyses or outcome estimates.
Clinical interpretation: Findings from early-phase safety studies are generally considered exploratory and inform decisions about future investigation.
11. Sources
- ClinicalTrials.gov record: ABC-01 (NCT02414776)