Trial Outcomes & Findings for Spesolimab (BI 655130) Single Dose in Generalized Pustular Psoriasis (NCT NCT02978690)
NCT ID: NCT02978690
Last Updated: 2023-08-21
Results Overview
Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.
COMPLETED
PHASE1
7 participants
Up to 140 days from the administration of spesolimab.
2023-08-21
Participant Flow
At the screening visit, patients had to be on stable maintenance treatment (for at least 4 weeks) with retinoids and/or methotrexate or not to receive any maintenance therapy at all. All patients were to use an effective birth control method throughout the trial.
This exploratory, Phase I, multi-centre, multi-national trial in male and female patients with a flare of Generalised Pustular Psoriasis (GPP) applied an open-label design.
Participant milestones
| Measure |
Spesolimab
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Overall Study
STARTED
|
7
|
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Overall Study
COMPLETED
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7
|
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Overall Study
NOT COMPLETED
|
0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Spesolimab (BI 655130) Single Dose in Generalized Pustular Psoriasis
Baseline characteristics by cohort
| Measure |
Spesolimab
n=7 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Age, Continuous
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38.6 years
STANDARD_DEVIATION 13.8 • n=5 Participants
|
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Sex: Female, Male
Female
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4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
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3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
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2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Up to 140 days from the administration of spesolimab.Population: Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.
Outcome measures
| Measure |
Spesolimab
n=7 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
|
Percentage of Patients With Adverse Reactions, Defined as Drug-related Adverse Events (AE)
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57.1 percentage of participants
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SECONDARY outcome
Timeframe: At baseline and at Week 2.Population: Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.
The GPPASI is an adaptation for Generalized Pustular Psoriasis (GPP) patients of the Psoriasis Area and Severity Index (PASI), an established measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool which provides a numeric scoring for patients overall GPP disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin that is affected and the severity of erythema, pustules, and scaling (desquamation) over four body regions. % GPPASI change from baseline=100\* (GPPASI at baseline - GPPASI at post-baseline visit)/(GPPASI at baseline). For %GPPASI change, positive numbers show reduction in GPPASI with higher values representing a larger improvement or recovery of disease, while negative numbers show an increase in GPPASI, i.e. worsening of disease.
Outcome measures
| Measure |
Spesolimab
n=7 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Percent Change From Baseline in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) Total Score at Week 2
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73.2 percent GPPASI change
Standard Deviation 16.2
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SECONDARY outcome
Timeframe: At Week 2.Population: Full Analysis Set No Response Imputed (FAS NRI). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. NRI: If there were data at the visits both immediately before and immediately after the visit with a missing outcome, a failure to achieve response imputed.
GPPGA relies on clinical assessment of the GPP patient's skin presentation. It is a modified Physician Global Assessment (PGA), a physician's assessment of psoriatic lesions, which has been adapted to the evaluation of GPP patients. The investigator (or qualified site personnel) scores the erythema, pustules and scaling of all psoriatic lesions from 0 - 4. Each component is graded separately, the average is calculated and the final GPPGA is determined from this composite score. A lower score then indicates a lesser severity, with 0 being clear and 1 being almost clear. To receive a score of 0 or 1, the patient should be afebrile, in addition to skin presentation requirements.
Outcome measures
| Measure |
Spesolimab
n=7 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Proportion of Patients With Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Total Score of 0 (Clear) or 1 (Almost Clear) at Week 2
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0.714 proportion of patients
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SECONDARY outcome
Timeframe: At baseline and at Week 2.Population: Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.
Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scale score at Week 2 is presented. The FACIT-Fatigue scale is a brief and reliable instrument for monitoring fatigue and its effects on patients. It is a comprehensive compilation of questions that measure health-related quality of life in patients with chronic illnesses. It comprises 13 questions, the responses to which are each recorded on a 5-point Likert scale. Scores range from 0 to 52, with lower scores representing greater fatigue, i.e. higher changes from baseline indicate higher improvement (compared to baseline)
Outcome measures
| Measure |
Spesolimab
n=7 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
|
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Week 2
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12.3 units on a scale
Standard Deviation 10.1
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SECONDARY outcome
Timeframe: At baseline and at Week 2.Population: Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.
The pain VAS is a unidimensional measure of pain intensity. It is a continuous scale comprised of a horizontal or vertical line, usually 10 centimeters (100 millimeters (mm) in length, anchored by word descriptors at each end ('no pain', 'very severe pain'). The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the 'no pain' anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity, i.e., a negative change from baseline indicates an improvement (compared to baseline).
Outcome measures
| Measure |
Spesolimab
n=7 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 2
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-45.9 units on a scale
Standard Deviation 32.3
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SECONDARY outcome
Timeframe: Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.Population: Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).
Area under the concentration-time curve of spesolimab in plasma over the time interval from 0 extrapolated to infinity (AUC0-∞) is reported.
Outcome measures
| Measure |
Spesolimab
n=6 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Area Under the Concentration-time Curve of Spesolimab in Plasma Over the Time Interval From 0 Extrapolated to Infinity (AUC0-∞)
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2350 microgram (μg)·day/ milliliter (mL)
Geometric Coefficient of Variation 41.1
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SECONDARY outcome
Timeframe: Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.Population: Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).
Maximum measured concentration of spesolimab in plasma (Cmax) is reported.
Outcome measures
| Measure |
Spesolimab
n=6 Participants
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Maximum Measured Concentration of Spesolimab in Plasma (Cmax)
|
203 microgram (μg)/milliliter (mL)
Geometric Coefficient of Variation 11.1
|
Adverse Events
Spesolimab
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Spesolimab
n=7 participants at risk
Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.
Patients were to be followed up for 140 days (20 weeks) after dosing.
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|---|---|
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Blood and lymphatic system disorders
Eosinophilia
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28.6%
2/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
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Gastrointestinal disorders
Abdominal pain
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
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|
Gastrointestinal disorders
Diarrhoea
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Gastrointestinal disorders
Vomiting
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
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General disorders
Asthenia
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
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General disorders
Chills
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28.6%
2/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
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General disorders
Oedema peripheral
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28.6%
2/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
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General disorders
Pain
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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General disorders
Pyrexia
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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Infections and infestations
Pharyngitis
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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Infections and infestations
Rash pustular
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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Infections and infestations
Upper respiratory tract infection
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28.6%
2/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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Infections and infestations
Urinary tract infection
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Infections and infestations
Viral tonsillitis
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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Injury, poisoning and procedural complications
Infusion related reaction
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
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Investigations
Blood creatine phosphokinase increased
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14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
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42.9%
3/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Renal and urinary disorders
Haematuria
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Renal and urinary disorders
Nephrolithiasis
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Renal and urinary disorders
Renal cyst
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Reproductive system and breast disorders
Pelvic pain
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Skin and subcutaneous tissue disorders
Eczema
|
28.6%
2/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Skin and subcutaneous tissue disorders
Pustular psoriasis
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Skin and subcutaneous tissue disorders
Skin fragility
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
|
Skin and subcutaneous tissue disorders
Skin striae
|
14.3%
1/7 • Up to 140 days from the administration of spesolimab.
Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).
|
Additional Information
Boehringer Ingelheim, Call Center
Boehringer Ingelheim
Results disclosure agreements
- Principal investigator is a sponsor employee Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER