Trial Outcomes & Findings for Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09 (NCT NCT02235493)

Last Updated: 2019-03-13

Results Overview

The MPOMA-G is a 5-component assessment that is used to evaluate gait performance. The first component has 4 sub-components. For 2 components and 2 sub-components, scores of 0 or 1 are assigned while scores of 0, 1 or 2 are assigned to the rest of the 2 components and 2 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.

Recruitment status

COMPLETED

Target enrollment

6 participants

Primary outcome timeframe

The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.

Results posted on

2019-03-13

Participant Flow

Participant milestones

Participant milestones
Measure	Retrospective Observational Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \< 18 years of age\].
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Retrospective Observational n=6 Participants Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \< 18 years of age\].
Age, Customized	28.2 months STANDARD_DEVIATION 10.6 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Race/Ethnicity, Customized White, Hispanic or Latino	2 participants n=5 Participants
Race/Ethnicity, Customized White, Not Hispanic or Latino	4 participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants
Modified Performance-Oriented Mobility Assessment - Gait subtest (MPOMA-G)	6.00 units on a scale n=5 Participants
Deceased/Alive Status - Deceased	0 participants n=5 Participants
Deceased/Alive Status - Alive	6 participants n=5 Participants

PRIMARY outcome

Timeframe: The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.

Outcome measures

Outcome measures
Measure	Retrospective Observational n=6 Participants Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \< 18 years of age\].
Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall	1.50 units on a scale Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.

The POMA-G is a 7-component assessment that is used to evaluate gait performance. The second component has 4 sub-components. Scores of 0, 1 or 2 are assigned to 2 components while scores of 0 or 1 are assigned to the rest of the 4 components and 4 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.

Outcome measures

Outcome measures
Measure	Retrospective Observational n=6 Participants Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \< 18 years of age\].
Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall	1.50 units on a scale Interval 0.0 to 2.0

Adverse Events

Retrospective Observational

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Senior Medical Director, Clinical Development

Alexion Pharmaceuticals

Phone: 203-272-2596

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Participation in this single-center study involves a commitment to publish the data from the study in a cooperative publication prior to release of study results on an individual basis. Therefore, the PI is subject to certain disclosure and publication limitations.
Publication restrictions are in place

Restriction type: OTHER