Transfusional Iron OverloadPhase 2NCT01927913
Treatment of Iron Overload Requiring Chelation Therapy
A Phase 2 study testing SPD602 and Deferasirox in people with Transfusional Iron Overload and Iron Overload, run by Shire.
Trial summary
The purpose of this open-label study is to assess liver iron concentration using MRI imaging in subjects with beta-thalassemia when administered with either SPD602 or deferasirox for the treatment of chronic transfusional iron overload. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transfusional Iron Overload, Iron Overload, Iron Chelation
- What is being tested?
- SPD602 and Deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 20 Nov 2014to 30 Oct 2015
- 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
- These criteria are to be met at the Screening Visit (Visit 1), the Washout Visit (Visit 2), and the Baseline Visit (Visit 3), if reassessed.
- An understanding, ability, and willingness to fully comply with study procedures and restrictions.
- Ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent as applicable to participate in the study before completing any study-related procedures.
- Subjects 18 years of age or older at the time of signing consent.
- Female subjects should be one of the following:
- Post-menopausal (12 consecutive months of spontaneous amenorrhea)
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- Surgically sterile, or
- Females of child-bearing potential must have a negative serum Beta-HCG pregnancy test at the Screening Visit (Visit 1) and a negative urine pregnancy test at the Baseline Visit (Visit 3). Females of child-bearing potential must agree to abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception.
- Subjects with beta-thalassemia who have received at least 100mL/kg of packed red blood cells (or >20 transfusion units) and who have iron overload (transfusional hemosiderosis) requiring chronic treatment with an iron chelator.
- Serum ferritin >500µg/L at the Screening Visit (Visit 1).
- Baseline LIC (last MRI assessment prior to Day 1) greater than or equal to 2.0mg and less than 30.0mg iron per g (equivalent dry weight, liver) determined by FerriScan® R2 MRI.
- 8. Mean of the previous 3 pre-transfusion hemoglobin concentrations greater than or equal to 7.5g/dL assessed at the Screening Visit (Visit 1) (1 value from clinical laboratory tests taken at the Screening Visit [Visit 1] and the previous 2 historical values available).
Who cannot join
- Severe iron overload including: (a) cardiac T2* MRI less than 10.0ms; or (b) LIC by FerriScan® R2 MRI greater than or equal to 30.0mg/g liver (dw) as assessed at the Screening Visit (Visit 1).
- Iron overload from causes other than transfusional siderosis.
- Subjects with thalassemia intermedia
- Unable to undergo MRI assessment.
- Cardiac LVEF less than 50% at baseline testing by MRI.
- Subjects with documented liver failure (presence of portal hypertension, hepatic edema, ascites, cirrhosis), Child-Pugh C hepatic impairment, or biliary disorder.
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- Platelet count <100 x 109/L at the Screening Visit (Visit 1).
- Absolute neutrophils counts of <1500mm3 at the Screening Visit (Visit 1).
- Evidence of renal insufficiency eg, creatinine clearance <60mL/min or serum creatinine >1.5x ULN at the Screening Visit (Visit 1).
- Clinically significant laboratory assessments at the Screening Visit (Visit 1).
- Significant proteinuria: urinary protein/creatinine ratio >1.0 in a non-first void urine sample at the Screening Visit (Visit 1).
- Current or recurrent disease that could affect the action, absorption, or disposition of the investigational product, or clinical or laboratory assessments.
- Current or relevant history of physical or psychiatric illness, any medical disorder that may require treatment or make the subject unlikely to fully complete the study, or any condition, including pregnancy, that presents undue risk from the investigational product or procedures.
- Current use of any medication contraindicated in the deferasirox prescribing information/SmPC.
- Known or suspected intolerance or hypersensitivity to SSP-004184AQ, deferasirox, closely-related compounds, or any of the stated ingredients in either medication.
- Known history of alcohol or other substance abuse within the last year.
- Within 30 days prior to the Baseline Visit (Visit 3):
- Have used an investigational product
- Have been enrolled in a clinical study (including vaccine studies) that, in the investigator's opinion, may impact this study.
- History of malignancy within the past 5 years, with the exception of basal cell or squamous cell skin carcinoma or cervical carcinoma in situ or completely resected colon carcinoma in situ.
- Insufficient venous access that precludes prescribed blood draws for safety laboratory assessments.
- Pregnant or lactating females.
About this study
Official title: A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in B-thalassemia Subjects Administered SPD602 (SSP-004184AQ) or Exjade® (Deferasirox) for Treatment of Chronic Transfusional Iron Overload
The purpose of this open-label study is to assess liver iron concentration using MRI imaging in subjects with beta-thalassemia when administered with either SPD602 or deferasirox for the treatment of chronic transfusional iron overload.
- Conditions
- Transfusional Iron Overload, Iron Overload, Iron Chelation, Beta-thalassemia, Transfusional Hemosiderosis, Iron Metabolism Disorders
- Being tested
- SPD602, Deferasirox
- Medical subject headings
- Iron Overload, beta-Thalassemia, Iron Metabolism Disorders, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPD602Drug
Also called SSP-004184AQ
32, 50 or 75 mg/kg/day BID, capsule
DeferasiroxDrug
Also called Exjade®
Per approved country specific label
| Study group | Role | What they receive |
|---|---|---|
| SPD602 | Experimental | Not described Receives: Drug: SPD602 |
| Deferasirox | Active comparator | Not described Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Liver Iron Concentration (LIC) from Baseline Utilizing R2 Magnetic Resonance Imaging (MRI)
Measured: 48 Weeks
Secondary outcomes (7)
Response Rate Utilizing R2* MRI
Measured: 48 weeks
Change in Cardiac Iron Concentration (CIC) from baseline Utilizing T2* MRI
Measured: 48 weeks
Change in Serum Ferritin Levels from Baseline
Measured: 48 weeks
Change in Pancreas Iron Concentration (PIC) from Baseline Utilizing R2* MRI
Measured: 48 weeks
Left Ventricular Ejection Fraction (LVEF) Utilizing MRI
Measured: 48 weeks
Gastrointestinal Symptom Rating Scale
Measured: 48 weeks
Total Neuropathy Score nurse (TNSn)
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2013 (estimated)
- Study start20 Nov 2014 (estimated)
- Primary completion30 Oct 2015 (estimated)
- Study completion30 Oct 2015 (estimated)
Why it stopped: This study was withdrawn until the evaluation of the nonclinical rat findings is complete.
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 Transfusional Iron OverloadStudies of the same condition, open and under way.
- 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
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01927913
- Sponsor's protocol ID
- SPD602-204
- Other IDs
- FBS0701 (SSP-004184), 2013-000743-33
- Status last verified
- May 2021
- First submitted
- 20 Aug 2013
- Last updated
- 2 Jun 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.
PubMed 37975597
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in B-thalassemia Subjects Administered SPD602 (SSP-004184AQ) or Exjade® (Deferasirox) for Treatment of Chronic Transfusional Iron Overload
- ClinicalTrials.gov ID
- NCT01927913
- Acronym / sponsor ID
- SPD602-204
- Conditions
- Transfusional Iron Overload, Iron Overload, Iron Chelation, Beta-thalassemia, Transfusional Hemosiderosis, Iron Metabolism Disorders
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: This study was withdrawn until the evaluation of the nonclinical rat findings is complete.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 20 Nov 2014 (estimated)
- Primary completion
- 30 Oct 2015 (estimated)
- Study completion
- 30 Oct 2015 (estimated)
- First posted
- 23 Aug 2013 (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.
The purpose of this open-label study is to assess liver iron concentration using MRI imaging in subjects with beta-thalassemia when administered with either SPD602 or deferasirox for the treatment of chronic transfusional iron overload.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SPD602 | Experimental | Drug: SPD602 |
| Deferasirox | Active comparator | Drug: Deferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- These criteria are to be met at the Screening Visit (Visit 1), the Washout Visit (Visit 2), and the Baseline Visit (Visit 3), if reassessed.
- An understanding, ability, and willingness to fully comply with study procedures and restrictions.
- Ability to voluntarily provide written, signed, and dated (personally or via a legally authorized representative) informed consent as applicable to participate in the study before completing any study-related procedures.
- Subjects 18 years of age or older at the time of signing consent.
- Female subjects should be one of the following:
Key exclusion criteria (22 listed)
- Severe iron overload including: (a) cardiac T2* MRI less than 10.0ms; or (b) LIC by FerriScan® R2 MRI greater than or equal to 30.0mg/g liver (dw) as assessed at the Screening Visit (Visit 1).
- Iron overload from causes other than transfusional siderosis.
- Subjects with thalassemia intermedia
- Unable to undergo MRI assessment.
- Cardiac LVEF less than 50% at baseline testing by MRI.
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 |
|---|---|---|
| SPD602Also: SSP-004184AQ32, 50 or 75 mg/kg/day BID, capsule | Drug | SPD602 |
| DeferasiroxAlso: Exjade®Per approved country specific label | Drug | Deferasirox |
Primary outcome
Change in Liver Iron Concentration (LIC) from Baseline Utilizing R2 Magnetic Resonance Imaging (MRI)
Time frame: 48 Weeks
7 secondary outcomes
Response Rate Utilizing R2* MRI
Time frame: 48 weeks
Change in Cardiac Iron Concentration (CIC) from baseline Utilizing T2* MRI
Time frame: 48 weeks
Change in Serum Ferritin Levels from Baseline
Time frame: 48 weeks
Change in Pancreas Iron Concentration (PIC) from Baseline Utilizing R2* MRI
Time frame: 48 weeks
Left Ventricular Ejection Fraction (LVEF) Utilizing MRI
Time frame: 48 weeks
Gastrointestinal Symptom Rating Scale
Time frame: 48 weeks
Total Neuropathy Score nurse (TNSn)
Time frame: 48 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2021.
- Derived from this study: PubMed 37975597Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.
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.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SPD602 and Deferasirox in people with Transfusional Iron Overload and Iron Overload, run by Shire.
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 12 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: withdrawn, as of the sponsor's last update on 2 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
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 was withdrawn before enrolling participants, so there are no results.
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?
Change in Liver Iron Concentration (LIC) from Baseline Utilizing R2 Magnetic Resonance Imaging (MRI) (time frame: 48 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 NCT01927913, 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 2 Jun 2021.