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HypophosphatasiaPhase 2NCT02456038

Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

A Phase 2 study testing Asfotase Alfa (ALXN1215) in people with Hypophosphatasia, run by Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan at 12 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia Read more about this study

Who is it for?
Any age
Conditions the study is for
Hypophosphatasia
What is being tested?
Asfotase Alfa (ALXN1215)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
12 sitesJapan
Study dates
Aug 2014to Jul 2015
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
  • Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
  • Patient who has been diagnosed as HPP
  • Documented diagnosis of HPP as indicated by:
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  • Total serum alkaline phosphatase below the lower limit of normal for age
Show all 21Show fewer
  • Ultrasonographic features of prenatal, characterized by:
  • 1) severe short extremities (femur length <-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length >0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference <0.6) (3) Computed tomographic findings of prenatal, characterized by:
  • Generalized decreased ossification
  • Extreme shortening of tubular bones
  • Hypoplastic thorax (4) Radiographic evidence of HPP, characterized by:
  • 1) Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:
  • History or presence of:
  • Nontraumatic post-natal fracture
  • Delayed fracture healing
  • Nephrocalcinosis or history of elevated serum calcium
  • Functional craniosynostosis
  • Respiratory compromise or rachitic chest deformity
  • Vitamin B6 dependent seizures
  • Failure to thrive
  • Premature tooth loss (6) Patient who have the mutation of tissue non-specific ALP gene 4. Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures

Who cannot join

  • Current evidence of treatable form of rickets
  • Serum calcium or phosphate levels below the normal range
  • Pregnant women and nursing mothers
  • Patient who cannot enforce suitable contraceptive measures during the clinical trial
  • Prior treatment with bisphosphonates
  • Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
  • Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
  • Clinically significant disease that precludes study participation, in the opinion of the Investigator

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

Japan12 sites

  • Nippon Medical School Hospital

    Bunkyo-ku, Tokyo, 113-8603

  • National Hospital Organization Nagara Medical Center

    Gifu, Gifu, 502-8558

  • Hamamatsu University Hospital

    Hamamatsu, Shizuoka, 431-3192

  • Hiroshima University Hospital

    Hiroshima, Hiroshima, 734-8551

  • Osaka Medical Center and Research Institute for Maternal and Child Health

    Izumi, Osaka, 594-1101

  • Showa General Hospital

    Kodaira, Tokyo, 187-8510

  • Kurume University Hospital

    Kurume, Fukuoka, 830-0011

  • Niigata University Medical & Dental Hospital

    Niigata, Niigata, 951-8510

  • Saitama Children's Medical Center

    Saitama, Saitama, 339-0077

  • Hokkaido University Hospital

    Sapporo, Hokkaido-prefecture, 060-8648

  • Tohoku University Hospital

    Sendai, Miyagi, 980-8574

  • Osaka University Hospital

    Suita, Osaka, 565-0871

Other trials in Japan

About this study

Official title: A Multicenter Study of the Safety and Efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Patients With Hypophosphatasia (HPP)

The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia

Being tested
Asfotase Alfa (ALXN1215)
Medical subject headings
Hypophosphatasia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Asfotase Alfa (ALXN1215)Drug

    Also called ALXN1215

Study groups in NCT02456038
Study groupRoleWhat they receive
Asfotase Alfa (ALXN1215)ExperimentalAsfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times.
Receives: Drug: Asfotase Alfa (ALXN1215)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa

    Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients

    Measured: Up to 50 months or until regulatory approval

Secondary outcomes (6)

  1. Overall survival

    Overall survival is defined as the time from birth to time of death.

    Measured: Up to 50 months or until regulatory approval

  2. Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale

    Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale for all treated patients

    Measured: Up to 50 months or until regulatory approval

  3. Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa)

    For patients who are not mechanically ventilated at the time of enrollment, the percentage who are alive and ventilator-free after receiving asfotase alfa

    Measured: Up to 50 months or until regulatory approval

  4. Profile of asfotase alfa treatment on respiratory function

    Effect of asfotase alfa treatment on respiratory function as measured by ventilator status, time on respiratory support (including time on ventilator or supplemental oxygen), ventilator rate or oxygen volume, ventilator pressures, and fraction of inspired oxygen (FiO2) for all treated patients

    Measured: Up to 50 months or until regulatory approval

  5. Profile of asfotase alfa treatment on physical growth

    Effect of asfotase alfa treatment on physical growth as measured by body weight, length, arm span, head circumference, and chest circumference for all treated patients

    Measured: Up to 50 months or until regulatory approval

  6. Effect of asfotase alfa treatment on development

    Assessment of changes in gross motor development as measured by the developmental motor milestones for all treated patients

    Measured: Up to 50 months or until regulatory approval

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 May 2015 (estimated)
  2. Study startAug 2014
  3. Primary completionJul 2015
  4. Study completionJul 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02456038
Sponsor's protocol ID
HPPJEAP-01
Other IDs
UMIN000014816
Status last verified
Mar 2016
First submitted
26 Mar 2015
Last updated
31 Mar 2016
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Asfotase Alfa (ALXN1215) in people with Hypophosphatasia, run by Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan at 12 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 21 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa (time frame: Up to 50 months or until regulatory approval). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02456038.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2016.

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