Iron OverloadPhase 1NCT02274233
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
A Phase 1 study testing SP-420 in people with Iron Overload and Beta-Thalassemia, run by Sideris Pharmaceuticals at 7 sites in 5 countries.
Trial summary
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload, Beta-Thalassemia
- What is being tested?
- SP-420
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 5 countries
- Study dates
- Oct 2014to Sep 2015
- Phase
- Phase 1
- 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
- Subject has iron-overload secondary to β-thalassemia requiring chronic red blood cell transfusions and iron chelation therapy
- Subject weighs ≥35 kg
- Subject is willing to discontinue current iron chelation therapy at least 7 days prior to the first dose of SP-420 and for the duration of the study
- Serum ferritin ≥700 ng/mL and iron saturation ≥70% within 3 weeks before Baseline (Day 1)
- Cardiac T2* score >20 msec within 6 months before Baseline (Day 1)
- Willing to use contraception during the study
Who cannot join
- Pregnant or breast-feeding
- Serum creatinine greater than the upper limit of normal
- Platelet count <100 × 10^9/L
- Use of another investigational drug within the last 30 days
- Significant cardiac, renal, hepatic dysfunction or other clinically significant conditions that, in the opinion of the Investigator, would exclude the subject
Trial locations and contacts
7 sites in 5 countries.
7 sites in 5 countries
United States3 sites
Sideris Investigative Site
Boston, Massachusetts, 02115
Sideris Investigative Site
New York, New York, 10065
Sideris Investigative Site
Philadelphia, Pennsylvania, 19104
About this study
Official title: Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Conditions
- Iron Overload, Beta-Thalassemia
- Being tested
- SP-420
- Keywords
- Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation
- Medical subject headings
- Iron Overload, beta-Thalassemia, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SP-420Drug
| Study group | Role | What they receive |
|---|---|---|
| 1.5 mg/kg | Experimental | 1.5 mg/kg SP-420 once daily for 14 days Receives: Drug: SP-420 |
| 3 mg/kg | Experimental | 3 mg/kg SP-420 once daily for 14 days Receives: Drug: SP-420 |
| 6 mg/kg | Experimental | 6 mg/kg SP-420 once daily for 14 days Receives: Drug: SP-420 |
| 12 mg/kg | Experimental | 12 mg/kg SP-420 once daily for 14 days Receives: Drug: SP-420 |
| 24 mg/kg | Experimental | 24 mg/kg SP-420 once daily for 28 days Receives: Drug: SP-420 |
| 9 mg/kg | Experimental | 9 mg/kg SP-420 twice daily for 28 days Receives: Drug: SP-420 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events
Measured: Up to 35 days
Secondary outcomes (2)
Peak Plasma Concentration (Cmax) of SP-420
Measured: Day 7
Area under the plasma concentration versus time curve (AUC) of SP-420
Measured: Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2014 (estimated)
- Study startOct 2014
- Primary completionSep 2015
- Study completionSep 2015
Why it stopped: Changes in renal function parameters.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sideris Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc., Study director, Sideris Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02274233
- Sponsor's protocol ID
- SP-420-702
- Status last verified
- Sep 2015
- First submitted
- 21 Oct 2014
- Last updated
- 29 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Open-label, Dose-escalation Study of SP-420 in Subjects With Transfusion-dependent β-Thalassemia
- ClinicalTrials.gov ID
- NCT02274233
- Acronym / sponsor ID
- SP-420-702
- Conditions
- Iron Overload, Beta-Thalassemia
- Keywords
- Chelation, Iron Overload, Thalassemia, Transfusion, Iron Chelation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Changes in renal function parameters.
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Sideris PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2014
- Primary completion
- Sep 2015 (actual)
- Study completion
- Sep 2015 (actual)
- First posted
- 24 Oct 2014 (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 study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| 1.5 mg/kg | Experimental | Drug: SP-4201.5 mg/kg SP-420 once daily for 14 days |
| 3 mg/kg | Experimental | Drug: SP-4203 mg/kg SP-420 once daily for 14 days |
| 6 mg/kg | Experimental | Drug: SP-4206 mg/kg SP-420 once daily for 14 days |
| 12 mg/kg | Experimental | Drug: SP-42012 mg/kg SP-420 once daily for 14 days |
| 24 mg/kg | Experimental | Drug: SP-42024 mg/kg SP-420 once daily for 28 days |
| 9 mg/kg | Experimental | Drug: SP-4209 mg/kg SP-420 twice daily for 28 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Subject has iron-overload secondary to β-thalassemia requiring chronic red blood cell transfusions and iron chelation therapy
- Subject weighs ≥35 kg
- Subject is willing to discontinue current iron chelation therapy at least 7 days prior to the first dose of SP-420 and for the duration of the study
- Serum ferritin ≥700 ng/mL and iron saturation ≥70% within 3 weeks before Baseline (Day 1)
- Cardiac T2* score >20 msec within 6 months before Baseline (Day 1)
Key exclusion criteria (5 listed)
- Pregnant or breast-feeding
- Serum creatinine greater than the upper limit of normal
- Platelet count <100 × 10^9/L
- Use of another investigational drug within the last 30 days
- Significant cardiac, renal, hepatic dysfunction or other clinically significant conditions that, in the opinion of the Investigator, would exclude the subject
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 |
|---|---|---|
| SP-420 | Drug | 1.5 mg/kg, 12 mg/kg, 24 mg/kg, 3 mg/kg, 6 mg/kg, 9 mg/kg |
Primary outcome
Number of Participants with Adverse Events
Time frame: Up to 35 days
2 secondary outcomes
Peak Plasma Concentration (Cmax) of SP-420
Time frame: Day 7
Area under the plasma concentration versus time curve (AUC) of SP-420
Time frame: Day 7
Results and safety
The study was stopped early (reason given: Changes in renal function parameters.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2015.
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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.
Sideris Pharmaceuticals, Inc. Sideris Pharmaceuticals, Inc. study director
Sideris Pharmaceuticals, Inc.
7 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SP-420 in people with Iron Overload and Beta-Thalassemia, run by Sideris Pharmaceuticals at 7 sites in 5 countries.
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 6 inclusion and 5 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: stopped early, as of the sponsor's last update on 29 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Canada, Lebanon, Thailand, Turkey (Türkiye). 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 stopped early (reason given: Changes in renal function parameters.). Results are not available in the source record.
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?
Number of Participants with Adverse Events (time frame: Up to 35 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Sep 2015.