Friedreich AtaxiaPhase 2NCT02035020
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients
A Phase 2 study testing gamma interferon in people with Friedreich Ataxia, run by Azienda Policlinico Umberto I at 1 site in Italy.
Trial summary
The primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- gamma interferon
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteItaly
- Study dates
- May 2013to 30 Jul 2014
- 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 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- FRDA patients should have their diagnosis genetically confirmed.
- Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
- Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Male and/or female subjects between the ages of > 18 and < 45 years
- -
Who cannot join
- Pregnant or breastfeeding women.
- Significant concurrent medical conditions at the time of screening or baseline visit, including, but not limited to, the following:
- Any major illness/condition or evidence of an unstable clinical condition (eg, renal, hepatic, hematologic, GI, endocrine, pulmonary, immunologic, or local active infection/infectious illness) that, in the investigator's judgment, will substantially increase the risk to the subject if he or she participates in the study.
- Class III or IV congestive heart failure as defined by the New York Heart Association.
- Acute coronary syndrome (eg, myocardial infarction, unstable angina pectoris) and any history of significant cerebrovascular disease within 24 weeks before screening.
- Presence of a transplanted organ.
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- Previous assumption of IFN gamma 1b.
- Abnormality in any of the below hematology or chemistry profile values at screening:
- Positive hepatitis B surface antigen (HBsAg), Total hepatitis B core antibody (HBcAb; also called anti HBc), and/or hepatitis C antibody (HCVAb) with confirmation by hepatitis C virus ribonucleic acid (HCV RNA).
- ALT/AST levels > or = 1.5X ULN.
- Total bilirubin level > or = 1.5 times the ULN.
- Hemoglobin level < or = 80 gL (8.0 g/dL).
- Platelet count < or = 100 x 109/L (100,000 cells/mm³) or > or = 1000 x 109/L (1,000,000 cells/mm³).
- White blood cell count < or = 3.5 x 109/L (3500 cells/mm³).
- Absolute neutrophil count (ANC) <2000 cells/mm³.
- Serum creatinine level > or = 177 μmol/ L (2 mg/dL).
- Glycosylated hemoglobin (HbA1c >10%).
- Current or history of serious psychiatric disorder or alcohol or drug abuse.
- Participation in other studies within 30 days before screening and/or during study participation.
- Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or ability to comply with study procedures, investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) Patients
The primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
Read the detailed description
This is a Phase 2 clinical trial. A total of 10 FRDA patients will be recruited All subjects will be treated with a dose of 100-150-200-micrograms of IFN gamma 1b (Imukin®) subcutaneously, with an interval of 14 days, for a total of 3 injections.
- Conditions
- Friedreich Ataxia
- Being tested
- gamma interferon
- Keywords
- frataxin, gamma interferon, Friedreich ataxia
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gamma interferonDrug
Also called Imukin
IFN gamma 1b (Immukin ®) will be administered by subcutaneous route at day 0, 14 and 28 at a dose of 100, 150 and 200 ug respectively.
| Study group | Role | What they receive |
|---|---|---|
| Gamma interferon | Experimental | IFN gamma 1b (Immukin ®) will be administered by subcutaneous route at day 0, 14 and 28 at a dose of 100, 150 and 200 ug respectively. Receives: Drug: gamma interferon |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in cellular frataxin
The primary endpoint is to test the increase of cellular frataxin after treatment with IFN gamma. Quantitation of cellular frataxin will be performed after 24 hours and 7 days from each study drug administration
Measured: 24 hours and 7 days from each study drug administration
Secondary outcomes (1)
Safety Blood sample
Secondary endpoint is the safety and tolerability of IFN gamma in FRDA patients. The on treatment adverse events and withdrawals due to adverse effects will be reported. Any subject who receives at least 1 dose of investigational product will be included in the evaluation for safety
Measured: day 0-14-28-35
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2014 (estimated)
- Study startMay 2013
- Primary completion30 Jul 2014
- Study completion30 Jul 2014
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 Friedreich AtaxiaStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Azienda Policlinico Umberto I
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Carlo Casali, Azienda Policlinico Umberto I
- Investigators
- Carlo Casali, MD, Principal investigator, Policlinico Umberto I°
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02035020
- Sponsor's protocol ID
- GIFT/1
- Status last verified
- Apr 2017
- First submitted
- 12 Jan 2014
- Last updated
- 21 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Tomassini B, Arcuri G, Fortuni S, Sandi C, Ezzatizadeh V, Casali C, Condo I, Malisan F, Al-Mahdawi S, Pook M, Testi R. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum Mol Genet. 2012 Jul 1;21(13):2855-61. doi: 10.1093/hmg/dds110. Epub 2012 Mar 23.
PubMed 22447512
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) Patients
- ClinicalTrials.gov ID
- NCT02035020
- Acronym / sponsor ID
- GIFT/1
- Conditions
- Friedreich Ataxia
- Keywords
- frataxin, gamma interferon, Friedreich ataxia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Azienda Policlinico Umberto IUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- 30 Jul 2014 (actual)
- Study completion
- 30 Jul 2014 (actual)
- First posted
- 14 Jan 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 primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
Detailed description
This is a Phase 2 clinical trial. A total of 10 FRDA patients will be recruited All subjects will be treated with a dose of 100-150-200-micrograms of IFN gamma 1b (Imukin®) subcutaneously, with an interval of 14 days, for a total of 3 injections.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gamma interferon | Experimental | Drug: gamma interferonIFN gamma 1b (Immukin ®) will be administered by subcutaneous route at day 0, 14 and 28 at a dose of 100, 150 and 200 ug respectively. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- FRDA patients should have their diagnosis genetically confirmed.
- Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
- Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
- Male and/or female subjects between the ages of > 18 and < 45 years
- -
Key exclusion criteria (21 listed)
- Pregnant or breastfeeding women.
- Significant concurrent medical conditions at the time of screening or baseline visit, including, but not limited to, the following:
- Presence of a transplanted organ.
- Previous assumption of IFN gamma 1b.
- Abnormality in any of the below hematology or chemistry profile values 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 |
|---|---|---|
| gamma interferonAlso: ImukinIFN gamma 1b (Immukin ®) will be administered by subcutaneous route at day 0, 14 and 28 at a dose of 100, 150 and 200 ug respectively. | Drug | Gamma interferon |
Primary outcome
Change in cellular frataxin
Time frame: 24 hours and 7 days from each study drug administration
The primary endpoint is to test the increase of cellular frataxin after treatment with IFN gamma. Quantitation of cellular frataxin will be performed after 24 hours and 7 days from each study drug administration
1 secondary outcome
Safety Blood sample
Time frame: day 0-14-28-35
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2017.
- Background: PubMed 22447512Tomassini B, Arcuri G, Fortuni S, Sandi C, Ezzatizadeh V, Casali C, Condo I, Malisan F, Al-Mahdawi S, Pook M, Testi R. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum Mol Genet. 2012 Jul 1;21(13):2855-61. doi: 10.1093/hmg/dds110. Epub 2012 Mar 23.
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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.
Carlo Casali, MD principal investigator
Policlinico Umberto I°
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 gamma interferon in people with Friedreich Ataxia, run by Azienda Policlinico Umberto I at 1 site in Italy.
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 21 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 21 Apr 2017. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 cellular frataxin (time frame: 24 hours and 7 days from each study drug administration). 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 NCT02035020, 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 21 Apr 2017.