Generalized Myasthenia GravisPhase 2NCT04982289
Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis
A Phase 2 study testing ALXN1830 in people with Generalized Myasthenia Gravis, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- ALXN1830
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2022to 31 Jan 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Diagnosis of myasthenia gravis.
- Positive serologic test for anti-acetylcholine receptor antibodies.
- Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at Screening.
- MG-ADL profile must be ≥ 5.
- Participants receiving stable treatment with azathioprine; other immunosuppressive therapies.
- Total IgG level at Screening ≥ 600 milligrams/deciliter.
Who cannot join
- History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to Screening.
- Any untreated thymic malignancy, carcinoma, or thymoma.
- Intravenous immunoglobulin within the 6 weeks, and/or use of plasmapheresis/plasma exchange prior to randomization (Day 1).
- Use of rituximab within the 3 months (90 days) prior to Screening.
- Participants who have received previous treatment with any biological agent or other anti-neonatal fragment crystallizable receptor therapy within 5 half-lives or 90 days after last dose (whichever is longer).
- Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the participant's full participation in the study, pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2a Randomized, Double-blind, Placebo-controlled, Parallel Groups, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALXN1830 Administered Subcutaneously in Adult Patients With Generalized Myasthenia Gravis
This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG).
Read the detailed description
Participants have the option to enroll in an Open-label Extension (OLE) Period to receive ALXN1830 up to 58 weeks (including follow-up period).
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- ALXN1830, Placebo
- Keywords
- Generalized Myasthenia Gravis
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN1830Drug
Administered as an SC infusion.
PlaceboOther
Administered as an SC infusion.
| Study group | Role | What they receive |
|---|---|---|
| ALXN1830 Dosing Arm 1 | Experimental | Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks. Receives: Drug: ALXN1830 |
| ALXN1830 Dosing Arm 2 | Experimental | Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks. Receives: Drug: ALXN1830 |
| ALXN1830 Dosing Arm 3 | Experimental | Participants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks. Receives: Drug: ALXN1830; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24
Measured: Up to Week 24
AEs And SAEs Up To Week 82
Measured: Up to Week 82 (OLE)
Change From Baseline In Serum Total Immunoglobulin G (IgG)
Measured: Up to Week 24
Secondary outcomes (9)
Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score
Measured: Up to Week 24
Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score
Measured: Up to Week 24
Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks
Measured: Up to Week 24
Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks
Measured: Up to Week 8
Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score
Measured: Up to Week 24
Serum Trough Concentrations Of ALXN1830
Measured: Up to Week 24
Change From Baseline In IgG Subtypes
Measured: Up to Week 24
Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830
Measured: Up to Week 24
Titers Of ADA And Nab Against ALXN1830
Measured: Up to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2021
- Study start1 Jan 2022 (estimated)
- Primary completion31 Jan 2023 (estimated)
- Study completion31 Jan 2024 (estimated)
Why it stopped: Sponsor decision to terminate program
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized Myasthenia GravisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 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
- NCT04982289
- Sponsor's protocol ID
- ALXN1830-MG-201
- Status last verified
- Feb 2022
- First submitted
- 21 Jun 2021
- Last updated
- 11 Feb 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Randomized, Double-blind, Placebo-controlled, Parallel Groups, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALXN1830 Administered Subcutaneously in Adult Patients With Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT04982289
- Acronym / sponsor ID
- ALXN1830-MG-201
- Conditions
- Generalized Myasthenia Gravis
- Keywords
- Generalized Myasthenia Gravis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Sponsor decision to terminate program
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Jan 2022 (estimated)
- Primary completion
- 31 Jan 2023 (estimated)
- Study completion
- 31 Jan 2024 (estimated)
- First posted
- 29 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics (PD), immunogenicity, and efficacy of subcutaneous (SC) ALXN1830 in adults with generalized myasthenia gravis (gMG).
Detailed description
Participants have the option to enroll in an Open-label Extension (OLE) Period to receive ALXN1830 up to 58 weeks (including follow-up period).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ALXN1830 Dosing Arm 1 | Experimental | Drug: ALXN1830Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks. |
| ALXN1830 Dosing Arm 2 | Experimental | Drug: ALXN1830Participants will receive ALXN1830. Treatment will be received for 16 weeks followed by an Observation Period (no treatment) for 8 weeks. |
| ALXN1830 Dosing Arm 3 | Experimental | Drug: ALXN1830, Other: PlaceboParticipants will receive placebo for 8 weeks, then ALXN1830 for 8 weeks, followed by an Observation Period (no treatment) for 8 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of myasthenia gravis.
- Positive serologic test for anti-acetylcholine receptor antibodies.
- Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at Screening.
- MG-ADL profile must be ≥ 5.
- Participants receiving stable treatment with azathioprine; other immunosuppressive therapies.
Key exclusion criteria (6 listed)
- History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to Screening.
- Any untreated thymic malignancy, carcinoma, or thymoma.
- Intravenous immunoglobulin within the 6 weeks, and/or use of plasmapheresis/plasma exchange prior to randomization (Day 1).
- Use of rituximab within the 3 months (90 days) prior to Screening.
- Participants who have received previous treatment with any biological agent or other anti-neonatal fragment crystallizable receptor therapy within 5 half-lives or 90 days after last dose (whichever is longer).
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 |
|---|---|---|
| ALXN1830Administered as an SC infusion. | Drug | ALXN1830 Dosing Arm 1, ALXN1830 Dosing Arm 2, ALXN1830 Dosing Arm 3 |
| PlaceboAdministered as an SC infusion. | Other | ALXN1830 Dosing Arm 3 |
Primary outcome
Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24
Time frame: Up to Week 24
AEs And SAEs Up To Week 82
Time frame: Up to Week 82 (OLE)
Change From Baseline In Serum Total Immunoglobulin G (IgG)
Time frame: Up to Week 24
9 secondary outcomes
Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score
Time frame: Up to Week 24
Change From Baseline In Quantitative Myasthenia Gravis (QMG) Score
Time frame: Up to Week 24
Number Of Participants With At Least A 2-point Improvement In The MG-ADL Score Over 4 Consecutive Weeks
Time frame: Up to Week 24
Number Of Participants With At Least A 3-point Improvement In The QMG Score Over 4 Consecutive Weeks
Time frame: Up to Week 8
Change From Baseline In Neurological Disorders Fatigue Questionnaire (Neuro-Qol) Fatigue Score
Time frame: Up to Week 24
Serum Trough Concentrations Of ALXN1830
Time frame: Up to Week 24
Change From Baseline In IgG Subtypes
Time frame: Up to Week 24
Incidence Of Anti-drug Antibodies (ADA) And Neutralizing Antibodies (Nab) Against ALXN1830
Time frame: Up to Week 24
Titers Of ADA And Nab Against ALXN1830
Time frame: Up to Week 24
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2022.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALXN1830 in people with Generalized Myasthenia Gravis, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
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 6 inclusion and 6 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: withdrawn, as of the sponsor's last update on 11 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 phase mean?
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Adverse Events (AEs) And Serious Adverse Events (SAEs) Up To Week 24 (time frame: Up to Week 24). The study lists 3 primary outcomes in all. 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 11 Feb 2022.