Friedreich AtaxiaPhase 1NCT04519567
Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
A Phase 1 study testing CTI-1601 in people with Friedreich Ataxia, run by Larimar Therapeutics, Inc. at 1 site in United States.
Trial summary
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- CTI-1601
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Jul 2020to 16 Mar 2021
- 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 genetically confirmed Friedreich's ataxia diagnosis manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
- Subject is male or female, 18 years of age or older at screening
- Subject must have a mFARS_neuro score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (cane, walker, crutches, self-propelled wheelchair) and (a) be able to sit upright with thighs together and arms crossed without requiring support on more than two sides; (b) be able to transfer from bed to chair independently or with assistance if, in the opinion of the principal investigator, the degree of physical disability does not result in undue risk to the subject while participating in the study; and (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance.
- Subjects must weigh > 40 kilograms (kg).
Who cannot join
- Subjects who had a serious adverse event (SAE), an adverse event (AE) that is Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), or an AE considered clinically significant during participation in CLIN-1601-101 (NCT04176991).
- Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
- Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
- Subject requires use of amiodarone.
- Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
- Subject use of daily biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to study drug administration and/or throughout the entire study.
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- Subject has clinically significant arrhythmia on electrocardiogram (ECG), or evidence of predisposition to significant ventricular arrhythmia on ECG, or evidence of active and unstable coronary artery disease.
- Male subject who has a QT interval corrected for heart rate using Fridericia's formula (QTcF) > 450 milliseconds or female subject who has a QTcF > 470 milliseconds on an ECG.
- Subject has a screening echocardiogram left ventricular ejection fraction < 45 percent.
- Subject has a history of aspiration, aspiration pneumonia, or recurrent episodes of pneumonia (greater than or equal to 2 episodes of pneumonia) within the last 12 months.
Trial locations and contacts
1 site in 1 country.
United States1 site
Clinilabs Drug Development Corporation
Eatontown, New Jersey, 07724
About this study
Official title: A Phase 1 Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia
Read the detailed description
Multiple Ascending Dose (MAD), Double-Blind, Placebo Controlled Study.
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in subjects with Friedreich's ataxia.
Secondary Objectives:
- To evaluate the pharmacokinetics (PK) of CTI-1601 following, multiple, increasing, doses of subcutaneously (SC) administered CTI-1601.
- To evaluate the pharmacodynamics (PD) of CTI-1601 following, multiple, increasing, doses of SC administered CTI-1601.
- Conditions
- Friedreich Ataxia
- Being tested
- CTI-1601, Placebo
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTI-1601Biological
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
PlaceboBiological
Placebo Comparator
| Study group | Role | What they receive |
|---|---|---|
| CTI-1601 | Experimental | Not described Receives: Biological: CTI-1601 |
| Placebo | Placebo comparator | Not described Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment Emergent Adverse Events
Overall summary of Participants with Treatment Emergent Adverse Events
Measured: Through study completion, an average of 75 days
Number of Participants with Treatment Emergent Adverse Events by System Organ Classification and Preferred Term
Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 23.0)
Measured: Through study completion, an average of 75 days
Secondary outcomes (12)
Pharmacokinetics - Maximum observed plasma concentration after multiple doses
Summary assessment of changes in the maximum observed plasma concentration after multiple doses
Measured: At baseline and up to 15 days
Pharmacokinetics - Minimum or "trough" plasma concentration after multiple doses
Summary assessment of minimum or "trough" observed plasma concentration after multiple doses just prior to the administration of a subsequent dose
Measured: At baseline and up to 15 days
Pharmacokinetics - Area under the concentration time curve (AUC) from time 0 through the last measurable time point
Summary assessment of changes in the AUC from time 0 to the last measurable time point and during the dosing interval
Measured: At baseline and up to 15 days
Pharmacokinetics - Terminal half-life estimation
Summary assessment of changes in the terminal half-life estimation
Measured: At baseline and up to 15 days
Changes from Baseline in Frataxin Levels in Buccal Cell
Summary assessment of changes in frataxin levels in buccal cells
Measured: At baseline and up to 43 days
Changes from Baseline in Levels of Protein Markers in Buccal Cell
Summary assessment of changes in levels of protein markers in buccal cells
Measured: At baseline and up to 43 days
Changes from Baseline in Gene Expression in Buccal Cells
Summary assessment of changes in gene expression in buccal cells
Measured: At baseline and up to 43 days
Changes from Baseline in Frataxin Levels in Platelets
Summary assessment of changes in frataxin levels in platelets
Measured: At baseline and up to 13 days
Changes from Baseline in Gene Expression in Whole Blood
Summary assessment of changes in gene expression in whole blood
Measured: At baseline and up to 16 days
Changes from Baseline in Frataxin Levels in Skin Punch Cells
Summary assessment of changes in frataxin levels in skin punch cells
Measured: At baseline and up to 13 days
Changes from Baseline in Levels of Defined Protein Markers in Blood
Summary assessment of changes in levels of defined protein markers in blood
Measured: At baseline and up to 16 days
Changes from Baseline in Levels of Specialized Lipids in Blood
Summary assessment of changes in levels of specialized lipids in blood
Measured: At baseline and up to 16 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2020
- Study start31 Jul 2020
- Primary completion16 Mar 2021
- Study completion16 Mar 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Larimar Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Veristat, Inc., Metrum Research Group, LLC
- Responsible party
- Sponsor
- Investigators
- Magdy Shenouda, M.D., Principal investigator, Clinilabs, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04519567
- Sponsor's protocol ID
- CLIN-1601-102
- Status last verified
- Jun 2021
- First submitted
- 17 Aug 2020
- Last updated
- 30 Jun 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci. 2011 Apr 15;303(1-2):1-12. doi: 10.1016/j.jns.2011.01.010.
PubMed 21315377Delatycki MB, Corben LA. Clinical features of Friedreich ataxia. J Child Neurol. 2012 Sep;27(9):1133-7. doi: 10.1177/0883073812448230. Epub 2012 Jun 29.
PubMed 22752493Goodkin DE, Hertsgaard D, Seminary J. Upper extremity function in multiple sclerosis: improving assessment sensitivity with box-and-block and nine-hole peg tests. Arch Phys Med Rehabil. 1988 Oct;69(10):850-4.
PubMed 3178453Rudick R, Antel J, Confavreux C, Cutter G, Ellison G, Fischer J, Lublin F, Miller A, Petkau J, Rao S, Reingold S, Syndulko K, Thompson A, Wallenberg J, Weinshenker B, Willoughby E. Clinical outcomes assessment in multiple sclerosis. Ann Neurol. 1996 Sep;40(3):469-79. doi: 10.1002/ana.410400321.
PubMed 8797541Plasterer HL, Deutsch EC, Belmonte M, Egan E, Lynch DR, Rusche JR. Development of frataxin gene expression measures for the evaluation of experimental treatments in Friedreich's ataxia. PLoS One. 2013 May 17;8(5):e63958. doi: 10.1371/journal.pone.0063958. Print 2013.
PubMed 23691127
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT04519567
- Acronym / sponsor ID
- CLIN-1601-102
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Larimar Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Veristat, Inc., Metrum Research Group, LLC
- Study start
- 31 Jul 2020 (actual)
- Primary completion
- 16 Mar 2021 (actual)
- Study completion
- 16 Mar 2021 (actual)
- First posted
- 19 Aug 2020 (actual)
- 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.
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia
Detailed description
Multiple Ascending Dose (MAD), Double-Blind, Placebo Controlled Study.
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in subjects with Friedreich's ataxia.
Secondary Objectives:
- To evaluate the pharmacokinetics (PK) of CTI-1601 following, multiple, increasing, doses of subcutaneously (SC) administered CTI-1601.
- To evaluate the pharmacodynamics (PD) of CTI-1601 following, multiple, increasing, doses of SC administered CTI-1601.
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 |
|---|---|---|
| CTI-1601 | Experimental | Biological: CTI-1601 |
| Placebo | Placebo comparator | Biological: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Subject has genetically confirmed Friedreich's ataxia diagnosis manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
- Subject is male or female, 18 years of age or older at screening
- Subject must have a mFARS_neuro score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (cane, walker, crutches, self-propelled wheelchair) and (a) be able to sit upright with thighs together and arms crossed without requiring support on more than two sides; (b) be able to transfer from bed to chair independently or with assistance if, in the opinion of the principal investigator, the degree of physical disability does not result in undue risk to the subject while participating in the study; and (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance.
- Subjects must weigh > 40 kilograms (kg).
Key exclusion criteria (10 listed)
- Subjects who had a serious adverse event (SAE), an adverse event (AE) that is Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), or an AE considered clinically significant during participation in CLIN-1601-101 (NCT04176991).
- Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
- Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
- Subject requires use of amiodarone.
- Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to 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 |
|---|---|---|
| CTI-1601CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia | Biological | CTI-1601 |
| PlaceboPlacebo Comparator | Biological | Placebo |
Primary outcome
Number of Participants with Treatment Emergent Adverse Events
Time frame: Through study completion, an average of 75 days
Overall summary of Participants with Treatment Emergent Adverse Events
Number of Participants with Treatment Emergent Adverse Events by System Organ Classification and Preferred Term
Time frame: Through study completion, an average of 75 days
Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 23.0)
12 secondary outcomes
Pharmacokinetics - Maximum observed plasma concentration after multiple doses
Time frame: At baseline and up to 15 days
Pharmacokinetics - Minimum or "trough" plasma concentration after multiple doses
Time frame: At baseline and up to 15 days
Pharmacokinetics - Area under the concentration time curve (AUC) from time 0 through the last measurable time point
Time frame: At baseline and up to 15 days
Pharmacokinetics - Terminal half-life estimation
Time frame: At baseline and up to 15 days
Changes from Baseline in Frataxin Levels in Buccal Cell
Time frame: At baseline and up to 43 days
Changes from Baseline in Levels of Protein Markers in Buccal Cell
Time frame: At baseline and up to 43 days
Changes from Baseline in Gene Expression in Buccal Cells
Time frame: At baseline and up to 43 days
Changes from Baseline in Frataxin Levels in Platelets
Time frame: At baseline and up to 13 days
Changes from Baseline in Gene Expression in Whole Blood
Time frame: At baseline and up to 16 days
Changes from Baseline in Frataxin Levels in Skin Punch Cells
Time frame: At baseline and up to 13 days
Changes from Baseline in Levels of Defined Protein Markers in Blood
Time frame: At baseline and up to 16 days
Changes from Baseline in Levels of Specialized Lipids in Blood
Time frame: At baseline and up to 16 days
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 30 Jun 2021.
- Background: PubMed 21315377Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci. 2011 Apr 15;303(1-2):1-12. doi: 10.1016/j.jns.2011.01.010.
- Background: PubMed 22752493Delatycki MB, Corben LA. Clinical features of Friedreich ataxia. J Child Neurol. 2012 Sep;27(9):1133-7. doi: 10.1177/0883073812448230. Epub 2012 Jun 29.
- Background: PubMed 3178453Goodkin DE, Hertsgaard D, Seminary J. Upper extremity function in multiple sclerosis: improving assessment sensitivity with box-and-block and nine-hole peg tests. Arch Phys Med Rehabil. 1988 Oct;69(10):850-4.
- Background: PubMed 8797541Rudick R, Antel J, Confavreux C, Cutter G, Ellison G, Fischer J, Lublin F, Miller A, Petkau J, Rao S, Reingold S, Syndulko K, Thompson A, Wallenberg J, Weinshenker B, Willoughby E. Clinical outcomes assessment in multiple sclerosis. Ann Neurol. 1996 Sep;40(3):469-79. doi: 10.1002/ana.410400321.
- Background: PubMed 23691127Plasterer HL, Deutsch EC, Belmonte M, Egan E, Lynch DR, Rusche JR. Development of frataxin gene expression measures for the evaluation of experimental treatments in Friedreich's ataxia. PLoS One. 2013 May 17;8(5):e63958. doi: 10.1371/journal.pone.0063958. Print 2013.
- Friedreich's Ataxia Research AllianceLink provided by the sponsor.
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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.
Magdy Shenouda, M.D. principal investigator
Clinilabs, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CTI-1601 in people with Friedreich Ataxia, run by Larimar Therapeutics, Inc. at 1 site in United States.
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 10 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 30 Jun 2021. 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 United States. 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 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 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?
Number of Participants with Treatment Emergent Adverse Events (time frame: Through study completion, an average of 75 days). The study lists 2 primary outcomes in all. 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 NCT04519567, which also lists 5 linked publications.
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 30 Jun 2021.