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Trial record 1 of 1 for:    SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
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A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)

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ClinicalTrials.gov Identifier: NCT03075904
Recruitment Status : Terminated (Study objectives achieved)
First Posted : March 9, 2017
Results First Posted : February 5, 2020
Last Update Posted : February 5, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pemphigus
Pemphigus Vulgaris
Pemphigus Foliaceus
Intervention Drug: ALXN1830
Enrollment 8
Recruitment Details  
Pre-assignment Details This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized at a single dose level (10 milligram/kilogram [mg/kg]) in Cohort 1, before any participants were enrolled in Cohort 2. This results disclosure is for Cohort 1 only.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Period Title: Overall Study
Started 8
Received at Least 1 Dose of Study Drug 8
Completed [1] 4
Not Completed 4
Reason Not Completed
Physician Decision             3
Need for Medication not Permitted             1
[1]
All 8 participants received all 5 weekly doses of ALXN1830 10 mg/kg in the Treatment Period.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
All participants who were enrolled in the study.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
51.4  (16.43)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
5
  62.5%
Male
3
  37.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
8
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
3
  37.5%
White
5
  62.5%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Type of Pemphigus  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Vulgaris
7
  87.5%
Foliaceus
1
  12.5%
Age at Diagnosis of Pemphigus  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
41.3  (18.90)
1.Primary Outcome
Title Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Hide Description A TEAE was defined as any adverse event (AE) that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" (Grade 3) if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Time Frame Day 1 (after first dose) through Day 112
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least 1 dose of study drug.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description:
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Overall Number of Participants Analyzed 8
Measure Type: Count of Participants
Unit of Measure: Participants
TEAEs
7
  87.5%
Serious TEAEs
1
  12.5%
Discontinuations due to TEAEs
0
   0.0%
Deaths
0
   0.0%
2.Secondary Outcome
Title Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline
Hide Description Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean serum total IgG levels from Baseline observed during the study is presented.
Time Frame Baseline through Day 112
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description:
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: percentage of reduction
57.3
3.Secondary Outcome
Title Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline
Hide Description Pemphigus severity and disease activity was measured using the PDAI in regions where a validated questionnaire was available. The PDAI was administered during Treatment Period and Follow-up Period. PDAI total activity was comprised of scores for the skin, mucous membrane, and scalp subscales. The investigator determined a PDAI score as 0 to 250 points for total activity score (0 to 120 for skin, 0 to 10 for scalp, and 0 to 120 for mucosa). A higher score indicated higher impact on skin disease. The maximum percent reduction in PDAI total activity score from Baseline observed during the study is presented.
Time Frame Baseline through Day 112
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description:
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: percentage of reduction
45.7
4.Secondary Outcome
Title Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline
Hide Description Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean CIC levels from Baseline observed during the study is presented.
Time Frame Baseline through Day 112
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description:
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: percentage of reduction
51.4
5.Secondary Outcome
Title Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline
Hide Description Pharmacodynamic samples were collected for analysis throughout the study. The maximum percent reduction of mean anti-Dsg 1 and 3 antibodies from Baseline observed during the study is presented.
Time Frame Baseline through Day 112
Hide Outcome Measure Data
Hide Analysis Population Description
All participants who received at least 1 dose of study drug and had postdose pharmacodynamic data available.
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description:
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: percentage of reduction
anti-Dsg 1 8.9
anti-Dsg 3 20.4
Time Frame Baseline through Day 112
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1: ALXN1830
Hide Arm/Group Description Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
All-Cause Mortality
Cohort 1: ALXN1830
Affected / at Risk (%)
Total   0/8 (0.00%) 
Hide Serious Adverse Events
Cohort 1: ALXN1830
Affected / at Risk (%)
Total   1/8 (12.50%) 
General disorders   
Disease progression  1 [1]  1/8 (12.50%) 
Renal and urinary disorders   
Acute kidney injury  1 [1]  1/8 (12.50%) 
1
Term from vocabulary, MedDRA 19.1
Indicates events were collected by systematic assessment
[1]
Resolved and was considered to be not related to the study drug by the Investigator.
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1: ALXN1830
Affected / at Risk (%)
Total   7/8 (87.50%) 
Eye disorders   
Dry eye  1  1/8 (12.50%) 
Gastrointestinal disorders   
Diarrhoea  1  1/8 (12.50%) 
Nausea  1  2/8 (25.00%) 
Vomiting  1  1/8 (12.50%) 
General disorders   
Fatigue  1  2/8 (25.00%) 
Malaise  1  1/8 (12.50%) 
Pyrexia  1  1/8 (12.50%) 
Hepatobiliary disorders   
Hepatic cyst  1  1/8 (12.50%) 
Infections and infestations   
Herpes simplex  1  1/8 (12.50%) 
Staphylococcal infection  1  1/8 (12.50%) 
Tinea barbae  1  1/8 (12.50%) 
Injury, poisoning and procedural complications   
Infusion related reaction  1  1/8 (12.50%) 
Investigations   
Urine analysis abnormal  1  1/8 (12.50%) 
Nervous system disorders   
Headache  1  6/8 (75.00%) 
Sinus headache  1  1/8 (12.50%) 
Renal and urinary disorders   
Acute kidney injury  1  1/8 (12.50%) 
Skin and subcutaneous tissue disorders   
Hyperhidrosis  1  1/8 (12.50%) 
Rash  1  1/8 (12.50%) 
Vascular disorders   
Pallor  1  1/8 (12.50%) 
1
Term from vocabulary, MedDRA 19.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding study results for a period that is less than or equal to 60 days from the date that the communication is submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot unilaterally extend the embargo.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals, Inc.
Organization: Alexion Pharmaceuticals, Inc.
Phone: 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03075904    
Other Study ID Numbers: SYNT001-103
First Submitted: March 6, 2017
First Posted: March 9, 2017
Results First Submitted: January 16, 2020
Results First Posted: February 5, 2020
Last Update Posted: February 5, 2020