Friedreich AtaxiaPhase 2NCT05579691

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

A Phase 2 study testing CTI-1601 in people with Friedreich Ataxia, run by Larimar Therapeutics, Inc. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA). 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: 28 participants
Where: 1 siteUnited States
Study dates: 21 Sep 2022to 4 Dec 2023
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

Subject has a genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
Subject is biologically male or female, 18 years of age or older at screening.
Subject must have a mFARS score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (e.g., 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 PI, 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.
Subject must weigh > 40.0 kg.

Who cannot join

Subjects are excluded from the study if any of the following exclusion criteria are met:
If the subject previously participated in a study of CTI-1601 (CLIN-1601-101 (NCT04176991) or CLIN-1601-102 (NCT04519567)) the subject may not enroll in this study if they experienced one or more of the following: (a) Serious Adverse Event (SAE) related to study drug; (b) Adverse Event (AE) defined as Grade 3 or higher according to the CTCAE version 5.0 (or higher), related to study drug; (c) some other event that supports the exclusion of the subject from participating in this study as determined by the Sponsor (i.e., an AE considered clinically significant by the Sponsor regardless of whether it met SAE criteria and regardless of CTCAE grade).
Subject who is confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
Subject used an investigational drug or device within 90 days prior to screening.
Subject requires use of amiodarone.
Subject used erythropoietin, etravirine, or gamma interferon 90 days prior to Screening.

Show all 13Show fewer

Subject use of biotin supplementation that exceeds 30.0 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug.
Subject uses more than 3.0 grams of acetaminophen daily.
Subject receives medication that requires SC injection in the abdomen or thigh.
Subject received a vaccination within 14 days of administration of the first dose of study drug or is scheduled to receive a vaccination within 14 days after administration of the last dose of study drug. As an exception, influenza and tetanus vaccines must be administered more than 72 hours prior to the first dose of study drug or 72 hours after the administration of the last dose of study drug.
Subject has a screening ECHO LVEF < 45%.
Male subject has a QTcF > 450 milliseconds or female subject has a QTcF > 470 milliseconds on an ECG.
Subject currently receiving or having received omaveloxolone within 30 days prior to Screening.

Trial locations and contacts

1 site in 1 country.

United States1 site

Clinilabs Drug Development Corporation
Eatontown, New Jersey, 07724

Other trials in United States

About this study

Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Read the detailed description

This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of CTI-1601.

This study will consist of at least 2 cohorts with 12 to 15 subjects participating in each cohort. Subjects will be dosed once daily (QD) for 14 days followed by dosing every other day (QOD) through Day 28.

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
PlaceboOther
Placebo Comparator

Study groups in NCT05579691
Study group	Role	What they receive
CTI-160l	Experimental	CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia Receives: Biological: CTI-1601
Placebo	Placebo comparator	Placebo Comparator Receives: Other: 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 93 days

Secondary outcomes (6)

Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses
Summary assessment of changes in the maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses
Measured: At baseline and up to 29 days
Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point
Summary assessment of changes in the AUC of CTI-1601 from time 0 to the last measurable time point and during the dosing interval
Measured: At baseline and up to 29 days
Time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses
Summary assessment of the time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses
Measured: At baseline and up to 29 days
Time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses
Summary assessment of the time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses
Measured: At baseline and up to 29 days
Changes from baseline in frataxin levels in buccal cells
Summary assessment of changes in frataxin levels in buccal cells
Measured: At baseline and up to 58 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 29 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

21 Sep 20224 Dec 2023

First posted14 Oct 2022
Study start21 Sep 2022
Primary completion4 Dec 2023
Study completion4 Dec 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (9)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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 28 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05579691
Sponsor's protocol ID: CLIN-1601-200
Status last verified: Nov 2024
First submitted: 5 Oct 2022
Last updated: 29 Nov 2024
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Delatycki 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 22752493
Goodkin 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 3178453
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 21315377
Rudick 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 8797541
Deutsch EC, Santani AB, Perlman SL, Farmer JM, Stolle CA, Marusich MF, Lynch DR. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab. 2010 Oct-Nov;101(2-3):238-45. doi: 10.1016/j.ymgme.2010.07.001. Epub 2010 Jul 8.
PubMed 20675166
Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schols L, Giordano I, Burk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.
PubMed 32746884
Lawerman TF, Brandsma R, Burger H, Burgerhof JGM, Sival DA; the Childhood Ataxia and Cerebellar Group of the European Pediatric Neurology Society. Age-related reference values for the pediatric Scale for Assessment and Rating of Ataxia: a multicentre study. Dev Med Child Neurol. 2017 Oct;59(10):1077-1082. doi: 10.1111/dmcn.13507. Epub 2017 Aug 17.
PubMed 28815574
Lazaropoulos M, Dong Y, Clark E, Greeley NR, Seyer LA, Brigatti KW, Christie C, Perlman SL, Wilmot GR, Gomez CM, Mathews KD, Yoon G, Zesiewicz T, Hoyle C, Subramony SH, Brocht AF, Farmer JM, Wilson RB, Deutsch EC, Lynch DR. Frataxin levels in peripheral tissue in Friedreich ataxia. Ann Clin Transl Neurol. 2015 Aug;2(8):831-42. doi: 10.1002/acn3.225. Epub 2015 Jul 1.
PubMed 26339677
Pandolfo M. Friedreich ataxia. Arch Neurol. 2008 Oct;65(10):1296-303. doi: 10.1001/archneur.65.10.1296.
PubMed 18852343

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
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Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
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Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
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RecruitingObservational study
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All Friedreich Ataxia trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia
ClinicalTrials.gov ID: NCT05579691
Acronym / sponsor ID: CLIN-1601-200
Conditions: Friedreich Ataxia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 28 participants (actual)
Lead sponsor: Larimar Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: 21 Sep 2022 (actual)
Primary completion: 4 Dec 2023 (actual)
Study completion: 4 Dec 2023 (actual)
First posted: 14 Oct 2022 (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, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Detailed description

This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of 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-160l	Experimental	Biological: CTI-1601CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
Placebo	Placebo comparator	Other: PlaceboPlacebo Comparator

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 a genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
Subject is biologically male or female, 18 years of age or older at screening.
Subject must have a mFARS score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (e.g., 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 PI, 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.
Subject must weigh > 40.0 kg.

Key exclusion criteria (13 listed)

If the subject previously participated in a study of CTI-1601 (CLIN-1601-101 (NCT04176991) or CLIN-1601-102 (NCT04519567)) the subject may not enroll in this study if they experienced one or more of the following: (a) Serious Adverse Event (SAE) related to study drug; (b) Adverse Event (AE) defined as Grade 3 or higher according to the CTCAE version 5.0 (or higher), related to study drug; (c) some other event that supports the exclusion of the subject from participating in this study as determined by the Sponsor (i.e., an AE considered clinically significant by the Sponsor regardless of whether it met SAE criteria and regardless of CTCAE grade).
Subject who is confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
Subject used an investigational drug or device within 90 days prior to screening.
Subject requires use of amiodarone.
Subject used erythropoietin, etravirine, or gamma interferon 90 days 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-160l
PlaceboPlacebo Comparator	Other	Placebo

Primary outcome

Number of Participants with Treatment Emergent Adverse Events
Time frame: Through study completion, an average of 93 days
Overall summary of Participants with Treatment Emergent Adverse Events

6 secondary outcomes

Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses
Time frame: At baseline and up to 29 days
Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point
Time frame: At baseline and up to 29 days
Time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses
Time frame: At baseline and up to 29 days
Time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses
Time frame: At baseline and up to 29 days
Changes from baseline in frataxin levels in buccal cells
Time frame: At baseline and up to 58 days
Changes from baseline in frataxin levels in skin punch cells
Time frame: At baseline and up to 29 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 29 Nov 2024.
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 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 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 20675166Deutsch EC, Santani AB, Perlman SL, Farmer JM, Stolle CA, Marusich MF, Lynch DR. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab. 2010 Oct-Nov;101(2-3):238-45. doi: 10.1016/j.ymgme.2010.07.001. Epub 2010 Jul 8.
Background: PubMed 32746884Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schols L, Giordano I, Burk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.
Background: PubMed 28815574Lawerman TF, Brandsma R, Burger H, Burgerhof JGM, Sival DA; the Childhood Ataxia and Cerebellar Group of the European Pediatric Neurology Society. Age-related reference values for the pediatric Scale for Assessment and Rating of Ataxia: a multicentre study. Dev Med Child Neurol. 2017 Oct;59(10):1077-1082. doi: 10.1111/dmcn.13507. Epub 2017 Aug 17.
Background: PubMed 26339677Lazaropoulos M, Dong Y, Clark E, Greeley NR, Seyer LA, Brigatti KW, Christie C, Perlman SL, Wilmot GR, Gomez CM, Mathews KD, Yoon G, Zesiewicz T, Hoyle C, Subramony SH, Brocht AF, Farmer JM, Wilson RB, Deutsch EC, Lynch DR. Frataxin levels in peripheral tissue in Friedreich ataxia. Ann Clin Transl Neurol. 2015 Aug;2(8):831-42. doi: 10.1002/acn3.225. Epub 2015 Jul 1.
Background: PubMed 18852343Pandolfo M. Friedreich ataxia. Arch Neurol. 2008 Oct;65(10):1296-303. doi: 10.1001/archneur.65.10.1296.

Related trials for Friedreich Ataxia

RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
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RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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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 2 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 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 29 Nov 2024. 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 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?

Number of Participants with Treatment Emergent Adverse Events (time frame: Through study completion, an average of 93 days). 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 NCT05579691, which also lists 9 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 29 Nov 2024.