A Comparative Safety, Tolerability and PK Study of Branaplam (QSC205070)
Research type
Research Study
Full title
Randomized, single-dose, open-label, two-part, two-period, cross-over study to compare the pharmacokinetics, safety and tolerability of the pediatric and adult formulation of branaplam and to investigate the adult formulation in fed and fasted state in healthy participants
IRAS ID
1004936
Contact name
Klaas Romanino
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2021-000298-10
ISRCTN Number
ISRCTN77772103
Research summary
The Sponsor is developing a new recipe of the test medicine, branaplam, for the potential treatment of Huntington’s disease, a genetic condition that affects the nerves and how people move.
The test medicine has been previously developed for a disease that affects nerves in infants and children called spinal muscular atrophy and has been used in previous clinical trials in a recipe that is suitable for children. The new recipe has been developed to be suitable for adults.
This is a two-part, healthy volunteer study. Part 1 consists of two periods and will compare the levels of test medicine in the blood when given as the new adult recipe or the existing pediatric recipe and Part 2 consists of two periods and will look at the effect of food on how the new adult recipe of the test medicine is taken up by the body. How safe and well tolerated the test medicine is will also be assessed in both study parts.
This study will take place at one non-NHS site, enrolling up to 32 male and non-pregnant, non-lactating female volunteers aged between 18 and 60.
In Part 1, 16 volunteers will receive an oral dose of the pediatric recipe in one period and new adult recipe in another period, both in the fasted state.
In Part 2, 16 volunteers will receive an oral dose of the adult recipe of the test medicine in the fed state in one period and in the fasted state in another period.
Volunteers will be discharged from clinical unit on Day 5 and will return to the clinic for 3 return visits on Day 8, Day 11 and Day 15 of each period . Volunteers will also receive a follow up phone call 30 days post-final dose.
Volunteer’s blood and urine will be taken throughout the study for analysis of the test medicine and for their safety.
In both study parts, volunteers are expected to be involved in this study for approximately 13 weeks from screening to the follow up call.
REC name
London - London Bridge Research Ethics Committee
REC reference
22/LO/0106
Date of REC Opinion
11 Apr 2022
REC opinion
Further Information Favourable Opinion