Clinical Trials Register

Summary
EudraCT Number:	2016-003621-41
Sponsor's Protocol Code Number:	DESIRED
National Competent Authority:	Sweden - MPA
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2016-09-28
Trial 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

Sweden - MPA

A.2

EudraCT number

2016-003621-41

A.3

Full title of the trial

Desferal administration to improve the impaired reaction to hypoxia in diabetes (DESIRED)
A randomised, double-blind, placebo-controlled, cross-over study

A.3.1

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

Treatment with Desferal to improve the impaired reaction to oxygen deficiency in patients with diabetes

A.3.2

Name or abbreviated title of the trial where available

DESIRED

A.4.1

Sponsor's protocol code number

DESIRED

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	Department of Endocrinology, Metabolism and Diabetes, Karolinska University Hospital
B.1.3.4	Country	Sweden
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Vetenskapsrådet
B.4.2	Country	Sweden
B.4.1	Name of organisation providing support	Karolinska University Hospital, ALF-medel
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Department of Endocrinology, Metabolism and Diabetes, Karolinska University Hospital
B.5.2	Functional name of contact point	Sergiu Bogdan Catrina
B.5.3	Address:
B.5.3.1	Street Address	Karolinska University Hospital
B.5.3.2	Town/ city	Stockholm
B.5.3.3	Post code	17176
B.5.3.4	Country	Sweden
B.5.4	Telephone number	+460739130497
B.5.6	E-mail	sergiu-bogdan.catrina@ki.se

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	Desferal
D.2.1.1.2	Name of the Marketing Authorisation holder	Novartis Sverige AB
D.2.1.2	Country which granted the Marketing Authorisation	Sweden
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.4	Pharmaceutical form	Powder for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
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
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

The study will investigate the effect of deferoxamine on the impaired reaction to hypoxia in patients with diabetes mellitus type 1.

E.1.1.1

Medical condition in easily understood language

The study will investigate the effect of deferoxamine on the impaired reaction to oxygen deficiency in patients with diabetes.

E.1.1.2

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To investigate whether systemic administration of deferoxamine can reverse the impaired response to hypoxic challenge in patients with diabetes mellitus type 1.

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

•Patients with diabetes mellitus type 1 with a duration of the disease between 10 and 20 years
•HbA1c 55-100 mmol/mol
•Age 18-55
•Contraception: Female subjects must be postmenopausal, surgically sterile, or if premenopausal (and not surgically sterile), be prepared to use ≥1 effective method of contraception during the study and for 30 days after the last visit. Effective methods of contraception are considered to be those listed below: Double barrier method, i.e. (a) condom (male or female) or (b) diaphragm, with spermicide; or Intrauterine device; or Vasectomy (partner); or Hormonal (e.g. contraceptive pill, patch, intramuscular implant or injection); or Abstinence, if in line with the preferred and usual lifestyle of the subject.
•Signed informed consent

E.4

Principal exclusion criteria

•Major cardiovascular complications such as coronary heart disease, unstable or stable angina, myocardial infarction, ventricular arrhythmias, and atrial fibrillation in the last 3 months
•Decompensated congestive heart failure of functional class 3-4.
•Therapy with β-blockers
•Severe hypertension (≥180 mmHg systolic or ≥110 mmHg diastolic blood pressure)
•Proliferative retinopathy
•Sign for peripheric diabetic neuropathy (decreased/absent sensitivity to 10 g monofilament, vibration, plantar reflex)
•Definite autonomic dysfunction
•History of anemia, bleeding gastric ulcer, abundant menstruation
•Currently smoking
•History of alcohol or drug abuse
•Infections during the last month
•Malignancy
•Participant in another ongoing pharmacological study
•Unwillingness to participate following oral and written information
•If female: plans to become pregnant, known pregnancy or a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
•Any concomitant disease or condition that may interfere with the possibility for the subject to comply with or complete the study protocol
•Subjects with any other severe acute or chronic medical or psychiatric condition that make the subject inappropriate for the study in the judgment of the Investigator
•Treatment with Prochlorperazine

E.5 End points

E.5.1

Primary end point(s)

Endothelial Precursor Cells number increase after intermittent hypoxia exposure

E.5.1.1

Timepoint(s) of evaluation of this end point

24 hours after intermittent hypoxia exposure compared to baseline

E.5.2

Secondary end point(s)

1. Changes in the levels of angiogenetic chemokines (SDF-1, EPO, VEGF)
2. Changes in the levels of angiogenetic and hypoxic induced genes (VEGFA, SDF-1, BPNP3, GLUT3, PDK1)
3. Changes in electrophysiological parameters important for the cardiorespiratory response:
o R-R interval change
o the standard deviation of the R-R interval (SDNN)
o systolic and diastolic blood pressure
o end-tidal carbon dioxide (CO2-et)
o arterial oxygen saturation (SaO2)
o breathing rate
o tidal volume (Vt), minute ventilation (VE), and the ratio between Vt and inspiration time (Vt/Ti)
o baroreflex sensitivity (BRS)
o arterial function parameters: pulse wave velocity, augmentation index (AI), augmentation index corrected for heart rate (AI75)

E.5.2.1

Timepoint(s) of evaluation of this end point

1. 6 hours after intermittent hypoxia exposure compared to baseline
2. 3 and 6 hours after intermittent hypoxia exposure compared to baseline
3. Immediately after and 3 and 6 hours after intermittent hypoxia exposure compared to baseline

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

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

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

E.8.1.6

Cross over

Yes

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

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

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

LVLS

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

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)

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

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Standard of care treatment

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2016-11-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-12-05

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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