Porphyria Cutanea TardaPhase 2NCT01284946
Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea Tarda
A Phase 2 study testing Exjade in people with Porphyria Cutanea Tarda, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
While clinical phlebotomy is current standard practice for alleviating non-transfusion iron overload in patients with PCT, it may not be suitable for all patients. For example, some patients are unwilling to be adequately phlebotomized because of inconvenience, as phlebotomy can be cumbersome, especially during the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Porphyria Cutanea Tarda
- What is being tested?
- Exjade
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteFrance
- Study dates
- Jan 2011to Dec 2012
- 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 diagnosed with clinically overt Porphyria Cutanea Tarda, sporadic or familial as per the European Porphyria Network guidelines i.e. increased urinary and plasma porphyrins and faecal isocoproporphyrin detected by fluorescence emission spectroscopy,
- Skin fragility and bullae lesions,
- Age ≥ 18 years old,
- non-transfusion iron overload as depicted by a serum ferritin value ≥ 300 μg/L for men and ≥ 200 μg/L for women, and/or LIC ≥ 2 mg Fe/g dw for both men and women and with transferrin saturation ≥ 45%,
- Adequate liver function i.e. ALAT/ASAT and Alkaline Phosphatase ? 2.5 times ULN, bilirubin < 1.5 times ULN,
- Signed informed consent prior to beginning the specific procedures of the protocol,
- Ability to comply with all study-related procedures, medications, and evaluations,
- Sexually active women must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months). Since hormonal therapy may cause PCT, oral contraceptives will not be started during the course of the study and patients already on oral contraceptives will be advised to speak to their physician about discontinuing them and will not be enrolled in the study.
Who cannot join
- Clinical evidence of active Hepatitis B (positive HBsAg with negative HBsAb) and/or hepatitis C (positive HCV antibody and detectable HCV RNA with ALT above the normal range)
- Patients with on going alcoholic dependency > 60g/day
- Serum creatinine above the ULN
- Creatinine clearance < 60 ml/min, estimated according to Cockcroft-Gault formula or MDRD formula for adults
- Significant proteinuria as indicated by a urine protein: urine creatinine ratio > 0.5 mg/mg in a non-first void urine sample.
- Diabetes
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- Iron overload due to hereditary hemochromatosis
- History of blood transfusion during the 6 months prior to study entry,
- Males with hemoglobin <13 mg/dL, females with hemoglobin <12 mg/dL
- Active peptic ulcus
- Treatment with phlebotomy within 2 weeks of screening visit
- Prior Desferal® treatment within 1 month of the screening visit
- Patients currently or previously treated with deferiprone or deferasirox
- Patients with a diagnosis of a clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation
- Patient with clinically significant decrease of hearing
- Pregnant or lactating women or women of childbearing potential not using adequate contraception (pregnancy test mandatory and negative for patient with childbearing potential)
- Known hypersensitivity to the active ingredient of deferasirox or any excipients
- Contraindication to the administration of deferasirox as outlined in the approved prescribing information.
- Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of deferasirox
- Presence of a non-controlled severe disease affected vital organs as cardiac and/or pulmonary disease
- Patients with a known diagnosis of cirrhosis (confirmed by biopsy)
- Patients with active inflammatory diseases that may interfere with the accurate measurement of serum ferritin
- Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug within 7 days prior to the screening visit
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Hopital Louis Mourier, GI unit,
Colombes, Île-de-France Region, 92700
Benoit Coffin, Professor, 33147606061, benoit.coffin@lmr.aphp.fr
Deybach Jean-Charles, Professor
About this study
Official title: A Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron Overload
While clinical phlebotomy is current standard practice for alleviating non-transfusion iron overload in patients with PCT, it may not be suitable for all patients. For example, some patients are unwilling to be adequately phlebotomized because of inconvenience, as phlebotomy can be cumbersome, especially during the induction treatment phase requiring frequent clinic visits (twice a month, for at least 6 months) or because of venous access difficulties. Other patients are unable to undergo phlebotomy due to medical reasons such as anemia or cardiopulmonary disorders. It is postulated such patients with PCT who have non-transfusion iron overload could benefit from treatment with deferasirox (Exjade®), a once daily oral iron chelator licensed in several countries, including the EU, for treating transfusion iron overload in adult and pediatric patients. Although there is some data on the efficacy and safety of deferasirox in patients with HH, who, like those with PCT, have non-transfusional iron overload, there is a need to evaluate the safety and efficacy of deferasirox treatment of non-transfusion iron overload in patients with PCT.
Read the detailed description
The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.
The secondary objective is to assess the effectiveness of deferasirox treatment :
After 3 and 6 months to:
•Lower serum ferritin from abnormal to normal standard ranges specified for males and females in this patient population.
After 6 months to :
•Lower liver iron content after 24 weeks of treatment measured by liver MRI T2
After 3 and 6 months to :
- Improve clinical symptoms, i.e. improvement in skin lesions (reduction or no new bullae formation), and skin fragility (photographs will be used).
- Reduce porphyrin levels.
- Conditions
- Porphyria Cutanea Tarda
- Being tested
- Exjade
- Keywords
- PCT sporadic or familial, Skin fragility and bullae lesions, non-transfusion iron overload
- Medical subject headings
- Porphyria Cutanea Tarda
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExjadeDrug
Also called DEFERASIROX
Orodispersible Tablet, 10 mg/Kg/day ± 5 mg/Kg/day during 24 weeks Deferasirox should be taken daily 30 minutes before breakfast
| Study group | Role | What they receive |
|---|---|---|
| Exjade | Experimental | Safety and efficacy Receives: Drug: Exjade |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.
Measured: 6 months
Related drug adverse events
Incidence type and severity of drug related adverse events
Measured: 6 months
Secondary outcomes (2)
The change from baseline in serum ferritin after 12 and 24 weeks of treatment,The change from baseline in iron burden after 24 weeks of treatment measured by liver MRI T2,The evolution of clinical symptoms
Measured: 6 months
Chage from baseline in serum ferritin, iron burden, improvement in clincal symptoms, porphyrin levels
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2011 (estimated)
- Study startJan 2011
- Primary completionDec 2012 (estimated)
- Study completionDec 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Porphyria Cutanea TardaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Association pour l'Etude des Fonctions Digestives (AEFD)
- Investigators
- Deybach Jean-Charles, Professor, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01284946
- Sponsor's protocol ID
- AEFD2010-01
- Status last verified
- Jan 2011
- First submitted
- 26 Jan 2011
- Last updated
- 27 Jan 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Puy H, Gouya L, Deybach JC. Porphyrias. Lancet. 2010 Mar 13;375(9718):924-37. doi: 10.1016/S0140-6736(09)61925-5.
PubMed 20226990
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron Overload
- ClinicalTrials.gov ID
- NCT01284946
- Acronym / sponsor ID
- AEFD2010-01
- Conditions
- Porphyria Cutanea Tarda
- Keywords
- PCT sporadic or familial, Skin fragility and bullae lesions, non-transfusion iron overload
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Association pour l'Etude des Fonctions Digestives (AEFD)
- Study start
- Jan 2011
- Primary completion
- Dec 2012 (estimated)
- Study completion
- Dec 2012 (estimated)
- First posted
- 27 Jan 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.
While clinical phlebotomy is current standard practice for alleviating non-transfusion iron overload in patients with PCT, it may not be suitable for all patients. For example, some patients are unwilling to be adequately phlebotomized because of inconvenience, as phlebotomy can be cumbersome, especially during the induction treatment phase requiring frequent clinic visits (twice a month, for at least 6 months) or because of venous access difficulties. Other patients are unable to undergo phlebotomy due to medical reasons such as anemia or cardiopulmonary disorders. It is postulated such patients with PCT who have non-transfusion iron overload could benefit from treatment with deferasirox (Exjade®), a once daily oral iron chelator licensed in several countries, including the EU, for treating transfusion iron overload in adult and pediatric patients. Although there is some data on the efficacy and safety of deferasirox in patients with HH, who, like those with PCT, have non-transfusional iron overload, there is a need to evaluate the safety and efficacy of deferasirox treatment of non-transfusion iron overload in patients with PCT.
Detailed description
The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.
The secondary objective is to assess the effectiveness of deferasirox treatment :
After 3 and 6 months to:
•Lower serum ferritin from abnormal to normal standard ranges specified for males and females in this patient population.
After 6 months to :
•Lower liver iron content after 24 weeks of treatment measured by liver MRI T2
After 3 and 6 months to :
- Improve clinical symptoms, i.e. improvement in skin lesions (reduction or no new bullae formation), and skin fragility (photographs will be used).
- Reduce porphyrin levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Exjade | Experimental | Drug: ExjadeSafety and efficacy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male and female diagnosed with clinically overt Porphyria Cutanea Tarda, sporadic or familial as per the European Porphyria Network guidelines i.e. increased urinary and plasma porphyrins and faecal isocoproporphyrin detected by fluorescence emission spectroscopy,
Key exclusion criteria (23 listed)
- Clinical evidence of active Hepatitis B (positive HBsAg with negative HBsAb) and/or hepatitis C (positive HCV antibody and detectable HCV RNA with ALT above the normal range)
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 |
|---|---|---|
| ExjadeAlso: DEFERASIROXOrodispersible Tablet, 10 mg/Kg/day ± 5 mg/Kg/day during 24 weeks Deferasirox should be taken daily 30 minutes before breakfast | Drug | Exjade |
Primary outcome
The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT.
Time frame: 6 months
Related drug adverse events
Time frame: 6 months
Incidence type and severity of drug related adverse events
2 secondary outcomes
The change from baseline in serum ferritin after 12 and 24 weeks of treatment,The change from baseline in iron burden after 24 weeks of treatment measured by liver MRI T2,The evolution of clinical symptoms
Time frame: 6 months
Chage from baseline in serum ferritin, iron burden, improvement in clincal symptoms, porphyrin levels
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2011.
- Results publication: PubMed 20226990Puy H, Gouya L, Deybach JC. Porphyrias. Lancet. 2010 Mar 13;375(9718):924-37. doi: 10.1016/S0140-6736(09)61925-5.
Related trials for Porphyria Cutanea Tarda
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
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.
Deybach Jean-Charles, Professor principal investigator
Assistance Publique - Hôpitaux de Paris
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 Exjade in people with Porphyria Cutanea Tarda, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 8 inclusion and 23 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 27 Jan 2011. 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 France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Benoit Coffin, Professor (33147606061, benoit.coffin@lmr.aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary objective is to assess the safety of deferasirox in treating non-transfusion iron overload in patients with PCT. (time frame: 6 months). 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 NCT01284946, which also lists 1 linked publication.
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 27 Jan 2011.