Primary Immune ThrombocytopeniaPhase 2NCT03102593
A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP
A Phase 2 study testing ARGX-113 in people with Primary Immune Thrombocytopenia, run by argenx at 30 sites in 10 countries.
Trial summary
The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- ARGX-113
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 38 participants
- Where
- 30 sitesin 10 countries
- Study dates
- 13 Mar 2017to 9 Apr 2019
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients aged ≥ 18 to ≤ 85 years.
- Must receive SoC treatment for ITP that has been stable in dose and frequency for at least 4 weeks prior to Screening. SoC may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist.
- Confirmed diagnosis of ITP with blood platelet counts < 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.
Who cannot join
- Use of anticoagulants, or any drug with antiplatelet effect within 3 weeks prior to Screening.
- Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
- Use of Intravenous immunoglobulin G (IVIg) or anti-D immunoglobulin treatment within 4 weeks prior to screening.
- Use of recombinant thrombopoietin at any time.
- Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
- Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine, danazol, mycophenolate mofetil, mycophenolate sodium which must have been stable for at least 4 weeks prior to Screening.
Show all 11Show fewer
- Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
- Received vaccinations within 4 weeks prior to Screening or planned during the study.
- At Screening, have clinically significant laboratory abnormalities
- History of any thrombotic or embolic event within 12 months prior to Screening.
- Known auto-immune disease other than ITP.
Trial locations and contacts
30 sites in 10 countries.
30 sites in 10 countries
Hungary6 sites
Budapest
Budapest
Debrecen
Debrecen
Gyula
Gyula
Kaposvar
Kaposvár
Nyiregyhaza
Nyíregyháza
Pecs
Pécs
Ukraine4 sites
Dnipro
Dnipro
Ivano-Frankivsk
Ivano-Frankivsk
Nikolaev
Mykolayiv
Uzhgorod
Uzhhorod
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia
The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.
Read the detailed description
This is a randomized, double-blind, placebo-controlled Phase II study in which approximately 36 patients will be randomized in a 1:1:1 ratio to receive either ARGX-113 Dose A, or ARGX-113 Dose B body weight or placebo in 4 infusions administered 1-week apart in addition to Standard-of-Care (SoC) treatment. Patients aged 18 to 85 years (inclusive) with confirmed primary immune thrombocytopenia (ITP) who have a platelet count ˂ 30 × 109/L and who are receiving oral corticosteroids and/or permitted oral immunosuppressants and/or Thrombopoietin receptor (TPO-R) agonist as SoC which must be maintained on a stable dose and frequency for at least 4 weeks prior to Screening.
The study will include a 2-week Screening, a 3-week Treatment period, and an 21-week follow-up (FU) period. The study is followed by an open label period where patients will be given the option to be treated with ARGX-113 Dose A in cycles of 4 weekly infusions with a minimum of 4 weeks apart. Patients may receive rescue therapy during the study at the discretion the investigator when deemed medically necessary.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- ARGX-113, Placebo
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARGX-113Drug
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
PlaceboOther
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
| Study group | Role | What they receive |
|---|---|---|
| ARGX-113 Dose A + SoC | Experimental | Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo Receives: Drug: ARGX-113 |
| ARGX-113 Dose B +SoC | Experimental | Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo Receives: Drug: ARGX-113 |
| Placebo + SoC | Placebo comparator | Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of serious adverse events (SAEs).
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
Measured: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
Secondary outcomes (1)
Frequency and proportion of patients with initial response
Mean change from Baseline in platelet counts
Measured: Over the study period (up to 13 weeks).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2017
- Study start13 Mar 2017
- Primary completion9 Apr 2019
- Study completion9 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by argenxThe 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
- Model description
- Protocol designed to evaluate one or more interventions for treating a disease, syndrome or condition.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Quintiles, Inc.
- Responsible party
- Sponsor
- Investigators
- Adrian Newland, Study chair, Barts Hospital, Cancer Centre in London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03102593
- Sponsor's protocol ID
- ARGX-113-1603
- Other IDs
- 2016-003038-26, 2016
- Status last verified
- Jul 2023
- First submitted
- 14 Mar 2017
- Last updated
- 25 Jul 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Newland AC, Sanchez-Gonzalez B, Rejto L, Egyed M, Romanyuk N, Godar M, Verschueren K, Gandini D, Ulrichts P, Beauchamp J, Dreier T, Ward ES, Michel M, Liebman HA, de Haard H, Leupin N, Kuter DJ. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020 Feb;95(2):178-187. doi: 10.1002/ajh.25680. Epub 2019 Dec 10.
PubMed 31821591
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT03102593
- Acronym / sponsor ID
- ARGX-113-1603
- Conditions
- Primary Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Collaborators
- Quintiles, Inc.
- Study start
- 13 Mar 2017 (actual)
- Primary completion
- 9 Apr 2019 (actual)
- Study completion
- 9 Apr 2019 (actual)
- First posted
- 6 Apr 2017 (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.
The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.
Detailed description
This is a randomized, double-blind, placebo-controlled Phase II study in which approximately 36 patients will be randomized in a 1:1:1 ratio to receive either ARGX-113 Dose A, or ARGX-113 Dose B body weight or placebo in 4 infusions administered 1-week apart in addition to Standard-of-Care (SoC) treatment. Patients aged 18 to 85 years (inclusive) with confirmed primary immune thrombocytopenia (ITP) who have a platelet count ˂ 30 × 109/L and who are receiving oral corticosteroids and/or permitted oral immunosuppressants and/or Thrombopoietin receptor (TPO-R) agonist as SoC which must be maintained on a stable dose and frequency for at least 4 weeks prior to Screening.
The study will include a 2-week Screening, a 3-week Treatment period, and an 21-week follow-up (FU) period. The study is followed by an open label period where patients will be given the option to be treated with ARGX-113 Dose A in cycles of 4 weekly infusions with a minimum of 4 weeks apart. Patients may receive rescue therapy during the study at the discretion the investigator when deemed medically necessary.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Protocol designed to evaluate one or more interventions for treating a disease, syndrome or condition.
| Arm | Type | Receives |
|---|---|---|
| ARGX-113 Dose A + SoC | Experimental | Drug: ARGX-113Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo |
| ARGX-113 Dose B +SoC | Experimental | Drug: ARGX-113Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo |
| Placebo + SoC | Placebo comparator | Other: PlaceboPatients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or female patients aged ≥ 18 to ≤ 85 years.
- Must receive SoC treatment for ITP that has been stable in dose and frequency for at least 4 weeks prior to Screening. SoC may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist.
- Confirmed diagnosis of ITP with blood platelet counts < 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.
Key exclusion criteria (11 listed)
- Use of anticoagulants, or any drug with antiplatelet effect within 3 weeks prior to Screening.
- Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
- Use of Intravenous immunoglobulin G (IVIg) or anti-D immunoglobulin treatment within 4 weeks prior to screening.
- Use of recombinant thrombopoietin at any time.
- Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
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 |
|---|---|---|
| ARGX-113ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly | Drug | ARGX-113 Dose A + SoC, ARGX-113 Dose B +SoC |
| PlaceboARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly | Other | Placebo + SoC |
Primary outcome
Incidence and severity of serious adverse events (SAEs).
Time frame: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
1 secondary outcome
Frequency and proportion of patients with initial response
Time frame: Over the study period (up to 13 weeks).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2023.
- Results publication: PubMed 31821591Newland AC, Sanchez-Gonzalez B, Rejto L, Egyed M, Romanyuk N, Godar M, Verschueren K, Gandini D, Ulrichts P, Beauchamp J, Dreier T, Ward ES, Michel M, Liebman HA, de Haard H, Leupin N, Kuter DJ. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020 Feb;95(2):178-187. doi: 10.1002/ajh.25680. Epub 2019 Dec 10.
- Individual participant dataSharing planned: Undecided
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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.
Adrian Newland study chair
Barts Hospital, Cancer Centre in London
30 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARGX-113 in people with Primary Immune Thrombocytopenia, run by argenx at 30 sites in 10 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 3 inclusion and 11 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: finished, as of the sponsor's last update on 25 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in Hungary, Spain, Ukraine, France, Germany and 5 more countries. 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence and severity of serious adverse events (SAEs). (time frame: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03102593, which also lists 1 linked publication.
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 25 Jul 2023.