Beta Thalassemia Major AnemiaPhase 4STARTNCT03591575
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
A Phase 4 study testing Deferiprone oral solution in people with Beta Thalassemia Major Anemia and Iron Overload, run by Chiesi Canada Corp at 4 sites in 2 countries.
Trial summary
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Read more about this study
- Who is it for?
- Ages 6 months to 9 years
- Conditions the study is for
- Beta Thalassemia Major Anemia, Iron Overload
- What is being tested?
- Deferiprone oral solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 9 Nov 2018to 29 Sep 2020
- Phase
- Phase 4
- 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 6 months to 9 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female aged ≥ 6 months to < 10 years
- Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing
- Started on a red blood cell (RBC) transfusion regimen, with a minimum of 2 transfusions already completed
- Screening level of serum ferritin (SF) greater than >200 μg/L but not more than 600 μg/L. Since SF level may be impacted by the presence of infection, it must additionally be verified that the child has had no signs of infection in the previous 7 days, including the day of screening, and that the level of C-reactive protein (CRP) is no greater than 20% higher than the normal range for the patient's age. If there are signs of infection and/or the CRP level is above this threshold, the SF level must be checked again a minimum of one week later.
Who cannot join
- Prior use of iron chelation
- Diagnosis of hepatitis B or C, or HIV infection
- Evidence of abnormal liver or kidney function at screening: serum alanine transaminase (ALT) level > 5 times upper limit of normal or creatinine levels >2 times upper limit of normal
- Disorders associated with neutropenia (absolute neutrophil count < 1.5 x 10^9/L) prior to the initiation of study medication
- A serious, unstable illness, as judged by the investigator, during the previous 3 months before screening/baseline visit including but not limited to hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease.
- Presence of any medical condition which in the opinion of the investigator would cause participation in the study to be unwise.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Egypt3 sites
Ain Shams University Children's Hospital
Cairo
Pediatric Hospital of Cairo University
Cairo
The Clinical Research Center, Faculty of Medicine, Cairo University
Cairo
About this study
Official title: Safety and Efficacy of Early-start Deferiprone Treatment in Infants and Young Children Newly Diagnosed With Transfusion-dependent Beta Thalassemia
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Half the patients in the study will receive deferiprone, and the other half will receive placebo, for up to 12 months.
Read the detailed description
This study will give deferiprone to infants and young children with thalassemia who have started receiving regular blood transfusions but whose iron load is not yet at the level where chelation treatment would normally begin. The purpose is to see if doing this will postpone the build-up of iron without causing serious side effects. Half the children in the study will be given deferiprone at a dose that is lower than what is normally prescribed, and the other half will be given placebo. All patients will receive the assigned product three times a day for up to 12 months. Tests for signs of iron overload will be done monthly, and a patient whose iron load reaches the level where chelation therapy would normally begin will be immediately taken out of the study and started on standard chelation therapy.
- Conditions
- Beta Thalassemia Major Anemia, Iron Overload
- Being tested
- Deferiprone oral solution, Placebo
- Keywords
- thalassemia, iron overload, chelation, deferiprone, Ferriprox
- Medical subject headings
- Iron Overload, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferiprone oral solutionDrug
Also called Ferriprox
Liquid formulation of deferiprone, with a concentration of 80 mg/mL
PlaceboDrug
Also called Placebo for deferiprone oral solution
Liquid solution that matches deferiprone oral solution in appearance and taste
| Study group | Role | What they receive |
|---|---|---|
| Deferiprone | Experimental | Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses Receives: Drug: Deferiprone oral solution |
| Placebo | Placebo comparator | Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses Receives: Drug: Placebo |
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 Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
Measured: 12 months
Secondary outcomes (1)
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points
The outcome measure was the percentage of patients in each group still below the serum ferritin threshold at Months 4, 8, and 12. Without adequate chelation therapy, most patients receiving red blood cell transfusions are likely to exceed this level within a few months. However, a safety feature of the study design was that patients who reached the serum ferritin threshold were withdrawn (i.e., so that they could begin standard chelation therapy).
Measured: 4, 8, and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2018
- Study start9 Nov 2018
- Primary completion29 Sep 2020
- Study completion29 Sep 2020
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 Beta Thalassemia Major AnemiaStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Canada Corp
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mohsen El Alfy, MD, Principal investigator, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03591575
- Sponsor's protocol ID
- LA55-0417
- Acronym
- START
- Status last verified
- Mar 2024
- First submitted
- 9 Jul 2018
- Last updated
- 15 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Early-start Deferiprone Treatment in Infants and Young Children Newly Diagnosed With Transfusion-dependent Beta Thalassemia
- ClinicalTrials.gov ID
- NCT03591575
- Acronym / sponsor ID
- START / LA55-0417
- Conditions
- Beta Thalassemia Major Anemia, Iron Overload
- Keywords
- thalassemia, iron overload, chelation, deferiprone, Ferriprox
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Chiesi Canada CorpIndustry (pharma, biotech, devices)
- Study start
- 9 Nov 2018 (actual)
- Primary completion
- 29 Sep 2020 (actual)
- Study completion
- 29 Sep 2020 (actual)
- First posted
- 19 Jul 2018 (actual)
- Results
- Posted 15 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Half the patients in the study will receive deferiprone, and the other half will receive placebo, for up to 12 months.
Detailed description
This study will give deferiprone to infants and young children with thalassemia who have started receiving regular blood transfusions but whose iron load is not yet at the level where chelation treatment would normally begin. The purpose is to see if doing this will postpone the build-up of iron without causing serious side effects. Half the children in the study will be given deferiprone at a dose that is lower than what is normally prescribed, and the other half will be given placebo. All patients will receive the assigned product three times a day for up to 12 months. Tests for signs of iron overload will be done monthly, and a patient whose iron load reaches the level where chelation therapy would normally begin will be immediately taken out of the study and started on standard chelation therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Deferiprone | Experimental | Drug: Deferiprone oral solutionSubjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses |
| Placebo | Placebo comparator | Drug: PlaceboSubjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses |
Who may qualify
- Ages
- 6 months to 9 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Male or female aged ≥ 6 months to < 10 years
- Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing
- Started on a red blood cell (RBC) transfusion regimen, with a minimum of 2 transfusions already completed
- Screening level of serum ferritin (SF) greater than >200 μg/L but not more than 600 μg/L. Since SF level may be impacted by the presence of infection, it must additionally be verified that the child has had no signs of infection in the previous 7 days, including the day of screening, and that the level of C-reactive protein (CRP) is no greater than 20% higher than the normal range for the patient's age. If there are signs of infection and/or the CRP level is above this threshold, the SF level must be checked again a minimum of one week later.
Key exclusion criteria (6 listed)
- Prior use of iron chelation
- Diagnosis of hepatitis B or C, or HIV infection
- Evidence of abnormal liver or kidney function at screening: serum alanine transaminase (ALT) level > 5 times upper limit of normal or creatinine levels >2 times upper limit of normal
- Disorders associated with neutropenia (absolute neutrophil count < 1.5 x 10^9/L) prior to the initiation of study medication
- A serious, unstable illness, as judged by the investigator, during the previous 3 months before screening/baseline visit including but not limited to hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease.
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 oral solutionAlso: FerriproxLiquid formulation of deferiprone, with a concentration of 80 mg/mL | Drug | Deferiprone |
| PlaceboAlso: Placebo for deferiprone oral solutionLiquid solution that matches deferiprone oral solution in appearance and taste | Drug | Placebo |
Primary outcome
The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12
Time frame: 12 months
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
1 secondary outcome
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points
Time frame: 4, 8, and 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
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Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
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ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
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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.
Mohsen El Alfy, MD principal investigator
Ain Shams University
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Deferiprone oral solution in people with Beta Thalassemia Major Anemia and Iron Overload, run by Chiesi Canada Corp at 4 sites in 2 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 4 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 15 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Egypt, Indonesia. 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. Some participants in this study may receive a placebo. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 15 Mar 2024). 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 Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12 (time frame: 12 months). 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 15 Mar 2024.