Cutaneous T-Cell Lymphoma
Conditions
Keywords
CTCL, Mycosis fungoides, Hypericin, SGX301, MF
Brief summary
To evaluate the use of SGX301, a topical photosensitizing agent, to treat patients with patch/plaque phase cutaneous T-cell lymphoma (mycosis fungoides).
Interventions
DRUGSGX301 (synthetic hypericin)
0.25% SGX301 in USP Hydrophilic Ointment applied twice per week, covered by opaque bandage for 12-24 hours, then treated with an initial dose of 5 J/cm\^2 fluorescent light.
DRUGPlacebo
USP Hydrophilic Ointment applied twice per week, covered by opaque bandage for 12-24 hours, then treated with an initial dose of 5 J/cm\^2 fluorescent light.
Sponsors
Soligenix
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects must have a clinical diagnosis of CTCL (mycosis fungoides), Stage IA, Stage IB, or Stage IIA. * Subjects must have a minimum of three (3) evaluable, discrete lesions. * Subjects must be willing to refrain from sunbathing for the duration of the study.
Exclusion criteria
* History of sun hypersensitivity and photosensitive dermatoses including porphyria, systemic lupus erythematosus, Sjögren's syndrome, xeroderma pigmentosum, polymorphous light eruptions or radiation therapy within 30 days of enrolling. * Pregnancy or mothers who are breast feeding. * Males and females not willing to use effective contraception. * Unhealed sunburn. * Subjects receiving topical steroids or other topical treatments for CTCL within 2 weeks. * Subjects receiving systemic steroids, nitrogen mustard, psoralen UVA radiation therapy (PUVA), narrow band UVB light therapy (NB-UVB) or carmustine (BCNU) or other systemic therapies for CTCL within 3 weeks of enrollment. * Subjects with significant history of systemic immunosuppression due to drugs or infection with HIV or HTLV 1. * Subjects taking other investigational drugs or drugs of abuse within 30 days of entry into this study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Defined as a ≥50% Improvement in the Composite Assessment of Index Lesion Disease Severity (CAILS) Score When Compared to Patients Receiving Placebo | 8 weeks | The percentage of patients achieving a treatment response in each of the 2 treatment groups. A treatment response was defined as a ≥50% improvement in CAILS score at Week 8 when compared to the CAILS score at baseline. The Composite Assessment of Index Lesion Disease Severity (CAILS) score measures: Erythema (or redness) on a scale of 0 (no redness) to 8 (very red), Scaling on a scale of 0 (no scaling) to 8 (all of the lesion is covered by a very rough surface), Plaque Elevation on a scale of 0 (no evidence of plaque above normal skin level) to 3 (plaque shows marked elevation above normal skin level) and Surface Area on a scale of 0 (no lesion/surface area is 0 cm\^2) to 18 (the lesion is larger than 300 cm\^2). A lower score means a better outcome. The overall CAILS score was calculated by adding the total score as described above for each of the 3 lesions. The overall CAILS score has a range of 0 to 111. A lower score means a better outcome. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score (Cycle 1 and 2 SGX301 vs Cycle 1 Placebo) | 16 weeks | The percentage of patients achieving a treatment response at Week 16 that received SGX301 for both Cycle 1 and 2 compared to the response rate in patients that received Placebo in Cycle 1. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described. |
| Number of Responders and Non-Responders With a Treatment Response of 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score in Patients Who Received 3 Cycles of SGX301 | 24 weeks | The percentage of patients achieving a treatment response at Week 24 compared at Week 16 in SGX301 treatment group. A treatment response was defined as a ≥50% improvement in CAILS score when compared to the CAILS score at baseline. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described. |
| Patch Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo) | 16 weeks | The proportion of patch lesions achieving a treatment response at Week 16 in the SGX301 treatment group compared to Week 8 in the Placebo treatment group. A treatment response was defined as a ≥50% improvement in CAILS score when compared to the CAILS score at baseline for individual lesions. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described. |
| Plaque Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo) | 16 weeks | The proportion of plaque lesions achieving a treatment response at Week 16 in the SGX301 treatment group compared to Week 8 in the Placebo treatment group. A treatment response was defined as a ≥50% improvement in CAILS score when compared to the CAILS score at baseline for individual lesions. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| SGX301 Three treatment cycles, each six (6) weeks followed by a two (2) week rest period. Treatment uses 0.25% SGX301 in USP Hydrophilic Ointment (or placebo) applied twice per week followed by fluorescent light therapy.
Cycle 1: Patients randomized 2:1 to active/placebo will have three (3) index lesions treated and evaluated.
Cycle 2: All patients will have three (3) index lesions treated and evaluated with active SGX301 ointment.
Cycle 3: All patients will be given the opportunity to enter an open-label cycle of active SGX301 ointment treatment for all lesions (index and non-index).
SGX301 (synthetic hypericin): 0.25% SGX301 in USP Hydrophilic Ointment applied twice per week, covered by opaque bandage for 12-24 hours, then treated with an initial dose of 5 J/cm\^2 fluorescent light. | 116 |
| Placebo Placebo ointment is indistinguishable from ointment containing active SGX301 and is only used in Cycle 1. Treatment paradigm (ointment application and fluorescent light therapy) is identical.
Placebo: USP Hydrophilic Ointment applied twice per week, covered by opaque bandage for 12-24 hours, then treated with an initial dose of 5 J/cm\^2 fluorescent light. | 50 |
| Total | 166 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 2 | 0 |
| Overall Study | Lost to Follow-up | 16 | 9 |
| Overall Study | Noncompliance with Study Procedures | 2 | 0 |
| Overall Study | Physician Decision | 1 | 1 |
| Overall Study | Pregnancy | 1 | 0 |
| Overall Study | Withdrawal by Subject | 13 | 9 |
Baseline characteristics
| Characteristic | Total | SGX301 | Placebo |
|---|---|---|---|
| Age, Continuous | 58.4 years STANDARD_DEVIATION 16 | 57.9 years STANDARD_DEVIATION 15.94 | 59.4 years STANDARD_DEVIATION 16.26 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 13 Participants | 10 Participants | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 148 Participants | 103 Participants | 45 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 5 Participants | 3 Participants | 2 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 4 Participants | 4 Participants | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 39 Participants | 27 Participants | 12 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Other | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Unknown | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized White | 120 Participants | 84 Participants | 36 Participants |
| Sex: Female, Male Female | 70 Participants | 47 Participants | 23 Participants |
| Sex: Female, Male Male | 96 Participants | 69 Participants | 27 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk |
|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 116 | 0 / 50 | 0 / 155 | 0 / 110 | 0 / 161 |
| other Total, other adverse events | 56 / 116 | 26 / 50 | 66 / 155 | 49 / 110 | 108 / 161 |
| serious Total, serious adverse events | 1 / 116 | 1 / 50 | 0 / 155 | 3 / 110 | 4 / 161 |
Outcome results
Primary
Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Defined as a ≥50% Improvement in the Composite Assessment of Index Lesion Disease Severity (CAILS) Score When Compared to Patients Receiving Placebo
The percentage of patients achieving a treatment response in each of the 2 treatment groups. A treatment response was defined as a ≥50% improvement in CAILS score at Week 8 when compared to the CAILS score at baseline. The Composite Assessment of Index Lesion Disease Severity (CAILS) score measures: Erythema (or redness) on a scale of 0 (no redness) to 8 (very red), Scaling on a scale of 0 (no scaling) to 8 (all of the lesion is covered by a very rough surface), Plaque Elevation on a scale of 0 (no evidence of plaque above normal skin level) to 3 (plaque shows marked elevation above normal skin level) and Surface Area on a scale of 0 (no lesion/surface area is 0 cm\^2) to 18 (the lesion is larger than 300 cm\^2). A lower score means a better outcome. The overall CAILS score was calculated by adding the total score as described above for each of the 3 lesions. The overall CAILS score has a range of 0 to 111. A lower score means a better outcome.
Time frame: 8 weeks
Population: The analysis population was defined as all participants that were treated with at least one dose of treatment (SGX301 or Placebo)
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| SGX301 | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Defined as a ≥50% Improvement in the Composite Assessment of Index Lesion Disease Severity (CAILS) Score When Compared to Patients Receiving Placebo | Non-responder | 97 Participants |
| SGX301 | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Defined as a ≥50% Improvement in the Composite Assessment of Index Lesion Disease Severity (CAILS) Score When Compared to Patients Receiving Placebo | Responder | 19 Participants |
| Placebo | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Defined as a ≥50% Improvement in the Composite Assessment of Index Lesion Disease Severity (CAILS) Score When Compared to Patients Receiving Placebo | Responder | 2 Participants |
| Placebo | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Defined as a ≥50% Improvement in the Composite Assessment of Index Lesion Disease Severity (CAILS) Score When Compared to Patients Receiving Placebo | Non-responder | 48 Participants |
p-value: 0.04Regression, Logistic
Secondary
Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score (Cycle 1 and 2 SGX301 vs Cycle 1 Placebo)
The percentage of patients achieving a treatment response at Week 16 that received SGX301 for both Cycle 1 and 2 compared to the response rate in patients that received Placebo in Cycle 1. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described.
Time frame: 16 weeks
Population: The analysis population was defined as all participants that were treated with at least one dose of treatment (SGX301) in Cycle 2
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| SGX301 | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score (Cycle 1 and 2 SGX301 vs Cycle 1 Placebo) | Responder | 44 Participants |
| SGX301 | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score (Cycle 1 and 2 SGX301 vs Cycle 1 Placebo) | Non-responder | 66 Participants |
| Placebo | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score (Cycle 1 and 2 SGX301 vs Cycle 1 Placebo) | Responder | 2 Participants |
| Placebo | Number of Responders and Non-Responders With a Treatment Response in 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score (Cycle 1 and 2 SGX301 vs Cycle 1 Placebo) | Non-responder | 48 Participants |
p-value: <0.0001Regression, Logistic
Secondary
Number of Responders and Non-Responders With a Treatment Response of 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score in Patients Who Received 3 Cycles of SGX301
The percentage of patients achieving a treatment response at Week 24 compared at Week 16 in SGX301 treatment group. A treatment response was defined as a ≥50% improvement in CAILS score when compared to the CAILS score at baseline. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described.
Time frame: 24 weeks
Population: The analysis population was defined as all participants that were treated with at least one dose of treatment (SGX301) in Cycle 2
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| SGX301 | Number of Responders and Non-Responders With a Treatment Response of 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score in Patients Who Received 3 Cycles of SGX301 | Responder | 38 Participants |
| SGX301 | Number of Responders and Non-Responders With a Treatment Response of 3 Treated Lesions as Measured by the Composite Assessment of Index Lesion Disease Severity (CAILS) Score in Patients Who Received 3 Cycles of SGX301 | Non-responder | 40 Participants |
p-value: 0.046McNemar
Secondary
Patch Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo)
The proportion of patch lesions achieving a treatment response at Week 16 in the SGX301 treatment group compared to Week 8 in the Placebo treatment group. A treatment response was defined as a ≥50% improvement in CAILS score when compared to the CAILS score at baseline for individual lesions. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described.
Time frame: 16 weeks
Population: The analysis population was defined as all participants that were treated with at least one dose of treatment (SGX301) in Cycle 2
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGX301 | Patch Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo) | 65 lesions with response |
| Placebo | Patch Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo) | 14 lesions with response |
p-value: =0.0009Fisher Exact
Secondary
Plaque Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo)
The proportion of plaque lesions achieving a treatment response at Week 16 in the SGX301 treatment group compared to Week 8 in the Placebo treatment group. A treatment response was defined as a ≥50% improvement in CAILS score when compared to the CAILS score at baseline for individual lesions. The Composite Assessment of Index Lesion Disease Severity (CAILS) score was measured as previously described.
Time frame: 16 weeks
Population: The analysis population was defined as all lesions that were treated with at least one dose of treatment (SGX301) in Cycle 2
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| SGX301 | Plaque Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo) | 64 lesions with response |
| Placebo | Plaque Lesion Response Rates With Extended Treatment (Cycle 1 & 2 SGX301 vs Cycle 1 Placebo) | 7 lesions with response |
p-value: <0.0001Fisher Exact