Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia

Status Completed

Clinical Trial Summary

The goal of this clinical trial is to compare the effectiveness of two doses of ilofotase alfa, an enzyme replacement treatment, in patients with hypophosphatasia (HPP). The main question it aims to answer is if the harmful accumulating levels of extracellular inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP) can be reduced with ilofotase alfa. Researchers will compare the two doses of ilofotase alfa to see if treatment effects differ between the doses.

Clinical Trial Description

Recombinant human alkaline phosphatase (ilofotase alfa) is a full-length human chimeric alkaline phosphatase (ALP) that could benefit patients with hypophosphatasia (HPP), which is characterized by low activity of tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). This is a pilot trial for a potential future trial aimed at identifying whether treatment with ilofotase alfa can normalize circulating levels of PPi, PLP and other biochemical markers of TNSALP deficiency along with the safety/tolerability of different doses of ilofotase alfa. The trial is designed as a single-center, open-label, randomized, parallel group clinical trial in adult patients with HPP. Two different dose levels (0.8 mg/kg and 3.2 mg/kg) of ilofotase alfa will be assessed. Participants will receive a single dose of ilofotase alfa, administered as a 1-hour intravenous infusion on Study Day 1. Participants will stay at the research center for a total of 12 days; from 2 days before study drug administration (run-in) to 10 days after treatment. An additional follow-up assessment is scheduled 14 days after administration of ilofotase alfa. Blood and urine samples will be taken daily for drug concentration and laboratory measurements assessing safety and effectiveness of treatment. In addition physical examinations will be performed on Day 1 and as needed afterwards. ;

Study Design

Related Conditions & MeSH terms

Hypophosphatasia

Clinical Trial Details

NCT number	NCT05890794
Study type	Interventional
Source	AM-Pharma
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	May 15, 2023
Completion date	July 12, 2023

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT03418389` - Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
Recruiting	`NCT02237625` - Natural History Study of Patients With Hypophosphatasia (HPP)
Completed	`NCT02291497` - Burden of Disease in Hypophosphatasia (HPP)	N/A
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Active, not recruiting	`NCT04195763` - Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
Not yet recruiting	`NCT05596539` - Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
Completed	`NCT02796885` - Characterisation of Adult-Onset Hypophosphatasia
Completed	`NCT02751801` - Health Burden of Hypophosphatasia
Recruiting	`NCT05234567` - A Prospective Sub-Study of the Global Hypophosphatasia Registry
Not yet recruiting	`NCT06079359` - Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP	Phase 3
Completed	`NCT02797821` - Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)	Phase 2
Completed	`NCT01163149` - Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)	Phase 2
Completed	`NCT04925804` - Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
Completed	`NCT02531867` - Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan	Phase 4
Completed	`NCT01406977` - Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)	Phase 2
Recruiting	`NCT01793168` - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed	`NCT01176266` - Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)	Phase 2/Phase 3
Withdrawn	`NCT00894075` - Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)	Phase 2
Active, not recruiting	`NCT04222452` - The PORTRAIT Study
Recruiting	`NCT06079281` - Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa	Phase 3