NCT02484703

A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age

Terminated Phase 2 Last updated 6 July 2017

What this trial tests

Phase 2 trial testing Placebo in Down Syndrome in 45 participants. Terminated before completion.

Timeline

28 October 2015

Primary endpoint
3 August 2016

3 August 2016

Quick facts

Lead sponsor	Hoffmann-La Roche
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	45
Start date	28 October 2015
Primary completion	3 August 2016
Estimated completion	3 August 2016
Sites	9 locations across United States

Drugs / interventions tested

• Placebo
• RO5186582 — full drug profile →

Conditions studied

• Down Syndrome — all drugs for Down Syndrome →

Sponsor

Hoffmann-La Roche — full company profile →

Who can join

Adults 6 to 11, any sex, with Down Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments in a 1:1:1:1 ratio, with 9 children per treatment arm. The target ratio between 6-8 years and 9-11 years age groups is approximately 1:1 in each treatment arm, with a minimum of 3 children per age group in each treatment arm.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Down syndrome.
   Antonarakis SE, Skotko BG, Rafii MS, Strydom A, et al · · 2020 · cited 630× · PMID 32029743 · DOI 10.1038/s41572-019-0143-7
2. The GABAergic Hypothesis for Cognitive Disabilities in Down Syndrome.
   Contestabile A, Magara S, Cancedda L. · · 2017 · cited 95× · PMID 28326014 · DOI 10.3389/fncel.2017.00054
3. Accelerated bio-cognitive aging in Down syndrome: State of the art and possible deceleration strategies.
   Franceschi C, Garagnani P, Gensous N, Bacalini MG, et al · · 2019 · cited 51× · PMID 30768754 · DOI 10.1111/acel.12903
4. Aging with Down Syndrome-Where Are We Now and Where Are We Going?
   Alldred MJ, Martini AC, Patterson D, Hendrix J, et al · · 2021 · cited 29× · PMID 34682809 · DOI 10.3390/jcm10204687
5. Long-lasting correction of in vivo LTP and cognitive deficits of mice modelling Down syndrome with an α5-selective GABA_A inverse agonist.
   Duchon A, Gruart A, Albac C, Delatour B, et al · · 2020 · cited 27× · PMID 31652355 · DOI 10.1111/bph.14903
6. Challenges and Opportunities for Translation of Therapies to Improve Cognition in Down Syndrome.
   Lee SE, Duran-Martinez M, Khantsis S, Bianchi DW, et al · · 2020 · cited 18× · PMID 31706840 · DOI 10.1016/j.molmed.2019.10.001
7. Editorial: Intellectual Disabilities in Down Syndrome from Birth and Throughout Life: Assessment and Treatment.
   Potier MC, Reeves RH. · · 2016 · cited 13× · PMID 27378871 · DOI 10.3389/fnbeh.2016.00120
8. Context Fear Conditioning in Down Syndrome Mouse Models: Effects of Trisomic Gene Content, Age, Sex and Genetic Background.
   Ahmed MM, Block A, Busquet N, Gardiner KJ. · · 2021 · cited 5× · PMID 34680922 · DOI 10.3390/genes12101528