Study on the effect of TM5614 on congenital FGF23-related hypophosphatemic rickets / osteomalacia

JRCT ID: jRCTs031210704

Registered date:28/03/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: FGF23-related hypophosphatemic rickets / osteomalacia
• Date of first enrollment: 28/03/2022
• Target sample size: 5
• Study type: Interventional
• Intervention(s): TM5614 tablets will be administered to patients after breakfast for a total of 28 days, starting with 2 tablets (120 mg) once a day. However, the dose on the first day of administration does not have to be taken after breakfast.

Outcome(s)

• Primary Outcome: The change in serum phosphorus concentration from the pre-administration level to 14 days and 28 days after the start of treatment.

Secondary Outcome

serum intact FGF2, serum C-terminal FGF2, corrected Ca, bone type ALP, serum intact PTH, serum
1-25OH2D, urinal Ca per gCre, urinal Pi per gCre TmP per GFR

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.patients over the age of 18 who have given written consent to participate in this study voluntarily (If the patient is 20 years old or younger at the time of consent, written consent must also be obtained from a legal representative.
2.patients diagnosed with congenital FGF23-related hypophosphatemic rickets/osteomalacia
3.patients with a serum phosphorus level of less than 2.5 mg/dL
4.patients with serum intact FGF23 levels greater than or equal to 30 pg/mL
5.patients with TmP/GFR less than 2.5 mg/dL
6.patients with a corrected serum Ca level less than 10.8 mg/dL
7.patients with no history of fracture or pseudofracture (no corrective surgery), and with height greater than -2 SD (male 158 cm, female 148 cm)

Exclude criteria

1.patients with congenital FGF23-related hypophosphatemic rickets/osteomalacia who are using or have used the fully human monoclonal antibody burosumab
2.patients with active infection or chronic inflammatory disease
3.patients with uncontrolled hypertension
4.patients with uncontrolled diabetes mellitus.
5.patients with kidney disease on dialysis treatment
6.patients with a history of or complications of malignant tumors (excluding those with no recurrence or new onset for at least 5 years after treatment)
7.patients with complications of cirrhosis (Child-Pugh score class B and C)
8.patients with the following abnormalities in liver function
patients whose AST and ALT exceed 2.5 times the institutional standard values.
patients whose serum bilirubin exceeds 2.5 times the institutional standard
9.patients with an eGFR of 30 mL/min/1.73 m2 or more but less than 60 mL/min/1.73 m2
10.patients with the following cardiovascular diseases
patients with NYHA class II-IV congestive heart failure
patients with a history of myocardial infarction in the 6 months prior to enrollment
patients with symptomatic arrhythmia that requires treatment
11.patients with bleeding tendency: specifically, bleeding time, 5 minutes or more; PT-INR, 3.0 or more; activated partial thromboplastin time, 40 seconds or more
12.patients who are receiving prohibited concomitant medications such as anticoagulants and have difficulty in discontinuing such medications
13.patients who do not agree to use an appropriate method of contraception while participating in the study
14.patients who are participating in other clinical studies
15.patients who are considered ineligible by the principal investigator or subinvestigator for other reasons

Related Information

• Primary Sponsor: ASOU Yoshinori
• Source(s) of Monetary Support: Renascience Inc.