Deutetrabenazine for Cerebral Palsy

2
Effectiveness
3
Safety
Teva Investigational Site 50478, Stavropol, Russian Federation
Cerebral Palsy+2 More
Deutetrabenazine - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Cerebral Palsy

Study Summary

A Study of TEV-50717 (Deutetrabenazine) for the Treatment of Dyskinesia in Cerebral Palsy in Children and Adolescents

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Eligible Conditions

  • Cerebral Palsy
  • Asterixis
  • Dyskinesias
  • Cerebral Palsy, Dyskinetic

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether Deutetrabenazine will improve 1 primary outcome and 22 secondary outcomes in patients with Cerebral Palsy. Measurement will happen over the course of Baseline and Week 15.

Baseline and Week 15
Change in PEDI-CAT (ADL, parent/caregiver completed, content balanced version)
Change in the cerebral palsy (CP) module of the PedsQL (QoL, patient/caregiver)
Baseline to Week 15
CGI-S response
Change in MD-CRS Global Index calculated from MD-CRS parts I and II total scores
Change in UHDRS-TMC (Centrally rated)
Change in UHDRS-TMC (Physician rated)
Change in UHDRS-TMD (Centrally rated)
Change in UHDRS-TMD (Physician rated)
Change in UHDRS-TMS (Physician rated)
Change in the MD-CRS part I
Change in the MD-CRS part II
Screening to Week 17
Percentage of Participants with Adverse Events
Week 16
Change in CGI-I response
Week 15
CGI-I Scale
CaGI-I Scale
Week 15
CGI-I response
CaGI-I response
Change in CGI-S Scale (global, physician rated)
Change in PGI-I Scale (global, patient/caregiver)
PGI-I response
Week 16
Change in CGI-S response
Change in CaGI-I response
Change is PGI-I response

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Placebo
Deutetrabenazine
Placebo group

This trial requires 65 total participants across 2 different treatment groups

This trial involves 2 different treatments. Deutetrabenazine is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Deutetrabenazine
Drug
administered as oral tablets at a starting dose of 6 mg once daily
Placebo
Drug
Matching placebo

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: week 3 to week 16
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly week 3 to week 16 for reporting.

Closest Location

Teva Investigational Site 14290 - New Orleans, LA

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
and for 28 days after the last dose of study drug For the duration of the study and for 28 days after the last dose of study drug, postmenarchal females aged ≥12 years whose male partners are potentially fertile must use highly effective birth control methods. show original
At baseline, the participant weighs at least 26 pounds (12 kg). show original
The participant has had symptoms of cerebral palsy since infancy. show original
The participant is aged between six and eighteen years at the baseline. show original
The majority of participants in the study exhibit choreiform movements, as assessed by the EAB. show original
The participant has a diagnosis of DCP. show original
The participant can swallow the study medication whole. show original
Please contact the investigator for more information if you are interested in this study show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is cerebral palsy?

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The International Classification of Functioning, Disability and Health (ICF) v.2.3.1 provides an international framework for communication and classification between health professionals and those with cerebral palsy, based on the ICF Core Sets. These concepts are useful in developing strategies for health professionals and those with cerebral palsy to develop services and facilitate communication and support. In developing evidence-informed resource allocation strategies, evidence is best incorporated into the ICF with particular focus on its relevance to people with cerebral palsy.

Unverified Answer

What causes cerebral palsy?

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There are several causes of cerebral palsy, including a complex mix of inherited and environmental causes. Brain injury or an abnormality of brain growth may be present from conception, but other causes of CP include infections during gestation, birth trauma such as cephalohematoma, and hypoxic brain injury, in particular, during the first month after birth. CP can have multiple types of symptoms, including low muscle tone, motor delay, ataxia, and intellectual disability.

Unverified Answer

What are the signs of cerebral palsy?

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Children and young adults with CP experience signs that may be recognized by a neurologist or physical therapist. CP is associated with abnormal posture and limb development, muscle strength, spasticity, and walking function. CP is associated with the ability to walk independently into adulthood and acquire employment. CP is associated with long-term quality of life and quality of life satisfaction for people, their families, and care staff. CP affects about 40 million people worldwide. It is the most common cause of disability in children under the age of 18 years old. Most cases of CP result from spontaneous fetal or fetal-related complications in the pre- or postnatal period and a subset of cases may develop after birth.

Unverified Answer

What are common treatments for cerebral palsy?

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This article presents a thorough and compelling history of medical treatments for CP children. The current knowledge about medical regimens, in the form of current and historical treatment practices, offers parents with CP children a strong rationale for establishing a comprehensive medical team at home to manage their child's medical issues with CP. Copyright © 2015 John Wiley & Sons, Ltd.

Unverified Answer

How many people get cerebral palsy a year in the United States?

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Approximately 40 million children ni the United States are currently diagnosed with CP. These children constitute 16.7% of children in the United States. Patients with CP are more likely to develop language impairments and cognitive difficulties in the second decade of life.

Unverified Answer

Can cerebral palsy be cured?

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A cure is hardly conceivable in cerebral palsy. However, the possibility is much bigger in preventing deterioration of spasticity than of spasticity itself. The most common causes of brain deterioration are infections and head injury. The aim of rehabilitation of cerebral palsy is to reduce spasticity, not to cure it.

Unverified Answer

Have there been other clinical trials involving deutetrabenazine?

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There is a high level of anecdotal evidence of deutetrabenazine use in CP. There are also several anecdotal reports of deutetrabenazine use in AP, or AFA, and ASD. We believe that there is great potential for further clinical trials on deutetrabenazine.

Unverified Answer

Is deutetrabenazine typically used in combination with any other treatments?

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DTG use is prevalent and associated with substantial clinical improvement. DTG is frequently used in combination with other treatment modalities. The combination of DTG and anticonvulsants improves response rates with an overall frequency of epilepsy improvement of about 75%. This supports earlier reports that antiepileptic drugs potentiated clinical effects of DTG. The effect of DTG on seizure activity can be enhanced further by the combination with a ketogenic diet with the addition of a second antiepileptic medication, such as vigabatrin, and with the inclusion of vitamin B₁ in DTG treatment. Further research is needed on optimal use of DTG in this setting.

Unverified Answer

What is deutetrabenazine?

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This was a pilot, small trial. Treatment with deutetrabenazine was effective when compared with placebo; however this effect was not maintained at 12 months post-randomization.

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What is the primary cause of cerebral palsy?

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The most important factor in the pathogenesis of CP is intrauterine insults, followed by insults to the developing myelin covering of the spinal cord, and finally by a secondary, chronic encephalopathy

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Does cerebral palsy run in families?

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The prevalence of CP in siblings within a family is higher than the prevalence of CP in the general population. These familial transmissions suggest further genetic study into the cause of CP, and may offer clues on the mechanisms of brain development and injury.

Unverified Answer

How serious can cerebral palsy be?

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The medical experts at the hospitals agree that cerebral palsy is very serious. It is the medical professionals who deal with these children who are on shift as day-to-day doctors and nursing staff are available who have to make life-or-death decisions as the children deteriorate. The medical professionals do not look at cerebral palsy, but as an 'orphan' condition, not a disease as such, and they may make a biased decision to'rescind' the care of a patient with cerebral palsy. A major problem is that the medical community in India is reluctant to provide proper care or treatment because of the stigma and the long-term economic cost.

Unverified Answer
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