Clinical Trials Register

Summary
EudraCT Number:	2014-002128-28
Sponsor's Protocol Code Number:	STS-CSM-1/13
National Competent Authority:	Austria - BASG
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2014-12-04
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Austria - BASG

A.2

EudraCT number

2014-002128-28

A.3

Full title of the trial

A Prospective Multicenter Phase 2/3 Clinical Trial with Sodium Thiosulfate for the Treatment of Calciphylaxis

Prospektive, multizentrische Phase 2/3-Studie mit Natriumthiosulfat zur Behandlung von Calciphylaxie

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Phase 2/3 Clinical Trial with Sodium Thiosulfate for the Treatment of Calciphylaxis

Phase 2/3-Studie mit Natriumthiosulfat zur Behandlung von Calciphylaxie

A.4.1

Sponsor's protocol code number

STS-CSM-1/13

A.5.1

ISRCTN (International Standard Randomised Controlled Trial) Number

ISRCTN73380053

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Dr. F. Köhler Chemie GmbH
B.1.3.4	Country	Germany
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Dr. F. Köhler Chemie GmbH
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Celerion Austria GmbH
B.5.2	Functional name of contact point	Clinical Research Organisation
B.5.3	Address:
B.5.3.1	Street Address	Hainburger Strasse 33
B.5.3.2	Town/ city	Vienna
B.5.3.3	Post code	1030
B.5.3.4	Country	Austria
B.5.4	Telephone number	+431403 38 05 0
B.5.5	Fax number	+431403 38 0566
B.5.6	E-mail	sts-csm-1_13@celerion.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Sodium Thiosulfat 25%
D.2.1.1.2	Name of the Marketing Authorisation holder	Dr. F. Köhler Chemie GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/10/848
D.3 Description of the IMP
D.3.1	Product name	Sodium Thiosulfate
D.3.2	Product code	STS
D.3.4	Pharmaceutical form	Concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	SODIUM THIOSULFATE PENTAHYDRATE
D.3.9.3	Other descriptive name	SODIUM THIOSULFATE PENTAHYDRATE
D.3.9.4	EV Substance Code	SUB22204
D.3.10	Strength
D.3.10.1	Concentration unit	% percent
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Calciphylaxis

Calciphylaxie

E.1.1.1

Medical condition in easily understood language

Calciphylaxis

Calciphylaxie

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10051714
E.1.2	Term	Calciphylaxis
E.1.2	System Organ Class	10027433 - Metabolism and nutrition disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

Percent reduction of the total wound area after 24 weeks (V4) compared to baseline (V0) as assessed by 2 independent, blinded dermatologists using a serial photo documentation. The mean value of both assessments will be taken.

Prozentuale Verringerung der Gesamtwundfläche nach 24 Wochen (V4) im Vergleich zu Baseline (V0) auf der Grundlage der Beurteilung von 2 unabhängigen, verblindeten Dermatologen anhand einer Fotoseriendokumentation. Relevant ist der Mittelwert aus den beiden Beurteilungen.

E.2.2

Secondary objectives of the trial

Status of skin lesions, Pain, Clincal Global Impression, Improvement leading to eligibility of the patient for kidney transplantation, New Lesions, Bone Mineral Density, Survival, Safety Parameters.

Status der Hautläsionen, Schmerz, KlinischerGesamteindruck, Verbesserung, die zur Eignung des Patienten für eine Nierentransplantation führt, Auftreten neuer Läsionen, Knochendichte, Überleben, Sicherheitsparameter

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

(1) All patients ≥ 18 years
(2) Male or female HD patients with a diagnosis of calciphylaxis. (Patients on peritoneal dialysis or patients with the requirement for renal replacement therapy, who are diagnosed with calciphylaxis, may be switched to HD and included in the study after switching).
(3) Able to understand character and individual consequences of the clinical trial and to provide written informed consent to participate in the study

(1) Patienten≥ 18 Jahre
(2) Männliche oder weibliche Hämodialyse (HD)-Patienten mit diagnostizierter Calciphylaxie. (Wurde bei Patienten auf Peritoneal-Dialyse oder bei Patienten, die eine Nierentransplantation benötigen, eine Calciphylaxie diagnostiziert, so können diese auf HD umgestellt und nach der Umstellung in die Studie eingeschlossen werden).
(3) Patienten, die in der Lage sind, Charakter und individuelle
Konsequenzen der klinischen Studie zu verstehen, und - nach erfolgter
Aufklärung -, ihre schriftliche Einwilligung zur Teilnahme an der Studie
zu geben.

E.4

Principal exclusion criteria

(1) Sodium metabisulfite hypersensitivity, among others the history of bronchial asthma due to known sodium metabisulfite hypersensitivity
(2) Females who are pregnant (positive pregnancy test at screening or during study phase), lactating, or if having reproductive potential (being not post-menopausal (no menses for 12 months without an alternative medical cause) or surgically sterilized) are considered potentially ineligible with respect to use highly effective methods of birth control throughout the study, which are also described in detail in the Patient Inform Consent Form. (Of note, STS has been demonstrated not to cross the blood-placenta barrier in gravid eves (Graeme et al., 1999); therefore we regard fetal damage also as unlikely in humans).
(3) Patients who have participated in any other investigational studies within 30 days previous to enrollment
(4) History of alcohol abuse, illicit drug use, significant mental illness, physical dependence to any opioid, or any history of drug abuse or addiction within 12 months of study enrolment.
(5) Good response to conventional treatment.
(6) Life expectancy less than 4 months in the judgement of the investigator

(1) Überempfindlichkeit gegen Natriummetabisulfit, u. a. anamnestisches Bronchialasthma wegen bekannter Überempfindlichkeit gegen Natriummetabisulfit.
(2) Frauen, welche schanger sind (positiver Schwangerschaftstest bei Screening oder während der Studienphase), stillen oder wenn sie im gebährfähigem Alter sind (nicht postmnopausal oder chirurgisch sterilisiert), und keine hochwirksame Methode der Geburtenkontrolle verwenden, welche detailliert in der Patienteninformation beschrieben werden.
(3) Patienten, die innerhalb von 30 Tagen vor Einschluss an anderen Studien teilgenommen haben
(4) Alkoholmissbrauch, Konsum illegaler Drogen, signifikante psychische Erkrankung oderkörperliche Opioid-Abhängigkeit in der Anamnese, oder Medikamenten-/drogenmissbrauch oder -abhängigkeit innerhalb von 12 Monaten vor Einschluss
(5) Gutes Ansprechen auf herkömmliche Behandlungen
(6) Lebenserwartung von weniger als 4 Monaten nach Einschätzung des Prüfarztes

E.5 End points

E.5.1

Primary end point(s)

See section E2.1 Primary Objectives

Siehe Sektion E2.1 Primäre Ziele

E.5.1.1

Timepoint(s) of evaluation of this end point

after 24 weeks of treatment

nach 24 Behandlungswochen

E.5.2

Secondary end point(s)

See section E2.2 Secondary Objectives

Siehe Sektion E2.2 Sekundäre Ziele

E.5.2.1

Timepoint(s) of evaluation of this end point

at 4, 8, 16, 24, 36, 48 weeks

nach 4, 8, 16, 24, 36, 48 Wochen

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

A Biobank will be established for calciphylaxis (blood).

Eine Biobank wird für Calciphylaxie-relevante Blutparameter wird eingerichtet.

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Austria

Germany

Switzerland

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

The study will be completed, when the last subject completes their Study Completion visit and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator.

Die Studie ist beendet, wenn der letzte Patient seine letzte Visite abgeschlossen hat und alle Untersuchungen dokumentiert und abgeschlossen sind.

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

F.3.3.1

Women of childbearing potential not using contraception