Iron Overload Due to Repeated Red Blood Cell TransfusionsPhase 2TWICENCT03802916
Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
A Phase 2 study testing Deferiprone DR tablets 1000 mg (Low dosage) and Deferiprone DR tablets 1000 mg (High dosage) in people with Iron Overload Due to Repeated Red Blood Cell Transfusions, run by ApoPharma at 5 sites in 4 countries.
Trial summary
Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload Due to Repeated Red Blood Cell Transfusions
- What is being tested?
- Deferiprone DR tablets 1000 mg (Low dosage) and Deferiprone DR tablets 1000 mg (High dosage)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 6 Mar 2019to 19 Dec 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 or female aged ≥ 18 years.
- Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.
- On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload.
- Absolute neutrophil count ≥1.5 x 10^9/L at screening.
- A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.
Who cannot join
- Receipt of any iron chelator other than Ferriprox (i.e., combination therapy) in the last 3 months, or planning to receive it at any time during the period of the study.
- ALT and/or AST value > 5 times the upper limit of normal (ULN) at screening
- Active case of hepatitis B or C at screening.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
United States2 sites
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
New York Presbyterian Hospital/Weill Cornell Medical Center
New York, New York, 10065
Greece1 site
National and Kapodistrian University of Athens, Aghia Sophia Children's Hospital
Goudi, Athens, 11527
Italy1 site
San Luigi Gonzaga University Hospital Reparto Microcitemie-Pediatria
Orbassano (TO), Regione Gonzole, 10043
About this study
Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload.
Read the detailed description
This study is looking at the safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day. Ferriprox doses range from 75 milligrams per kilogram of body weight (mg/kg) per day to 100 mg/kg per day. Half the patients in the study will be on a dosage that is closer to the low end of the range, and half will be on a dosage that is closer to the high end. Both groups will be switched for one month to deferiprone DR tablets at approximately the same total daily dosage that they have been taking for Ferriprox.
- Being tested
- Deferiprone DR tablets 1000 mg (Low dosage), Deferiprone DR tablets 1000 mg (High dosage)
- Keywords
- iron overload, chelation, deferiprone, Ferriprox
- Medical subject headings
- Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferiprone DR tablets 1000 mg (Low dosage)Drug
Deferiprone DR tablets 1000 mg
Deferiprone DR tablets 1000 mg (High dosage)Drug
Deferiprone DR tablets 1000 mg
| Study group | Role | What they receive |
|---|---|---|
| Low dosage | Experimental | Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart. Receives: Drug: Deferiprone DR tablets 1000 mg (Low dosage) |
| High dosage | Experimental | Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart. Receives: Drug: Deferiprone DR tablets 1000 mg (High dosage) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following:
- For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN)
- For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN
Measured: Day 28
Secondary outcomes (2)
The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.
Patients will be asked to report any events of GI distress during the study, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.
Measured: Day 28
The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.
At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.
Measured: Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2019
- Study start6 Mar 2019
- Primary completion4 Dec 2019
- Study completion19 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Iron Overload Due to Repeated Red Blood Cell TransfusionsStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Parallel Assignment
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03802916
- Sponsor's protocol ID
- LA61-0218
- Acronym
- TWICE
- Status last verified
- Jun 2021
- First submitted
- 7 Jan 2019
- Last updated
- 16 Jul 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload
- ClinicalTrials.gov ID
- NCT03802916
- Acronym / sponsor ID
- TWICE / LA61-0218
- Keywords
- iron overload, chelation, deferiprone, Ferriprox
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- 6 Mar 2019 (actual)
- Primary completion
- 4 Dec 2019 (actual)
- Study completion
- 19 Dec 2019 (actual)
- First posted
- 14 Jan 2019 (actual)
- Results
- Posted 18 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload.
Detailed description
This study is looking at the safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day. Ferriprox doses range from 75 milligrams per kilogram of body weight (mg/kg) per day to 100 mg/kg per day. Half the patients in the study will be on a dosage that is closer to the low end of the range, and half will be on a dosage that is closer to the high end. Both groups will be switched for one month to deferiprone DR tablets at approximately the same total daily dosage that they have been taking for Ferriprox.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Parallel Assignment
| Arm | Type | Receives |
|---|---|---|
| Low dosage | Experimental | Drug: Deferiprone DR tablets 1000 mg (Low dosage)Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart. |
| High dosage | Experimental | Drug: Deferiprone DR tablets 1000 mg (High dosage)Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart. |
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 or female aged ≥ 18 years.
- Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions.
- On a stable regimen (≥3 months) of Ferriprox tablets for the treatment of systemic iron overload.
- Absolute neutrophil count ≥1.5 x 10^9/L at screening.
- A record of at least 12 measured alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels.
Key exclusion criteria (3 listed)
- Receipt of any iron chelator other than Ferriprox (i.e., combination therapy) in the last 3 months, or planning to receive it at any time during the period of the study.
- ALT and/or AST value > 5 times the upper limit of normal (ULN) at screening
- Active case of hepatitis B or C at screening.
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 |
|---|---|---|
| Deferiprone DR tablets 1000 mg (Low dosage)Deferiprone DR tablets 1000 mg | Drug | Low dosage |
| Deferiprone DR tablets 1000 mg (High dosage)Deferiprone DR tablets 1000 mg | Drug | High dosage |
Primary outcome
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
Time frame: Day 28
Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: * For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN) * For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN
2 secondary outcomes
The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.
Time frame: Day 28
The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.
Time frame: Day 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferiprone DR tablets 1000 mg (Low dosage) and Deferiprone DR tablets 1000 mg (High dosage) in people with Iron Overload Due to Repeated Red Blood Cell Transfusions, run by ApoPharma at 5 sites in 4 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 5 inclusion and 3 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 16 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada, Greece, 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?
Yes. Results are posted in the registry (first posted 18 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern. (time frame: Day 28). 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 16 Jul 2021.