Effects of Botulinum Toxin Injections in Patients With Hereditary Spastic Paraplegia

NCT02604186 | Completed Phase 2

• 1 other identifier: CAAE: 48177315.9.1001.5404
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary spastic paraplegias constitute a heterogeneous group of diseases with the common predominant feature of spasticity of the lower limbs. The clinical picture is composed of difficulty walking, exaggerated deep reflexes, pathological reflexes such as the Babinski sign, sphincter disturbances and various degrees of weakness as well as sensory disturbances. Spasticity is the symptom that provoques greater incapacity. Although there have been recent advances in the genetic and pathogenic characterization of SPG there is scarcity of therapeutic options. The Botulinum Toxin (BTx) is a well established treatment for movement disorders such as cervical dystonia, blepharospasm, and arm spastic following stroke. Therefore, the investigators propose the execution of a randomized, double-blind, placebo-controlled, crossover study to evaluate the efficacy of the treatment with Btx over SPG patient's gait. The primary outcome measure will be gait velocity with the 10 meter walking test 8 weeks after injection. Each participant will be submitted to one injection session of Btx and one of placebo (consisting of sterile sodium chloride), each one separated by a period of 6 months. The primary and secondary outcomes will be evaluated by a blind investigator 8 weeks after each injection session.

Conditions

Hereditary Spastic Paraplegia

Keywords

Spasticity; Botulinum Toxin; Hereditary Spastic Paraplegia

Outcome Measures

Primary Outcomes (1)

• Change from baseline in 10 meter maximum gate velocity

  The primary outcome measure will be change from baseline in maximum gait velocity. Each patient will be asked to walk a 10 meter distance barefooted 3 times, as fast as he can. The average velocity between the 3 trials will be used as the final measure. Assistive devices are permitted.

  8 weeks after injections

Secondary Outcomes (7)

• Change from baseline in Spastic Paraplegia Rating Scale (SPRS),

  8 weeks after each procedure

• Change from baseline in Ashworth spasticity scale of adductors and triceps surae muscles

  8 weeks after each procedure

• Change from baseline in muscle strengh (Medical Research Council scale) concerning adductors and triceps surae muscles.

  8 weeks after each procedure

• Change from baseline in visual analogic scale of pain

  8 weeks after each procedure

• Change from baseline in brief pain inventory scale

  8 weeks after each procedure

Study Arms (2)

Botulinum Toxin Injections

EXPERIMENTAL

Botulinum Toxin injections at adductors and triceps surae

Other: Botulinum Toxin Injections

Placebo Injections

PLACEBO COMPARATOR

Placebo injections at adductors and triceps surae

Other: Placebo Injections

Interventions

Botulinum Toxin Injections OTHER

Each patient in the treatment group will receive 400 units of botulinum toxin. 100 units will be injected at adductors at each leg and 100 units will be applied in each triceps surae bilaterally.

Botulinum Toxin Injections

Placebo Injections OTHER

Each patient in the placebo group will receive sterile sodium chloride injections at adductors and triceps surae muscles bilaterally.

Placebo Injections

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age between 18 and 80 years
• Clinical diagnosis of Hereditary Spastic Paraplegia
• Ability to walk at least 10 meters: Assistive devices are permitted

You may not qualify if:

• Wheelchair bound patients
• Additional neurological symptoms that may significantly impact gait such as ataxia, polyneuropathy or dementia.
• Fixed tendon contractures
• Antecedents of allergy or adverse reaction to botulinum toxin
• Pregnancy or breastfeeding condition
• Mental retardation
• Dementia

Sponsors & Collaborators

• University of Campinas, Brazil: lead
• Conselho Nacional de Desenvolvimento Científico e Tecnológico: collaborator
• Cristália Produtos Químicos Farmacêuticos Ltda.: collaborator

Study Sites (1)

Univeristy of Campinas Hospital

Campinas, São Paulo, 13083-888, Brazil

Location

Related Publications (1)

• de Niet M, de Bot ST, van de Warrenburg BP, Weerdesteyn V, Geurts AC. Functional effects of botulinum toxin type-A treatment and subsequent stretching of spastic calf muscles: a study in patients with hereditary spastic paraplegia. J Rehabil Med. 2015 Feb;47(2):147-53. doi: 10.2340/16501977-1909.

  PMID: 25325386 BACKGROUND

MeSH Terms

Conditions

Spastic Paraplegia, Hereditary; Muscle Spasticity

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Muscular Diseases; Musculoskeletal Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Marcondes c França Júnior, M.D, PhD

  University of Campinas, Brazil

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, Phd

Study Record Dates

• First Submitted: November 9, 2015
• First Posted: November 13, 2015
• Study Start: March 9, 2016
• Primary Completion: March 15, 2017
• Study Completion: March 15, 2017
• Last Updated: November 7, 2017

Record last verified: 2017-11

Data Sharing

• IPD Sharing: Will not share

Locations

BR (1)