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| ID | Type | Description | Link |
|---|---|---|---|
| 2010-022780-35 | EudraCT Number |
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| Name | Class |
|---|---|
| Parexel | INDUSTRY |
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Chronic graft-versus-host disease (cGVHD) is a long-lasting complication that can occur after transplants. The transplanted cells seem to fight with the patient's own cells.
Extracorporeal photopheresis (ECP) is a fairly new procedure for cGVHD. The participant gets a port to hook up to a machine. The machine removes the white blood cells, mixes them with a light-sensitive drug, shines light on it, and puts all the blood back in.
This study will find out if patients respond better if they get ECP with methoxsalen, in addition to the pills normally used to treat cGVHD.
The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use.
This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment.
Adult patients with new-onset (≤3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ECP Methoxsalen + Standard of Care | Experimental | Participants receive methoxsalen administered via ECP in addition to standard of care |
|
| Standard of Care | Active Comparator | Participants receive standard of care only |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ECP Methoxsalen | Drug | Methoxsalen delivered by ECP |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With an Overall Response at Week 28 | Participants with overall response included those with partial response or complete response, according to study staff who did not know which treatment they received (blinded assessment). | Week 28 |
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Inclusion Criteria:
To be eligible for inclusion in this trial, the patient must have moderate to severe chronic graft-versus-host disease [defined by the National Institutes of Health (NIH) 2015 Consensus Criteria] that started within 3 years after hematopoietic stem cell transplantation (HSCT).
Exclusion Criteria:
Is intolerant to corticosteroids or hypersensitive to methoxsalen
Received certain treatments during time periods disallowed by protocol
Has infections and/or requires treatment that (per protocol or in the opinion of the investigator) might compromise:
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| Name | Affiliation | Role |
|---|---|---|
| Global Clinical Leader | Mallinckrodt | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Miami Hospital | Miami | Florida | 33136 | United States | ||
| Indiana Blood and Marrow Transplantation Research |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31332045 | Result | Jagasia M, Scheid C, Socie G, Ayuk FA, Tischer J, Donato ML, Batai A, Chen H, Chen SC, Chin T, Boodee H, Mitri G, Greinix HT. Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD. Blood Adv. 2019 Jul 23;3(14):2218-2229. doi: 10.1182/bloodadvances.2019000145. |
| Label | URL |
|---|---|
| Cited article made available by the US National Library of Medicine, National Institutes of Health | View source |
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All participants enrolled were included in the safety analysis set
Patients were enrolled in Austria, France, Germany, Italy, Hungary, Spain, the United Kingdom, and the United States. Enrollment started in November 2011, and the last patient visit occurred in March 2015.
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| ID | Title | Description |
|---|---|---|
| FG000 | ECP Methoxsalen + SoC | Participants receive ECP methoxsalen in addition to standard of care |
| FG001 | Standard of Care (SoC) | Participants receive only standard of care |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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Pilot Study for the 2015 NIH Consensus Criteria to inform the design of subsequent studies
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| Standard of Care | Drug | Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus) |
|
|
| Indianapolis |
| Indiana |
| 46237 |
| United States |
| Tulane University Health Sciences Center, 1430 Tulane Avenue, Rm 7551, | New Orleans | Louisiana | 70112 | United States |
| Karmanos Cancer Institute | Detroit | Michigan | 48201 | United States |
| Hackensack University Medical Center | Hackensack | New Jersey | 07601 | United States |
| New York Presbyterian Hospital | New York | New York | 10065 | United States |
| Stony brook university medical Center | Stony Brook | New York | 11794 | United States |
| Vanderbilt Ingram Cancer Center | Nashville | Tennessee | 37232 | United States |
| Medical City Dallas Hospital,Transplant center | Dallas | Texas | 75230 | United States |
| Parkland Memorial Hospital | Dallas | Texas | 75235 | United States |
| Methodist healthcare system of san Antonio | San Antonio | Texas | 78229 | United States |
| General Hospital of Vienna (Medical University of Vienna) | Vienna | Austria |
| Hopital Saint Antoine | Saint-Antoine | Paris | 75571 | France |
| CHU de Nantes | Nantes | France |
| Hospital Saint Louis | Paris | France |
| Universitätsklinik Köln | Cologne | 50937 | Germany |
| Universitäts-Klinikum Carl Gustav Carus | Dresden | 01309 | Germany |
| Universitatsklinikum Erlangen | Erlangen | 91054 | Germany |
| Universitätsklinikum Essen | Essen | 45122 | Germany |
| Universitatskrankenhaus Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| Universitätsklinikum Leipzig AöR | Leipzig | 04103 | Germany |
| Universitätklinikum Mannheim | Mannheim | 68167 | Germany |
| Medizinische Universitatsklinik | München | 81377 | Germany |
| Uniklinik für Kinder und Jugendmedizin | Tübingen | 72076 | Germany |
| EgyesÃtett Szent István és Szent László Kórház, Gyáli út 5-7, | Budapest | 1097 | Hungary |
| A.O.U. Policlinico- Vittorio Emanuele | Catania | Italy |
| Universita de Genova - Ospedale S. Martino | Genova | Italy |
| Hospital Marques de Valdecilla | Santander | Cantabria | 39008 | Spain |
| Santa Creu I Sant Pau | Barcelona | 08025 | Spain |
| Kings College Hospital | London | Greater London | SE5 9RS | United Kingdom |
| University of Birmingham: Queen Elizabeth Hospital | Birmingham | United Kingdom |
| Royal Marsden Hospital | London | United Kingdom |
| Baseline Population | All enrolled participants |
|
| Safety Population | All randomized participants who received at least 1 dose of study treatment, per treatment received. |
|
| Intent-to-treat Population | All randomized subjects with baseline and post-treatment measurements, per assigned treatment |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
All participants enrolled and included in the safety analysis set
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| ID | Title | Description |
|---|---|---|
| BG000 | ECP Methoxsalen + SoC | Participants receive ECP methoxsalen in addition to standard of care |
| BG001 | Standard of Care (SOC) | Participants receive only standard of care |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||
| Region of Enrollment | Number | 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 | Number of Participants With an Overall Response at Week 28 | Participants with overall response included those with partial response or complete response, according to study staff who did not know which treatment they received (blinded assessment). | Intention to treat population | Posted | Count of Participants | Participants | Week 28 |
|
|
|
|
Treatment emergent adverse events were recorded from the start of treatment through Week 28.
Treatment emergent adverse events (TEAEs) are unpleasant events not there before the medical treatment, or that were there, but got worse or happened more often after the treatment. Both serious and non-serious adverse events reported here are TEAEs. All-cause mortality is based on baseline population, while serious adverse events and non-serious adverse events are based on the safety population.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ECP Methoxsalen + SoC | Participants receive ECP methoxsalen in addition to standard of care | 4 | 29 | 8 | 28 | 26 | 28 |
| EG001 | Standard of Care (SoC) | Participants receive only standard of care | 0 | 31 | 9 | 28 | 24 | 28 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiac Failure | Cardiac disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Thyroiditis subacute | Endocrine disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Graft versus host disease in liver | Immune system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Escherichia infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Herpes simplex | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Toxoplasmosis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Viraemia | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Myopathy | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Polyneuropathy | Nervous system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Vocal cord polyp | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dry Eye | Eye disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (16.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Blood cholesterol increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hypoproteinaemia | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Myopathy | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dry Skin | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
|
Long term follow-up (LTFU) safety data were provided to health authorities in safety reports; not the 28-week protocol.
LTFU pre-specified outcome measures previously entered in error were removed.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information Call Center | Mallinckrodt Pharmaceuticals | 800-556-3314 | 5 | clinicaltrials@mnk.com |
| ID | Term |
|---|---|
| D000092122 | Bronchiolitis Obliterans Syndrome |
| ID | Term |
|---|---|
| D000092124 | Organizing Pneumonia |
| D001989 | Bronchiolitis Obliterans |
| D001988 | Bronchiolitis |
| D001991 | Bronchitis |
| D001982 | Bronchial Diseases |
| D012140 | Respiratory Tract Diseases |
| D008173 | Lung Diseases, Obstructive |
| D008171 | Lung Diseases |
| D006086 | Graft vs Host Disease |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D008730 | Methoxsalen |
| D017893 | Photopheresis |
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D011564 | Furocoumarins |
| D003374 | Coumarins |
| D001578 | Benzopyrans |
| D011714 | Pyrans |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006575 | Heterocyclic Compounds, 3-Ring |
| D011701 | PUVA Therapy |
| D014467 | Ultraviolet Therapy |
| D010789 | Phototherapy |
| D013812 | Therapeutics |
| D005112 | Extracorporeal Circulation |
| D013514 | Surgical Procedures, Operative |
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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| Title | Measurements |
|---|---|
|
| 65 years and over |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black |
|
| Asian |
|
| Other |
|
| Not Collected |
|
| Hungary |
|
| United States |
|
| Italy |
|
| United Kingdom |
|
| France |
|
| Germany |
|
| Spain |
|