A Phase 3 Study of Etelcalcetide in Children With Secondary Hyperparathyroidism Receiving Hemodialysis

NCT03969329 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: 20170724
• Study Type: interventional
• Target: 24
• Locations: 12 countries
• Sites: 23

Brief Summary

Assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of etelcalcetide in the treatment of secondary hyperparathyroidism (SHPT) in pediatric participants between ≥ 2 to \< 18 years of age, with chronic kidney disease (CKD) on hemodialysis.

Conditions

Secondary Hyperparathyroidism

Keywords

Secondary hyperparathyroidism (sHPT); Chronic kidney disease (CKD); Paediatric

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in iPTH at Weeks 20 to 26

  To evaluate the efficacy of etelcalcetide in reducing the iPTH level in children ages equal to or greater than 2 to less than 18 years with SHPT receiving maintenance hemodialysis.

  Week 20 to 26

Secondary Outcomes (8)

• Number of Participants Who Achieve a >30% Reduction From Baseline in Mean iPTH

  Week 20 to 26

• Percent Change From Baseline in Corrected Total Serum Ca and Serum Phosphorus

  Week 20 to 26

• Proportion of Participants Achieving Corrected Serum Ca Levels Less Than 8.0 mg/dL (2.0 mmol/L)

  During the treatment period (up to 31 weeks)

• Proportion of Participants with Hypocalcemia

  During the treatment period (up to 31 weeks)

• Etelcalcetide Plasma Concentrations Before and at the end of Dialysis After Single and Multiple Doses

  During the treatment period (up to 31 weeks)

Study Arms (1)

Etelcalcetide

EXPERIMENTAL

Participants will receive etelcalcetide in addition to standard of care.

Drug: Etelcalcetide

Interventions

Etelcalcetide DRUG

Etelcalcetide has been shown to be safe and efficacious in treating adult CKD patients with SHPT by simultaneously controlling iPTH, calcium (Ca), and phosphorus and has recently been approved for use in adult patients with SHPT treated with hemodialysis in both the United States and Europe.

Also known as: Parsabiv - brand name

Etelcalcetide

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant's legally acceptable representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines prior to any trial-specific activities/procedures being initiated.
• Male or female participants greater than or equal to 2 to less than 18 years of age at the time of enrollment.
• Targeted dry weight greater than or equal to 7 kg at the time of screening Week -1.
• Diagnosed with CKD and SHPT undergoing hemodialysis/hemodiafiltration TIW or four times a week (QIW) at the time of screening greater than or equal to 1 month.
• Diagnosis of SHPT with the mean of the 2 consecutive central laboratory iPTH values greater than 300 pg/mL during screening, on separate days and within 2 weeks of enrollment obtained from the central laboratory during screening.
• Serum corrected Ca value greater than or equal to 9.0 mg/dL obtained from the central laboratory during screening.
• Dialysate Ca level greater than or equal to 2.5 mEq/L for at least 1 month prior to screening and throughout the duration of the trial.
• participant receiving active vitamin D sterols must have had no more than a maximum dose change of 50% within the 2 weeks prior to screening laboratory assessments, remain stable through enrollment, and be expected to maintain stable doses for the duration of the trial, except for adjustments allowed per protocol.
• participant receiving phosphate binders must have had no more than a maximum dose change of 50% within the 2 weeks prior to screening laboratory assessments, remain stable through enrollment, and be expected to maintain stable dose for the duration of the trial, except for adjustments allowed per protocol.
• Subject receiving Ca supplements must have had no more than a maximum dose change of 50% within the 2 weeks prior to screening laboratory assessments, remain stable through enrollment, and be expected to maintain stable dose for the duration of the trial, except for adjustments allowed per protocol.
• SHPT not due to vitamin D deficiency, per investigator assessment.

You may not qualify if:

• Disease Related:
• History of congenital long QT syndrome, second or third degree heart block, ventricular tachyarrhythmia's, history of symptomatic ventricular dysrhythmias Torsades de Pointes or other conditions associated with prolonged QT interval.
• Anticipated or scheduled parathyroidectomy during the trial period.
• Anticipated or scheduled kidney transplant during the trial period.
• Participant has received a parathyroidectomy within 6 months prior to enrollment.
• Other Medical Conditions:
• Current malignancy or history of other malignancy, except non-melanoma skin cancers within the last 5 years.
• Prior/Concomitant Therapy:
• Use of concomitant medications that may prolong the QTc (eg, ondansetron, albuterol, sotalol, amiodarone, erythromycin, or clarithromycin). Refer to CredibleMeds.org for guidance. Certain medications may be allowed based on review by the Medical Monitor and require additional electrocardiogram (ECG) monitoring and potential electrolyte monitoring.
• Receipt of cinacalcet therapy within 30 days prior to screening and through enrollment.
• Any previous use of etelcalcetide prior to screening and through enrollment (Original protocol, Amendment 1, and Amendment 2 only).
• Receipt of etelcalcetide therapy within 6 months prior to screening assessments and through enrollment (Amendment 3 and later only).
• All herbal medicines (eg, St. John's wort), vitamins, and supplements consumed by the participant within the 30 days prior to enrollment, and continuing use if applicable, will be reviewed by the Principal Investigator and the Amgen Medical Monitor. Written documentation of the review and Amgen acknowledgment is required for participant participation.
• Use of any over-the-counter or prescription medications within the 14 days or 5 half-lives (whichever is longer) prior to enrollment that are not established therapies for participants with renal disease or other conditions secondary to renal disease will be reviewed by the Principal Investigator and the Amgen Medical Monitor. Written documentation of the review and Amgen acknowledgment is required for participant participation. Paracetamol for analgesia will be allowed.
• Prior/Concurrent Clinical Trial Experience:

Sponsors & Collaborators

• Amgen: lead

Study Sites (23)

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

TERMINATED

Fakultni nemocnice v Motole

Prague, 150 06, Czechia

COMPLETED

Hospices Civils de Lyon Hopital Femme Mere Enfant

Bron, 69677, France

TERMINATED

Hopital Armand Trousseau

Paris, 75012, France

COMPLETED

Kindernierenzentrum Bonn

Bonn, 53127, Germany

COMPLETED

Universitaetsklinikum Koeln

Cologne, 50937, Germany

RECRUITING

Universitaetsklinikum Hamburg Eppendorf

Hamburg, 20246, Germany

RECRUITING

Medizinische Hochschule Hannover

Hanover, 30625, Germany

COMPLETED

Universitaetsklinikum Heidelberg, Zentrum fuer Kinder und Jugendmedizin

Heidelberg, 69120, Germany

RECRUITING

General Children Hospital Panagioti and Aglaias Kyriakou

Athens, 11527, Greece

RECRUITING

Ippokrateio General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

RECRUITING

Semmelweis Egyetem

Budapest, 1083, Hungary

TERMINATED

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar

Szeged, 6720, Hungary

TERMINATED

Azienda Ospedaliera Universitaria Meyer

Florence, 50139, Italy

TERMINATED

Childrens Hospital, Affiliate of Vilnius University Hospital Santaros Klinikos

Vilinus, 08406, Lithuania

TERMINATED

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, 30-663, Poland

TERMINATED

Unidade Local de Saude de Santo Antonio, EPE - Hospital de Santo Antonio

Porto, 4050-651, Portugal

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Andalusia, 41013, Spain

TERMINATED

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, 08035, Spain

RECRUITING

Royal Hospital for Sick Children

Glasgow, G51 4TF, United Kingdom

RECRUITING

Leeds Teaching Hospitals NHS Trust

Leeds, LS1 3EX, United Kingdom

RECRUITING

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

TERMINATED

University Hospital Southampton NHS Foundation Trust

Southampton, SO16 6YD, United Kingdom

RECRUITING

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Hyperparathyroidism, Secondary; Renal Insufficiency, Chronic

Interventions

etelcalcetide hydrochloride

Condition Hierarchy (Ancestors)

Hyperparathyroidism; Parathyroid Diseases; Endocrine System Diseases; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Central Study Contacts

Amgen Call Center

CONTACT

866-572-6436; medinfo@amgen.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will remain on treatment for 26 weeks from day 1 or until the time of renal transplant or parathyroidectomy, whichever occurs first. Participants will receive a starting dose of etelcalcetide 0.07 mg/kg or 5 mg, whichever is lower, 3 times a week (TIW) intravenously. The dose of etelcalcetide will be titrated every 4 weeks (weeks 5, 9, 13, and 17) based on intact parathyroid hormone (iPTH), calcium, and safety to a maximum dose of 0.21 mg/kg or 15 mg, whichever is lower. The lowest protocol specified dose (PSD) for this trial is 0.035 mg/kg or 2.5 mg, whichever is lower.

Study Record Dates

• First Submitted: May 29, 2019
• First Posted: May 31, 2019
• Study Start: December 20, 2019
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: March 23, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities.There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.

Locations

HU (2); CZ (1); ES (2); LI (1); IT (1); GR (2); BE (1); PL (1); FR (2); GB (4); PT (1); DE (5)