A Study to Investigate the Efficacy and Safety of NT 201 (Botulinum Toxin) Compared With Placebo for the Treatment of Adult Participants With Essential Tremor in the Arm

Merz Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Essential Tremor of the Upper Limb

Treatments: Drug: NT 201; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04766723

M602011069

2021-001988-24 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to determine whether a single treatment with administration of NT 201 (botulinum toxin) is superior to placebo (no medicine) for one-sided treatment of essential tremor in the arm (Unilateral Period). Participants will be assigned to the treatment groups by chance and neither the participants nor the research staff who interact with them will know the allocation.

The following treatment cycle will investigate the safety and tolerability of two-sided treatment with NT 201 (botulinum toxin) (Open Label Bliaterial Period). All participants will receive NT 201 treatment.

Enrollment: 78 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Score of ≥ 2 (at least 1 cm tremor amplitude) in at least two out of three maneuvers of test item 4 (upper limb tremor) confirmed by an independent TETRAS expert by means of video assessment.

Exclusion criteria

• History or presence of day-to-day fluctuations in ET which would jeopardize meaningful tremor assessment over time, e.g. severe tremor on one day and minimal or no tremor on another day.
• Other neurological signs, such as dystonia, ataxia, or parkinsonism, which in the judgment of the investigator could interfere with the ET diagnosis and/or assessment of ET in ULs.
• Tremor types other than ET

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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78 participants in 2 patient groups, including a placebo group

NT 201 (IncobotulinumtoxinA, Xeomin)

Experimental group

Description:

Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular injection of NT 201 (130-165 units) into muscles of the upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units) into muscles of the upper limbs.

Treatment:

Drug: NT 201

Placebo

Placebo Comparator group

Description:

Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units per arm) into muscles of the upper limbs.

Treatment:

Drug: NT 201

Drug: Placebo