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Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease (REBUILD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04189315
Recruitment Status : Withdrawn (R&D portfolio review and in light of the global impact of the COVID-19 pandemic)
First Posted : December 6, 2019
Last Update Posted : June 4, 2020
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE December 4, 2019
First Posted Date  ICMJE December 6, 2019
Last Update Posted Date June 4, 2020
Estimated Study Start Date  ICMJE April 2020
Estimated Primary Completion Date August 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 4, 2019)
Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1 [ Time Frame: Baseline, Week 36 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 4, 2019)
  • Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2 [ Time Frame: Baseline, Week 36 ]
  • Change From Baseline To Week 36 In The 36-item Short-Form Survey (SF-36) Physical Component Summary (PCS) Score In Groups 1 And 2 [ Time Frame: Baseline, Week 36 ]
  • Change From Baseline To Week 36 In The Repeated Chair Stand Test (A Component Of The Short Physical Performance Battery [SPPB]) In Groups 1 And 2 [ Time Frame: Baseline, Week 36 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease
Official Title  ICMJE A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia
Brief Summary The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hypophosphatasia
Intervention  ICMJE Drug: asfotase alfa
Asfotase alfa is administered subcutaneously.
Study Arms  ICMJE
  • Experimental: Group 1 Asfotase Alfa
    Participants will be administered asfotase alfa per approved dose for 36 weeks.
    Intervention: Drug: asfotase alfa
  • Experimental: Group 2 Asfotase Alfa
    Participants will be administered asfotase alfa per approved dose for 12 weeks and then asfotase alfa at a lower dose for 24 weeks.
    Intervention: Drug: asfotase alfa
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: June 1, 2020)
0
Original Estimated Enrollment  ICMJE
 (submitted: December 4, 2019)
50
Estimated Study Completion Date  ICMJE August 2022
Estimated Primary Completion Date August 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male or female aged ≥ 18 years at the time of signing the informed consent form
  • Clinical diagnosis of pediatric-onset HPP based on signs and symptoms consistent with HPP
  • Past medical history that includes at least one nonvertebral fracture (or pseudofracture) incurred without evidence of significant trauma.
  • The presence of a current fracture is not necessary, but for participants with current unhealed fracture(s) or pseudofracture(s) of the lower extremity(ies) (that is, femoral, tibial, fibular, metatarsal) documentation must be provided of the presence of these fractures for at least 3 months prior to screening (with or without surgical intervention)

Exclusion Criteria:

  • Medical condition, serious concurrent or recurrent illness and/or injury, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or the assessment of study endpoints, including all protocol required evaluations and follow up activities, or may put the patient at risk
  • Primary or secondary hyperparathyroidism or hypoparathyroidism
  • History of hypersensitivity to any ingredient contained in asfotase alfa
  • Oral bisphosphonate use within 6 to 12 months (depending on the half-life of the drug as assessed by the investigator) and intravenous (IV) bisphosphonate use within 12 months prior to screening
  • Denosumab use within 18 months prior to screening
  • Asfotase alfa use within 6 months prior to screening
  • Teriparatide/parathyroid hormone analog use within 2 months prior to screening
  • Treatment with strontium or sclerostin inhibitors within 6 months prior to the first dose of study drug
  • Vitamin B6 use for at least 2 weeks prior to screening
  • Serum 25-hydroxy (25-OH) vitamin D below 20 nanogram (ng)/milliliter (mL), with repletion and recheck allowed at screening (results from local laboratory may be used if within 4 weeks of screening)
  • Female patients who are pregnant, planning to become pregnant, or breastfeeding
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04189315
Other Study ID Numbers  ICMJE AA-HPP-406
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Alexion Pharmaceuticals
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP