Hereditary Hemorrhagic TelangiectasiaPhase 2THALI-HHTNCT01485224
Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia
A Phase 2 study testing Thalidomide in people with Hereditary Hemorrhagic Telangiectasia and Epistaxis, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 1 site in Italy.
Trial summary
Hereditary hemorrhagic telangiectasia (HHT) (OMIM 187300 and 600376), also known as Rendu-Osler-Weber syndrome, is an autosomal dominant disease and has a prevalence between 1:5000 and 1:8000 in different populations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia, Epistaxis
- What is being tested?
- Thalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteItaly
- Study dates
- Nov 2011to 11 Oct 2016
- 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
- Diagnosis of HHT, according to the diagnostic criteria world-wide recognized (Curacao criteria), with severe recurrent epistaxis (grade 2-3 according to the criteria proposed by Pagella et al., i.e. at least one episode of overt bleeding/week requiring at least one blood transfusion during the last three months), and refractory to mini-invasive surgical procedures, i.e. argon plasma coagulation. For these patients, there is no effective treatment option currently available
- Age > 18 years
- Ability of signing written informed consent
- Women of childbearing potential:
- declared intention not to start a pregnancy throughout the study and for four weeks following the date of the last dose of thalidomide (safe contraception, see Celgene guidelines, "Programma di Prevenzione della Gravidanza")
- negative serum pregnancy test obtained within 48 hours prior to the first dose of Thalidomide
Show all 10Show fewer
- declared intention to undergo pregnancy tests periodically while on the study medication
- Males with female partner of childbearing potential:
- declared intention not to father throughout the study and for one week following the date of the last dose of thalidomide (safe contraception, see Celgene guidelines, "Programma di Prevenzione della Gravidanza")
- Estimated life expectancy must be greater than 10 months
Who cannot join
- Pregnant or lactating women, or potentially fertile (both males and females) who have not agreed to avoid pregnancy during the trial period and for four weeks (females) or one week (males) following the date of the last dose of thalidomide
- Neurological diseases
- Psychiatric illness that would prevent granting of informed consent
- Active cardiovascular disease
- High risk for thromboembolic events (comorbidities, such as diabetes or uncontrolled infections, malignancy, immobility, prior history of thromboembolic events, use of erythropoietic agents or other agents such as hormone replacement therapy, central venous catheter, anti-cardiolipin, or anti-beta2 glycoprotein antibodies)
- Patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption since thalidomide capsules contain lactose
Trial locations and contacts
1 site in 1 country.
Italy1 site
Clinica Medica 3, Fondazione IRCCS Policlinico S. Matteo
Pavia, 27100
About this study
Official title: Efficacy of Thalidomide in the Treatment of Severe Recurrent Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
Hereditary hemorrhagic telangiectasia (HHT) (OMIM 187300 and 600376), also known as Rendu-Osler-Weber syndrome, is an autosomal dominant disease and has a prevalence between 1:5000 and 1:8000 in different populations. Clinically, the occurrence of mucocutaneous and gastrointestinal telangiectasias and of systemic arteriovenous malformations is commonly observed. Recurrent and severe epistaxis, due to the presence of telangiectasias in nasal mucosa, is the most common presentation of HHT, frequently leading to severe anemia requiring intravenous iron and blood transfusions. Although not life threatening, severe epistaxis has a great impact on quality of life in HHT patients and it represents the most important impediment in daily activities, that poses therapeutic challenge. Recently, angiogenesis has been implicated in the pathogenesis of HHT. Circulating concentrations of both TGF-beta and vascular endothelial growth factor (VEGF) are significantly elevated and therefore, anti-angiogenic substances may be effective in the treatment of vascular malformations in this disease. Thalidomide functions as a potent immunosuppressive and antiangiogenic agent. The aim of this study is to assess the clinical effects of thalidomide therapy on the severity of epistaxis in subjects with HHT who are refractory to standard therapies.
Read the detailed description
In the management of HHT epistaxis, multiple approaches have been tried, including electrocautery, laser, embolization, arterial ligation, but all approaches are largely palliative with variable results, many requiring repeated interventions. Except for nasal closure, surgical options offer, at best, limited hemorrhage-free intervals, but no definitive results and all have side effects. Moreover, currently, there is no established medical treatment available for these patients. To limit blood loss, the few medical treatments used include manipulation of the coagulation and fibrinolytic pathways or topical applications of anti-inflammatory drugs. However, multiple lesions disseminated over the entire mucosal surface are common in affected individuals, making local treatment difficult. Re-bleeding consumes a disproportionate share of healthcare resources devoted to multiple admissions, repeated endoscopies and blood transfusions.
Recently, angiogenesis has been implicated in the pathogenesis of HHT. Circulating concentrations of both TGF-beta and vascular endothelial growth factor (VEGF) are significantly elevated and therefore, anti-angiogenic substances may be effective in the treatment of vascular malformations in this disease.
Thalidomide functions as a potent immunosuppressive and antiangiogenic agent by inhibiting the phagocytic ability of inflammatory cells and the production of cytokines, such as tumor necrosis factor-alpha (TNF-a). It has been shown to be effective in the treatment of inflammatory diseases, in conditions associated with human immunodeficiency virus (HIV) infection, and in various cancers. Bleeding inhibition has been observed in HHT patients who received thalidomide as an antiangiogenic cancer therapy. A recent paper has reported that thalidomide treatment induced vessel maturation in an experimental model of HHT and reduced severe nosebleeds in six of the seven HHT patients studied. On the other hand, spectacular improvements have been described in patients with intestinal angiodysplasias, treated with thalidomide. In isolated case reports, patients with severe recurrent intestinal bleeding refractory to standard treatment achieved prolonged complete remission with thalidomide at a dose of 100 to 300 mg/day for a few months and tolerance was good. Cessation of bleeding was associated with a reduction in serum VEGF levels. These observations suggest that thalidomide might be useful for treatment of HHT patients and address significant unmet medical needs. Unfortunately, this drug exposes patients to the risk of severe side effects.
Drug metabolism is under control of a number of enzymes specific for each drug; among these enzymes, many show variable levels of activity and we can thus recognize in the population extensive (high or fast) metabolizers (EM) intermediate (IM) and poor (low or slow) metabolizers (PM). This is true also for thalidomide, whose metabolism is in part controlled by the enzyme CYP2C19.
The aims of our study are to test the hypothesis that thalidomide reduces the bleeding tendency of HHT patients and to verify to which extent CYP2C19 polymorphism modulates both response to treatment and side effects.
- Conditions
- Hereditary Hemorrhagic Telangiectasia, Epistaxis
- Being tested
- Thalidomide
- Keywords
- Hereditary hemorrhagic telangiectasia, Epistaxis, Thalidomide
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic, Epistaxis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
Also called Thalidomide Celgene
Eligible patients will receive thalidomide at a starting dose of 50 mg/day by mouth at bedtime for 4 weeks. In the event of unsatisfactory/no response, thalidomide dosage will be progressively increased by 50 mg/day every 4 weeks until complete or partial response, to a maximum dose of 200 mg/day.
Treatment will be continued until one of the following criteria is met:
- 8 additional weeks of treatment after the achievement of complete response
- 16 additional weeks of treatment after the achievement of partial response
- 24 weeks of treatment completed without response
- unacceptable toxicity. Then, patients will be followed off of thalidomide for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide | Experimental | Single arm study:
Eligible patients will receive thalidomide at a starting dose of 50 mg/day by mouth at bedtime for 4 weeks. In the event of unsatisfactory/no response, thalidomide dosage will be progressively increased by 50 mg/day every 4 weeks until complete or partial response, to a maximum dose of 200 mg/day.
Treatment will be continued until one of the following criteria is met:
* 8 additional weeks of treatment after the achievement of complete response
* 16 additional weeks of treatment after the achievement of partial response
* 24 weeks of treatment completed without response
* unacceptable toxicity. Then, patients will be followed off of thalidomide for 24 weeks. Receives: Drug: Thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients showing a decrease in the frequency, intensity and duration of epistaxis and in the blood transfusion requirement.
Cessation of nose bleeding will be defined as complete response. Reduction in the severity of any bleeding parameter less than complete response will represent partial response.
Measured: up to 24 weeks
Secondary outcomes (6)
Size and number of telangiectasias evaluated by endoscopy of nasal mucosa (recording images of the size and localization of telangiectasias)
Measured: up to 24 weeks
Minimum dose of the drug that reduces bleeding
Measured: up to 24 weeks
Time to response
Measured: up to 24 weeks
Time to relapse after the end of treatment
Measured: up to 24 weeks after the end of treatment
Number of adverse events
Measured: up to 1 year
Correlations between biological parameters, response to treatment and side effects profile
- Correlations between the mutations that are responsible for HHT and response to treatment
- Correlations between polymorphisms of CYP2C19 and response to treatment
- Correlations between polymorphisms of CYP2C19 and side effects profile
Measured: up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2011 (estimated)
- Study startNov 2011
- Primary completionOct 2014
- Study completion11 Oct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Hereditary Hemorrhagic TelangiectasiaStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Policlinico San Matteo di PaviaThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Carlo Balduini, Fondazione IRCCS Policlinico San Matteo di Pavia
- Investigators
- Carlo Balduini, M.D., Principal investigator, Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01485224
- Sponsor's protocol ID
- EudratCT 2011-004096-36
- Acronym
- THALI-HHT
- Status last verified
- Aug 2017
- First submitted
- 28 Nov 2011
- Last updated
- 29 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Lebrin F, Srun S, Raymond K, Martin S, van den Brink S, Freitas C, Breant C, Mathivet T, Larrivee B, Thomas JL, Arthur HM, Westermann CJ, Disch F, Mager JJ, Snijder RJ, Eichmann A, Mummery CL. Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia. Nat Med. 2010 Apr;16(4):420-8. doi: 10.1038/nm.2131. Epub 2010 Apr 4.
PubMed 20364125Invernizzi R, Quaglia F, Klersy C, Pagella F, Ornati F, Chu F, Matti E, Spinozzi G, Plumitallo S, Grignani P, Olivieri C, Bastia R, Bellistri F, Danesino C, Benazzo M, Balduini CL. Efficacy and safety of thalidomide for the treatment of severe recurrent epistaxis in hereditary haemorrhagic telangiectasia: results of a non-randomised, single-centre, phase 2 study. Lancet Haematol. 2015 Nov;2(11):e465-73. doi: 10.1016/S2352-3026(15)00195-7. Epub 2015 Oct 27.
PubMed 26686256
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Thalidomide in the Treatment of Severe Recurrent Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
- ClinicalTrials.gov ID
- NCT01485224
- Acronym / sponsor ID
- THALI-HHT / EudratCT 2011-004096-36
- Conditions
- Hereditary Hemorrhagic Telangiectasia, Epistaxis
- Keywords
- Hereditary hemorrhagic telangiectasia, Epistaxis, Thalidomide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Fondazione IRCCS Policlinico San Matteo di PaviaUniversity, hospital or other
- Study start
- Nov 2011
- Primary completion
- Oct 2014 (actual)
- Study completion
- 11 Oct 2016 (actual)
- First posted
- 5 Dec 2011 (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.
Hereditary hemorrhagic telangiectasia (HHT) (OMIM 187300 and 600376), also known as Rendu-Osler-Weber syndrome, is an autosomal dominant disease and has a prevalence between 1:5000 and 1:8000 in different populations. Clinically, the occurrence of mucocutaneous and gastrointestinal telangiectasias and of systemic arteriovenous malformations is commonly observed. Recurrent and severe epistaxis, due to the presence of telangiectasias in nasal mucosa, is the most common presentation of HHT, frequently leading to severe anemia requiring intravenous iron and blood transfusions. Although not life threatening, severe epistaxis has a great impact on quality of life in HHT patients and it represents the most important impediment in daily activities, that poses therapeutic challenge. Recently, angiogenesis has been implicated in the pathogenesis of HHT. Circulating concentrations of both TGF-beta and vascular endothelial growth factor (VEGF) are significantly elevated and therefore, anti-angiogenic substances may be effective in the treatment of vascular malformations in this disease. Thalidomide functions as a potent immunosuppressive and antiangiogenic agent. The aim of this study is to assess the clinical effects of thalidomide therapy on the severity of epistaxis in subjects with HHT who are refractory to standard therapies.
Detailed description
In the management of HHT epistaxis, multiple approaches have been tried, including electrocautery, laser, embolization, arterial ligation, but all approaches are largely palliative with variable results, many requiring repeated interventions. Except for nasal closure, surgical options offer, at best, limited hemorrhage-free intervals, but no definitive results and all have side effects. Moreover, currently, there is no established medical treatment available for these patients. To limit blood loss, the few medical treatments used include manipulation of the coagulation and fibrinolytic pathways or topical applications of anti-inflammatory drugs. However, multiple lesions disseminated over the entire mucosal surface are common in affected individuals, making local treatment difficult. Re-bleeding consumes a disproportionate share of healthcare resources devoted to multiple admissions, repeated endoscopies and blood transfusions.
Recently, angiogenesis has been implicated in the pathogenesis of HHT. Circulating concentrations of both TGF-beta and vascular endothelial growth factor (VEGF) are significantly elevated and therefore, anti-angiogenic substances may be effective in the treatment of vascular malformations in this disease.
Thalidomide functions as a potent immunosuppressive and antiangiogenic agent by inhibiting the phagocytic ability of inflammatory cells and the production of cytokines, such as tumor necrosis factor-alpha (TNF-a). It has been shown to be effective in the treatment of inflammatory diseases, in conditions associated with human immunodeficiency virus (HIV) infection, and in various cancers. Bleeding inhibition has been observed in HHT patients who received thalidomide as an antiangiogenic cancer therapy. A recent paper has reported that thalidomide treatment induced vessel maturation in an experimental model of HHT and reduced severe nosebleeds in six of the seven HHT patients studied. On the other hand, spectacular improvements have been described in patients with intestinal angiodysplasias, treated with thalidomide. In isolated case reports, patients with severe recurrent intestinal bleeding refractory to standard treatment achieved prolonged complete remission with thalidomide at a dose of 100 to 300 mg/day for a few months and tolerance was good. Cessation of bleeding was associated with a reduction in serum VEGF levels. These observations suggest that thalidomide might be useful for treatment of HHT patients and address significant unmet medical needs. Unfortunately, this drug exposes patients to the risk of severe side effects.
Drug metabolism is under control of a number of enzymes specific for each drug; among these enzymes, many show variable levels of activity and we can thus recognize in the population extensive (high or fast) metabolizers (EM) intermediate (IM) and poor (low or slow) metabolizers (PM). This is true also for thalidomide, whose metabolism is in part controlled by the enzyme CYP2C19.
The aims of our study are to test the hypothesis that thalidomide reduces the bleeding tendency of HHT patients and to verify to which extent CYP2C19 polymorphism modulates both response to treatment and side effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thalidomide | Experimental | Drug: ThalidomideSingle arm study: Eligible patients will receive thalidomide at a starting dose of 50 mg/day by mouth at bedtime for 4 weeks. In the event of unsatisfactory/no response, thalidomide dosage will be progressively increased by 50 mg/day every 4 weeks until complete or partial response, to a maximum dose of 200 mg/day. Treatment will be continued until one of the following criteria is met: * 8 additional weeks of treatment after the achievement of complete response * 16 additional weeks of treatment after the achievement of partial response * 24 weeks of treatment completed without response * unacceptable toxicity. Then, patients will be followed off of thalidomide for 24 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of HHT, according to the diagnostic criteria world-wide recognized (Curacao criteria), with severe recurrent epistaxis (grade 2-3 according to the criteria proposed by Pagella et al., i.e. at least one episode of overt bleeding/week requiring at least one blood transfusion during the last three months), and refractory to mini-invasive surgical procedures, i.e. argon plasma coagulation. For these patients, there is no effective treatment option currently available
- Age > 18 years
- Ability of signing written informed consent
- Women of childbearing potential:
- declared intention not to start a pregnancy throughout the study and for four weeks following the date of the last dose of thalidomide (safe contraception, see Celgene guidelines, "Programma di Prevenzione della Gravidanza")
Key exclusion criteria (6 listed)
- Pregnant or lactating women, or potentially fertile (both males and females) who have not agreed to avoid pregnancy during the trial period and for four weeks (females) or one week (males) following the date of the last dose of thalidomide
- Neurological diseases
- Psychiatric illness that would prevent granting of informed consent
- Active cardiovascular disease
- High risk for thromboembolic events (comorbidities, such as diabetes or uncontrolled infections, malignancy, immobility, prior history of thromboembolic events, use of erythropoietic agents or other agents such as hormone replacement therapy, central venous catheter, anti-cardiolipin, or anti-beta2 glycoprotein antibodies)
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 |
|---|---|---|
| ThalidomideAlso: Thalidomide CelgeneEligible patients will receive thalidomide at a starting dose of 50 mg/day by mouth at bedtime for 4 weeks. In the event of unsatisfactory/no response, thalidomide dosage will be progressively increased by 50 mg/day every 4 weeks until complete or partial response, to a maximum dose of 200 mg/day. Treatment will be continued until one of the following criteria is met: * 8 additional weeks of treatment after the achievement of complete response * 16 additional weeks of treatment after the achievement of partial response * 24 weeks of treatment completed without response * unacceptable toxicity. Then, patients will be followed off of thalidomide for 24 weeks. | Drug | Thalidomide |
Primary outcome
Percentage of patients showing a decrease in the frequency, intensity and duration of epistaxis and in the blood transfusion requirement.
Time frame: up to 24 weeks
Cessation of nose bleeding will be defined as complete response. Reduction in the severity of any bleeding parameter less than complete response will represent partial response.
6 secondary outcomes
Size and number of telangiectasias evaluated by endoscopy of nasal mucosa (recording images of the size and localization of telangiectasias)
Time frame: up to 24 weeks
Minimum dose of the drug that reduces bleeding
Time frame: up to 24 weeks
Time to response
Time frame: up to 24 weeks
Time to relapse after the end of treatment
Time frame: up to 24 weeks after the end of treatment
Number of adverse events
Time frame: up to 1 year
Correlations between biological parameters, response to treatment and side effects profile
Time frame: up to 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2017.
- Background: PubMed 20364125Lebrin F, Srun S, Raymond K, Martin S, van den Brink S, Freitas C, Breant C, Mathivet T, Larrivee B, Thomas JL, Arthur HM, Westermann CJ, Disch F, Mager JJ, Snijder RJ, Eichmann A, Mummery CL. Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia. Nat Med. 2010 Apr;16(4):420-8. doi: 10.1038/nm.2131. Epub 2010 Apr 4.
- Derived from this study: PubMed 26686256Invernizzi R, Quaglia F, Klersy C, Pagella F, Ornati F, Chu F, Matti E, Spinozzi G, Plumitallo S, Grignani P, Olivieri C, Bastia R, Bellistri F, Danesino C, Benazzo M, Balduini CL. Efficacy and safety of thalidomide for the treatment of severe recurrent epistaxis in hereditary haemorrhagic telangiectasia: results of a non-randomised, single-centre, phase 2 study. Lancet Haematol. 2015 Nov;2(11):e465-73. doi: 10.1016/S2352-3026(15)00195-7. Epub 2015 Oct 27.
- Individual participant dataSharing planned: No
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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.
Carlo Balduini, M.D. principal investigator
Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy
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 Thalidomide in people with Hereditary Hemorrhagic Telangiectasia and Epistaxis, run by Fondazione IRCCS Policlinico San Matteo di Pavia at 1 site in Italy.
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 10 inclusion and 6 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: finished, as of the sponsor's last update on 29 Aug 2017. 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 Italy. 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. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of patients showing a decrease in the frequency, intensity and duration of epistaxis and in the blood transfusion requirement. (time frame: up to 24 weeks). 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 NCT01485224, which also lists 2 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 29 Aug 2017.