fb

Why the first drug trial for dyskinetic cerebral palsy failed, and what comes next

10/13/2026
Medically reviewed by: Kelsey Pabst, Registered Nurse
Why the first drug trial for dyskinetic cerebral palsy failed, and what comes next

On December 22, 2025, Neurocrine Biosciences announced that its Phase 3 trial of valbenazine in dyskinetic cerebral palsy did not meet its main goal. The drug, sold as Ingrezza, did no better than a placebo (a capsule with no medicine in it) at easing chorea, the quick, jerky movements a person cannot control.

The trial, called KINECT-DCP, enrolled 86 people aged 6 to 70. Neurocrine described it as the largest double-blind, placebo-controlled trial ever completed in dyskinetic cerebral palsy, the form of CP marked by involuntary movement. A win would have made valbenazine the first medicine approved for these movements in CP.

As of October 5, 2026, the full numbers are still not public. No results are posted on ClinicalTrials.gov, and the study does not appear in the final program of the American Academy for Cerebral Palsy and Developmental Medicine (AACPDM) meeting held in Philadelphia, September 27 to 30, 2026.

“These results are disappointing, as there are no approved treatments for people living with dyskinetic cerebral palsy.”
— Sanjay Keswani, Chief Medical Officer, Neurocrine Biosciences (press release, Dec 22, 2025)

What the trial tested

Valbenazine is a VMAT2 inhibitor. VMAT2 is a protein nerve cells use to store and release dopamine and similar chemical messengers. Participants took one capsule a day, by mouth or through a feeding tube, either valbenazine or placebo, for 14 weeks. Everyone had to have choreiform (chorea-like) movements from CP.

The main measure was the Total Maximal Chorea score, a scale borrowed from Huntington’s disease, averaged over weeks 12 and 14. Secondary measures included dystonia scores, caregiver and patient ratings, personal goals and pain. Neurocrine said side effects matched the drug’s known safety profile.

Why a negative result matters

Valbenazine is FDA approved for adults with tardive dyskinesia (2017) and Huntington’s disease chorea (2023). Its label states that safety and effectiveness have not been established in children. KINECT-DCP now gives families a tested answer: over 14 weeks, it did not beat placebo for chorea in CP.

It is the second drug of this type to fall short. In a Teva trial of deutetrabenazine in 63 children and teens aged 6 to 18 (results posted September 2023), the movement score fell 1.5 points on drug and 0.4 on placebo, on a scale from 0 to 28. That gap was not statistically significant.

Options for dyskinetic CP today

The AACPDM care pathway (April 2024) covers dystonia, the slow twisting movements and stuck postures, and states that treatment aimed at rapid movements such as chorea is outside its scope. For dystonia that affects the whole body and interferes with daily life, it suggests a trial of oral baclofen first, with trihexyphenidyl or benzodiazepines as alternatives, and clonidine or gabapentin when pain or sleep is affected. When medicines fail, intrathecal baclofen or deep brain stimulation (DBS) can be considered. Dystonia in one area may be treated with botulinum toxin injections. The authors note the evidence for all of these is limited.

What comes next

Neurocrine has said it will present the full results at a scientific meeting. Those numbers could show whether any group responded. At UCSF, a study of cerebellar DBS (wires placed in the cerebellum, at the back of the brain) is enrolling 10 people aged 7 to 25 with dyskinetic CP, comparing real and sham stimulation for 3 months each. Its main results are expected in 2029.

Sources

Related reading on this site: deep brain stimulation for dyskinetic cerebral palsy and medications for cerebral palsy.

These results are disappointing, as there are no approved treatments for people living with dyskinetic cerebral palsy. — Sanjay Keswani, Chief Medical Officer, Neurocrine Biosciences (press release, Dec 22, 2025)
Previous post
What was on the program at AACPDM's 80th Annual Meeting in P...
Related Articles

More news & updates

View all resources →