A Phase 1/2 interventional study of ALXN1830 in Pemphigus, Pemphigus Vulgaris and Pemphigus Foliaceus, sponsored by Alexion Pharmaceuticals, Inc.. Terminated at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-02-05.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 1/2, Interventional, and Treatment
This was a multicenter, open-label safety study to determine the dose regimen of SYNT001 (ALXN1830) administered intravenously in participants with pemphigus (vulgaris or foliaceus).
This study planned to evaluate 2 cohorts: up to 8 participants to receive 5 weekly intravenous (IV) doses of ALXN1830 at 10 milligram/kilogram (mg/kg) (Cohort 1) and up to 12 participants to receive 3 x 30 mg/kg weekly doses of ALXN1830 IV (loading) followed by 5 x 10 mg/kg doses of ALXN1830 IV every other week or 10 weekly doses of ALXN1830 IV (maintenance) (Cohort 2).
This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized in participants with pemphigus at a single dose level (10 mg/kg) in Cohort 1, before any participants were enrolled in Cohort 2.
The study consisted of 3 periods: Screening, Treatment, and Follow-Up.
76 studies on the registry are indexed under Pemphigus; 18 are open to participants now.
This study's enrollment of 8 is below the median of 30 across 53 interventional studies indexed under Pemphigus.
Browse Pemphigus studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted before Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants must have meet the following criteria to be included:
Exclusion Criteria:
Participants meeting any of the following criteria were excluded:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
Drug: ALXN1830
Participants were to receive 3 doses of ALXN1830 30 mg/kg administered weekly (loading) followed by 5 doses of ALXN1830 10 mg/kg administered every other week or 10 weekly doses of ALXN1830 IV (maintenance).
Drug: ALXN1830
Administered via IV infusion.
Also known as: SYNT001
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
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
Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline
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
Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline
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
Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline
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
Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline
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
| Milestone | Cohort 1: ALXN1830 |
|---|---|
| Started | 8 |
| Received at least 1 dose of study drug | 8 |
| Completed | 4 |
| Not completed | 4 |
| Withdrew: Physician decision | 3 |
| Withdrew: Need for medication not permitted | 1 |
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.
| Participants | Cohort 1: ALXN1830 |
|---|---|
| TEAEs | 7 |
| Serious TEAEs | 1 |
| Discontinuations due to TEAEs | 0 |
| Deaths | 0 |
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.
| percentage of reduction | Cohort 1: ALXN1830 |
|---|---|
| Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline | 57.3 |
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.
| percentage of reduction | Cohort 1: ALXN1830 |
|---|---|
| Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline | 45.7 |
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.
| percentage of reduction | Cohort 1: ALXN1830 |
|---|---|
| Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline | 51.4 |
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.
| percentage of reduction | Cohort 1: ALXN1830 |
|---|---|
| anti-Dsg 1 | 8.9 |
| anti-Dsg 3 | 20.4 |
Collected over Baseline through Day 112. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: ALXN1830 | 0/8 (0%) | 1/8 (12.5%) | 7/8 (87.5%) |
| Event | Cohort 1: ALXN1830 |
|---|---|
| Disease progressionGeneral disorders | 1/8 |
| Acute kidney injuryRenal and urinary disorders | 1/8 |
| Event | Cohort 1: ALXN1830 |
|---|---|
| HeadacheNervous system disorders | 6/8 |
| NauseaGastrointestinal disorders | 2/8 |
| FatigueGeneral disorders | 2/8 |
| Dry eyeEye disorders | 1/8 |
| DiarrhoeaGastrointestinal disorders | 1/8 |
| VomitingGastrointestinal disorders | 1/8 |
| MalaiseGeneral disorders | 1/8 |
| PyrexiaGeneral disorders | 1/8 |
| Hepatic cystHepatobiliary disorders | 1/8 |
| Herpes simplexInfections and infestations | 1/8 |
All participants who were enrolled in the study.
| Age, Continuous(years) | Cohort 1: ALXN1830 |
|---|---|
| Mean | 51.4 ± 16.43 |
| Sex: Female, Male(Participants) | Cohort 1: ALXN1830 |
|---|---|
| Female | 5 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: ALXN1830 |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 8 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1: ALXN1830 |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 5 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Type of Pemphigus(Participants) | Cohort 1: ALXN1830 |
|---|---|
| Vulgaris | 7 |
| Foliaceus | 1 |
| Age at Diagnosis of Pemphigus(years) | Cohort 1: ALXN1830 |
|---|---|
| Mean | 41.3 ± 18.90 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jul 2017 to primary completion in Jan 2019, 17 months.
Results were first posted in Feb 2020, 1 year after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jan 2020. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Orilanolimab
Alexion Pharmaceuticals, Inc. →