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| ID | Type | Description | Link |
|---|---|---|---|
| MOM-M281-006 | Other Identifier | Janssen Research & Development, LLC | |
| 2019-000720-17 | EudraCT Number | ||
| 2023-505321-14-00 | Registry Identifier | EUCT number |
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The main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).
The study consists of a 24-week double-blind, placebo control period, a 144-week open-label extension period and follow-up period of 8 weeks after last study drug administration. Eligible participants will be randomized to placebo or nipocalimab (2 dose levels) during the double-blind period and nipocalimab (2 dose levels) during the open-label extension period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| M281 administered every 4 weeks (double-blind period) | Experimental | Participants will receive M281 administered every 4 weeks alternating with placebo every 4 weeks during the 24 weeks double-blind period. |
|
| M281 administered every 2 weeks (double-blind period) | Experimental | Participants will receive M281 administered every 2 weeks during the 24 weeks double-blind period. |
|
| Placebo administered every 2 weeks (double-blind period) | Experimental | Participants will receive M281 matching placebo administered every 2 weeks during the 24 weeks double-blind period. |
|
| M281 administered every 4 weeks (open-label extension period) | Experimental | Participants will receive M281 administered every 4 weeks during the 144 weeks open-label extension period. |
|
| M281 administered every 2 weeks (open-label extension period) | Experimental | Participants will receive M281 administered every 2 weeks during the 144 weeks open-label extension period. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| M281 | Drug | M281 injection administered as intravenous infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb) | Up to Week 20 of the double-blind period |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Total Score From the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale at the Time of Durable Response | The FACIT-Fatigue is a self-administered 13-item questionnaire that assess patient-reported fatigue associated with chronic illness therapy. It assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement. |
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Inclusion criteria:
Exclusion criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Janssen Research & Development, LLC Clinical Trial | Janssen Research & Development, LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| MemorialCare Medical Group | Fountain Valley | California | 92708 | United States | ||
| University of Southern California |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33786812 | Derived | Liu AP, Cheuk DK. Disease-modifying treatments for primary autoimmune haemolytic anaemia. Cochrane Database Syst Rev. 2021 Mar 26;3(3):CD012493. doi: 10.1002/14651858.CD012493.pub2. |
| Label | URL |
|---|---|
| Participant recruitment poster | View source |
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|
| Placebo | Drug | Placebo administered as intravenous infusion |
|
| Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in the Total Score From the FACIT-Fatigue Scale at the end of the Double-blind Period (Week 24) | The FACIT-Fatigue is a self-administered 13-item questionnaire that assess patient-reported fatigue associated with chronic illness therapy. It assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement. | Baseline (Day 1, Week 0) through Week 24 of the double-blind period |
| Change from Baseline in Average Daily Dose of Prednisone or Equivalent | Change from baseline in average daily dose of prednisone or equivalent at week 24 among participants on prednisone or equivalent at baseline will be reported. | Baseline (Day 1, Week 0) and at Week 24 |
| Number of Participants That Simultaneously Attain Normal Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin Levels at a Minimum of 3 Consecutive Visits After Baseline | Baseline (Day 1, Week 0) through Week 24 |
| Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at any Time During the Study | Baseline (Day 1, Week 0) through Week 24 |
| Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at 3 Consecutive Visits | Baseline (Day 1, Week 0) through Week 24 |
| Percentage of Participants who Achieve the Durable Response in Improvement of Hgb During the Double-blind Period and Maintain that Response for Up to 24 Weeks, Without the Need of Rescue Therapy | Percentage of participants who achieve the durable response in improvement of Hgb during the double-blind period and maintain that response for up to 24 weeks, without the need of rescue therapy will be reported. | Up to 24 weeks |
| Change From Baseline in Hgb Concentration | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in Reticulocyte Count | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in Hemolytic Marker - Haptoglobin | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in Hemolytic Marker - Indirect Bilirubin | Baseline (Day 1, Week 0) through Week 24 |
| Time to Hgb Response | Baseline (Day 1, Week 0) through Week 24 |
| Mean Time During Which the Primary Endpoint is Maintained | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in the Total Score, Item Scores, and Impact and Experience Domains From the FACIT-Fatigue Scale | The FACIT-Fatigue scale is a 13-item self-administered questionnaire that assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement. | Baseline (Day 1, Week 0) through Week 24 of the double-blind period |
| Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score | The EQ-5D-5L quality of life questionnaire will be used to assess health related quality of life status. The 5 dimensions are mobility, self-care, usual activities, pain/discomfort, and anxiety/depression; each dimension is rated by the patient on a 5 level scale (no problems, slight problems, moderate problems, severe problems, extreme problems). | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in Medical Outcomes Study Short Form 36 Item Health Survey Version 2 Acute (SF-36v2) Score | The SF-36v2 will be used to assess general quality of life. The 36 items on the SF-36 health survey encompass the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. The 8 domains can be aggregated into 2 summary scales that reflect physical and mental health: a physical component summary (PCS) and a mental component summary (MCS). Responses to all items are rated on a 3-, 5- or 6-point Likert scale. Higher scores indicate a higher level of functioning. A positive change from baseline score indicates an improvement. | Baseline (Day 1, Week 0) through Week 24 |
| Change From Baseline in Patient Global Impression of Severity (PGIS) | The PGIS will be used to assess the severity of warm autoimmune hemolytic anemia (wAIHA) fatigue symptoms. The PGIS is a 5-point response scale. Participant will be asked to rate their fatigue over the past 7 days using the following 5-point scale: 1 = None, 2 = Mild, 3 = Moderate, 4 = Severe, and 5 = Very severe. | Baseline (Day 1, Week 0) through Week 24 |
| Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score | The PGIC will assess if there has been an improvement or decline in patient-reported status since the beginning of the treatment. The PGIC is a 7-point response scale. Participants will be asked to rate their current fatigue as compared to when they started the study, using the following 7-point scale: 1 = Much better, 2 = Moderately better, 3 = A little better, 4 = No change, 5 = A little worse, 6 = Moderately worse, and 7 = Much worse. | At Week 24 |
| Hgb Range at Steady State | It will be estimated using a model-based longitudinal analysis of Hgb/hemolysis parameters in relationship to IgG level and dose regimen. | Baseline (Day 1, Week 0) through Week 24 |
| Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent | Absolute change from baseline in average daily dose of prednisone or equivalent at Week 24 among all participants will be reported. | Baseline (Day 1, Week 0) and at Week 24 |
| Percentage of participants who Achieve Corticosteroid Reduction to less than or equal to (<=) 7.5 milligrams per day (mg/day) of Oral Prednisone (or Equivalent), Among Participants with Prednisone or Equivalent greater than (>) 7.5 mg/day at Baseline | Percentage of participants who achieve corticosteroid reduction to <= 7.5 mg/day of oral prednisone (or equivalent) at Week 24 of the double-blind period, among participants with prednisone or equivalent >7.5 mg/day at baseline will be reported. | At Week 24 |
| Los Angeles |
| California |
| 90033 |
| United States |
| Compassionate Cancer Care | Riverside | California | 92501 | United States |
| American Institute of Research | Whittier | California | 90603 | United States |
| University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | United States |
| GNP Research | Cooper City | Florida | 33024 | United States |
| University of Florida College of Medicine | Gainesville | Florida | 32610 | United States |
| 21st Century Oncology | Jacksonville | Florida | 32204 | United States |
| Lakes Research | Miami Lakes | Florida | 33014 | United States |
| AdventHealth Cancer Institute | Orlando | Florida | 32804 | United States |
| Children's Research Institute | St. Petersburg | Florida | 33701 | United States |
| Rush University Medical Center | Chicago | Illinois | 60612 | United States |
| Alliance for Multispeciality Research | Merriam | Kansas | 66204 | United States |
| Tulane University School of Medicine | New Orleans | Louisiana | 70112 | United States |
| Henry Ford Medical Center | Detroit | Michigan | 48202 | United States |
| Research Medical Center | Kansas City | Missouri | 64132 | United States |
| Montefiore Medical Center | Lake Success | New York | 11042 | United States |
| Monter Cancer Center | Lake Success | New York | 11042 | United States |
| Hematology Oncology Associates of Rockland | Nyack | New York | 10960 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| East Carolina University | Greenville | North Carolina | 27834 | United States |
| Taussig Cancer Insititute Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| The Ohio State University- James Cancer Hospital | Columbus | Ohio | 43210 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Hospital of the University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| West Penn Hospital | Pittsburgh | Pennsylvania | 15224 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Northwest Medical Specialists | Tacoma | Washington | 98405 | United States |
| University of Wisconsin Carbone Cancer Center | Madison | Wisconsin | 53792 | United States |
| Hospital das Clínicas da Faculdade de Medicina de Botucatu | Botucatu | 18610-070 | Brazil |
| Impar Servicos Hospitalares S/A - Hospital Brasilia | Brasília | 71681-603 | Brazil |
| CTO Centro de Tratamento Oncologico Unidade Belem | Campinas | 13083-887 | Brazil |
| Hospital Das Clinicas Da Universidade Federal De Goias | Goiânia | 74605-020 | Brazil |
| Complexo Hospitalar de Niteroi | Niterói | 24020-096 | Brazil |
| Oncoclinicas - Unidade OC Oncoclinicas Multihemo Ilha do Leite | Recife | 50040-000 | Brazil |
| Nucleo de Oncologia da Bahia | Salvador | 40170-110 | Brazil |
| Hospital Sao Rafael | Salvador | 41253 190 | Brazil |
| CEPHO Centro de Estudos e Pesquisa de Hematologia e Oncologia | Santo André | 09060 650 | Brazil |
| Hospital Santa Marcelina | Santo André | 09060-870 | Brazil |
| Fundacao Faculdade Regional de Medicina de Sao Jose do Rio Preto Hospital de Base | São José do Rio Preto | 15090 000 | Brazil |
| Sociedade Beneficente de Senhoras Hospital Sirio Libanes | São Paulo | 01308 050 | Brazil |
| Universidade Federal de Sao Paulo | São Paulo | 04024-002 | Brazil |
| Hospital Das Clinicas Da Faculdade De Medicina Da USP | São Paulo | 05403 000 | Brazil |
| Beijing Chao-yang Hospital, Capital Medical University | Beijing | 100020 | China |
| Peking University People's Hospital | Beijing | 100044 | China |
| The First Hospital of Jilin University | Changchun | 130021 | China |
| West China Hospital of Sichuan University | Chengdu | 610041 | China |
| Guangzhou First Municipal People's Hospital | Guangzhou | 510180 | China |
| Hainan General Hospital | Haikou | 570311 | China |
| First Affiliated Hospital Medical School of Zhejiang University | Hangzhou | 310003 | China |
| Lanzhou University Second Hospital | Lanzhou | 730030 | China |
| Jiangsu Province Hospital | Nanjing | 210029 | China |
| Affiliated Hospital of Nantong University | Nantong | 226001 | China |
| Shanghai Zhongshan Hospital | Shanghai | 200032 | China |
| Huashan Hospital affiliated to Fudan University | Shanghai | 200040 | China |
| The First Affiliated Hospital of Soochow University | Suzhou | 215006 | China |
| Tianjin Medical University General Hospital | Tianjin | 300052 | China |
| Union Hospital Tongji Medical College of Huazhong University of Science and Technology | Wuhan | 430022 | China |
| The First Affiliated Hospital of Xian Jiaotong University | Xi'an | 710061 | China |
| Fakultni nemocnice Brno | Brno | 625 00 | Czechia |
| Fakultni nemocnice Kralovske Vinohrady | Prague | 100 34 | Czechia |
| Ustav Hematologie A Krevni Transfuze | Prague | 128 00 | Czechia |
| Mansoura University Hospital | Al Mansurah | 35516 | Egypt |
| Alexandria University | Alexandria | 21561 | Egypt |
| Cairo university | Cairo | 11211 | Egypt |
| National Cancer Institute Cairo | Cairo | 11796 | Egypt |
| Ain Shams University Hospital | Cairo | 1181 | Egypt |
| Nasser Institute Hospital for Research and Treatment | Cairo | 12655 | Egypt |
| CHRU Hopital Amiens Picardie Sud | Amiens | 80054 | France |
| ICH Hopital A. Morvan | Brest | 29609 | France |
| CHU Dijon Bourgogne | Dijon | 21000 | France |
| CHU Grenoble | La Tronche | 38700 | France |
| CHU Nantes - Hotel Dieu | Nantes | 44093 | France |
| CHU De Poitiers | Poitiers | 86021 | France |
| Onkologische Schwerpunktpraxis | Berlin | 10707 | Germany |
| Universitatsklinikum Essen | Essen | 45147 | Germany |
| Rotkreuzklinikum München | München | 80634 | Germany |
| General Hospital of Athens G Gennimatas | Athens | 115 27 | Greece |
| Laiko General Hospital of Athens | Athens | 11527 | Greece |
| Attikon University General Hospital of Attica | Athens | 12462 | Greece |
| University Hospital Of Larissa | Larissa | 41110 | Greece |
| University General Hospital of Rio Patras | Pátrai | 26504 | Greece |
| University General Hospital of Thessaloniki | Thessaloniki | 54636 | Greece |
| Semmelweis Egyetem | Budapest | 1083 | Hungary |
| Del Pesti Centrumkorhaz Orszagos Hematologiai es Infektologiai Intezet Szent Laszlo Telephely | Budapest | 1097 | Hungary |
| Debreceni Egyetem Klinikai Kozpont | Debrecen | 4032 | Hungary |
| Petz Aladar Megyei Oktato Korhaz | Gyõr | 9023 | Hungary |
| Somogy Megyei Kaposi Mor Oktato Korhaz | Kaposvár | 7400 | Hungary |
| Ha'Emek Medical Center | Afula | 1834111 | Israel |
| Soroka Medical Center | Beersheba | 8410101 | Israel |
| Rambam Health Care Campus | Haifa | 3109601 | Israel |
| Shaare Zedek Medical Center | Jerusalem | 9103102 | Israel |
| Hadassah Medical Center | Jerusalem | 9112001 | Israel |
| Hospital Sanz Medical Center Laniado Hospital | Kiryat Tzanz | 4244916 | Israel |
| Rabin Medical Center, Beilinson Hospital | Petach Tikvah | 49100 | Israel |
| Tel Aviv Sourasky Medical Center | Tel Aviv | 64239 | Israel |
| PO C e G Mazzoni AST Ascoli Piceno | Ascoli Piceno | 63100 | Italy |
| ASST Spedali Civili di Brescia | Brescia | 25123 | Italy |
| Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi | Catania | 95122 | Italy |
| AOU Policlinico G Rodolico S Marco | Catania | 95125 | Italy |
| Azienda Ospedaliero Universitaria Careggi | Florence | 50134 | Italy |
| Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Fondazione IRCCS San Gerardo dei Tintori | Monza | 20900 | Italy |
| Azienda Ospedaliero Universitaria Maggiore della Carita di Novara | Novara | 28100 | Italy |
| Umberto I Policlinico di Roma | Roma | 00161 | Italy |
| Fondazione Policlinico Universitario A Gemelli IRCCS | Roma | 00168 | Italy |
| Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino | Torino | 10126 | Italy |
| Azienda Ulss 8 Berica- Ospedale Di Vicenza | Vicenza | 36100 | Italy |
| Fukushima Medical University Hospital | Fukushima | 960 1295 | Japan |
| Saitama Medical University Hospital | Iruma-gun | 350-0495 | Japan |
| St Marianna University Hospital | Kanagawa | 216 8511 | Japan |
| Japan Community Health care Organization Kyushu Hospital | Kitakyusyu-Shi | 806-8501 | Japan |
| Yamanashi Prefectural Central Hospital | Kofu | 400-0027 | Japan |
| Ogaki Municipal Hospital | Ōgaki | 503-8502 | Japan |
| Sapporo Medical University Hospital | Sapporo | 060-8543 | Japan |
| Tohoku University Hospital | Sendai | 980 8574 | Japan |
| Osaka University Hospital | Suita | 565 0871 | Japan |
| Toyama Prefectural Central Hospital | Toyama | 930 8550 | Japan |
| Wakayama Medical University Hospital | Wakayama | 641 8510 | Japan |
| Tottori University Hospital | Yonago | 683-8504 | Japan |
| Hospital Ampang | Ampang | 68000 | Malaysia |
| Hospital Sultanah Aminah | Johor Bharu | 80100 | Malaysia |
| Hospital Queen Elizabeth | Kota Kinabalu | 88200 | Malaysia |
| University Malaya Medical Centre | Kuala Lumpur | 50603 | Malaysia |
| Hospital Tengku Ampuan Afzan | Kuantan | 25100 | Malaysia |
| Hospital Umum Sarawak | Kuching | 93586 | Malaysia |
| Sunway Medical Centre | Petaling Jaya | 47500 | Malaysia |
| Amsterdam Universitair Medische Centra Locatie Vrije Universiteit Medisch Centrum | Amsterdam | 1081 HV Amsterdam | Netherlands |
| Academic Medical Centre Amsterdam | Amsterdam | 1105 AZ | Netherlands |
| Maastricht Universitair Medisch Centrum | Maastricht | 6229 HX | Netherlands |
| Interhem | Bialystok | 15-732 | Poland |
| Klinika Hematologii i Transplantologii, UCK | Gdansk | 80-214 | Poland |
| Pratia Onkologia Katowice | Katowice | 40 519 | Poland |
| Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im M Kopernika w Lodzi | Lodz | 93 510 | Poland |
| Szpital Wojewodzki w Opolu | Opole | 45-061 | Poland |
| MTZ Clinical Research Powered by Pratia | Warsaw | 02-172 | Poland |
| Kyungpook National University Hospital | Daegu | 41944 | South Korea |
| Chungnam National University Hospital | Daejeon | 35015 | South Korea |
| Samsung Medical Center | Seoul | 06351 | South Korea |
| Asan Medical Center | Seoul | 138-040 | South Korea |
| Hosp. Univ. Principe de Asturias | Alcalá de Henares | 28805 | Spain |
| Hosp. Univ. Germans Trias I Pujol | Badalona | 08916 | Spain |
| Hosp Clinic de Barcelona | Barcelona | 08036 | Spain |
| Complejo Asistencial Univ. de Burgos | Burgos | 09006 | Spain |
| Hosp Reina Sofia | Córdoba | 14004 | Spain |
| Hosp. Univ. de Gran Canaria Dr. Negrin | Las Palmas de Gran Canaria | 35010 | Spain |
| Hosp. Univ. Infanta Leonor | Madrid | 28031 | Spain |
| Hosp. Univ. Ramon Y Cajal | Madrid | 28034 | Spain |
| Hospital Clinico San Carlos | Madrid | 28040 | Spain |
| Hosp. Univ. 12 de Octubre | Madrid | 28041 | Spain |
| Hosp. Univ. La Paz | Madrid | 28046 | Spain |
| Hospital Universitario Puerta de Hierro Majadahonda | Majadahonda | 28222 | Spain |
| Hosp Regional Univ de Malaga | Málaga | 29010 | Spain |
| Hosp Virgen de La Victoria | Málaga | 29010 | Spain |
| Hosp. Virgen Del Rocio | Málaga | 29010 | Spain |
| Hosp. Son Llatzer | Palma de Mallorca | 07198 | Spain |
| Clinica Univ. de Navarra | Pamplona | 31008 | Spain |
| Hosp. Quiron Madrid Pozuelo | Pozuelo de Alarcón | 28223 | Spain |
| Hosp. Univ. I Politecni La Fe | Valencia | 46026 | Spain |
| Hosp. Univ. Miguel Servet | Zaragoza | 50009 | Spain |
| Cherkassy Regional Oncology Dispensary, Department of Hematology | Cherkassy | 18009 | Ukraine |
| Dnipropetrovsk State Medical Academy, Dnipropetrovsk City Multifield Clinical Hospital # 4 | Dnipro | 49102 | Ukraine |
| Medical Center 'Ok Clinic' of LLC 'International Institute of Clinical Studies' | Kyiv | 02091 | Ukraine |
| Kyiv City Clinical Hospital #9, Department of infectious diseases | Kyiv | 04112 | Ukraine |
| Ternopil University Hospital of Ternopil Regional Council | Ternopil | 46002 | Ukraine |
| Leeds Teaching Hospitals NHS Trust | Leeds | LF9 7TF | United Kingdom |
| Liverpool University Hospitals NHS Foundation Trust | Liverpool | L7 8XP | United Kingdom |
| Barts Health NHS Trust | London | E1 2ES | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust | Oxford | OX3 7LE | United Kingdom |
| University Hospitals Plymouth NHS Trust | Plymouth | PL6 8DH | United Kingdom |
| University Hospital Southampton NHS Foundation Trust | Southampton | SO166YD | United Kingdom |