A translation into French language can be found here Safe-KIDDS (French).
The Safe-KIDDS trial is the second clinical trial of odiparcil in MPS VI patients. The first trial, iMProveS, in adolescent and adult MPS VI patients has been completed and headline results reported in December 2019. The treatment was well tolerated by the patients whether they were taking enzyme replacement therapy (ERT) at the time of treatment with odiparcil or not. We observed a clear correlation between the dose of odiparcil and the level of glycosaminoglycans (GAGs) cleared in the urine as well as first signs of improvement in several organs such as the heart, lung and the eye. For more information on the trial results please click here .
The primary objective of this phase Ib / II Safe-KIDDS trial is to show that odiparcil can be safely administered to children with MPS VI who are over the age of five years and to determine the right dose for treatment. We are also collecting pharmacodynamics (PD) and pharmacokinetics (PK) data and are looking to observe signs of efficacy by examining several biomarkers, growth, mobility, pulmonary, cardiac, visual and auditory function and quality of life. Odiparcil will be given to the children as an add-on to ERT.
The trial consists of three periods.
For Part 1 of the trial 9 to 12 children between the age of 5 and 15 years will be enrolled. This first part is an open label phase which means that all children will receive three ascending doses of odiparcil. The duration of phase 1 duration is three weeks (one week per dose).
Part 2 is a double-blind, placebo-controlled, 3-arm phase with two doses of odiparcil which were determined by data collected in Part 1. 24 patients will be enrolled and randomised 1:1:1 in the odiparcil dose 1/odiparcil dose 2/placebo groups. The duration of Part 2 is 1 year.
Part 3 is a continuation of part 2 for 6-12 months. Patients from part 2 will either pursue their treatment if they were treated with odiparcil in part 2 or will be randomised in one of the 2 odiparcil-receiving groups (dose 1 or dose 2) if they received placebo in part 2. There will be no placebo group in part 3.
Eventually children who have completed Part 3 may join a potential extension phase during which all children will receive odiparcil.
Patients eligible to enroll must be (at a minimum)
- between 5 and 15 years old
- have a confirmed MPS VI diagnosis
- have been taking ERT (galsulfase) for at least 12 months
- female patients with childbearing potential agreeing to use a highly effective method of birth control during the study
Safe-KIDDS is a multi-national trial with trial centers in Birmingham (UK) with Dr Julian Raiman (Julian.Raiman@nhs.net), in Lyon (France) with Dr Nathalie Guffon (email@example.com), in Porto (Portugal) with Dr Elisa Leao Teles (firstname.lastname@example.org), in Barcelona (Spain) with Dr Mireia del Toro (email@example.com) and in Rotterdam (The Netherlands) with Dr Esmee Oussoren (firstname.lastname@example.org). If you would like to participate in this trial please contact one of the trial investigators listed above or ask your doctor to contact one of them.
Patient travel logistics are supported by Rare Disease Research Partners.
Patient associations can be found in almost all countries where people are effected by MPS. A reference to national MPS societies can be found on the web site of the MPS Society (UK)