This study has been transitioned to CTIS with ID 2024-515832-59-00 check the CTIS register for the current data. To assess the long-term safety and tolerability of treatment with efgartigimod PH20 SC in participants with BP
ID
Source
Brief title
Condition
- Autoimmune disorders
- Skin and subcutaneous tissue disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
• Incidence and severity of treatment-emergent adverse events (TEAEs), serious
AEs (SAEs), and AEs of special interest (AESIs)
• Rate of treatment discontinuation because of safety concerns
Secondary outcome
To assess the long-term efficacy and durability of response with efgartigimod
PH20 SC treatment in participants with BP :
• Proportions of participants who:
* Achieve complete remission (CR) and have been off oral corticosteroids (OCS)
for >=8 weeks
* Achieve CR or partial remission (PR) and have been off OCS therapy for >=8
weeks
* Achieve CR and have been on minimal OCS therapy for >=8 weeks. (Minimal OCS
therapy is defined as <=0.1 mg/kg a day of prednisone [or an equivalent dose of
another OCS])
* Are in CR and have been off both OCS and efgartigimod PH20 SC for >=8 weeks
* Are in CR or PR and have been off both OCS and efgartigimod PH20 SC for >=8
weeks
• Duration of sustained remission
• Proportion of participants who relapse
• Time to relapse
• Incidence and severity of relapse
• Bullous Pemphigoid Disease Area Index (BPDAI) activity scores, Investigator
Global Assessment of Bullous Pemphigoid (IGA-BP) scores, and itch numerical
rating scale (NRS) over time
• Rate of treatment discontinuation due to efficacy concerns
Background summary
BP is a subepidermal AIBD that predominantly affects older adults. It is a
chronic disease that significantly affects morbidity and QoL; additionally, the
disease can worsen spontaneously, even when the patient is treated with the
current standard of care. The pathogenesis of BP is driven by IgG and IgE
autoantibodies against the hemidesmosomal proteins BP180 and BP230, acting as
key antigens for pathogenic autoantibodies.
The current standard of care for BP is treatment with TCS or OCS, either of
which can be combined with conventional immunosuppressant therapy.
Unfortunately, corticosteroid therapy in patients with BP typically causes
comorbidities, which can be severe and even life-threatening, especially in
older adults.
In summary, there is currently an unmet medical need for new BP treatments that
provide rapid CDA and remission, minimize (or even prevent) relapse, and reduce
the burdens placed on patients caused by cumulative corticosteroid exposure.
This OLE study aims to evaluate the safety, tolerability, long-term efficacy,
immunogenicity, PRO measures, and PD of efgartigimod PH20 SC in adult
participants with BP, who completed the antecedent study ARGX 113 2009.
Study objective
This study has been transitioned to CTIS with ID 2024-515832-59-00 check the CTIS register for the current data.
To assess the long-term safety and tolerability of treatment with efgartigimod
PH20 SC in participants with BP
Study design
This is a phase 3, prospective, global, multicenter, OLE study to investigate
the long-term safety, tolerability, efficacy, quality of life (QoL),
pharmacodynamics (PD), and immunogenicity of efgartigimod PH20 SC in adult
participants with BP who have completed ARGX-113-2009. All participants who
complete the end-of-treatment period (EoTP) visit at week 36 in ARGX 113 2009
will be invited to enroll. The study will be conducted in the same sites as
ARGX 113 2009.
In ARGX-113-2009, participants received efgartigimod PH20 SC or placebo with
concurrent OCS, or rescue therapy (defined as concurrent BP therapy in this
protocol). Depending on their clinical status at the time of rollover,
participants may be treated with efgartigimod PH20 SC and participants who
relapse during the study may also be treated with efgartigimod PH20 SC.
Intervention
At rollover, participants will be treated with efgartigimod PH20 SC according
to their clinical status at the EoTP visit of ARGX-113-2009. A summary of the
treatment interventions is provided in Table 5 in the protocol on page 38 -41
Study burden and risks
There is currently an unmet medical need for new BP treatments that provide
rapid CDA and remission, minimize (or even prevent) relapse, and reduce the
burdens placed on patients caused by cumulative corticosteroid exposure. This
OLE study aims to evaluate the safety, tolerability, long-term efficacy,
immunogenicity, PRO measures, and PD of efgartigimod PH20 SC in adult
participants with BP, who completed the antecedent study ARGX 113 2009.
The favorable balance between the risks and anticipated efficacy/benefits
supports the administration of efgartigimod PH20 SC to participants with BP in
ARGX-113-2010.
More detailed information about the known and expected benefits and risks of
efgartigimod*both as an IV formulation and as efgartigimod PH20 SC and
reasonably expected AEs can be found in the current IB.
See also section 2.3 in the protocol for detailed information about the Benefit
and Risk Assessment.
Industriepark Zwijnaarde 7
Zwijnaarde B-9052
BE
Industriepark Zwijnaarde 7
Zwijnaarde B-9052
BE
Listed location countries
Age
Inclusion criteria
1. Has completed the week 36 visit of ARGX-113-2009
2. Is capable of providing signed informed consent and complying with protocol
requirements
3. Agrees to use contraceptive measures consistent with local regulations and
the following:
a. Male participants: An acceptable method of contraception is a condom. All
nonsterilized male participants must use this method from signing of the ICF
until the date of the last dose of IMP.
b. Women of childbearing potential must have a negative urine pregnancy test at
baseline before receiving IMP. Women must use one of the contraception methods
described in the protocol from signing the ICF until the last dose of IMP
Exclusion criteria
1. Clinically significant disease, recent major surgery (within 3 months of
baseline), or intention to have surgery during the study; or any other medical
condition that, in the investigator*s opinion would confound the results of the
study or put the participant at undue risk
2. Known hypersensitivity to IMP or 1 of its excipients
3. Permanently discontinued IMP in ARGX-113-2009 due to an AE considered
related to IMP and for whom the benefit/risk balance is not considered
positive
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2024-515832-59-00 |
| EudraCT | EUCTR2021-003063-10-NL |
| CCMO | NL83164.028.23 |