efgartigimod PH20 SC for Thrombocytopenia

Phase-Based Estimates
2
Effectiveness
3
Safety
Investigator Site 0010116, Bentonville, AR
+2 More
efgartigimod PH20 SC - Biological
Eligibility
18+
All Sexes
Eligible conditions
Thrombocytopenia

Study Summary

A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

See full description

Eligible Conditions

  • Thrombocytopenia
  • Purpura, Thrombocytopenic, Idiopathic
  • Primary Immune Thrombocytopenia (ITP)

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether efgartigimod PH20 SC will improve 4 primary outcomes and 27 secondary outcomes in patients with Thrombocytopenia. Measurement will happen over the course of 52 weeks.

216 weeks
ECG: PR, QT and QRS interval in the overall population
Incidence and prevalence of antibodies to efgartigimod
Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)
Laboratory safety evaluations: CRP analysis in the overall population
Presence of neutralizing antibodies (NAb) against efgartigimod
Titers of antibodies to efgartigimod
Vital sign measurement: blood pressure in the overall population
5 weeks (week 19-24)
In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10E9/L for at least 4 of the 6 visits between week 19 and week 24
52 weeks
Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits
Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits
Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L
For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10E9/L
In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline
Incidence of the World Health Organization (WHO)-classified bleeding events
Mean change from baseline in platelet count at each visit
Number of caregivers who administered the injection to the patient at home over time
Number of patients who performed self-administration at home over time
Number of self- or caregiver-supported administrations at home
Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
Percentage of caregivers who administered the injection to the patient at home over time
Percentage of patients who performed self-administration at home over time
Percentage of self- or caregiver-supported administrations at home
Pharmacodynamics markers: total IgG
Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline
Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period
Rate of receipt of rescue therapy (rescue per patient per month)
Serum efgartigimod concentration observed predose (Ctrough)
Severity of the World Health Organization (WHO)-classified bleeding events
The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline
7 weeks (week 17-24)
In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of ≥50×10E9/L for at least 6 of the 8 visits between week 17 and week 24

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Control
efgartigimod PH20 SC

This trial requires 156 total participants across 2 different treatment groups

This trial involves 2 different treatments. Efgartigimod PH20 SC is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

efgartigimod PH20 SC
Biological
Patients receiving efgartigimod PH20 SC treatment
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Efgartigimod alfa
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 7 weeks (week 17-24)
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 7 weeks (week 17-24) for reporting.

Closest Location

Investigator Site 0010116 - Bentonville, AR

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period.
Note: If a participant has had an SAE during the ARGX-113-2004 trial, their eligibility should be evaluated by the investigator and the sponsor's trial physician. The decision of enrolling the participant will be evaluated case by case.
Male participants.
4. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).
You are able to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits). show original
You are female and of childbearing potential show original
You have completed a 52-week treatment period.\n show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is purpura, thrombocytopenic, idiopathic?

Add answer

Results from a recent clinical trial of 50 clinically normal persons found a significant correlation between occurrence of purpura, thrombocytopenia and idiopathic thrombocytopenic purpura. There seems to be strong environmental influence on idiopathic thrombocytopenic purpura.

Unverified Answer

How many people get purpura, thrombocytopenic, idiopathic a year in the United States?

Add answer

<5% of the pediatric cases had a first underlying neoplastic disease. This percentage did not vary by age, gender, location of hospital or race/ethnicity of patient. We hypothesize that the pathogenesis of this illness in children is complex and multifactorial.

Unverified Answer

What are the signs of purpura, thrombocytopenic, idiopathic?

Add answer

A complete set of the above criteria can be useful when diagnosing INIT. Clinical and laboratory findings will depend on the underlying cause of thrombocytopenia and purpura. It is recommended that INIT be considered when purpura and thrombocytopenia is the presentation of INIT.

Unverified Answer

What causes purpura, thrombocytopenic, idiopathic?

Add answer

Purpura, thrombocytopenic, idiopathic (or PTHI) is a constellation of rare disorders with a range of causes, including paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenic purpura, and multiple myeloma. PNH is an acquired disorder that is very rare. Autoimmune thrombocytopenia-hemolytic anemia can lead to intravascular hemolysis with platelet destruction, hemoglobinemia, and a microangiopathic hemolytic anemia with thrombocytopenia without other underlying abnormalities.

Unverified Answer

What are common treatments for purpura, thrombocytopenic, idiopathic?

Add answer

Immunoglobulin injections are not recommended as they are highly toxic with an estimated rate of serious adverse effects up to 30% of the time. Cyclosporinic A has been found to be effective over 10 years after the start but has a potential rate of fatal cardiac side effects. Prednisolone, corticosteroid has been found to be effective over 10 years after the start, although this should be repeated every 3–4 weeks until good results are recorded. Anticoagulation is very effective, although there is a chance of bleeding.

Unverified Answer

Can purpura, thrombocytopenic, idiopathic be cured?

Add answer

Treatment of purpura, thrombocytopenic, idiopathic is justified and well accepted. It can be treated non-operatively. If there is no associated underlying disorder, it can be effectively managed.

Unverified Answer

Does purpura, thrombocytopenic, idiopathic run in families?

Add answer

Results from a recent paper we found that a variable fraction of PVTI patients in Israel are related to PD1-3. Although our findings need to be validated by the larger sample presented here, they are similar to those presented in Western countries and suggest the need for PVTI and PD1-3 evaluation.

Unverified Answer

What does efgartigimod ph20 sc usually treat?

Add answer

Efgartigimod is effective for treating HSP and PE in a minority of patients. Physicians should anticipate that more patients will benefit, but these patients may require high-dose intravenous immunoglobulin or plasma exchange.

Unverified Answer

How does efgartigimod ph20 sc work?

Add answer

This is the first direct comparison of efgartigimod vs placebo. [Figures are based on total of the treated groups for the endpoints (primary and secondary endpoints) being treated with all-drug versus monotherapy groups plus placebo. The percentage for each endpoint is as follows:\n- MIRRAD score = 59.5% versus 43.5% P < 0.0001\n- CIRS score ≥ 9: 45.7% versus 25.9% P = 0.01\n- CIRS score ≤ 8: 53.3% versus 44.9% P = 0.

Unverified Answer

What is the primary cause of purpura, thrombocytopenic, idiopathic?

Add answer

There are several possibilities for the development of purpura in the newborn setting including autoimmune hemolytic anemia, drug-induced, parvovirus infection, and, most consistently, thrombocytopenic hemolytic anemia caused by an identifiable trigger (drug, transfusion, or surgery). In this regard, the infant may develop purpura from thrombosis or hypercoagulation in the liver or lungs that then lead directly to the development of purpura or thrombocytopenic purpura. This purpura is commonly observed in cases of maternal, fetal, or neonatal alloimmune thrombocytopenia.

Unverified Answer

Is efgartigimod ph20 sc typically used in combination with any other treatments?

Add answer

Egftagimod ph05 and EGFR1-4 inhibitor are used typically in combination with any other treatments. Interestingly, no EGFR antagonistic therapy was used as sole-monotherapy in the present series. Nevertheless, this retrospective study does not exclude the possibility of an active EGFR antagonistic therapy being added as a second-line therapy in advanced metastatic pancreatic cancer.

Unverified Answer

Have there been any new discoveries for treating purpura, thrombocytopenic, idiopathic?

Add answer

We believe that new treatments for purpura, thrombocytopenic, idiopathic (PTI) are still evolving but are slowly emerging. Treatment for thrombocytopenic purpura (TTP) has been improved but is still underdevelopment.

Unverified Answer
See if you qualify for this trial
Get access to this novel treatment for Thrombocytopenia by sharing your contact details with the study coordinator.