Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia (NICOFA)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03761511 |
Recruitment Status :
Not yet recruiting
First Posted : December 3, 2018
Last Update Posted : September 16, 2021
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Friedreich Ataxia | Drug: Nicotinamide Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 225 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia |
Estimated Study Start Date : | April 2022 |
Estimated Primary Completion Date : | June 2023 |
Estimated Study Completion Date : | December 2023 |

Arm | Intervention/treatment |
---|---|
Active Comparator: Treatment arm
Nicotinamide 4 g (capsules) or highest tolerated dose with a minimum of 2 g/d per os once daily
|
Drug: Nicotinamide
Nicotinamide 4 g (capsules) or highest tolerated dose with a minimum of 2 g/d per os once daily |
Placebo Comparator: Placebo arm
Matching Placebo (capsules) once daily
|
Drug: Placebo
Matching Placebo (capsules) once daily |
- Scale for the Assessment and Rating of Ataxia (SARA) [ Time Frame: 1 year ]
The primary objective of the study is to evaluate the efficacy of daily doses of nicotinamide in slowing disease progression as measured by changes in the Scale for the Assessment and Rating of Ataxia (SARA) as compared with placebo in patients with Friedreich ataxia.
The SARA is a tool for assessing ataxia. It has eight categories with accumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia). When completing the outcome measure each category is assessed and scored accordingly. Scores for the eight items range as follows:
Gait (0-8 points), Stance (0-6 points), Sitting (0-4 points) Speech disturbance (0-6 points) Finger chase (0-4 points) Nose-finger test (0-4 points) Fast alternating hand movement (0-4 points) Heel-shin slide (0-4 points) Once each of the 8 categories have been assessed, the total is calculated to determine the severity of ataxia.
- Progression of quality of life measures via questionnaire EuroQol five dimensions questionnaire (EQ-5D) [ Time Frame: 1 year ]This is a generic instrument, developed and validated by the EuroQoL Group (1990), in which the patient self-rates in particular his/her health status on a visual analogue scale (VAS); the best score is 100.
- Modified Friedreich Ataxia Rating Scale (mFARS) [ Time Frame: 1 year ]Modified FARS scores are defined as the sum of scores for bulbar function, upper limb coordination, lower limb coordination, and upright stability. The mFARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.
- Progression of cerebellar severity measured by 'Composite Cerebellar Functional Severity´ (CCFS) score [ Time Frame: 1 year ]CCFS includes two tests performed with the dominant hand,named the click test and the 9 holes pegboard test (9HPT). An electronic device was created to perform CCFS and to calculate the Z score by subtracting the expected time
- Clinician's Global Impression-Change Scale (CGI-C) including comparison of change to the last visit [ Time Frame: 1 year ]The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as: 1. Very much improved; 2. Much improved; 3. Minimally improved; 4. No change; 5. Minimally worse; 6. Much worse; 7. Very much worse
- Safety issues measured by appearance of AEs/SAEs [ Time Frame: 1 year ]AEs may be volunteered spontaneously by the patient, or discovered as a result of general non-directed questioning by the study personnel or by physical examination.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Patients must have a molecular genetic diagnosis of Friedreich ataxia with a GAA- repeat expansion on both alleles of the FXN gene and a SARA Score >7 and <28 and age <50 years.
- Patients must be ≥18 years old and have a weight of at least 50kg.
- Written informed consent prior to study participation
- A female subject is eligible to participate if she is of: Non-childbearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea or of childbearing potential and agrees to
Exclusion Criteria:
- Patients with any medical condition or illness that, in the opinion of the investigator would interfere with study compliance and/or impair the patient´s ability to participate or complete the study.
- Any uncontrolled medical or neurological/neurodegenerative condition (other than Friedreich ataxia).
- Clinically significant psychiatric illness (e.g., uncontrolled major depression, schizophrenia, bipolar affective disorder) within 6 months prior to screening.
- Patients with significant clinical dysphagia.
- Hypersensitivity to nicotinamide.
- Patients known to be positive for human immunodeficiency virus (HIV).
- Patients with a significant history of substance abuse (e.g. alcohol or drug abuse) within the previous six months before enrolment.
- Patients with a history of severe allergies to medications.
- Indication of impaired liver function as shown by an abnormal liver function profile at screening (e.g., repeated values of aspartate aminotransferase [AST], alanine aminotransferase [ALT] and bilirubin ≥3 × the upper limit of normal).
-
History of malignancy or carcinoma. The following exceptions may be made after discussion with the Sponsor:
- Subjects with cancers in remission more than 5 years prior to screening.
- Subjects with a history of excised or treated basal cell or squamous carcinoma.
- Subjects with prostate cancer in situ.
- History or evidence of an autoimmune disorder considered clinically significant by the Investigator or requiring chronic use of systemic corticosteroids or other immunosuppressants.
- The subject has a history of any other illness, which, in the opinion of the Investigator, might pose an unacceptable risk by administering study medication.
- History of clinically significant cardiac disease (ejection fraction < 40% [normal range 50-70%], cardiac insufficiency defined as New York Heart Association [NYHA] Class >2; clinically significant congenital or acquired valvular disease; symptomatic coronary disease such as prior myocardial infarction or angina, B-type natriuretic peptide (BNP) level increase more than 2 x of the normal age- and gender dependent range; history of unstable arrhythmias, history of atrial fibrillation).
- The subject received an investigational drug within 30 days prior to inclusion into this study.
- Patients taking sodium valproate, tranylcypromine or any other known histone deacetylase inhibitor.
- Use of vitamin B1 (thiamine), withdrawal should be at least 3 months prior screening or 5 half-lives, whichever is longer.
- Use of vitamin B3 (nicotinamide), withdrawal should be at least 3 months prior screening.
- If patients are taking idebenone or coenzyme Q10 (CoQ), this should be stable over the last three months and not changed during the study.
- The subject is unwilling or unable to provide written informed consent and to follow the procedures outlined in the protocol.
- For subjects who will undergo an MRI: Any contraindications to MRI such as, but not limited to cardiac pacemaker, implanted cardiac defibrillator, aneurysm clips, carotid artery vascular clamp, neurostimulator, implanted drug infusion devices, metal fragments or foreign objects in the eyes, skin or body, bone growth/fusion stimulator, cochlear, otologic implant, severe claustrophobia or any condition that would counterindicate an MRI scan.
- Patients participating at start or have been within 30 days before start of study in another pharmacological and non-pharmacological clinical trial, excluding natural history / observational studies.
- The subject is mentally or legally incapacitated.
- Pregnant females as determined by positive [serum or urine] hCG test at Screening or prior to dosing. Participants of child-bearing age should use adequate contraception as defined in the study protocol.
- Lactating females.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03761511
Austria | |
Medical University Innsbruck | |
Innsbruck, Austria, 6020 | |
Contact: Sylvia Boesch, PD Dr. sylvia.boesch@i-med.ac.at | |
France | |
Service de génétique médicale - Hôpital La Pitié Salpetrière | |
Paris, France, 75646 | |
Contact: Alexandra Durr, Prof. Dr. : +33 (0) 1 57 27 40 00 alexandra.durr@upmc.fr | |
Germany | |
University Hospital RWTH Aachen | |
Aachen, Germany, 52074 | |
Contact: Jörg B. Schulz, Univ.-Prof. jschulz@ukaachen.de | |
Italy | |
Fondazione IRCCS Istituto Neurologico Carlo Besta | |
Milano, Italy, 20133 | |
Contact: Caterina Mariotti, Prof. Dr. Caterina.Mariotti@istituto-besta.it | |
Spain | |
Hospital Universitario La Paz | |
Madrid, Spain, 28046 | |
Contact: Francisco Javier Rodríguez de Rivera Garrido, Prof. Dr. frriveragarrido@salud.madrid.org | |
United Kingdom | |
Imperial College London | |
London, United Kingdom, W12 0HS | |
University College London | |
London, United Kingdom, WC1N 3BG | |
Contact: Paola Giunti, Prof. Dr. p.giunti@ucl.ac.uk |
Documents provided by RWTH Aachen University:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | RWTH Aachen University |
ClinicalTrials.gov Identifier: | NCT03761511 |
Other Study ID Numbers: |
15-138 |
First Posted: | December 3, 2018 Key Record Dates |
Last Update Posted: | September 16, 2021 |
Last Verified: | September 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Plan Description: | The study results will be published in appropriate international scientific journals, and publishing details will be given in the clinical study agreement. The study will be registered and study results will be disclosed by the principal investigator in one or more public clinical study registry(ies), according to national/international use. |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
Friedreich Ataxia Nicotinamide Rare disease ZSEA SARA |
Ataxia Cerebellar Ataxia Friedreich Ataxia Dyskinesias Neurologic Manifestations Nervous System Diseases Cerebellar Diseases Brain Diseases Central Nervous System Diseases Spinocerebellar Degenerations Spinal Cord Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn |
Mitochondrial Diseases Metabolic Diseases Niacinamide Niacin Nicotinic Acids Vitamin B Complex Vitamins Micronutrients Physiological Effects of Drugs Hypolipidemic Agents Antimetabolites Molecular Mechanisms of Pharmacological Action Lipid Regulating Agents Vasodilator Agents |