ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 10 for:    "infantile-onset ascending hereditary spastic paralysis"
Previous Study | Return to List | Next Study

Effects of Botulinum Toxin Injections in Patients With Hereditary Spastic Paraplegia (SPASTOX)

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. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02604186
Recruitment Status : Completed
First Posted : November 13, 2015
Last Update Posted : November 7, 2017
Sponsor:
Collaborators:
Conselho Nacional de Desenvolvimento Científico e Tecnológico
Cristália Produtos Químicos Farmacêuticos Ltda.
Information provided by (Responsible Party):
Marcondes Cavalcante França Júnior, University of Campinas, Brazil

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.


Condition or disease Intervention/treatment Phase
Hereditary Spastic Paraplegia Other: Botulinum Toxin Injections Other: Placebo Injections Phase 2 Phase 3

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 55 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Botulinum Toxin in Patients With Hereditary Spastic Paraplegia: a Randomized, Double-blind, Placebo-controlled, Crossover Study
Actual Study Start Date : March 9, 2016
Actual Primary Completion Date : March 15, 2017
Actual Study Completion Date : March 15, 2017


Arm Intervention/treatment
Experimental: Botulinum Toxin Injections
Botulinum Toxin injections at adductors and triceps surae
Other: Botulinum Toxin Injections
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.

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




Primary Outcome Measures :
  1. Change from baseline in 10 meter maximum gate velocity [ Time Frame: 8 weeks after injections ]
    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.


Secondary Outcome Measures :
  1. Change from baseline in Spastic Paraplegia Rating Scale (SPRS), [ Time Frame: 8 weeks after each procedure ]
    The same neurologist will examine the patient to evaluate change at the SPRS scale

  2. Change from baseline in Ashworth spasticity scale of adductors and triceps surae muscles [ Time Frame: 8 weeks after each procedure ]
    The same neurologist will examine the patient to evaluate change from baseline

  3. Change from baseline in muscle strengh (Medical Research Council scale) concerning adductors and triceps surae muscles. [ Time Frame: 8 weeks after each procedure ]
    The same neurologist will examine the patient to evaluate change from baseline

  4. Change from baseline in visual analogic scale of pain [ Time Frame: 8 weeks after each procedure ]
    this scale will be applied by a neurologic physiotherapist

  5. Change from baseline in brief pain inventory scale [ Time Frame: 8 weeks after each procedure ]
    this scale will be applied by a neurologic physiotherapist

  6. Change from baseline in modified fatigue impact scale [ Time Frame: 8 weeks after each procedure ]
    this scale will be applied by a neurologic physiotherapist

  7. Change from baseline in 10 meter comfortable walking velocity [ Time Frame: 8 weeks after each procedure ]
    this scale will be applied by a neurologic physiotherapist



Information from the National Library of Medicine

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 to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

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): NCT02604186


Locations
Brazil
Univeristy of Campinas Hospital
Campinas, SP, Brazil, 13083-888
Sponsors and Collaborators
University of Campinas, Brazil
Conselho Nacional de Desenvolvimento Científico e Tecnológico
Cristália Produtos Químicos Farmacêuticos Ltda.
Investigators
Principal Investigator: Marcondes c França Júnior, M.D, PhD University of Campinas, Brazil

Publications:
Responsible Party: Marcondes Cavalcante França Júnior, MD, Phd, University of Campinas, Brazil
ClinicalTrials.gov Identifier: NCT02604186     History of Changes
Other Study ID Numbers: CAAE: 48177315.9.1001.5404
First Posted: November 13, 2015    Key Record Dates
Last Update Posted: November 7, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Marcondes Cavalcante França Júnior, University of Campinas, Brazil:
Spasticity
Botulinum Toxin
Hereditary Spastic Paraplegia

Additional relevant MeSH terms:
Muscle Spasticity
Paraplegia
Spastic Paraplegia, Hereditary
Muscular Diseases
Musculoskeletal Diseases
Muscle Hypertonia
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Paralysis
Hereditary Sensory and Motor Neuropathy
Nervous System Malformations
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Botulinum Toxins
Acetylcholine Release Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs