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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-004032-21 | EudraCT Number |
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This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Efgartigimod PH20 SC | Experimental | Patients receiving efgartigimod PH20 SC treatment |
|
| Placebo PH20 SC | Placebo Comparator | Patients receiving placebo PH20 SC treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efgartigimod PH20 SC | Biological | Subcutaneous injection with efgartigimod PH20 SC |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24. | Up to 6 weeks (between Weeks 19 and 24) |
| Measure | Description | Time Frame |
|---|---|---|
| Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^9/L in the chronic ITP population. | Up to 24 weeks |
| Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 |
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Inclusion criteria:
Permitted concurrent ITP medications include corticosteroids, danazol, vinca alkaloids, oral immunosuppressants, dapsone, fostamatinib, and/or oral TPO-RAs.
-Agree to use contraceptive measures consistent with local regulations and the protocol
Exclusion criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator Site 0010116 | Springdale | Arkansas | 72758 | United States | ||
| Investigator site 0010036 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41943179 | Derived | Cooper N, Broome CM, Miyakawa Y, McDonald V, Al-Samkari H, Khelif A, Cataland SR, Barcellini W, Yang R, Mei H, Matthijssens F, Hultberg A, Ciurlia G, Gandini D, Ayguasanosa J, De Beuf K, Pastouret S, Ghanima W, Rodeghiero F, Bussel JB; ADVANCE SC Investigator Trial Group. Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial. Am J Hematol. 2026 Jul;101(7):1494-1506. doi: 10.1002/ajh.70303. Epub 2026 Apr 6. |
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A regionally diverse participant population was enrolled (including but not limited to North America, East Asia, Europe, and the rest of world [ROW]).
A total of 207 participants were randomized to IMP: 137 participants in the efgartigimod PH20 subcutaneous (SC) arm and 70 participants in the placebo PH20 SC arm.
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| ID | Title | Description |
|---|---|---|
| FG000 | Efgartigimod PH20 SC | Participants receiving efgartigimod PH20 SC treatment. |
| FG001 | Placebo PH20 SC | Participants receiving placebo PH20 SC treatment. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 6, 2023 | Oct 8, 2024 |
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| Placebo PH20 SC | Other | Subcutaneous injection with placebo PH20 SC |
|
A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24. |
| Up to 6 weeks (between Weeks 19 and 24) |
| Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24 | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥6 of the 8 analysis visits between weeks 17 and 24. | Up to 8 weeks (between Weeks 17 and 24) |
| Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period | A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 analysis visits at any time during the 24-week treatment period. | Up to 24 weeks |
| Extent of Disease Control Until Week 12 in the Overall Population | Extent of disease control was defined as the number of cumulative weeks until Week 12 with platelet counts of ≥50×10^9/L in the overall population. | Up to 12 weeks |
| Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12 | A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 analysis visits at any time until Week 12. | Up to 12 weeks |
| Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population | Change from Baseline at time point t = value at time point t - Baseline value. Baseline was defined as the last available value prior to first administration of the investigational medicinal product (IMP). | Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33) |
| Time to Platelet Count Response in the Overall Population | Time to platelet count response, defined as the time to have 2 consecutive platelet counts of ≥50 × 10^9/L via Kaplan-Meier estimates. | Up to 24 weeks |
| Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L with at least ≥20×10^9/L above Baseline in the overall population. | Up to 24 weeks |
| Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population for Participants With Baseline Platelet Count of <15×10^9/L | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L with at least ≥20×10^9/L above Baseline in the overall population. | Up to 24 weeks |
| Number of the World Health Organization (WHO)-Classified Bleeding Events (Grade ≥1) in the Overall Population | Assessed using the WHO bleeding scale. The WHO bleeding scale is a five-point scale where Grade 0 = no bleeding; Grade 1 = petechial bleeding; Grade 2 = mild blood loss; Grade 3 = gross blood loss (requires transfusion); and Grade 4 = debilitating blood loss, associated with fatality. | Up to 24 weeks |
| Percentage of Participants With a Platelet Count International Working Group (IWG) Response | IWG complete response was defined as platelet counts of ≥100 × 10^9/L and the absence of bleeding events (WHO Grading = 0 [no bleeding]) for at least 2 separate, consecutive analysis visits at least 7 days apart. IWG response was defined as platelet counts of ≥30 × 10^9/L and a 2-fold increase of platelet count from Baseline and the absence of bleeding events (WHO grading = 0) for at least 2 separate, consecutive analysis visits that were at least 7 days apart. Initial response was defined as platelet counts of ≥30 × 10^9/L and a 2-fold increase from the Baseline platelet count at analysis visit 5. | Up to 24 weeks |
| Rate of Receipt of Rescue Therapy (Rescue Per Participant Per Month) in the Overall Population | Rescue therapy was defined as an occurrence where the participant needed treatment with 1 or more rescue treatments. An occurrence was defined as a period of maximum 5 days where 1 or more rescue treatments were administered simultaneously or consecutively to the trial participant. The following rescue treatments were permitted: methylprednisolone, dexamethasone, prednisone, normal immunoglobulins, anti-D (Rho) immunoglobins, or platelet transfusions. | Up to 24 weeks |
| Percentage of Participants for Whom Dose and/or Frequency of Concurrent ITP Therapies Have Increased at Week 12 or Later in the Overall Population | A change in ITP therapy was defined as either an increase in the dose and/or frequency of a concurrent ITP therapy relative to Baseline or the initiation of a new concurrent ITP therapy. | Up to 13 weeks (between Weeks 12 and 24) |
| Change From Baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) at Week 24 in the Overall Population | The FACIT-fatigue scale is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during his/her usual daily activities over the past week. The level of fatigue was measured by recording item responses on a 5-point Likert scale ranging from 0 "not at all" to 4 "very much". All items were summed to create a single fatigue score with a range from 0 to 52, where a higher FACIT-F score indicated more severe symptoms. A negative change score from Baseline indicated improvement in quality of life (QoL). | Baseline and Week 24 |
| Change From Baseline in Functional Assessment of Cancer Therapy Questionnaire-Th6 (Fact-Th6) at Week 24 in the Overall Population | The FACT-Th6 uses a 5-level Likert scale (0=not at all to 4=very much), with participants rating their degree of concern in the past 7 days. The 6 selected items pertain to ability to do usual activities, worry about problems with bleeding or bruising, worry about the possibility of serious bleeding, avoidance of physical or social activity because of concern with bleeding or bruising and frustration due to the inability to carry out usual activities. All items were summed to create a single score with a range from 0 to 24, where a higher score indicated less severe symptoms. A positive change score from Baseline indicated improvement in QoL. | Baseline and Weeks 4, 8, 12, 16, 20, and 24 |
| Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population | The SF-36 is a 36-item scale constructed to survey health-related QoL on 8 domains: limitations in physical activities due to health problems; limitations in social activities due to physical or emotional problems; limitations in usual role activities due to physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities due to emotional problems; vitality (energy and fatigue); and general health perceptions. The scores from the 8 domains were evaluated independently and aggregated into 2 norm-based summary component measures of physical and mental health. The summary component scores could range from 0 to 100, where a higher score indicated improvement in QoL. A positive change score from Baseline indicated improvement in QoL. | Baseline and Week 24 |
| Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population | Anti-drug antibody (ADA) incidence was defined as the percentage of participants with treatment-induced or treatment boosted ADA (denominator: number of evaluable participants). ADA prevalence was defined as the percentage of participants with treatment-unaffected ADA, treatment-induced ADA or treatment-boosted ADA (denominator: number of evaluable participants). | Up to 35 weeks |
| Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population | A titer was determined in the samples with a positive assay response. | Weeks 3, 7, 11, 15, 19, 23, 24, SEFU1 (up to Week 29), and SEFU2 (up to Week 33) |
| Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population | Samples were tested for the presence of NAb against efgartigimod and/or rHuPH20 and titers for NAb against rHuPH20. NAb incidence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive" and "baseline positive-postbaseline positive". NAb prevalence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive," "baseline positive-postbaseline positive," or "baseline positive-postbaseline negative". | Up to 35 weeks |
| Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population | All pharmacokinetic (PK) samples were collected predose, on the day of IMP administration. | Predose on Weeks 1, 2, 3, 17, 19, 21, 23, and 24 |
| Percentage Change From Baseline in Total IgG in the Overall Population | Samples were collected predose, on the day of IMP administration. | Baseline and Weeks 1, 2, 3, 17, 19, 21, 23, and 24 |
| Number of Participants With Antiplatelet Antibodies in the Overall Population | The antiplatelet antibody was positive if optical density value >0.129. | Weeks 7, 15, 23, and 24 |
| Los Angeles |
| California |
| 90033 |
| United States |
| Investigator Site 0010045 | Washington D.C. | District of Columbia | 20007 | United States |
| Investigator Site 0010104 | Weston | Florida | 33331 | United States |
| Investigator site 0010112 | Chicago | Illinois | 60612 | United States |
| Investigator site 0010193 | Chicago | Illinois | 60637 | United States |
| Investigator Site 0010079 | Lisle | Illinois | 60187 | United States |
| Investigator Site 0010062 | Fort Wayne | Indiana | 46804 | United States |
| Investigator site 0010042 | Iowa City | Iowa | 52242 | United States |
| Investigator Site 0010083 | Detroit | Michigan | 48202 | United States |
| Investigator Site 0010102 | Minneapolis | Minnesota | 55455 | United States |
| Investigator site 0010040 | Columbus | Ohio | 43210 | United States |
| Investigator Site 0010095 | Oklahoma City | Oklahoma | 73142 | United States |
| Investigator Site 0010115 | Pittsburgh | Pennsylvania | 15222 | United States |
| Investigator Site 0540001 | Buenos Aires | Argentina |
| Investigator Site 0540004 | Buenos Aires | Argentina |
| Investigator Site 0540003 | Córdoba | Argentina |
| Investigator Site 0610009 | Adelaide | Australia |
| Investigator Site 0610004 | Bedford Park | Australia |
| Investigator Site 0610002 | Box Hill | Australia |
| Investigator Site 0610010 | Clayton | Australia |
| Investigator Site 0610012 | Garran | Australia |
| Investigator Site 0610001 | Hobart | Australia |
| Investigator Site 0610011 | Perth | Australia |
| Investigator Site 0610003 | West Perth | Australia |
| Investigator Site 0610005 | Westmead | Australia |
| Investigator Site 3590017 | Plovdiv | Bulgaria |
| Investigator Site 3590015 | Sofia | Bulgaria |
| Investigator Site 0560002 | Santiago | Chile |
| Investigator Site 0560004 | Temuco | Chile |
| Investigator Site 0560003 | Viña del Mar | Chile |
| Investigator Site 0860003 | Beijing | China |
| Investigator Site 0860013 | Beijing | China |
| Investigator Site 0860008 | Bengbu | China |
| Investigator Site 0860055 | Huizhou | China |
| Investigator Site 0860009 | Kunming | China |
| Investigator Site 0860012 | Nanchang | China |
| Investigator Site 0860014 | Shanxi | China |
| Investigator Site 0860015 | Shenzhen | China |
| Investigator Site 0860001 | Tianjin | China |
| Investigator Site 0860006 | Wenzhou | China |
| Investigator Site 0860010 | Wuhan | China |
| Investigator Site 0860002 | Wuxi | China |
| Investigator Site 0860005 | Zhejiang | China |
| Investigator Site 0860011 | Zhengzhou | China |
| Investigator Site 0860058 | Zhenjiang | China |
| Investigator site 0860062 | Zhenjiang | China |
| Investigator Site 0450005 | Roskilde | Denmark |
| Investigator Site 0330009 | Créteil | France |
| Investigator Site 0330018 | Montpellier | France |
| Investigator Site 9950006 | Tbilisi | Georgia |
| Investigator Site 9950007 | Tbilisi | Georgia |
| Investigator Site 9950008 | Tbilisi | Georgia |
| Investigator Site 9950009 | Tbilisi | Georgia |
| Investigator Site 9950011 | Tbilisi | Georgia |
| Investigator Site 9950019 | Tbilisi | Georgia |
| Investigator site 0490008 | Essen | 45147 | Germany |
| Investigator Site 0490012 | Giessen | Germany |
| Investigator Site 0300008 | Athens | Greece |
| Investigator Site 0300010 | Athens | Greece |
| Investigator Site 0300007 | Pátrai | Greece |
| Investigator Site 0300009 | Thessaloniki | Greece |
| Investigator Site 3530002 | Dublin | Ireland |
| Investigator Site 3530003 | Dublin | Ireland |
| Investigator Site 3530001 | Galway | Ireland |
| Investigator Site 9720013 | Ashkelon | Israel |
| Investigator Site 9720010 | Haifa | Israel |
| Investigator Site 9720012 | Haifa | Israel |
| Investigator Site 9720008 | Jerusalem | Israel |
| Investigator Site 9720011 | Jerusalem | Israel |
| Investigator Site 9720007 | Petah Tikva | Israel |
| Investigator Site 9720009 | Tel Aviv | Israel |
| Investigator Site 0390037 | Alessandria | Italy |
| Investigator Site 0390043 | Ferrara | Italy |
| Investigator Site 0390045 | Meldola | Italy |
| Investigator site 0390014 | Milan | 20122 | Italy |
| Investigator Site 0390032 | Milan | Italy |
| Investigator Site 0390041 | Naples | Italy |
| Investigator Site 0390044 | Naples | Italy |
| Investigator Site 0390015 | Novara | Italy |
| Investigator Site 0390035 | Potenza | Italy |
| Investigator Site 0390011 | Reggio Calabria | Italy |
| Investigator site 0390018 | Reggio Emilia | 42100 | Italy |
| Investigator Site 0390046 | Rome | Italy |
| Investigator Site 0390033 | Terni | Italy |
| Investigator Site 0390036 | Varese | Italy |
| Investigator Site 0810056 | Chiba | Japan |
| Investigator Site 0810015 | Hirakata | Japan |
| Investigator Site 0810010 | Hiroshima | Japan |
| Investigator Site 0810053 | Kanagawa | Japan |
| Investigator Site 0810051 | Kitakyushu | Japan |
| Investigator Site 0810054 | Kumamoto | Japan |
| Investigator Site 0810018 | Maebashi | Japan |
| Investigator Site 0810057 | Morioka | Japan |
| Investigator Site 0810012 | Ōgaki | Japan |
| Investigator Site 0810017 | Saitama | Japan |
| Investigator Site 0810016 | Shibukawa | Japan |
| Investigator Site 0810023 | Shimotsuke | Japan |
| Investigator Site 0810039 | Shinagawa-Ku | Japan |
| Investigator Site 0810038 | Tama | Japan |
| Investigator Site 0810052 | Tokyo | Japan |
| Investigator Site 0810048 | Tsukuba | Japan |
| Investigator Site 0810044 | Yamanashi | Japan |
| Investigator Site 9620002 | Amman | Jordan |
| Investigator Site 9620001 | Irbid | Jordan |
| Investigator Site 0520002 | Aguascalientes | Mexico |
| Investigator Site 0520004 | Chihuahua City | Mexico |
| Investigator Site 0520007 | México | Mexico |
| Investigator Site 0520003 | Monterrey | Mexico |
| Investigator Site 0520001 | Oaxaca City | Mexico |
| Investigator Site 0640001 | Auckland | New Zealand |
| Investigator Site 0640005 | Christchurch | New Zealand |
| Investigator Site 0640002 | Palmerston North | New Zealand |
| Investigator Site 0470002 | Bergen | Norway |
| Investigator Site 0470003 | Oslo | Norway |
| Investigator Site 0480013 | Katowice | 40519 | Poland |
| Investigator Site 0480014 | Lublin | Poland |
| Investigator Site 0480026 | Nowy Sącz | Poland |
| Investigator Site 0480037 | Skorzewo | Poland |
| Investigator Site 0480039 | Torun | Poland |
| Investigator Site 0480033 | Warsaw | Poland |
| Investigator Site 3510006 | Braga | Portugal |
| Investigator Site 3510003 | Coimbra | Portugal |
| Investigator Site 3510002 | Lisbon | Portugal |
| Investigator Site 3510005 | Lisbon | Portugal |
| Investigator Site 3510007 | Lisbon | Portugal |
| Investigator Site 3510001 | Porto | Portugal |
| Investigator Site 3510004 | Porto | Portugal |
| Investigator Site 0400005 | Bucharest | Romania |
| Investigator Site 0400006 | Bucharest | Romania |
| Investigator Site 0400009 | Bucharest | Romania |
| Investigator Site 0400012 | Bucharest | Romania |
| Investigator Site 0400016 | Cluj-Napoca | Romania |
| Investigator Site 0400007 | Craiova | Romania |
| Investigator Site 0400011 | Sibiu | Romania |
| Investigator Site 0400008 | Târgu Mureş | Romania |
| Investigator Site 0070006 | Kaluga | Russia |
| Investigator Site 0070040 | Kirov | Russia |
| Investigator Site 0070026 | Moscow | Russia |
| Investigator Site 0070038 | Nizhny Novgorod | Russia |
| Investigator Site 0070037 | Novosibirsk | Russia |
| Investigator Site 0070024 | Pyatigorsk | Russia |
| Investigator Site 0070025 | Saint Petersburg | Russia |
| Investigator Site 0070039 | Smolensk | Russia |
| Investigator Site 0070015 | Syktyvkar | Russia |
| Investigator Site 0070012 | Tula | Russia |
| Investigator Site 3810006 | Belgrade | Serbia |
| Investigator Site 3810008 | Kragujevac | Serbia |
| Investigator Site 0270005 | George | South Africa |
| Investigator Site 0270003 | Johannesburg | South Africa |
| Investigator Site 0270004 | Observatory | South Africa |
| Investigator Site 0270001 | Pretoria | South Africa |
| Investigator Site 0270002 | Randburg | South Africa |
| Investigator Site 0820005 | Seongnam | South Korea |
| Investigator Site 0820003 | Seoul | South Korea |
| Investigator Site 0820004 | Seoul | South Korea |
| Investigator Site 0820006 | Seoul | South Korea |
| Investigator Site 0820007 | Seoul | South Korea |
| Investigator Site 0820008 | Seoul | South Korea |
| Investigator Site 0340024 | Alava | Spain |
| Investigator Site 0340007 | Barcelona | 08035 | Spain |
| Investigator Site 0340006 | Barcelona | Spain |
| Investigator Site 0340023 | Barcelona | Spain |
| Investigator Site 0340037 | Madrid | Spain |
| Investigator Site 0340022 | Murcia | Spain |
| Investigator Site 0340036 | Sabadell | Spain |
| Investigator site 0340013 | Seville | 41013 | Spain |
| Investigator Site 0340004 | Valencia | Spain |
| Investigator Site 8860001 | New Taipei City | Taiwan |
| Investigator Site 8860003 | Taoyuan | Taiwan |
| Investigator Site 0660002 | Bangkok | Thailand |
| Investigator Site 0660003 | Bangkok | Thailand |
| Investigator Site 0660005 | Bangkok | Thailand |
| Investigator Site 0660008 | Bangkok | Thailand |
| Investigator Site 0660001 | Bangkok Noi | Thailand |
| Investigator Site 0660004 | Chiang Mai | Thailand |
| Investigator Site 0660009 | Khon Kaen | Thailand |
| Investigator Site 0660006 | Pathum Thani | Thailand |
| Investigator Site 2160006 | Sfax | Tunisia |
| Investigator Site 2160001 | Sousse | Tunisia |
| Investigator Site 2160002 | Tunis | Tunisia |
| Investigator Site 0900007 | Adapazarı | Turkey (Türkiye) |
| Investigator Site 0900003 | Ankara | Turkey (Türkiye) |
| Investigator Site 0900006 | Ankara | Turkey (Türkiye) |
| Investigator Site 0900008 | Ankara | Turkey (Türkiye) |
| Investigator Site 0900015 | Ankara | Turkey (Türkiye) |
| Investigator Site 0900016 | Edirne | Turkey (Türkiye) |
| Investigator Site 0900013 | Istanbul | Turkey (Türkiye) |
| Investigator Site 0900004 | Izmir | Turkey (Türkiye) |
| Investigator Site 0900014 | Kocaeli | Turkey (Türkiye) |
| Investigator Site 0900018 | Malatya | Turkey (Türkiye) |
| Investigator Site 0900010 | Mersin | Turkey (Türkiye) |
| Investigator Site 0900009 | Samsun | Turkey (Türkiye) |
| Investigator Site 0900017 | Tekirdağ | Turkey (Türkiye) |
| Investigator Site 0900019 | Trabzon | Turkey (Türkiye) |
| Investigator site 0440011 | Bradford | BD9 6RJ | United Kingdom |
| Investigator Site 0440005 | Coventry | United Kingdom |
| Investigator Site 0440008 | London | United Kingdom |
| Investigator Site 0440041 | London | United Kingdom |
| Investigator Site 0440014 | Truro | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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Safety Analysis Set (SAF): All participants who received at least 1 dose or part of a dose.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Efgartigimod PH20 SC | Participants receiving efgartigimod PH20 SC treatment. |
| BG001 | Placebo PH20 SC | Participants receiving placebo PH20 SC treatment. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24. | Full Analysis Set (FAS)-Chronic: Participants from the FAS (all randomized participants) including only participants with chronic ITP. | Posted | Number | percentage of participants | Up to 6 weeks (between Weeks 19 and 24) |
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| Secondary | Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^9/L in the chronic ITP population. | FAS-Chronic: Participants from the FAS (all randomized participants) including only participants with chronic ITP. | Posted | Median | Full Range | weeks | Up to 24 weeks |
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| Secondary | Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24. | FAS: All randomized participants. | Posted | Number | percentage of participants | Up to 6 weeks (between Weeks 19 and 24) |
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| Secondary | Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24 | A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥6 of the 8 analysis visits between weeks 17 and 24. | FAS: All randomized participants. | Posted | Number | percentage of participants | Up to 8 weeks (between Weeks 17 and 24) |
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| Secondary | Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period | A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 analysis visits at any time during the 24-week treatment period. | FAS: All randomized participants. | Posted | Number | percentage of participants | Up to 24 weeks |
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| Secondary | Extent of Disease Control Until Week 12 in the Overall Population | Extent of disease control was defined as the number of cumulative weeks until Week 12 with platelet counts of ≥50×10^9/L in the overall population. | FAS: All randomized participants. | Posted | Median | Full Range | weeks | Up to 12 weeks |
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| Secondary | Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12 | A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 analysis visits at any time until Week 12. | FAS: All randomized participants. | Posted | Number | percentage of participants | Up to 12 weeks |
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| Secondary | Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population | Change from Baseline at time point t = value at time point t - Baseline value. Baseline was defined as the last available value prior to first administration of the investigational medicinal product (IMP). | FAS: All randomized participants with available data. | Posted | Mean | Standard Deviation | platelets x10^9/L | Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33) |
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| Secondary | Time to Platelet Count Response in the Overall Population | Time to platelet count response, defined as the time to have 2 consecutive platelet counts of ≥50 × 10^9/L via Kaplan-Meier estimates. | FAS: All randomized participants. Participants with no occurrence of platelet count response, early discontinuation of treatment, or dose and/or frequency of concurrent ITP therapy increased or a new ITP therapy were censored. If multiple censoring conditions apply, the earliest censoring date is considered. | Posted | Median | 95% Confidence Interval | days | Up to 24 weeks |
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| Secondary | Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L with at least ≥20×10^9/L above Baseline in the overall population. | FAS: All randomized participants. | Posted | Median | Full Range | weeks | Up to 24 weeks |
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| Secondary | Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population for Participants With Baseline Platelet Count of <15×10^9/L | Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L with at least ≥20×10^9/L above Baseline in the overall population. | FAS: All randomized participants with Baseline Platelet Count of <15×10^9/L. | Posted | Median | Full Range | weeks | Up to 24 weeks |
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| Secondary | Number of the World Health Organization (WHO)-Classified Bleeding Events (Grade ≥1) in the Overall Population | Assessed using the WHO bleeding scale. The WHO bleeding scale is a five-point scale where Grade 0 = no bleeding; Grade 1 = petechial bleeding; Grade 2 = mild blood loss; Grade 3 = gross blood loss (requires transfusion); and Grade 4 = debilitating blood loss, associated with fatality. | FAS: All randomized participants. | Posted | Median | Full Range | bleeding events | Up to 24 weeks |
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| Secondary | Percentage of Participants With a Platelet Count International Working Group (IWG) Response | IWG complete response was defined as platelet counts of ≥100 × 10^9/L and the absence of bleeding events (WHO Grading = 0 [no bleeding]) for at least 2 separate, consecutive analysis visits at least 7 days apart. IWG response was defined as platelet counts of ≥30 × 10^9/L and a 2-fold increase of platelet count from Baseline and the absence of bleeding events (WHO grading = 0) for at least 2 separate, consecutive analysis visits that were at least 7 days apart. Initial response was defined as platelet counts of ≥30 × 10^9/L and a 2-fold increase from the Baseline platelet count at analysis visit 5. | FAS: All randomized participants. | Posted | Number | percentage of participants | Up to 24 weeks |
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| Secondary | Rate of Receipt of Rescue Therapy (Rescue Per Participant Per Month) in the Overall Population | Rescue therapy was defined as an occurrence where the participant needed treatment with 1 or more rescue treatments. An occurrence was defined as a period of maximum 5 days where 1 or more rescue treatments were administered simultaneously or consecutively to the trial participant. The following rescue treatments were permitted: methylprednisolone, dexamethasone, prednisone, normal immunoglobulins, anti-D (Rho) immunoglobins, or platelet transfusions. | FAS: All randomized participants. | Posted | Mean | Standard Deviation | rescues per participant per month | Up to 24 weeks |
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| Secondary | Percentage of Participants for Whom Dose and/or Frequency of Concurrent ITP Therapies Have Increased at Week 12 or Later in the Overall Population | A change in ITP therapy was defined as either an increase in the dose and/or frequency of a concurrent ITP therapy relative to Baseline or the initiation of a new concurrent ITP therapy. | FAS: All randomized participants. | Posted | Number | percentage of participants | Up to 13 weeks (between Weeks 12 and 24) |
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| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) at Week 24 in the Overall Population | The FACIT-fatigue scale is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during his/her usual daily activities over the past week. The level of fatigue was measured by recording item responses on a 5-point Likert scale ranging from 0 "not at all" to 4 "very much". All items were summed to create a single fatigue score with a range from 0 to 52, where a higher FACIT-F score indicated more severe symptoms. A negative change score from Baseline indicated improvement in quality of life (QoL). | FAS: All randomized participants. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline and Week 24 |
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| Secondary | Change From Baseline in Functional Assessment of Cancer Therapy Questionnaire-Th6 (Fact-Th6) at Week 24 in the Overall Population | The FACT-Th6 uses a 5-level Likert scale (0=not at all to 4=very much), with participants rating their degree of concern in the past 7 days. The 6 selected items pertain to ability to do usual activities, worry about problems with bleeding or bruising, worry about the possibility of serious bleeding, avoidance of physical or social activity because of concern with bleeding or bruising and frustration due to the inability to carry out usual activities. All items were summed to create a single score with a range from 0 to 24, where a higher score indicated less severe symptoms. A positive change score from Baseline indicated improvement in QoL. | FAS: All randomized participants. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline and Weeks 4, 8, 12, 16, 20, and 24 |
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| Secondary | Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population | The SF-36 is a 36-item scale constructed to survey health-related QoL on 8 domains: limitations in physical activities due to health problems; limitations in social activities due to physical or emotional problems; limitations in usual role activities due to physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities due to emotional problems; vitality (energy and fatigue); and general health perceptions. The scores from the 8 domains were evaluated independently and aggregated into 2 norm-based summary component measures of physical and mental health. The summary component scores could range from 0 to 100, where a higher score indicated improvement in QoL. A positive change score from Baseline indicated improvement in QoL. | FAS: All randomized participants. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline and Week 24 |
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| Secondary | Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population | Anti-drug antibody (ADA) incidence was defined as the percentage of participants with treatment-induced or treatment boosted ADA (denominator: number of evaluable participants). ADA prevalence was defined as the percentage of participants with treatment-unaffected ADA, treatment-induced ADA or treatment-boosted ADA (denominator: number of evaluable participants). | SAF: All participants who received at least 1 dose or part of a dose including only antibody-evaluable participants. | Posted | Number | percentage of participants | Up to 35 weeks |
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| Secondary | Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population | A titer was determined in the samples with a positive assay response. | SAF: All participants who received at least 1 dose or part of a dose with available data. | Posted | Mean | Standard Deviation | titer | Weeks 3, 7, 11, 15, 19, 23, 24, SEFU1 (up to Week 29), and SEFU2 (up to Week 33) |
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| Secondary | Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population | Samples were tested for the presence of NAb against efgartigimod and/or rHuPH20 and titers for NAb against rHuPH20. NAb incidence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive" and "baseline positive-postbaseline positive". NAb prevalence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive," "baseline positive-postbaseline positive," or "baseline positive-postbaseline negative". | SAF: All participants who received at least 1 dose or part of a dose with available data. | Posted | Number | percentage of participants | Up to 35 weeks |
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| Secondary | Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population | All pharmacokinetic (PK) samples were collected predose, on the day of IMP administration. | PK Analysis Set: Safety analysis set excluding placebo participants and including participants with at least one serum post dose PK measurement. | Posted | Mean | Standard Deviation | μg/mL | Predose on Weeks 1, 2, 3, 17, 19, 21, 23, and 24 |
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| Secondary | Percentage Change From Baseline in Total IgG in the Overall Population | Samples were collected predose, on the day of IMP administration. | Pharmacodynamic (PD) Analysis Set: Safety analysis set including participants with at least one serum post dose PD measurement. | Posted | Mean | Standard Deviation | percentage of total IgG | Baseline and Weeks 1, 2, 3, 17, 19, 21, 23, and 24 |
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| Secondary | Number of Participants With Antiplatelet Antibodies in the Overall Population | The antiplatelet antibody was positive if optical density value >0.129. | PD Analysis Set: Safety analysis set including participants with at least one serum post dose PD measurement and tested positive for antiplatelet antibodies at Baseline. | Posted | Count of Participants | Participants | Weeks 7, 15, 23, and 24 |
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Up to 35 weeks
The total number of participants affected by other (not including serious) adverse events (AEs) include participants who experienced other AEs under the frequency threshold of 5%.
SAF: All participants who received at least 1 dose or part of a dose.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Efgartigimod PH20 SC | Participants receiving efgartigimod PH20 SC treatment. | 1 | 137 | 14 | 137 | 130 | 137 |
| EG001 | Placebo PH20 SC | Participants receiving placebo PH20 SC treatment. | 0 | 70 | 10 | 70 | 65 | 70 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Autoimmune thyroiditis | Endocrine disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Amaurosis fugax | Eye disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vitreous haemorrhage | Eye disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Inguinal hernia strangulated | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Arthritis infective | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Meningitis aseptic | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Intermenstrual bleeding | Reproductive system and breast disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Oral blood blister | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Injection site haemorrhage | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Injection site rash | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Vessel puncture site haemorrhage | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
| |
| Blood urine present | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Purpura | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (26.0) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Regulatory manager | Argenx | Please email | regulatory@argenx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 7, 2023 | Oct 8, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Black or African American |
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| White |
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| Other |
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