Systemic SclerosisPhase 2NCT02551042
CSL Behring Sclero XIII
A Phase 2 study testing Fibrogammin®P, coagulation factor XIII concentrate (Human) and 0.9% sodium chloride in people with Systemic Sclerosis, run by University College, London at 1 site in United Kingdom.
Trial summary
Many patients with Scleroderma (Systemic sclerosis) experience damage to blood vessels, mainly to the small arteries. A common manifestation of this is Raynaud's phenomenon (fingers or toes turning white then blue in the cold) and digital ulcers (open sores on the fingertips). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Sclerosis
- What is being tested?
- Fibrogammin®P, coagulation factor XIII concentrate (Human) and 0.9% sodium chloride
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Sep 2015to Sep 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female adults.
- Subjects with a diagnosis of systemic sclerosis (scleroderma, SSc) according to the 2013 American College of Rheumatology European League Against Rheumatism (ACR EULAR) classification criteria. They will be classified according to LeRoy criteria as limited or diffuse subset.
- Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test.
- Subjects will have serological status for hepatitis A and B assessed at screening.
- Patients who have given their free and informed consent. -≥ 18 years.
Who cannot join
- Participants must:
- Be willing to use an effective method of contraception (hormonal or barrier method of birth control; abstinence) from the time consent is signed until 6 weeks after treatment discontinuation (females of childbearing potential and males)
- Have a negative pregnancy test within 7 days prior to being registered for trial treatment (females of childbearing potential). NOTE: Subjects are considered not of child bearing potential if they are surgically sterile (i.e. they have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are postmenopausal.
- Not be breastfeeding (females).
- Participants must not:
- Have allergies to excipients of the investigational medicinal product (IMP) and placebo
Show all 22Show fewer
- Have uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure ≥ 160 mmHg or sitting diastolic blood pressure ≥ 100 mmHg.
- Have portal hypertension or chronic liver disease defined as mild to severe hepatic impairment (Child-Pugh Class A-C). Subjects positive for Hepatitis C with evidence of active viral replication on sensitive polymerase chain reaction (PCR) testing are also excluded.
- Have hepatic dysfunction, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of the normal range (× ULN) at the Screening Visit.
- Have chronic renal insufficiency as defined by a serum creatinine ≥ 2.5 mg/dL (≤ 221 micromol/L) or requires dialysis.
- Have a haemoglobin concentration ≤ 10 g/dL (≤ 100 g/L) at the Screening Visit.
- Have a history of left-sided heart disease and/or clinically significant cardiac disease, including but not limited to any of the following: aortic or mitral valve disease (stenosis or regurgitation) defined as more than minimum aortic insufficiency and more than moderate mitral regurgitation (stenosis or regurgitation≥ grade 1); pericardial constriction; restrictive or congestive cardiomyopathy; left ventricular ejection fraction ≤ 40 % by multiple gated acquisition scan (MUGA), angiography, or echocardiography; left ventricular shortening fraction ≤ 22 % by echocardiography; or symptomatic coronary disease with demonstrable ischemia.
- Have a history of malignancies within 5 years of Screening Visit with the exception of localized skin or cervical carcinomas.
- Have psychiatric, addictive, or other disorder that compromises the ability to give informed consent for participating in this study. This includes subjects with a recent history of abusing alcohol or illicit drugs.
- Be receiving ongoing treatment with hyperbaric oxygen
- Have pulmonary artery hypertension (PAH)
- Have received IV Iloprost within the last 2 months
- Have been treated with sympathectomy or toxin botulinum A within the last 3 months
- Have had thrombosis, stroke, pulmonary embolism or myocardial infarction in the last 6 months
- Have a diagnosis of diabetes mellitus requiring dietary restriction of carbohydrate.
- Be on a low sodium diet on medical advice.
- Be participating in another clinical trial involving an investigational medicinal product.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Royal Free London NHS Foundation Trust
London, NW3 2QG
About this study
Official title: A Phase II, Double-blind, Randomized, Placebo-controlled Study to Investigate Pharmacokinetics (PK), Safety and Efficacy of Intravenous Factor XIII Treatment in Patients With Systemic Sclerosis
Many patients with Scleroderma (Systemic sclerosis) experience damage to blood vessels, mainly to the small arteries. A common manifestation of this is Raynaud's phenomenon (fingers or toes turning white then blue in the cold) and digital ulcers (open sores on the fingertips). The purpose of this study is to see how effective the study drug Human Factor XIII Concentrate is in treating patients who have these and other common manifestation of Scleroderma. It will be given in addition to the accepted treatments used for this disease.
Read the detailed description
This is a phase II, double-blind, randomized, placebo-controlled study to investigate pharmacokinetics (PK), safety and efficacy of intravenous factor XIII treatment in patients with systemic sclerosis.
Scleroderma (Systemic sclerosis) is a multisystem rheumatic disease that is characterised by progressive vascular damage e.g Raynaud's phenomenon and digital ulcers and organ fibrosis e.g. skin thickening and pulmonary fibrosis.The disease is associated with significant morbidity and mortality and current therapeutic options are only partially effective, including Cyclophosphamide for skin or lung fibrosis and Bosentan which reduces but does not heal digital ulcers.
There is no cure available and there is therefore a high need for new therapeutic options.Administration of human Factor XIII (FXIII) concentrate in patients with scleroderma demonstrated promising results in the 1980s and 1990s . However these studies were not performed according to current Good Clinical Practice (GCP) guidelines and involved relatively small sample sizes.
This is a single site study, therefore all study participants will be seen at the Royal Free London National Health Service (NHS) Foundation Trust.Total study duration is 36 months and will involve 2 phases: an initial single dose, pharmacokinetic (PK) phase in 8 subjects over 6 weeks and a multiple dose, active treatment phase in 18 subjects over 24 weeks. During the treatment phase subjects will be randomized at 2:1 ratio to either FXIII or Placebo and will receive biweekly injection of either factor XIII Concentrate or placebo.
- Conditions
- Systemic Sclerosis
- Being tested
- Fibrogammin®P, coagulation factor XIII concentrate (Human), 0.9% sodium chloride
- Keywords
- Digital Ulcers, Raynauds Phenomenon, Factor XIII
- Medical subject headings
- Scleroderma, Systemic, digital ulcers, Raynaud Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fibrogammin®P, coagulation factor XIII concentrate (Human)Drug
Also called Factor XIII
IV infusion
0.9% sodium chlorideDrug
Also called Normal saline
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Active treatment arm | Experimental | Fibrogammin®P, coagulation factor XIII concentrate (Human) IV infusion Receives: Drug: Fibrogammin®P, coagulation factor XIII concentrate (Human) |
| Placebo arm | Placebo comparator | Placebo will be 0.9 % Sodium chloride solution IV infusion Receives: Drug: 0.9% sodium chloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary outcome assessed by skin involvement measured with modified Rodnan skin score
Measured: 24 weeks
Primary outcome assessed by skin involvement measured with Raynaud condition score
Measured: 24 weeks
Secondary outcomes (16)
Pulmonary function measured by pulmonary function test
Pulmonary function measured by pulmonary function test
Measured: 24 weeks
Hand function measured with Cochin hand function
Hand function measured with Cochin hand function
Measured: 24 weeks
Quality of life measured with Short Form-36 (SF-36) quality of life questionnaire
Quality of life measured with SF36 quality of life questionnaire
Measured: 24 weeks
Number of new digital ulcers (DU)
Prevention of new DU: Number of new DU developed during a 24-week period of treatment
Measured: 24 weeks
Complete healing of digital ulcers (DU)
Healing of DU: Complete healing of DUs present at baseline; each DU is considered as an entity
Measured: 24 weeks
Digital ulcer pain assessment
DU Pain assessment at 4, 8, 12, 16, 24 weeks of treatment: Pain will be assessed by analogue scale for pain (Visual Analogue Scale, VAS)
Measured: 24 weeks
Digital ulcer pain assessment
DU Pain assessment at 4, 8, 12, 16, 24 weeks of treatment: Pain will be assessed by Raynaud's severity (Raynaud's condition score)
Measured: 24 weeks
Digital ulcer worsening: hospitalisation required
Number of overnight hospitalisations for digital ulcers
Measured: 24 weeks
Digital ulcer worsening: surgical intervention required
Number of additional surgical treatments for digital ulcer in outpatient clinic
Measured: 24 weeks
Digital ulcer worsening: Digital ulcer infection
Number of digital ulcers with infections
Measured: 24 weeks
Digital ulcer worsening: Gangrene
Number of episodes of gangrene
Measured: 24 weeks
Digital ulcer worsening: Amputation
Number of amputations
Measured: 24 weeks
Digital ulcer worsening: Need of local sympathectomy
Number of local sympathectomies
Measured: 24 weeks
Digital ulcer worsening: Need of toxin Botulinum A
Number of treatments with Botulinum toxin A
Measured: 24 weeks
Digital ulcer worsening: Need of oral or parenteral antibiotic
Number of treatments needed with oral or parenteral antibiotic
Measured: 24 weeks
Digital ulcer worsening: Need of intravenous (IV) Iloprost : this is considered treatment failure
Number of treatments needed with intravenous (IV) Iloprost : this is considered treatment failure
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2015 (estimated)
- Study startSep 2015
- Primary completionSep 2016 (estimated)
- Study completionSep 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (33)Papers the sponsor has linked to this study.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by University College, LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring
- Responsible party
- Sponsor
- Investigators
- Christopher Denton, PhD, Principal investigator, Royal Free London NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02551042
- Sponsor's protocol ID
- 13/0417
- Other IDs
- 2014-001101-40, 2014
- Status last verified
- May 2016
- First submitted
- 17 Dec 2014
- Last updated
- 30 May 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
LeRoy EC, Black C, Fleischmajer R, Jablonska S, Krieg T, Medsger TA Jr, Rowell N, Wollheim F. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol. 1988 Feb;15(2):202-5. No abstract available.
PubMed 3361530Nihtyanova SI, Brough GM, Black CM, Denton CP. Clinical burden of digital vasculopathy in limited and diffuse cutaneous systemic sclerosis. Ann Rheum Dis. 2008 Jan;67(1):120-3. doi: 10.1136/ard.2007.072686. Epub 2007 Jul 27.
PubMed 17660220Levy JH, Greenberg C. Biology of Factor XIII and clinical manifestations of Factor XIII deficiency. Transfusion. 2013 May;53(5):1120-31. doi: 10.1111/j.1537-2995.2012.03865.x. Epub 2012 Aug 28.
PubMed 22928875Yee VC, Pedersen LC, Le Trong I, Bishop PD, Stenkamp RE, Teller DC. Three-dimensional structure of a transglutaminase: human blood coagulation factor XIII. Proc Natl Acad Sci U S A. 1994 Jul 19;91(15):7296-300. doi: 10.1073/pnas.91.15.7296.
PubMed 7913750Nugent DJ. Prophylaxis in rare coagulation disorders -- factor XIII deficiency. Thromb Res. 2006;118 Suppl 1:S23-8. doi: 10.1016/j.thromres.2006.02.009. Epub 2006 Apr 17.
PubMed 16616323Ivaskevicius V, Seitz R, Kohler HP, Schroeder V, Muszbek L, Ariens RA, Seifried E, Oldenburg J; Study Group. International registry on factor XIII deficiency: a basis formed mostly on European data. Thromb Haemost. 2007 Jun;97(6):914-21.
PubMed 17549292Dallabrida SM, Falls LA, Farrell DH. Factor XIIIa supports microvascular endothelial cell adhesion and inhibits capillary tube formation in fibrin. Blood. 2000 Apr 15;95(8):2586-92.
PubMed 10753838Dardik R, Shenkman B, Tamarin I, Eskaraev R, Harsfalvi J, Varon D, Inbal A. Factor XIII mediates adhesion of platelets to endothelial cells through alpha(v)beta(3) and glycoprotein IIb/IIIa integrins. Thromb Res. 2002 Feb 15;105(4):317-23. doi: 10.1016/s0049-3848(02)00014-2.
PubMed 12031826Dardik R, Solomon A, Loscalzo J, Eskaraev R, Bialik A, Goldberg I, Schiby G, Inbal A. Novel proangiogenic effect of factor XIII associated with suppression of thrombospondin 1 expression. Arterioscler Thromb Vasc Biol. 2003 Aug 1;23(8):1472-7. doi: 10.1161/01.ATV.0000081636.25235.C6. Epub 2003 Jun 12.
PubMed 12805075Dardik R, Leor J, Skutelsky E, Castel D, Holbova R, Schiby G, Shaish A, Dickneite G, Loscalzo J, Inbal A. Evaluation of the pro-angiogenic effect of factor XIII in heterotopic mouse heart allografts and FXIII-deficient mice. Thromb Haemost. 2006 Mar;95(3):546-50. doi: 10.1160/TH05-06-0409.
PubMed 16525585Khanna D, Furst DE, Hays RD, Park GS, Wong WK, Seibold JR, Mayes MD, White B, Wigley FF, Weisman M, Barr W, Moreland L, Medsger TA Jr, Steen VD, Martin RW, Collier D, Weinstein A, Lally EV, Varga J, Weiner SR, Andrews B, Abeles M, Clements PJ. Minimally important difference in diffuse systemic sclerosis: results from the D-penicillamine study. Ann Rheum Dis. 2006 Oct;65(10):1325-9. doi: 10.1136/ard.2005.050187. Epub 2006 Mar 15.
PubMed 16540546Kilian O, Fuhrmann R, Alt V, Noll T, Coskun S, Dingeldein E, Schnettler R, Franke RP. Plasma transglutaminase factor XIII induces microvessel ingrowth into biodegradable hydroxyapatite implants in rats. Biomaterials. 2005 May;26(14):1819-27. doi: 10.1016/j.biomaterials.2004.06.015.
PubMed 15576156Mirochnik Y, Kwiatek A, Volpert OV. Thrombospondin and apoptosis: molecular mechanisms and use for design of complementation treatments. Curr Drug Targets. 2008 Oct;9(10):851-62. doi: 10.2174/138945008785909347.
PubMed 18855619Nor JE, Mitra RS, Sutorik MM, Mooney DJ, Castle VP, Polverini PJ. Thrombospondin-1 induces endothelial cell apoptosis and inhibits angiogenesis by activating the caspase death pathway. J Vasc Res. 2000 May-Jun;37(3):209-18. doi: 10.1159/000025733.
PubMed 10859480Streit M, Velasco P, Riccardi L, Spencer L, Brown LF, Janes L, Lange-Asschenfeldt B, Yano K, Hawighorst T, Iruela-Arispe L, Detmar M. Thrombospondin-1 suppresses wound healing and granulation tissue formation in the skin of transgenic mice. EMBO J. 2000 Jul 3;19(13):3272-82. doi: 10.1093/emboj/19.13.3272.
PubMed 10880440
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Double-blind, Randomized, Placebo-controlled Study to Investigate Pharmacokinetics (PK), Safety and Efficacy of Intravenous Factor XIII Treatment in Patients With Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT02551042
- Acronym / sponsor ID
- 13/0417
- Conditions
- Systemic Sclerosis
- Keywords
- Digital Ulcers, Raynauds Phenomenon, Factor XIII
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Collaborators
- CSL Behring
- Study start
- Sep 2015
- Primary completion
- Sep 2016 (estimated)
- Study completion
- Sep 2018 (estimated)
- First posted
- 16 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Many patients with Scleroderma (Systemic sclerosis) experience damage to blood vessels, mainly to the small arteries. A common manifestation of this is Raynaud's phenomenon (fingers or toes turning white then blue in the cold) and digital ulcers (open sores on the fingertips). The purpose of this study is to see how effective the study drug Human Factor XIII Concentrate is in treating patients who have these and other common manifestation of Scleroderma. It will be given in addition to the accepted treatments used for this disease.
Detailed description
This is a phase II, double-blind, randomized, placebo-controlled study to investigate pharmacokinetics (PK), safety and efficacy of intravenous factor XIII treatment in patients with systemic sclerosis.
Scleroderma (Systemic sclerosis) is a multisystem rheumatic disease that is characterised by progressive vascular damage e.g Raynaud's phenomenon and digital ulcers and organ fibrosis e.g. skin thickening and pulmonary fibrosis.The disease is associated with significant morbidity and mortality and current therapeutic options are only partially effective, including Cyclophosphamide for skin or lung fibrosis and Bosentan which reduces but does not heal digital ulcers.
There is no cure available and there is therefore a high need for new therapeutic options.Administration of human Factor XIII (FXIII) concentrate in patients with scleroderma demonstrated promising results in the 1980s and 1990s . However these studies were not performed according to current Good Clinical Practice (GCP) guidelines and involved relatively small sample sizes.
This is a single site study, therefore all study participants will be seen at the Royal Free London National Health Service (NHS) Foundation Trust.Total study duration is 36 months and will involve 2 phases: an initial single dose, pharmacokinetic (PK) phase in 8 subjects over 6 weeks and a multiple dose, active treatment phase in 18 subjects over 24 weeks. During the treatment phase subjects will be randomized at 2:1 ratio to either FXIII or Placebo and will receive biweekly injection of either factor XIII Concentrate or placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active treatment arm | Experimental | Drug: Fibrogammin®P, coagulation factor XIII concentrate (Human)Fibrogammin®P, coagulation factor XIII concentrate (Human) IV infusion |
| Placebo arm | Placebo comparator | Drug: 0.9% sodium chloridePlacebo will be 0.9 % Sodium chloride solution IV infusion |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male and female adults.
- Subjects with a diagnosis of systemic sclerosis (scleroderma, SSc) according to the 2013 American College of Rheumatology European League Against Rheumatism (ACR EULAR) classification criteria. They will be classified according to LeRoy criteria as limited or diffuse subset.
- Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test.
- Subjects will have serological status for hepatitis A and B assessed at screening.
- Patients who have given their free and informed consent. -≥ 18 years.
Key exclusion criteria (22 listed)
- Be willing to use an effective method of contraception (hormonal or barrier method of birth control; abstinence) from the time consent is signed until 6 weeks after treatment discontinuation (females of childbearing potential and males)
- Have a negative pregnancy test within 7 days prior to being registered for trial treatment (females of childbearing potential). NOTE: Subjects are considered not of child bearing potential if they are surgically sterile (i.e. they have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are postmenopausal.
- Not be breastfeeding (females).
- Have allergies to excipients of the investigational medicinal product (IMP) and placebo
- Have uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure ≥ 160 mmHg or sitting diastolic blood pressure ≥ 100 mmHg.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fibrogammin®P, coagulation factor XIII concentrate (Human)Also: Factor XIIIIV infusion | Drug | Active treatment arm |
| 0.9% sodium chlorideAlso: Normal salineIV infusion | Drug | Placebo arm |
Primary outcome
Primary outcome assessed by skin involvement measured with modified Rodnan skin score
Time frame: 24 weeks
Primary outcome assessed by skin involvement measured with Raynaud condition score
Time frame: 24 weeks
16 secondary outcomes
Pulmonary function measured by pulmonary function test
Time frame: 24 weeks
Hand function measured with Cochin hand function
Time frame: 24 weeks
Quality of life measured with Short Form-36 (SF-36) quality of life questionnaire
Time frame: 24 weeks
Number of new digital ulcers (DU)
Time frame: 24 weeks
Complete healing of digital ulcers (DU)
Time frame: 24 weeks
Digital ulcer pain assessment
Time frame: 24 weeks
Digital ulcer pain assessment
Time frame: 24 weeks
Digital ulcer worsening: hospitalisation required
Time frame: 24 weeks
Digital ulcer worsening: surgical intervention required
Time frame: 24 weeks
Digital ulcer worsening: Digital ulcer infection
Time frame: 24 weeks
Digital ulcer worsening: Gangrene
Time frame: 24 weeks
Digital ulcer worsening: Amputation
Time frame: 24 weeks
Digital ulcer worsening: Need of local sympathectomy
Time frame: 24 weeks
Digital ulcer worsening: Need of toxin Botulinum A
Time frame: 24 weeks
Digital ulcer worsening: Need of oral or parenteral antibiotic
Time frame: 24 weeks
Digital ulcer worsening: Need of intravenous (IV) Iloprost : this is considered treatment failure
Time frame: 24 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2016.
- Background: PubMed 3361530LeRoy EC, Black C, Fleischmajer R, Jablonska S, Krieg T, Medsger TA Jr, Rowell N, Wollheim F. Scleroderma (systemic sclerosis): classification, subsets and pathogenesis. J Rheumatol. 1988 Feb;15(2):202-5. No abstract available.
- Background: PubMed 17660220Nihtyanova SI, Brough GM, Black CM, Denton CP. Clinical burden of digital vasculopathy in limited and diffuse cutaneous systemic sclerosis. Ann Rheum Dis. 2008 Jan;67(1):120-3. doi: 10.1136/ard.2007.072686. Epub 2007 Jul 27.
- Background: PubMed 22928875Levy JH, Greenberg C. Biology of Factor XIII and clinical manifestations of Factor XIII deficiency. Transfusion. 2013 May;53(5):1120-31. doi: 10.1111/j.1537-2995.2012.03865.x. Epub 2012 Aug 28.
- Background: PubMed 7913750Yee VC, Pedersen LC, Le Trong I, Bishop PD, Stenkamp RE, Teller DC. Three-dimensional structure of a transglutaminase: human blood coagulation factor XIII. Proc Natl Acad Sci U S A. 1994 Jul 19;91(15):7296-300. doi: 10.1073/pnas.91.15.7296.
- Background: PubMed 16616323Nugent DJ. Prophylaxis in rare coagulation disorders -- factor XIII deficiency. Thromb Res. 2006;118 Suppl 1:S23-8. doi: 10.1016/j.thromres.2006.02.009. Epub 2006 Apr 17.
- Background: PubMed 17549292Ivaskevicius V, Seitz R, Kohler HP, Schroeder V, Muszbek L, Ariens RA, Seifried E, Oldenburg J; Study Group. International registry on factor XIII deficiency: a basis formed mostly on European data. Thromb Haemost. 2007 Jun;97(6):914-21.
- Background: PubMed 10753838Dallabrida SM, Falls LA, Farrell DH. Factor XIIIa supports microvascular endothelial cell adhesion and inhibits capillary tube formation in fibrin. Blood. 2000 Apr 15;95(8):2586-92.
- Background: PubMed 12031826Dardik R, Shenkman B, Tamarin I, Eskaraev R, Harsfalvi J, Varon D, Inbal A. Factor XIII mediates adhesion of platelets to endothelial cells through alpha(v)beta(3) and glycoprotein IIb/IIIa integrins. Thromb Res. 2002 Feb 15;105(4):317-23. doi: 10.1016/s0049-3848(02)00014-2.
- Background: PubMed 12805075Dardik R, Solomon A, Loscalzo J, Eskaraev R, Bialik A, Goldberg I, Schiby G, Inbal A. Novel proangiogenic effect of factor XIII associated with suppression of thrombospondin 1 expression. Arterioscler Thromb Vasc Biol. 2003 Aug 1;23(8):1472-7. doi: 10.1161/01.ATV.0000081636.25235.C6. Epub 2003 Jun 12.
- Background: PubMed 16525585Dardik R, Leor J, Skutelsky E, Castel D, Holbova R, Schiby G, Shaish A, Dickneite G, Loscalzo J, Inbal A. Evaluation of the pro-angiogenic effect of factor XIII in heterotopic mouse heart allografts and FXIII-deficient mice. Thromb Haemost. 2006 Mar;95(3):546-50. doi: 10.1160/TH05-06-0409.
- Background: PubMed 16540546Khanna D, Furst DE, Hays RD, Park GS, Wong WK, Seibold JR, Mayes MD, White B, Wigley FF, Weisman M, Barr W, Moreland L, Medsger TA Jr, Steen VD, Martin RW, Collier D, Weinstein A, Lally EV, Varga J, Weiner SR, Andrews B, Abeles M, Clements PJ. Minimally important difference in diffuse systemic sclerosis: results from the D-penicillamine study. Ann Rheum Dis. 2006 Oct;65(10):1325-9. doi: 10.1136/ard.2005.050187. Epub 2006 Mar 15.
- Background: PubMed 15576156Kilian O, Fuhrmann R, Alt V, Noll T, Coskun S, Dingeldein E, Schnettler R, Franke RP. Plasma transglutaminase factor XIII induces microvessel ingrowth into biodegradable hydroxyapatite implants in rats. Biomaterials. 2005 May;26(14):1819-27. doi: 10.1016/j.biomaterials.2004.06.015.
- NovoThirteen® Summary of Product CharacteristicsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Scleroderma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher Denton, PhD principal investigator
Royal Free London NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fibrogammin®P, coagulation factor XIII concentrate (Human) and 0.9% sodium chloride in people with Systemic Sclerosis, run by University College, London at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 30 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Christopher Denton, PhD (02073177544, c.denton@ucl.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary outcome assessed by skin involvement measured with modified Rodnan skin score (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02551042, which also lists 33 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2016.