Trial Outcomes & Findings for Safety, Pharmacodynamics (PD), Pharmacokinetics (PK) Study of SHP141 in 1A, 1B, or 2A Cutaneous T-Cell Lymphoma (CTCL) (NCT NCT01433731)
NCT ID: NCT01433731
Last Updated: 2016-03-24
Results Overview
Response assessed by change in lesion severity using Composite Assessment of Index Lesion Severity (CAILS) Assessment Tool which measures clinical signs of CTCL by erythema; scaling; plaque elevation; hypo- or hyperpigmentation, each on a scale of 0-8; and lesion size (cm2), on a scale of 0 (no lesion; 0 cm2) to 18 (300 cm2). Up to five index lesions are each scored, and a subtotal CAILS score is provided for each index lesion. A total score is calculated by summing these subtotals. Response criteria measure the change in CAILS score from baseline to follow-up as follows: Complete Response (CR): 100% decrease in CAILS score; Partial Response (PR): 50% - 99% decrease in CAILS score; Stable Disease (SD): \< 25% increase to \< 50% decrease in CAILS score; Progressive Disease (PD) ≥ 25% increase in CAILS score.
COMPLETED
PHASE1
18 participants
Weekly through day 28 (days 1, 7, 14, 21, 28) and again day 42
2016-03-24
Participant Flow
Participant milestones
| Measure |
Placebo for SHAPE (SHP-141)
placebo for SHAPE (SHP-141): topical gelled solution
|
SHAPE (SHP-141) 0.1% BID
SHAPE (SHP-141): topical gelled solution at 0.1% concentration twice daily
|
SHAPE (SHP-141) 0.5% BID
SHAPE (SHP-141): topical gelled solution at 0.5% concentration twice daily
|
SHAPE (SHP-141) 1.0% BID
SHAPE (SHP-141): topical gelled solution at 1.0% concentration twice daily
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
5
|
5
|
5
|
|
Overall Study
COMPLETED
|
3
|
5
|
5
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety, Pharmacodynamics (PD), Pharmacokinetics (PK) Study of SHP141 in 1A, 1B, or 2A Cutaneous T-Cell Lymphoma (CTCL)
Baseline characteristics by cohort
| Measure |
Placebo for SHAPE (SHP-141)
n=3 Participants
placebo for SHAPE (SHP-141): topical gel
|
SHAPE (SHP-141) 0.1% BID
n=5 Participants
SHAPE (SHP-141): topical gelled solution at 0.1% concentration twice daily
|
SHAPE (SHP-141) 0.5% BID
n=5 Participants
SHAPE (SHP-141): topical gelled solution at 0.5% concentration twice daily
|
SHAPE (SHP-141) 1.0% BID
n=5 Participants
SHAPE (SHP-141): topical gelled solution at 1.0% concentration twice daily
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
5 participants
n=5 Participants
|
5 participants
n=4 Participants
|
18 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Weekly through day 28 (days 1, 7, 14, 21, 28) and again day 42Response assessed by change in lesion severity using Composite Assessment of Index Lesion Severity (CAILS) Assessment Tool which measures clinical signs of CTCL by erythema; scaling; plaque elevation; hypo- or hyperpigmentation, each on a scale of 0-8; and lesion size (cm2), on a scale of 0 (no lesion; 0 cm2) to 18 (300 cm2). Up to five index lesions are each scored, and a subtotal CAILS score is provided for each index lesion. A total score is calculated by summing these subtotals. Response criteria measure the change in CAILS score from baseline to follow-up as follows: Complete Response (CR): 100% decrease in CAILS score; Partial Response (PR): 50% - 99% decrease in CAILS score; Stable Disease (SD): \< 25% increase to \< 50% decrease in CAILS score; Progressive Disease (PD) ≥ 25% increase in CAILS score.
Outcome measures
| Measure |
SHAPE (SHP-141)
n=15 Participants
Histone deacetylase inhibitor
SHAPE (SHP-141): topical gel
|
Placebo for SHAPE (SHP-141)
n=3 Participants
placebo for SHAPE (SHP-141): topical gel
|
|---|---|---|
|
Percentage of Patients With Complete or Partial Response as Measured by Change in Lesion Severity Using CAILS (Composite Assessment of Index Lesion Severity)
|
27 percentage of participants
|
0 percentage of participants
|
Adverse Events
Placebo for SHAPE (SHP-141)
SHAPE (SHP-141) 0.1% BID
SHAPE (SHP-141) 0.5% BID
SHAPE (SHP-141) 1.0% BID
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo for SHAPE (SHP-141)
n=3 participants at risk
placebo for SHAPE (SHP-141): topical gelled solution
|
SHAPE (SHP-141) 0.1% BID
n=5 participants at risk
SHAPE (SHP-141): topical gelled solution at 0.1% concentration twice daily
|
SHAPE (SHP-141) 0.5% BID
n=5 participants at risk
SHAPE (SHP-141): topical gelled solution at 0.5% concentration twice daily
|
SHAPE (SHP-141) 1.0% BID
n=5 participants at risk
SHAPE (SHP-141): topical gelled solution at 1.0% concentration twice daily
|
|---|---|---|---|---|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Skin and subcutaneous tissue disorders
Skin burning sensation
|
33.3%
1/3 • 42 days
|
40.0%
2/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Nervous system disorders
Burning sensation
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Nervous system disorders
Dysaesthesia
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Nervous system disorders
Paresthesia
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Investigations
Blood uric acid increased
|
33.3%
1/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Investigations
GGT increased
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Investigations
Lymphocyte count decreased
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Investigations
Urine analysis abnormal
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/3 • 42 days
|
40.0%
2/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Injury, poisoning and procedural complications
Incision site blister
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Injury, poisoning and procedural complications
Joint dislocation
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Injury, poisoning and procedural complications
Radius fracture
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Injury, poisoning and procedural complications
Wound dehiscence
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
General disorders
Applications site warmth
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
General disorders
Edema peripheral
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
General disorders
Pyrexia
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
|
Cardiac disorders
Supraventricular extrasystoles
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Infections and infestations
Rhinitis
|
33.3%
1/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
0.00%
0/3 • 42 days
|
20.0%
1/5 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/3 • 42 days
|
0.00%
0/5 • 42 days
|
0.00%
0/5 • 42 days
|
20.0%
1/5 • 42 days
|
Additional Information
Vice President, Clinical Research
TetraLogic Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place