Huntington's Disease Clinical Trial
Official title:
A Double Blind, Randomized, Cross Over, Placebo Controlled Phase 2 Clinical Trial to Asses Neuroprotection by Cannabinoids in Huntington's Disease
Huntington's disease (HD) is a progressive neurodegenerative disorder, related to an
abnormal expansion of CAG triplets in the huntingtin gene, characterized by motor, cognitive
and behavioral abnormalities, without known effective symptomatic treatment and without
known disease slowing strategy. The most severe neuropathological lesions observed in HD
take place in the striatum, one brain area important in motor control and rich in
cannabinoid receptors (CBR). CBR are subdivided in two classes: CB1R are located in neurons
and play a role in neuronal function; CB2R in brain are located mostly in microglia and
modulate neuroinflammation.
CBR disappear early in the course of HD, before there is a massive drop out of cells in the
striatum. Cannabinoid transmission is also an early event in brains of animal models of HD.
In R6/2 mice, which carry large CAG expansions and develop an early and severe HD phenotype
the suppression of the CB1R gene further accelerate the development of a severe clinical
syndrome and the characteristic brain inclusions and abnormalities of synaptic density. R6/2
treated mice treated with cannabinoids improve their clinical phenotype, their brain
lesions, the synaptic density and the levels of BNDF, a neurotrophic factor which enhances
survival and resistance of striatal neurons.
Preliminary studies of cannabinoids in patients with HD have shown that these compounds are
safe in these patients. Those studies, however, did not show efficacy because 1) they were
underpowered from the statistical point of view, 2) were performed with isolated pure
cannabinoids, instead of the more physiological stimulation with a mixture of compounds, and
3) they did use insensitive clinical parameters instead of sensitive end points, such as
pathogenically important biomarkers.
The investigators propose a phase II trial with combination of cannabinoids with evaluation
of safety, by the profile of adverse events, and efficacy, according to changes of important
biomarkers
n/a
Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04120493 -
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
|
Phase 1/Phase 2 | |
Completed |
NCT02956148 -
Follow-up Measurement of Brain PDE10A Enzyme Levels in Huntington´s Disease Gene Expansion Carriers
|
Early Phase 1 | |
Terminated |
NCT02494778 -
A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease
|
Phase 2 | |
Completed |
NCT02208934 -
Study To Assess the Safety and Tolerability of Single Ascending Oral Doses of PBF-999 in Healthy Young Male Volunteers
|
Phase 1 | |
Completed |
NCT02216474 -
Brain Stimulation in Movement Disorders
|
N/A | |
Completed |
NCT02197130 -
Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
|
Phase 2 | |
Completed |
NCT01806896 -
Study Evaluating The Safety, Tolerability And Brain Function Of 2 Doses Of PF-0254920 In Subjects With Early Huntington's Disease
|
Phase 2 | |
Terminated |
NCT00712426 -
Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)
|
Phase 3 | |
Completed |
NCT00670709 -
Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease
|
||
Completed |
NCT00029874 -
Minocycline in Patients With Huntington's Disease
|
Phase 1/Phase 2 | |
Terminated |
NCT02231580 -
Study Exploring Safety, Pharmacokinetic and Pharmacodynamic of BN82451 in Male Huntington's Disease Patients
|
Phase 2 | |
Completed |
NCT02215616 -
A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod
|
Phase 2 | |
Not yet recruiting |
NCT02551705 -
Functional Imaging of Social Cognition in Premanifest Huntington's Disease
|
N/A | |
Active, not recruiting |
NCT02101957 -
Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)
|
Phase 2/Phase 3 | |
Completed |
NCT00990613 -
A Study Evaluating The Absorption Of Dimebon Into The Body From A Dimebon Solution Applied To The Skin
|
Phase 1 | |
Completed |
NCT00975481 -
A Study To Evaluate The Abuse Potential Of Single Oral Doses Of Dimebon (Latrepirdine) In Healthy Recreational Polydrug Users
|
Phase 1 | |
Completed |
NCT01521832 -
Escalating Dose Study in Healthy Volunteers With SEN0014196
|
Phase 1 | |
Completed |
NCT00387270 -
Safety Study of the Novel Drug Dimebon to Treat Patients With Huntington's Disease
|
Phase 1/Phase 2 | |
Completed |
NCT00095355 -
Effects of Lithium and Divalproex`on Brain-Derived Neurotrophic Factor in Huntington's Disease
|
Phase 2 | |
Completed |
NCT00026988 -
Creatine Therapy for Huntington's Disease
|
Phase 1/Phase 2 |