A Multi-Center Study to Evaluate the Efficacy and Safety of KX2-391 Ointment 1% on Actinic Keratosis on Face or Scalp (AK004)

Browse trials for

• Keratosis, Actinic 364
• Keratosis 348

Keywords

• Actinic Keratosis
• Keratosis
• Keratosis, Actinic
• Precancerous Conditions
• Neoplasms
• Sun Damaged Skin
• Actinic Keratoses

Study arms

• Experimental
  KX2-391 Ointment 1%

  KX2-391 Ointment was applied once daily for 5 consecutive days on the face or scalp.

  Drug: KX2-391 Ointment 1%

• Placebo comparator
  Placebo

  The Vehicle Ointment was applied once daily for 5 consecutive days on the face or scalp.

  Drug: Placebo

Interventions

• DrugKX2-391 Ointment 1%

  Dose: 1% (250 mg single-use packets); Dosage form: Ointment; Route of administration: Topical

• DrugPlacebo

  Dosage form: Ointment; Route of administration: Topical

Primary outcomes

1. Percentage of Participants With Complete (100%) Clearance of Actinic Keratosis (AK) Lesions

   Complete clearance rate was defined as the percentage of participants at Day 57 with no clinically visible AK lesions in the treatment area.

   Time frame: Day 57

Secondary outcomes

1. Percentage of Participants With Partial Clearance Rate of Actinic Keratosis Lesions at Day 57

   Partial clearance rate of AK lesions was defined as the percentage of participants with a greater than or equal to (\>=) 75% reduction in the number of AK lesions identified at Baseline (Day 1 predose) in the treatment area.

   Time frame: Day 57

2. Overall Change From Baseline in Actinic Keratosis Lesion Counts at Days 8, 15, 29 and 57

   Overall the change from baseline in lesion count at each visit were summarized and reported using descriptive statistics by treatment location (face or scalp).

   Time frame: Days 8, 15, 29 and 57

3. Percentage of Participants With Recurrence of Actinic Keratosis Lesions Who Achieved Complete Clearance at Day 57

   Recurrence rate was estimated based on Kaplan-Meier method, with recurrence define as appearance of any AK lesions in the treatment area, including those recurred or newly identified.

   Time frame: 3, 6, 9 and 12 months post-Day 57

4. Number of Participants With Maximal Post Baseline Local Skin Reaction (LSR)

   Maximal post baseline LSR was defined as the highest grade of any LSR reported at any post baseline visits for a participant. The LSR assessment was an Investigator's (or sub-investigator's) assessment of the following signs on the treatment area: erythema, flaking/scaling, crusting, swelling, vesiculation/pustulation, and erosion/ulceration. The LSRs were graded on a 4-point scale ranging from 0=absent, 1=mild (slightly, barely perceptible), 2=moderate (distinct presence), and 3=severe (marked, intense).

   Time frame: Day 57

5. Number of Participants With Pigmentation and Scarring in the Treatment Area

   Absence or presence of pigmentation (i.e., hypopigmentation and hyperpigmentation) and scarring in the treatment area were assessed.

   Time frame: Baseline (Day 1 predose), Days 5, 8, 15, 29 and 57

6. Number of Participants With Adverse Event (AE), Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Events of Special Interests

   An AE was defined as any untoward medical occurrence in participant which does not necessarily have causal relationship with treatment. An AE was any unfavorable and unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with use of medicinal product, whether or not considered related to medicinal product. An SAE was any untoward medical occurrence that at any dose resulted in death; was life threatening; required persistent/significant disability/incapacity; resulted in initial or prolonged in patient hospitalization; was congenital anomaly/birth defect or otherwise considered medically important. TEAEs (serious and non-serious) were defined as either those AEs with onset after first dose or those pre-existing AEs that worsen after first dose. Events of special interest included skin cancers (including basal cell carcinoma, squamous cell carcinoma, melanoma and their location and treatment area), ocular exposure, overdose, and pregnancy.

   Time frame: Baseline (Day 1 predose) up to Day 57

7. Number of Participants With Adverse Events, Serious Adverse Events, Events of Special Interests Within the Treatment Area After Day 57 and up to 12 Months Post-Day 57

   An AE was defined as any untoward medical occurrence in participant which does not necessarily have causal relationship with treatment. An AE was any unfavorable and unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with use of medicinal product, whether or not considered related to medicinal product. An SAE was any untoward medical occurrence that at any dose resulted in death; was life threatening; required persistent/significant disability/incapacity; resulted in initial or prolonged in patient hospitalization; was congenital anomaly/birth defect or otherwise considered medically important. Events of special interest included skin cancers (including basal cell carcinoma, squamous cell carcinoma, melanoma and their location and treatment area), ocular exposure, overdose, and pregnancy.

   Time frame: From Day 57 up to 12-months post-Day 57

8. Number of Participants With Clinically Significant Safety Observations- Hematology, Blood Chemistry, Urinalysis

   Assessed laboratory parameters included hematology, blood chemistry and urinalysis. Clinical significance and abnormal observations were determined by the investigator.

   Time frame: From Baseline (Day 1 predose) up to Day 57

9. Number of Participants With Clinically Significant Safety Observations- Vital Signs

   Vital signs included measurement of pulse rate, systolic and diastolic blood pressure, respiratory rate, and body temperature. Clinical significance was determined by the investigator.

   Time frame: From Baseline (Day 1 predose) up to Day 57

10. Number of Participants With Clinically Significant Safety Observations- Physical Examination

    A physical examination included weight and height measurements was performed. Clinical significance was determined by the investigator.

    Time frame: From Baseline (Day 1 predose) up to Day 57

11. Number of Participants With Clinically Significant Safety Observations- Electrocardiograms (ECGs)

    ECG parameters included heart rhythm, heart rate, QRS intervals, QT intervals, RR intervals and corrected QT (QTc) intervals. Clinical significance was determined by the investigator.

    Time frame: From Baseline (Day 1 predose) up to Day 57

Baseline characteristics

Publications

• Blauvelt A, Kempers S, Lain E, Schlesinger T, Tyring S, Forman S, Ablon G, Martin G, Wang H, Cutler DL, Fang J, Kwan MR; Phase 3 Tirbanibulin for Actinic Keratosis Group. Phase 3 Trials of Tirbanibulin Ointment for Actinic Keratosis. N Engl J Med. 2021 Feb 11;384(6):512-520. doi: 10.1056/NEJMoa2024040. PubMed 33567191 ↗

Study documents

• Study protocol · Feb 18, 2018
• Statistical analysis plan · Jun 4, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No