JOURNAL ONKOLOGIE – STUDIE

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

Studien-Informationen Einschlusskriterien Studien-Rationale Geprüfte Regime

Rekrutierend

NCT-Nummer:
NCT03969329

Studienbeginn:
Dezember 2019

Letztes Update:
09.08.2024

Wirkstoff:
Etelcalcetide

Indikation (Clinical Trials):
Neoplasm Metastasis, Hyperparathyroidism, Hyperparathyroidism, Secondary

Geschlecht:
Alle

Altersgruppe:
Kinder (0-17)

Phase:
Phase 3

Sponsor:
Amgen

Collaborator:
-

Studienleiter

MD
Study Director
Amgen

Kontakt

Amgen Call Center
Kontakt:
Phone: 866-572-6436
E-Mail: medinfo@amgen.com» Kontaktdaten anzeigen

Studienlocations
(3 von 21)

Kindernierenzentrum Bonn
53127 Bonn
(Nordrhein-Westfalen)
GermanyRekrutierend» Google-Maps

Universitaetsklinikum Hamburg Eppendorf
20246 Hamburg
(Hamburg)
GermanyRekrutierend» Google-Maps

Leberkrebszentrum Medizinische Hochschule Hannover
Carl-Neuberg-Straße 1
30625 Hannover
DeutschlandAbgeschlossen» Google-Maps

Universitaetsklinikum Heidelberg, Zentrum fuer Kinder und Jugendmedizin
69120 Heidelberg
(Baden-Württemberg)
GermanyRekrutierend» Google-Maps

Universitaetsklinikum Koeln
50937 Koeln
(Nordrhein-Westfalen)
GermanyRekrutierend» Google-Maps

Universitair Ziekenhuis Gent
9000 Gent
BelgiumAbgebrochen» Google-Maps

Fakultni nemocnice v Motole
150 06 Praha 5
CzechiaRekrutierend» Google-Maps

Hospices Civils de Lyon Hopital Femme Mere Enfant
69677 Bron cedex
FranceAbgebrochen» Google-Maps

Hôpital Armand Trousseau
75012 Paris
FranceAbgeschlossen» Google-Maps

General Children Hospital Panagioti and Aglaias Kyriakou
11527 Athens
GreeceRekrutierend» Google-Maps

Ippokrateio General Hospital of Thessaloniki
54642 Thessaloniki
GreeceRekrutierend» Google-Maps

Semmelweis Egyetem
1083 Budapest
HungaryAbgebrochen» Google-Maps

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar
6720 Szeged
HungaryAbgebrochen» Google-Maps

Azienda Ospedaliera Universitaria Meyer
50139 Firenze
ItalyRekrutierend» Google-Maps

Childrens Hospital, Affiliate of Vilnius University Hospital Santaros Klinikos
08406 Vilinus
LithuaniaAbgebrochen» Google-Maps

Uniwersytecki Szpital Dzieciecy w Krakowie
30-663 Krakow
PolandRekrutierend» Google-Maps

Unidade Local de Saude de Santo Antonio, EPE - Hospital de Santo Antonio
4050-651 Porto
PortugalRekrutierend» Google-Maps

Hospital Universitario Virgen del Rocio
41013 Sevilla
SpainRekrutierend» Google-Maps

Hospital Universitari Vall d Hebron
08035 Barcelona
SpainRekrutierend» Google-Maps

Royal Hospital for Sick Children
G51 4TF Glasgow
United KingdomRekrutierend» Google-Maps

Great Ormond Street Hospital for Children
WC1N 3JH London
United KingdomAbgebrochen» Google-Maps

Alle anzeigen

Studien-Informationen

Brief Summary:

Assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of

etelcalcetide in the treatment of secondary hyperparathyroidism (SHPT) in pediatric

subjects between ≥ 2 to < 18 years of age, with chronic kidney disease (CKD) on

hemodialysis

Ein-/Ausschlusskriterien

Inclusion Criteria:

- Subject's legally acceptable representative has provided informed consent when the

subject is legally too young to provide informed consent and the subject has

provided written assent based on local regulations and/or guidelines prior to any

study-specific activities/procedures being initiated.

- Male or female subjects 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 chronic kidney disease (CKD) and secondary hyperparathyroidism (SHPT)

undergoing hemodialysis/hemodiafiltration three times per week (TIW) at the time of

screening greater than or equal to 1 month.

- Diagnosis of secondary hyperparathyroidism (SHPT) with the mean of the 2 consecutive

central laboratory intact parathyroid hormone (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 calcium (cCa) value greater than or equal to 9.0 mg/dL obtained from

the central laboratory during screening.

- Dialysate calcium (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 study.

- Subject 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 study, except for adjustments allowed per protocol.

- Subject 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

study, except for adjustments allowed per protocol.

- Subject receiving calcium (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 study, except for adjustments allowed per protocol.

- Secondary hyperparathyroidism (SHPT) not due to vitamin D deficiency, per

investigator assessment.

Exclusion Criteria:

- 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 study period.

- Anticipated or scheduled kidney transplant during the study period.

- Subject 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.

- Receipt of cinacalcet therapy within 30 days prior to screening and through

enrollment.

- Receipt of etelcalcetide therapy within 6 months prior to screening and through

enrollment.

- All herbal medicines (eg, St. John's wort), vitamins, and supplements consumed by

the subject 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 subject 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 subjects 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 subject participation. Paracetamol for analgesia will be allowed.

- Prior/Concurrent Clinical Study Experience:

- Currently receiving treatment in another investigational device or drug study, or

less than 30 days since ending treatment on another investigational device or drug

study(ies). Other investigational procedures while participating in this study are

excluded.

- Diagnostic Assessments During Screening:

- Subject has significant abnormalities on the most recent central laboratory test

during the screening period prior to enrollment per the Investigator including but

not limited to the following: a. Serum transaminase (alanine aminotransferase [ALT]

or serum glutamic pyruvic transaminase [SGPT], aspartate aminotransferase [AST], or

serum glutamic oxaloacetic transaminase [SGOT]) greater than 1.5 times the upper

limit of normal (ULN).

- Corrected QT interval greater than 500 ms, using Bazett's formula.

- Corrected QT interval greater than or equal to 450 to less than or equal to 500 ms,

using Bazett's formula, unless written permission to enroll is provided by the

investigator after consultation with a pediatric cardiologist.

- Subject has a clinically significant electrocardiogram (ECG) abnormality (eg,

unstable arrhythmia) during screening that, in the opinion of the investigator,

could pose a risk to subject safety or interfere with the study evaluation.

- Within the 3 Months Prior to Screening:

- New onset or worsening of a pre-existing seizure disorder.

- Subjects on anti-convulsant medication must be on a stable and therapeutic dose for

3 months prior to screening (if blood level monitoring is clinically available, then

the subject must have a therapeutic blood level within 1 week of enrollment).

Other Exclusions:

- Female subject is pregnant or breastfeeding or planning to become pregnant or

breastfeed during treatment and for an additional 3 months after the last dose of

etelcalcetide. (Females of childbearing potential should only be included in the

study after a confirmed menstrual period and a negative highly sensitive serum

pregnancy test within 7 days prior to the first dose of investigational product).

- Female subjects of childbearing potential unwilling to use 1 highly effective or

acceptable method of effective contraception during treatment and for an additional

3 months after the last dose of investigational product. Refer to Appendix 5 for

additional contraceptive information.

- Female subjects of childbearing potential with a positive pregnancy test assessed at

screening by a serum pregnancy test.

- Subject has known sensitivity to etelcalcetide or excipients to be administered

during dosing.

- Subject likely to not be available to complete all protocol-required study visits or

procedures, and/or to comply with all required study procedures to the best of the

subject and investigator's knowledge.

- History or evidence of any other clinically significant disorder, condition or

disease (with the exception of those outlined above) or unacceptable physical

findings, that, in the opinion of the investigator or Amgen physician, if consulted,

would pose a risk to subject safety or interfere with the study evaluation

procedures or completion.

- Subject previously has entered this study or previously received treatment with

etelcalcetide.

- Anemia, which in the opinion of the investigator makes it not advisable to undergo

sequential blood draws.

- History of unstable chronic heart failure within the last 1 year prior to screening.

Studien-Rationale

Primary outcome:

1. Percent change in intact parathyroid hormone (iPTH) from baseline during the efficacy assessment period (EAP) at weeks 20 to 26 (Time Frame - 20 to 26 weeks):
To evaluate the efficacy of etelcalcetide in reducing the intact parathyroid hormone (iPTH) level in children ages equal to or greater than 2 to less than 18 years with secondary hyperparathyroidism (SHPT) receiving maintenance hemodialysis

Secondary outcome:

1. Achievement of a greater than 30% reduction from baseline in mean intact parathyroid hormone (iPTH) during the efficacy assessment period (EAP) (Time Frame - 20 to 26 weeks):
To evaluate the efficacy of etelcalcetide

2. Percent change from baseline in corrected total serum calcium (Ca) and serum phosphorus from baseline during the EAP (Time Frame - 20 to 26 weeks):
To characterize change in laboratory markers of chronic kidney disease

3. Proportion of subjects achieving corrected serum calcium (Ca) levels less than 8.0 mg/dL (2.0 mmol/L) at any time during the study (Time Frame - During the treatment period):
To characterize the safety of etelcalcetide treatment based on laboratory values

4. Proportion of subjects with hypocalcemia (corrected serum calcium levels less than 8.4 mg/dL) (Time Frame - During the treatment period):
To characterize the safety of etelcalcetide treatment based on laboratory values

5. Etelcalcetide plasma concentrations before and at the end of dialysis after single and multiple doses (Time Frame - During the treatment period):
Etelcalcetide plasma concentrations before and at the end of dialysis after single and multiple doses

6. Etelcalcetide PK parameter of maximum-observed concentration (Cmax) (Time Frame - During the treatment period):
Etelcalcetide plasma concentrations before and at the end of dialysis after single and multiple doses

7. Etelcalcetide pharmacokinetic (PK) parameter of plasma trough concentrations (Cmin) (Time Frame - During the treatment period):
To characterize the pharmacokinetic (PK) of etelcalcetide treatment after single and multiple doses

8. Subject incidence of all treatment-emergent adverse events (Time Frame - During the treatment period):
To characterize the safety of etelcalcetide treatment based on adverse events. Nature, frequency, severity, and relationship to treatment of all adverse events, including those of special interest reported during the study

Geprüfte Regime

Etelcalcetide (Parsabiv - brand name):
Etelcalcetide has been shown to be safe and efficacious in treating adult chronic kidney disease (CKD) patients with secondary hyperparathyroidism (SHPT) by simultaneously controlling intact parathyroid hormone (iPTH), calcium (Ca), and phosphorus and has recently been approved for use in adult patients with secondary hyperparathyroidism (SHPT) treated with hemodialysis in both the United States and Europe

Quelle: ClinicalTrials.gov

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