Trial Outcomes & Findings for A Study of Efalizumab in Participants With Moderate to Severe Chronic Psoriasis Who Have Failed, Have a Contraindication to, or Are Intolerant of Other Systemic Therapies (NCT NCT00249808)

Last Updated: 2018-03-20

Results Overview

The PGA assesses the global response of all psoriatic lesions to therapy by comparing the participant's present condition to baseline. PGA response includes: Cleared (100 percent \[%\] improvement; remission of all clinical signs and symptoms, except for residual manifestations such as mild erythema); Excellent (75% to 99% improvement of all clinical signs and symptoms, except for residual manifestations such as mild erythema); Good (50% to 74% improvement of all clinical signs and symptoms); Fair (25% to 49% improvement of all clinical signs and symptoms); Slight (1% to 24% improvement of all clinical signs and symptoms); Unchanged (clinical signs and symptoms unchanged); Worse (clinical signs and symptoms deteriorated). Percentage of participants with PGA ratings of Good or Better (i.e, Good, Excellent or Cleared) are reported.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

1266 participants

Primary outcome timeframe

Week 12

Results posted on

2018-03-20

Participant Flow

Participant milestones

Participant milestones
Measure	Efalizumab Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (first treatment \[FT\]). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Overall Study STARTED	1266
Overall Study COMPLETED	1084
Overall Study NOT COMPLETED	182

Reasons for withdrawal

Reasons for withdrawal
Measure	Efalizumab Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (first treatment \[FT\]). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Overall Study Other	27
Overall Study Protocol Violation	3
Overall Study Lost to Follow-up	8
Overall Study Adverse Event	81
Overall Study Lack of Efficacy	52
Overall Study Did Not Provide Post-Baseline Data	11

Baseline Characteristics

A Study of Efalizumab in Participants With Moderate to Severe Chronic Psoriasis Who Have Failed, Have a Contraindication to, or Are Intolerant of Other Systemic Therapies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Efalizumab n=1255 Participants Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (FT). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Age, Continuous	46.2 years STANDARD_DEVIATION 12.9 • n=5 Participants
Sex: Female, Male Female	395 Participants n=5 Participants
Sex: Female, Male Male	860 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 12

Population: ITT population.

Outcome measures

Outcome measures
Measure	Efalizumab n=1255 Participants Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (FT). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Percentage of Participants With Physician's Global Assessment (PGA) Ratings of Good or Better (FT)	68.0 percentage of participants Interval 65.3 to 70.5

SECONDARY outcome

Timeframe: Up to 8 weeks after end of FT (up to Week 20)

Population: Observation population included all participants had stopped treatment with efalizumab following the FT (Week 12) and who had responded to treatment during (PGA good or better ) FT. Here, overall number of participants analyzed = participants who were evaluable for this outcome.

Rebound was defined as worsening of disease as assessed by Psoriasis Area and Severity Index (PASI) score \>125% of baseline or new pustular, erythrodermic, or more inflammatory psoriasis occurring within 2 months of stopping therapy. PASI is an instrument used to assess the extent of cutaneous psoriasis and to measure the effects of therapy. The PASI divides the body into four anatomical regions: head, trunk, upper limbs, and lower limbs. For each region, the evaluator assesses the severity of erythema, induration/thickness and scaling and determines the percentage of the region affected by disease. A numerical PASI score is derived that evaluates the severity of symptoms in terms of the total body surface area affected. Total PASI score ranges from 0 to 72, with higher scores indicating more severe disease.

Outcome measures

Outcome measures
Measure	Efalizumab n=135 Participants Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (FT). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Percentage of Participants With Psoriasis Rebound	11.0 percentage of participants Interval 65.3 to 70.5

SECONDARY outcome

Timeframe: During study (40 weeks)

Population: ITT population. Here, overall number of participants analyzed = participants who failed to respond (PGA less than good) at Week 12.

Exacerbation was defined as disease worsening either during or after treatment which was more inflammatory in nature compared to baseline and occurred either within pre-existing plaques, at previously uninvolved sites, or as new morphologies of disease.

Outcome measures

Outcome measures
Measure	Efalizumab n=402 Participants Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (FT). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Percentage of Participants With Psoriasis Exacerbation	12.9 percentage of participants Interval 65.3 to 70.5

Adverse Events

Efalizumab

Serious events: 60 serious events

Other events: 623 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Efalizumab n=1266 participants at risk Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (FT). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Skin and subcutaneous tissue disorders Psoriasis	1.1% 14/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Erythrodermic Psoriasis	0.24% 3/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Dermatitis Allergic	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Dermatitis Exfoliative	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Erythema	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Follicular Mucinosis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Rash	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Skin and subcutaneous tissue disorders Skin Exfoliation	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Psoriatic Arthropathy	0.47% 6/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Arthralgia	0.16% 2/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Arthritis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Monarthritis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Rotator Cuff Syndrome	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Synovial Cyst	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Meningitis Aseptic	0.16% 2/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Abscess Limb	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Bronchitis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Erysipelas	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Oesophageal Candidiasis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Pneumonia Bacterial	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Skin Bacterial Infection	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Upper Respiratory Tract Infection	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Infections and infestations Urinary Tract Infection	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Pyrexia	0.32% 4/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Asthenia	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Face Oedema	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Influenza Like Illness	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Injection Site Reaction	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Acute Myocardial Infarction	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Angina Pectoris	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Angina Unstable	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Atrial Fibrillation	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Coronary Artery Disease	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Coronary Artery Stenosis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Cardiac disorders Myocardial Infarction	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Gastrointestinal disorders Abdominal Pain	0.16% 2/1266 Safety population included all participants who received at least one injection of efalizumab.
Gastrointestinal disorders Colitis Ulcerative	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Gastrointestinal disorders Enteritis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Gastrointestinal disorders Oesophagitis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Gastrointestinal disorders Small Intestinal Obstruction	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Nervous system disorders Headache	0.16% 2/1266 Safety population included all participants who received at least one injection of efalizumab.
Nervous system disorders Cerebrovascular Accident	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Nervous system disorders Peripheral Sensorimotor Neuropathy	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Nervous system disorders Syncope	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Immune system disorders Drug Rash With Eosinophilia And Systemic Symptoms	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Immune system disorders Hypersensitivity	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Injury, poisoning and procedural complications Accidental Overdose	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Injury, poisoning and procedural complications Wound Dehiscence	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon Adenoma	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous Cell Carcinoma	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Ear and labyrinth disorders Vertigo	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Eye disorders Optic Ischaemic Neuropathy	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Hepatobiliary disorders Cholecystitis	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Psychiatric disorders Depression	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.08% 1/1266 Safety population included all participants who received at least one injection of efalizumab.

Other adverse events

Other adverse events
Measure	Efalizumab n=1266 participants at risk Participants received efalizumab 1.0 mg/kg (with an initial conditioning dose of 0.7 mg/kg) once weekly by subcutaneous injection for 12 weeks (FT). Depending on the response at Week 12, participants could have received additional 8 to 12 weekly injections of efalizumab 1.0 mg/kg.
Nervous system disorders Headache	25.5% 323/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Pyrexia	9.6% 121/1266 Safety population included all participants who received at least one injection of efalizumab.
General disorders Influenza Like Illness	8.5% 108/1266 Safety population included all participants who received at least one injection of efalizumab.
Musculoskeletal and connective tissue disorders Arthralgia	5.6% 71/1266 Safety population included all participants who received at least one injection of efalizumab.

Additional Information

Merck KGaA Communication Center,

Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany

Phone: 496151725200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place