None listed
Conditions
Brief summary
The aim of this study is to evaluate the efficacy and long-term safety of modified-release calcium formate to reduce elevated plasma homocysteine (Hcy) levels in adults patients with homocystinuria. The hypothesis, seen in preclinical models, is that modified-release formate can rapidly (within weeks), sustainably, and safely lower plasma homocysteine levels. This outcome would improve long-term outcomes in patients with homocystinuria
Interventions
Patients eligible to participate will receive 2.6 g calcium formate (corresponding to 1.8 g formate) per day for the first two weeks. Daily dosing will be divided into 1.3 g calcium formate in the morning and 1.3 g in the evening. At week 3, dosing may be up-titrated (increments of 300 mg formate and not exceeding 2.4 g formate/day). The decision to up titrate will be dependent on plasma homocysteine levels measured at the day 14 visit. The total duration of the intervention is 52 weeks.
The i
Patients eligible to participate will receive 2.6 g calcium formate (corresponding to 1.8 g formate) per day for the first two weeks. Daily dosing will be divided into 1.3 g calcium formate in the morning and 1.3 g in the evening. At week 3, dosing may be up-titrated (increments of 300 mg formate and not exceeding 2.4 g formate/day). The decision to up titrate will be dependent on plasma homocysteine levels measured at the day 14 visit. The total duration of the intervention is 52 weeks. The investigational treatment will be administered orally as 434 mg calcium formate (300 mg formate) per tablet evenly divided between the morning and the evening up to the required dose. Tablets will be returned after each clinic visit and compared to the expected number of tablets returned to calculate compliance.
Sponsors
SubRed Therapeutics Pty Ltd
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female aged 18- 65 years, inclusive. 2. Homocystinuria as confirmed by prior diagnosis. 3. Plasma Hcy more than 50 µM (rounded to the nearest whole number) and documentation of previous Hcy more than 70 µM. 4. Plasma methionine levels less than 800 µmol/L 5. Willing to maintain a stable diet (plus/minus 20% variability of daily protein intake) with no significant modifications while on study and complete a daily diet diary as requested.
Exclusion criteria
1. Changes to current medications or commencement of any new medications, including prescription drugs (except hormonal contraception), within 28 days prior to the Screening Visit and during the study treatment phase. 2. Currently taking calcium supplements and where these supplements cannot be replaced by calcium formate 3. Known history of hypercalcemia 4. Known history of any severe cardiovascular disorder not including previous thromboembolic events 5. History of hypersensitivity to formate supplements that, in the judgment of the investigator, puts the participant at unacceptable risk for adverse events (AEs) 6. Known intolerance to any of the investigational product excipients, particularly lactose
Outcome results
None listed