Warm Autoimmune Hemolytic AnemiaPhase 1/2NCT03075878
A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA)
A Phase 1/2 study testing ALXN1830 in people with Warm Autoimmune Hemolytic Anemia, run by Alexion Pharmaceuticals, Inc. at 10 sites in 2 countries.
Trial summary
This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Warm Autoimmune Hemolytic Anemia
- What is being tested?
- ALXN1830
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 10 Jan 2018to 6 Aug 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants had to meet the following criteria to be included:
- Willing and able to read, understand, and sign an informed consent form
- Confirmed diagnosis of WAIHA by enrolling physician
- Must have used medically acceptable contraception
Who cannot join
- Participants who met any of the following criteria were excluded:
- Participant unable or unwilling to comply with the protocol
- Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)
- Positive for human immunodeficiency virus or hepatitis C antibody
- Positive for hepatitis B surface antigen
- Any exposure to an investigational drug or device within the 30 days prior to screening
Show all 9Show fewer
- Intravenous immunoglobulin treatment within 30 days of screening
- Plasmapheresis or immunoadsorption within 30 days of screening
- Participant had any current medical condition that, in the opinion of the Investigator, may have compromised their safety or compliance, precluded successful conduct of the study, or interfered with interpretation of the results
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States9 sites
Alexion Study Site
Boston, Massachusetts, 02114
Alexion Study Site
Cleveland, Ohio, 44106
Alexion Study Site
Los Angeles, California, 90033
Alexion Study Site
Philadelphia, Pennsylvania, 19104
Alexion Study Site
Pittsburgh, Pennsylvania, 15232
Alexion Study Site
Pittsfield, Massachusetts, 01201
Alexion Study Site
Rochester, Minnesota, 55905
Alexion Study Site
San Francisco, California, 94143
Alexion Study Site
Seattle, Washington, 98195
About this study
Official title: A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)
This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA.
Read the detailed description
This study planned to evaluate 2 cohorts: Cohort 1, up to 8 participants to receive IV doses of ALXN1830 (SYNT001 Dose 1); Cohort 2, up to 12 participants to receive IV doses of ALXN1830 (SYNT001 Dose 2).
This study was terminated after the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy were characterized in participants with WAIHA in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2.
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Being tested
- ALXN1830
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1830Drug
Also called SYNT001
Administered via IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: ALXN1830 | Experimental | SYNT001 Dose 1 Receives: Drug: ALXN1830 |
| Cohort 2: ALXN1830 | Experimental | SYNT001 Dose 2 Receives: Drug: ALXN1830 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
A TEAE was defined as any adverse event 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" 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. All serious TEAEs were not considered related to the study drug.
Measured: Day 0 (after first dose) through Day 112
Secondary outcomes (4)
Maximum Serum Concentration (Cmax) On Day 0 And Day 28
The Cmax was determined directly from the concentration-time profile. Starting on Days 0 and 28, serum samples were collected just prior to the start of study drug infusion (predose), at 5 minutes, at 2, 4, 6, 24, and 48 hours, and at 5 days postdose after the end-of-study drug infusion. Results are reported in micrograms/milliliter (ug/mL).
Measured: Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose
Change From Baseline In Reticulocyte Count At Day 33
Reticulocyte count was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in cells/liter (cells*10^12/L). A decrease in reticulocyte count indicated a potential improvement in condition.
Measured: Baseline, Day 33
Change From Baseline In Hemoglobin At Day 33
Hemoglobin was measured as a PD biomarker. Pharmacodynamic samples were collected for analyses throughout the study prior to infusion of study drug for Cohort 1. Measurements for PD biomarkers were derived from the laboratory results. Results are reported in grams/deciliter (g/dL). An increase in hemoglobin indicated a potential improvement in condition.
Measured: Baseline, Day 33
Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level
Immunogenicity analyses are reported for Day 112. Testing was carried out to detect binding antidrug antibodies by anti-ALXN1830 antibody level. Results are reported as the reciprocal of the titer where they cross the study cut point.
Measured: Day 112
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2017
- Study start10 Jan 2018
- Primary completion15 Apr 2019
- Study completion6 Aug 2019
Why it stopped: Focus resources on a planned phase 2/3 study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Warm Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03075878
- Sponsor's protocol ID
- SYNT001-102
- Status last verified
- May 2020
- First submitted
- 3 Mar 2017
- Last updated
- 13 May 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1B/2, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Patients With Warm Autoimmune Hemolytic Anemia (WAIHA)
- ClinicalTrials.gov ID
- NCT03075878
- Acronym / sponsor ID
- SYNT001-102
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Focus resources on a planned phase 2/3 study
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 10 Jan 2018 (actual)
- Primary completion
- 15 Apr 2019 (actual)
- Study completion
- 6 Aug 2019 (actual)
- First posted
- 9 Mar 2017 (actual)
- Results
- Posted 13 May 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This main study objective was to evaluate the safety and tolerability of intravenous (IV) SYNT001 (ALXN1830) in participants with WAIHA.
Detailed description
This study planned to evaluate 2 cohorts: Cohort 1, up to 8 participants to receive IV doses of ALXN1830 (SYNT001 Dose 1); Cohort 2, up to 12 participants to receive IV doses of ALXN1830 (SYNT001 Dose 2).
This study was terminated after the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy were characterized in participants with WAIHA in Cohort 1 (SYNT001 Dose 1), before any participants were enrolled in Cohort 2.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: ALXN1830 | Experimental | Drug: ALXN1830SYNT001 Dose 1 |
| Cohort 2: ALXN1830 | Experimental | Drug: ALXN1830SYNT001 Dose 2 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Willing and able to read, understand, and sign an informed consent form
- Confirmed diagnosis of WAIHA by enrolling physician
- Must have used medically acceptable contraception
Key exclusion criteria (9 listed)
- Participant unable or unwilling to comply with the protocol
- Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)
- Positive for human immunodeficiency virus or hepatitis C antibody
- Positive for hepatitis B surface antigen
- Any exposure to an investigational drug or device within the 30 days prior to screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALXN1830Also: SYNT001Administered via IV infusion. | Drug | Cohort 1: ALXN1830, Cohort 2: ALXN1830 |
Primary outcome
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
Time frame: Day 0 (after first dose) through Day 112
A TEAE was defined as any adverse event 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" 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. All serious TEAEs were not considered related to the study drug.
4 secondary outcomes
Maximum Serum Concentration (Cmax) On Day 0 And Day 28
Time frame: Predose, 5 minutes, 2, 4, 6, 24, and 48 hours, and 5 days postdose
Change From Baseline In Reticulocyte Count At Day 33
Time frame: Baseline, Day 33
Change From Baseline In Hemoglobin At Day 33
Time frame: Baseline, Day 33
Immunogenicity Of ALXN1830 At Day 112, As Assessed By Anti-ALXN1830 Antibody Level
Time frame: Day 112
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ALXN1830 in people with Warm Autoimmune Hemolytic Anemia, run by Alexion Pharmaceuticals, Inc. at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Jordan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 May 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs) (time frame: Day 0 (after first dose) through Day 112). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2020.