A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD+)
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| ClinicalTrials.gov Identifier: NCT05681481 |
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Recruitment Status :
Recruiting
First Posted : January 12, 2023
Last Update Posted : April 9, 2024
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Sponsor:
argenx
Information provided by (Responsible Party):
argenx
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Brief Summary:
ARGX-113-2010 is an open-label extension study with the aim to provide supporting evidence that efgartigimod PH20 SC is a safe and effective long-term treatment for bullous pemphigoid (BP), providing symptom control and eventually remission, while also reducing the cumulative exposure to oral corticosteroids (OCS).
All participants who complete the end-of-treatment period (EoTP) visit at week 36 in ARGX-113-2009 will be invited to enroll.
In ARGX-113-2009, participants received efgartigimod PH20 SC or placebo with concurrent OCS, or rescue therapy (without efgartigimod PH20 SC or placebo). Depending on their clinical status at the time of rollover into ARGX-113-2010, participants may stop, continue or initiate efgartigimod PH20 SC treatment. In ARGX-113-2010, participants will stop efgartigimod PH20 SC treatment when they achieve complete remission (CR) or partial remission (PR) while being off other concurrent BP therapy for at least 8 weeks. Participants not in CR or PR while off OCS for ≥8 weeks and not on rescue therapy will either start or continue efgartigimod PH20 SC treatment, while maintaining the treatment allocation of ARGX-113-2009 blinded. Participants may also be retreated with efgartigimod PH20 SC after a relapse. In this study, loading doses of 2000 mg (on day 1 and day 8 of a treatment course) and weekly maintenance doses of 1000 mg will be used.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Bullous Pemphigoid | Biological: efgartigimod PH20 SC Drug: Prednisone | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 160 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long Term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid |
| Actual Study Start Date : | March 22, 2023 |
| Estimated Primary Completion Date : | January 9, 2026 |
| Estimated Study Completion Date : | March 6, 2026 |
Resource links provided by the National Library of Medicine
Drug Information
available for:
Prednisone
| Arm | Intervention/treatment |
|---|---|
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Experimental: efgartigimod PH20 SC
participants receiving efgartigimod PH20 SC on top of Prednisone
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Biological: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer Drug: Prednisone Oral Prednisone |
Primary Outcome Measures :
- Incidence of treatment-emergent adverse events [ Time Frame: Up to 56 weeks ]Incidence of treatment-emergent adverse events
- Severity of treatment-emergent adverse events [ Time Frame: Up to 56 weeks ]Severity will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events ( CTCAE) definitions (current version): Grade 1 (mild) to Grade 5 (death related to adverse event)
- Incidence of serious adverse events [ Time Frame: Up to 56 weeks ]Incidence of serious adverse events
- Severity of serious adverse events [ Time Frame: Up to 56 weeks ]Severity of serious adverse events
- Incidence of adverse events of special interest [ Time Frame: Up to 56 weeks ]Incidence of adverse events of special interest
- Severity of adverse events of special interest [ Time Frame: Up to 56 weeks ]Severity will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events ( CTCAE) definitions (current version): Grade 1 (mild) to Grade 5 (death related to adverse event)
- Rate of treatment discontinuation because of safety concerns [ Time Frame: Up to 56 weeks ]Rate of treatment discontinuation because of safety concerns
Secondary Outcome Measures :
- Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks
- Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks
- Proportion of participants achieving complete remission while on minimal oral corticosteroids therapy for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]Minimal oral corticosteroid therapy is defined as ≤0.10 mg/kg/day of prednisone (or an equivalent dose of another oral corticosteroid)
- Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks
- Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks
- Duration of sustained remission [ Time Frame: Up to 56 weeks ]Duration of sustained remission
- Proportion of participants who relapse [ Time Frame: Up to 56 weeks ]Proportion of participants who relapse
- Time to relapse [ Time Frame: Up to 56 weeks ]Time to relapse
- Incidence of relapse [ Time Frame: Up to 56 weeks ]Incidence of relapse
- Severity of relapse [ Time Frame: Up to 56 weeks ]Severity of relapse will be assessed based on the Bullous Pemphigoid Disease Area Index (BPDAI)
- Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time
- Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time
- Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time
- Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time
- Itch Numerical Rating Scale (NRS) over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]Itch Numerical Rating Scale (NRS) over time
- Itch Numerical Rating Scale (NRS) over time [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]Itch Numerical Rating Scale (NRS) over time
- Rate of treatment failure [ Time Frame: Up to 56 weeks ]Rate of treatment failure
- Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
- Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. ]Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
- Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
- Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. ]Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
- Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time [ Time Frame: For participants not requiring treatment with efgartigimod at rollover: at weeks 0, 24 and 48. ]Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
- Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. ]Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
- EQ-5D-5L scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]EQ-5D-5L scores over time
- EQ-5D-5L scores over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. ]EQ-5D-5L scores over time
- Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time
- Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. ]Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time
- Dermatology Life Quality Index (DLQI) scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]Dermatology Life Quality Index (DLQI) scores over time
- Dermatology Life Quality Index (DLQI) scores over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. ]Dermatology Life Quality Index (DLQI) scores over time
- Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
- Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks to efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
- Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56. ]Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
- Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 8 weeks until and after efgartigimod PH20 SC stop and at weeks 48, 52 and 56. ]Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
- Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56. ]Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
- Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 8 weeks until and after efgartigimod PH20 SC stop and at weeks 48, 52 and 56. ]Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Has completed the week 36 visit of ARGX-113-2009
- Is capable of providing signed informed consent and complying with protocol requirements
- Agrees to use contraceptive measures consistent with local regulations and the following: Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP and must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP
Exclusion Criteria:
- Clinically significant disease, recent major surgery (within 3 months of baseline), or intends 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
- Known hypersensitivity to IMP or 1 of its excipients
- 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05681481
Contacts
| Contact: Sabine Coppieters, MD | 857-350-4834 | clinicaltrials@argenx.com |
Locations
Show 31 study locations
Sponsors and Collaborators
argenx
| Responsible Party: | argenx |
| ClinicalTrials.gov Identifier: | NCT05681481 |
| Other Study ID Numbers: |
ARGX-113-2010 |
| First Posted: | January 12, 2023 Key Record Dates |
| Last Update Posted: | April 9, 2024 |
| Last Verified: | April 2024 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | Yes |
Additional relevant MeSH terms:
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Pemphigoid, Bullous Skin Diseases, Vesiculobullous Skin Diseases Autoimmune Diseases Immune System Diseases Prednisone Anti-Inflammatory Agents |
Glucocorticoids Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs Antineoplastic Agents, Hormonal Antineoplastic Agents |