Friedreich's AtaxiaPhase 2NCT01016366
Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia
A Phase 2 study testing Lu AA24493 in people with Friedreich's Ataxia, run by H. Lundbeck A/S at 7 sites in 3 countries.
Trial summary
The primary purpose of the study is to determine whether carbamylated erythropoietin is a safe treatment for patients who suffer from Friedreich's Ataxia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- Lu AA24493
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 7 sitesin 3 countries
- Study dates
- Oct 2009to Apr 2011
- 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
- The patient has been diagnosed with FRDA and has had a genetic test demonstrating >400 GAA nucleotide triplet repeats on the shorter of the two frataxin alleles
- The patient has a SARA (Stance) sub-score of <=6
- The patient has a SARA (Gait) sub-score of <=6
- Man or woman, aged 18 years or over
- If female then woman should agree not to try to become pregnant during the study, and use adequate protection/abstinence or not be of child bearing potential
Who cannot join
- Clinically significant unstable illnesses such as liver, kidney, heart, stomach problems unrelated to their disease of FRDA
- Disallowed medications
- Serious underlying disease
- Clinically significant abnormal vital signs unrelated to the underlying disease of FRDA
- Abnormal laboratory blood results considered by the doctor as clinically significant, e.g.anaemia
- Treatment with idebenone within 6 weeks prior to screening
Show all 13Show fewer
- Treatment with erythropoietin within 16 weeks prior to screening
- Clinically significant abnormal ECG
- Received or donated blood within previous 3 months
- Participation within another clinical trial within past 30 days
- Pregnancy or breast feeding
- History of drug allergies or hypersensitivities
- Current (or within past 6 months) disorder related to drug or alcohol abuse (as defined DSM-IV-TR)
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
Germany4 sites
DE004
Bochum, 44791
DE002
Bonn, 53127
DE001
Munich, 80336
DE003
Tübingen, 72026
About this study
Official title: Randomised, Double Blind, Placebo Controlled Study of Lu AA24493 in Patients With Friedreich's Ataxia to Evaluate Safety and Tolerability and to Explore Efficacy
The primary purpose of the study is to determine whether carbamylated erythropoietin is a safe treatment for patients who suffer from Friedreich's Ataxia.
Read the detailed description
Friedreich's Ataxia (FRDA) is a hereditary, progressive neurodegenerative disorder caused by mutations in the gene encoding frataxin. The mutation results in a severe reduction in levels of the mitochondrial protein, frataxin. A decline in frataxin levels and its associated consequences is believed to be the primary cause of symptoms in FRDA patients. The clinical symptoms of FRDA include progressive gait and limb ataxia, dysarthria, diabetes mellitus and hypertrophic cardiomyopathy. First symptoms usually appear between the age of 5 and 15 years. As the disease progresses the patient becomes confined to a wheel chair and at later stages the patients become increasingly incapacitated. There is currently no effective treatment for FRDA.
The naturally occurring hormone, erythropoietin (EPO), is able to protect various neuronal tissues from ischemic injury. Recombinant human erythropoietin (EPO) increases frataxin expression in lymphocytes from patients with FRDA. Also, EPO treatment of FRDA patients resulted in a favourable outcome compared to baseline as assessed by the levels of frataxin and biomarkers of oxidative stress. In a pilot study with EPO in FRDA patients, the treatment was well tolerated apart from the expected haematological (haematopoietic) side effects. Lu AA24493 (CEPO) is a modified (carbamylated) version of EPO, which is neuroprotective but without the haematopoietic side effects. Lu AA24493 is being developed for treatment of patients with FRDA.
Although the target for the non-haematological effects of Lu AA24493 (and EPO) is currently unknown, Lu AA24493 (CEPO) can protect cells and tissue from various types of injuries. Furthermore, in vitro Lu AA24493 (CEPO) increases the frataxin levels in lymphocytes from FRDA patients as well as from control patients. This study aims to evaluate the safety of 2 weeks treatment (6 doses, 3 doses per week) of CEPO in patients with FRDA and to explore efficacy by using neurological rating scales and by exploring levels of frataxin and biomarkers of oxidative stress.
- Conditions
- Friedreich's Ataxia
- Being tested
- Lu AA24493, Placebo
- Keywords
- Friedreich's Ataxia, FRDA, Neurodegenerative, Erythropoietin, Carbamylated, Neuroprotection, Frataxin
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lu AA24493Drug
Also called CEPO
Vials with solution for i.v. injection. 325mcg Lu AA24493 dosed 3 times per week for two weeks. Vials will be supplied in concentrations ready for injection.
PlaceboDrug
Vials with solution for i.v. injection. Placebo dosed 3 times per week for two weeks.
| Study group | Role | What they receive |
|---|---|---|
| Lu AA24493 | Experimental | Not described Receives: Drug: Lu AA24493 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with Friedreich's Ataxia
Measured: 2 week treatment phase + 4 week follow up period
Secondary outcomes (5)
To explore biomarkers of efficacy, including frataxin, 8-OHdG & peroxides
Measured: 2 week treatment phase + 4 week follow up period
To explore efficacy by neurological assessment (Scale for the Assessment and Rating of Ataxia (SARA), Friedreich's Ataxia Rating Scale (FARS))
Measured: 2 week treatment phase + 4 week follow up period
To explore efficacy by the Clinical Global Impression scales (CGI-I/S)
Measured: 2 week treatment phase + 4 week follow up period
To explore population pharmacokinetic parameters of Lu AA24493
Measured: 2 week treatment phase + 4 week follow up period
To evaluate the immunogenicity of Lu AA24493
Measured: 2 week treatment phase + 4 week follow up period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2009 (estimated)
- Study startOct 2009
- Primary completionMar 2011
- Study completionApr 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- More trials run by H. Lundbeck A/SThe sponsor's other registered studies.
- 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- H. Lundbeck A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Email contact via H. Lundbeck A/S, Study director, LundbeckClinicalTrials@lundbeck.com
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01016366
- Sponsor's protocol ID
- 12631A
- Other IDs
- 2008-003662-25, 2008
- Status last verified
- Nov 2016
- First submitted
- 18 Nov 2009
- Last updated
- 8 Nov 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomised, Double Blind, Placebo Controlled Study of Lu AA24493 in Patients With Friedreich's Ataxia to Evaluate Safety and Tolerability and to Explore Efficacy
- ClinicalTrials.gov ID
- NCT01016366
- Acronym / sponsor ID
- 12631A
- Conditions
- Friedreich's Ataxia
- Keywords
- Friedreich's Ataxia, FRDA, Neurodegenerative, Erythropoietin, Carbamylated, Neuroprotection, Frataxin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- H. Lundbeck A/SIndustry (pharma, biotech, devices)
- Study start
- Oct 2009
- Primary completion
- Mar 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 19 Nov 2009 (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 purpose of the study is to determine whether carbamylated erythropoietin is a safe treatment for patients who suffer from Friedreich's Ataxia.
Detailed description
Friedreich's Ataxia (FRDA) is a hereditary, progressive neurodegenerative disorder caused by mutations in the gene encoding frataxin. The mutation results in a severe reduction in levels of the mitochondrial protein, frataxin. A decline in frataxin levels and its associated consequences is believed to be the primary cause of symptoms in FRDA patients. The clinical symptoms of FRDA include progressive gait and limb ataxia, dysarthria, diabetes mellitus and hypertrophic cardiomyopathy. First symptoms usually appear between the age of 5 and 15 years. As the disease progresses the patient becomes confined to a wheel chair and at later stages the patients become increasingly incapacitated. There is currently no effective treatment for FRDA.
The naturally occurring hormone, erythropoietin (EPO), is able to protect various neuronal tissues from ischemic injury. Recombinant human erythropoietin (EPO) increases frataxin expression in lymphocytes from patients with FRDA. Also, EPO treatment of FRDA patients resulted in a favourable outcome compared to baseline as assessed by the levels of frataxin and biomarkers of oxidative stress. In a pilot study with EPO in FRDA patients, the treatment was well tolerated apart from the expected haematological (haematopoietic) side effects. Lu AA24493 (CEPO) is a modified (carbamylated) version of EPO, which is neuroprotective but without the haematopoietic side effects. Lu AA24493 is being developed for treatment of patients with FRDA.
Although the target for the non-haematological effects of Lu AA24493 (and EPO) is currently unknown, Lu AA24493 (CEPO) can protect cells and tissue from various types of injuries. Furthermore, in vitro Lu AA24493 (CEPO) increases the frataxin levels in lymphocytes from FRDA patients as well as from control patients. This study aims to evaluate the safety of 2 weeks treatment (6 doses, 3 doses per week) of CEPO in patients with FRDA and to explore efficacy by using neurological rating scales and by exploring levels of frataxin and biomarkers of oxidative stress.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lu AA24493 | Experimental | Drug: Lu AA24493 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- The patient has been diagnosed with FRDA and has had a genetic test demonstrating >400 GAA nucleotide triplet repeats on the shorter of the two frataxin alleles
- The patient has a SARA (Stance) sub-score of <=6
- The patient has a SARA (Gait) sub-score of <=6
- Man or woman, aged 18 years or over
- If female then woman should agree not to try to become pregnant during the study, and use adequate protection/abstinence or not be of child bearing potential
Key exclusion criteria (13 listed)
- Clinically significant unstable illnesses such as liver, kidney, heart, stomach problems unrelated to their disease of FRDA
- Disallowed medications
- Serious underlying disease
- Clinically significant abnormal vital signs unrelated to the underlying disease of FRDA
- Abnormal laboratory blood results considered by the doctor as clinically significant, e.g.anaemia
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 |
|---|---|---|
| Lu AA24493Also: CEPOVials with solution for i.v. injection. 325mcg Lu AA24493 dosed 3 times per week for two weeks. Vials will be supplied in concentrations ready for injection. | Drug | Lu AA24493 |
| PlaceboVials with solution for i.v. injection. Placebo dosed 3 times per week for two weeks. | Drug | Placebo |
Primary outcome
To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with Friedreich's Ataxia
Time frame: 2 week treatment phase + 4 week follow up period
5 secondary outcomes
To explore biomarkers of efficacy, including frataxin, 8-OHdG & peroxides
Time frame: 2 week treatment phase + 4 week follow up period
To explore efficacy by neurological assessment (Scale for the Assessment and Rating of Ataxia (SARA), Friedreich's Ataxia Rating Scale (FARS))
Time frame: 2 week treatment phase + 4 week follow up period
To explore efficacy by the Clinical Global Impression scales (CGI-I/S)
Time frame: 2 week treatment phase + 4 week follow up period
To explore population pharmacokinetic parameters of Lu AA24493
Time frame: 2 week treatment phase + 4 week follow up period
To evaluate the immunogenicity of Lu AA24493
Time frame: 2 week treatment phase + 4 week follow up period
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 8 Nov 2016.
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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.
Email contact via H. Lundbeck A/S study director
LundbeckClinicalTrials@lundbeck.com
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lu AA24493 in people with Friedreich's Ataxia, run by H. Lundbeck A/S at 7 sites in 3 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 13 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 8 Nov 2016. 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 Germany, Italy, Austria. 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 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?
To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with Friedreich's Ataxia (time frame: 2 week treatment phase + 4 week follow up period). 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 8 Nov 2016.