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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Recruitment Status : Withdrawn (R&D portfolio review and in light of the global impact of the COVID-19 pandemic)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

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).

Condition or disease Intervention/treatment Phase
Hypophosphatasia Drug: asfotase alfa Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Estimated Study Start Date : April 2020
Estimated Primary Completion Date : August 2022
Estimated Study Completion Date : August 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Group 1 Asfotase Alfa
Participants will be administered asfotase alfa per approved dose for 36 weeks.
Drug: asfotase alfa
Asfotase alfa is administered subcutaneously.

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.
Drug: asfotase alfa
Asfotase alfa is administered subcutaneously.




Primary Outcome Measures :
  1. Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1 [ Time Frame: Baseline, Week 36 ]

Secondary Outcome Measures :
  1. Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2 [ Time Frame: Baseline, Week 36 ]
  2. 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 ]
  3. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04189315    
Other Study ID Numbers: AA-HPP-406
First Posted: December 6, 2019    Key Record Dates
Last Update Posted: June 4, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Hypophosphatasia
HPP
Pediatric-Onset
Asfotase Alfa
Additional relevant MeSH terms:
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Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases