A multicentre, randomised, double-blind, placebo-controlled, parallel-group trial to evaluate the efficacy and safety of BP1.4979 in adult patients with essential tremor

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bioprojet Pharma

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

21 Jul 2025 → ongoing

Decision date (initial)

2025-06-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

The primary objective is to evaluate the efficacy of BP1.4979 in treating Essential Tremor (ET).

Secondary objectives 3

1. To assess the effect of BP1.4979 on functional disability associated with essential tremor.
2. To evaluate the impact of Investigational BP1.4979 on patient-reported quality of life.
3. To assess the safety and tolerability of BP1.4979.

Conditions and MedDRA coding

Essential tremor

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Written informed consent obtained prior to any trial-related procedures.
2. Male or female ≥18-85 years old.
3. Confirmed diagnosis of ET, characterized by meeting the following criteria: (a) the presence of a bilateral upper limb action tremor that occurs in isolation; (b) a minimum duration of three (3) years; and (c) the tremor may or may not be present in other areas such as the voice, or lower limbs.
4. Patient with ET, characterized by a TETRAS-P score of at least 1.5 in either the forward posture, wing beating posture, or finger-to-nose movement of at least one upper extremity. This assessment will be conducted using the Performance subscale of The Essential Tremor Rating Assessment Scale, following the criteria established by the Tremor Investigation Group.
5. If taking anti-tremor medication(s), patient must have been on a consistent and unchanged dose of anti-tremor medication for at least four (4) weeks prior to the screening. Additionally, she/he must be willing to maintain their current medication dosage throughout the entire duration of the study.
6. The patient should not have undergone any previous surgical procedures specifically for tremor treatment.
7. There should be a minimum interval of four (4) months between the patient’s last botulinum injection and the screening.
8. The patient should not exhibit significant imbalance due to the tremors or have an increased risk of falls.
9. Patient must have a cooperative attitude and be able to comply with the entire trial requirements and procedures (e.g., trial-related questionnaire, drug compliance, not use prohibited concomitant medications).
10. Female patient: post-menopausal woman having at least 12 months of natural (spontaneous) amenorrhea without any alternative medical cause, or women of childbearing potential (WOCBP, defined as all fertile woman, following menarche and until becoming post-menopausal unless permanently sterile; permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy) using a highly effective method of contraception for the duration of the trial and for one (1) month after stopping the investigational medication.
11. If required, patient must be insured by appropriate national health insurance system (mandatory for France).

Exclusion criteria 20

1. Severe tremor, defined as patient with ET characterized by a TETRAS-P score of ≥ 3 in either the forward posture, wing beating posture, or finger-to-nose movement of at least one upper extremity, or tremor of trunk.
2. Isolated head tremor not accompanied with tremor of any other body part.
3. Patient who has a medical history or clinical evidence of any other conditions, whether medical, neurological, or psychiatric, that could potentially explain or contribute to the presence of tremors. Examples of such conditions include but are not limited to Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, cerebellar disease (including spinocerebellar ataxias), primary dystonia, Fragile X Tremor/Ataxia syndrome or a family history of Fragile X syndrome, traumatic brain injury, psychogenic tremor, alcohol or benzodiazepine abuse or withdrawal, multiple sclerosis, polyneuropathy, endocrine disorders such as hyperthyroidism or unstable treatment of hypothyroidism, food-induced tremors, or tremors related to the use of supplements (e.g., tremors caused by beta agonists or caffeine).
4. Patient who takes a medication which may induce tremor. For reference, some of the common medications and substances which may potentially cause physiologic tremors are: Amiodarone, certain antidepressants (e.g., serotonin reuptake inhibitors, serotonin-noradrenaline reuptake inhibitors, tricyclic antidepressants), antiseizure medications such as bromides carbamazepine, lacosamide, lamotrigine, phenytoin, valproic acid, certain beta-agonists such as albuterol and terbutaline, certain glucocorticoids such as dexamethasone or prednisone, the mood stabilizer lithium, sympathomimetics such as amphetamine salts, epinephrine, methylphenidate, thyroid hormone replacement therapies such as levothyroxine, substances such as caffeine, cocaine and nicotine, and certain other treatments such as cyclosporine, tacrolimus, theophylline.
5. Patient who may have had direct or indirect injury or trauma to the nervous system within 3 months before the onset of tremor.
6. Patient who takes concomitant treatment with more than three drugs to treat ET.
7. Patient who has undergone any prior procedures for the treatment of ET such as deep brain stimulation, brain lesioning, or magnetic resonance (MR)-guided procedures, including MR-guided focused ultrasound.
8. Patient who has a historical or clinical evidence of tremor with a psychogenic origin, which includes conditions like eating disorders or major depression, among others.
9. Patient with a history of suicidal behaviour within the past two years or who is currently assessed to be at risk for suicide as assessed by the C-SSRS score of “YES” on questions 4 or 5, and/or based on clinical evaluation by the investigator, is not eligible for the study.
10. Female patient: pregnant or lactating woman. [Pregnancy is confirmed by a positive serum human chorionic gonadotrophin laboratory test (> 5mIU/mL)].
11. History of significant cardiovascular disease, particularly recent history of myocardial infarction or unstable coronary artery disease, arrhythmias, congestive heart failure, uncontrolled arterial hypertension. Patient with a known history of long QT syndrome with or without history of syncope.
12. Patient with a clinically significant deviation(s) from normal on 12-lead ECG that results in an active medical problem, as determined by the Investigator at screening or has a corrected QT interval using Fridericia’s formula (QTcF) ≥450 msec for males or ≥470 msec for females.
13. Patient with unstable or uncontrolled disease that might affect the patient’s safety and/or interfere with the conduct of the study according to the Investigator’s judgement.
14. Patient with concomitant prolactin-dependent tumour (e.g., pituitary tumour or breast cancer)
15. Patient with history of malignancy within the past 5 years with the exception of adequately treated or excised non metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.
16. Established diagnosis of human immunodeficiency virus (HIV), hepatitis B viral infection or is positive for hepatitis surface antigen (HbsAg) or hepatitis B core antibody (HbcAb) at screening or established diagnosis of hepatitis C viral infection or is positive for hepatitis C antibody at screening.
17. Patient who has a laboratory abnormality at screening as follows: ALT, AST values > 2 x upper limit of normal (ULN), Serum creatinine value >1.5 x ULN, GFR < 50 ml/min/1.73m2 (CKD-EPI formula), Absolute neutrophils count <1.0x109 /L, Platelets < 100x109 /L or who has any other uncontrolled clinically significant laboratory abnormalities that would affect interpretation of the study data or the patient’s participation in the study.
18. History of hypersensitivity to any of the study drug constituents.
19. Current or recent history (less than one year) of alcohol or drug abuse.
20. Patient having received any other investigational drug within the preceding 30 days, or a longer and more appropriate time as determined by the Investigator (e.g., approximately five half-lives of the previous investigational drug).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary efficacy endpoint is the change in the total score of the Performance Subscale of The Essential Tremor Rating Assessment Scale (TETRAS-P) after 4 weeks of treatment.

Secondary endpoints 1

1. No secondary endpoints are planned in this trial.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bioprojet Pharma

Sponsor organisation

Bioprojet Pharma

Address

9 Rue Rameau

City

Paris

Postcode

75002

Country

France

Scientific contact point

Organisation

Bioprojet Pharma

Contact name

Regulatory Affairs Director

Public contact point

Organisation

Bioprojet Pharma

Contact name

Regulatory Affairs Director

Locations

1 EU/EEA country · 14 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 21 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications