Clinical trial • Phase IV • Endocrinology | Rare Disease

TIRATRICOL for Monocarboxylate transporter 8 (MCT8) deficiency

Phase IV trial of TIRATRICOL for Monocarboxylate transporter 8 (MCT8) deficiency. open-label, none/not specified-controlled. 13 participants.

Overview

open-label, none/not specified-controlled Phase IV trial across 2 sites in Netherlands, Czechia.

Recruits 13 paediatric patients.

Vulnerable Population: Participants are infants and young children (0–30 months), identified as a vulnerable population. Consent is required from parents or the legal guardian ("Signed and dated informed consent form from the parents or legal guardian"). Study documentation includes parent/legal representative informed consent forms and addenda (e.g. "L1_SIS and ICF_Parent_for publication", "L1_SIS_and ICF_Addendum_Legal Representative_for publication") and translations/extensions for multiple languages, indicating handling of consent by legal representatives; assent by the child is not applicable given participant age.

{"criterion_text":"- 1. Signed and dated informed consent form from the parents or legal guardian.\n- 2. Parents stated willingness to comply with all study procedures and availability for the duration of the study.\n- 3. The participant should be aged between 0 and 30 months on the day of inclusion.\n- 4. The participant should be male and have a pathogenic mutation in the MCT8 gene."}

{"criterion_text":"- 1.\tPrevious treatment with tiratricol.\n- 2.\tPrevious treatment with LT4 and/or PTU and/or other anti-thyroid medication for a period longer than three months. Patients previously treated with LT4 for a shorter period than 3 months may be included in the study (baseline visit) six weeks (or longer) after last dose of LT4 if two consecutive analyses show stable TFT*. Patients treated with PTU and/or other anti-thyroid medication for a shorter period than three months may be included in the study (baseline visit) six weeks (or longer) after last dose.\n- 3.\tMajor illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.\n- 4.\tKnown allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactase deficiency or malabsorption of glucose or galactose (the IMP contains lactose).\n- 5.\tTreatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit *Stable TFT (T3, T4, fT4), determined as a maximal variation of 20%, should be demonstrated at two separate occasions at least two weeks apart, measured on the same platform."}

{"endpoint_text":"- Part I of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.","definition_or_measurement_approach":"Measured using the Gross Motor Function Measure (GMFM-88) total score and Bayley Scales of Infant Development (BSID)-III Gross Motor Skill Domain at specified timepoints (week 96 for Part I; at 3 years and 4 years for Part II) compared to natural history scores from the Triac Trial I study."}

{"endpoint_text":"- Part I and II:GMFM-88 individual item score 10 (\"lifts head upright\") and item score 24 (\"sit on mat\") at week 96, at 3 and 4 years respectively, compared to baseline; GMFM Domain B (Sitting) - summary score of all items 18-37 at week 96, at 3 and 4 years respectively, compared to baseline; Motor milestone responder analysis of Section 2 of the Hammersmith Infant Neurological Examination (HINE) at week 96, at 3 and 4 years respectively.","definition_or_measurement_approach":"Specific GMFM-88 item scores (items 10 and 24), GMFM Domain B summary score (items 18–37), and motor milestone responder analysis from Section 2 of the HINE at the specified timepoints compared to baseline."}

Planned Sample Size: 13
Recruitment Window Months: 69
Consent Approach: Informed consent must be provided by the parents or legal guardian ("Signed and dated informed consent form from the parents or legal guardian"). Study includes parent/legal representative information and consent forms and addenda (documents listed: e.g. "L1_SIS and ICF_Parent_for publication", "L1_SIS_and ICF_Addendum_Legal Representative_for publication", plus language-specific versions for CZE/GER/POL and other supporting materials). Consent materials are provided in multiple language versions (English and language-specific extensions/translations listed: Czech, German, Polish and other localized documents).

{"country":"Netherlands","full_name":"Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)","duties_or_roles":"code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
{"country":"Sweden","full_name":"Viedoc Technologies AB","duties_or_roles":"code: 7","organisation_type":"Non-Pharmaceutical company"}
{"country":"Spain","full_name":"Premier Research Group S.L.","duties_or_roles":"codes: 1, 12, 5, 6, 8","organisation_type":"Pharmaceutical company"}
{"country":"France","full_name":"Cenexi","duties_or_roles":"code: 15 (Manufacture IMPs)","organisation_type":"Pharmaceutical company"}

Other published trials that may interest you.