Clinical Trials Register

Summary
EudraCT Number:	2007-000171-41
Sponsor's Protocol Code Number:	0000726
National Competent Authority:	Slovakia - SIDC (Slovak)
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2008-04-10
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

Slovakia - SIDC (Slovak)

A.2

EudraCT number

2007-000171-41

A.3

Full title of the trial

A Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study Evaluating the Efficacy and Safety of ALTU-135 Treatment in Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

A.4.1

Sponsor's protocol code number

0000726

A.7

Trial is part of a Paediatric Investigation Plan

Information not present in EudraCT

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Altus Pharmaceuticals Inc
B.1.3.4	Country	United States
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
B.4.2	Country
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation
B.5.2	Functional name of contact point

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	No
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/3/04/222
D.3 Description of the IMP
D.3.1	Product name	ALTU-135
D.3.2	Product code	ALTU-135
D.3.4	Pharmaceutical form	Capsule*
D.3.4.1	Specific paediatric formulation	Information not present in EudraCT
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NA
D.3.9.1	CAS number	9001-62-1
D.3.9.3	Other descriptive name	Lipase CLEC
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	32,500 U
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NA
D.3.9.1	CAS number	9000-90-2
D.3.9.3	Other descriptive name	Amylase
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	3,750 U
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NA
D.3.9.1	CAS number	9074-07-1
D.3.9.3	Other descriptive name	Protease
D.3.10	Strength
D.3.10.1	Concentration unit	U unit(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25,000 U
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	Information not present in EudraCT
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	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	Information not present in EudraCT
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	Information not present in EudraCT
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	Yes
D.3.11.13.1	Other medicinal product type	purified enzymes of microbial origin

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	Capsule*
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	9.1
E.1.2	Level	LLT
E.1.2	Classification code	10011766
E.1.2	Term	Cystic fibrosis pancreatic

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

To determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI.

Primary:
• Mean change in CFA from Baseline Period to Double-Blind Treatment Period.
Secondary:
• Mean change in CNA from Baseline Period to Double-Blind Treatment Period.
• Proportion of patients who have a maximum increase in blood glucose of ≥ 10 mg/dl in
the SCT (non-diabetic patients only) during the Double-Blind Treatment Period.
Mean changes in stool weight and frequency from Baseline Period to Double-Blind
Treatment Period.
Tertiary:
• Mean difference in maximum change in blood glucose level following starch challenge
from Baseline Period to Double-Blind Treatment Period.
• Mean percent change in CFA and CNA from Baseline Period to Double-Blind Treatment
Period.
• Mean relative improvement in CFA and CNA from Baseline Period to Double-Blind
Treatment Period.

E.2.2

Secondary objectives of the trial

• To determine the efficacy of ALTU-135 for the treatment of protein malabsorption in
patients with CF-related exocrine PI.
• To determine the efficacy of ALTU-135 treatment in improving the absorption of
carbohydrates in patients with CF-related exocrine PI.
• To determine the efficacy of ALTU-135 treatment in decreasing stool weight and
frequency in patients with CF-related exocrine PI.
Safety Endpoints:
• Safety endpoints include frequency and severity of treatment-emergent AEs; mean
changes in clinical laboratory parameters and vital signs; toxicity shifts in LFT
parameters; proportion of patients with abnormal clinical laboratory and vital signs
results.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Each patient must meet the following criteria to be enrolled in this study.
1. ≥ 7 years of age.
2. Females of childbearing potential must be willing to use birth control (IUD; oral,
transdermal or parenteral contraceptives; abstinence).
3. Diagnosis of CF based upon the following criteria:
a. two clinical features consistent with CF; and
b. either genotype with two identifiable mutations consistent with CF,
c. or sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis.
4. Clinically stable with no evidence of acute upper or lower respiratory tract infection.
5. PI determined by fecal elastase < 100 µg/g stool measured at the Screening Visit.
6. Able to take pancreatic enzyme supplementation in the form of capsules.
7. Able to perform the testing (e.g., stool collections) and inpatient stays required for this
study, as judged by the Investigator.
8. Willing and able to provide informed consent or assent.
9. Baseline CFA ≤ 80%.

E.4

Principal exclusion criteria

Patients who meet any of the following criteria will be excluded from the study.
1. < 7 years of age.
2. CFA > 80% at Baseline.
3. Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence) during the study.
4. History of fibrosing colonopathy.
5. History of liver transplant, lung transplant or significant surgical resection of the bowel. Note: Significant resection of the bowel is defined as any resection of the terminal ileum, or ileo-cecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (e.g., increased, or new, need for supplementation compared to pre-op in order to maintain the same nutritional status) should also be excluded.
6. Any acute or chronic diarrheal illness unrelated to PI (e.g. infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease).
7. Unable to discontinue enteral tube feedings during the study.
8. Known hypersensitivity to food additives.
9. Inability to consume the PP diets, in the judgment of the Investigator.
10. Participation in an investigational study of a drug, biologic, or device not currently
approved for marketing within 30 days prior to the Screening Visit.
11. ALT or AST level > 5x ULN, or total bilirubin level > 1.5x ULN at the Screening Visit
or at Baseline (except for patients with Gilbert Syndrome).
12. Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF‡.
13. DIOS in the last six months prior to the Screening Visit.
14. Unable to discontinue the use of pancreatic enzymes.
15. Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the patient.
16. Patient is unlikely to complete the study, as determined by the Investigator.

E.5 End points

E.5.1

Primary end point(s)

Mean change in CFA from Baseline Period to Double-Blind Treatment Period.
to determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI.

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

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

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

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.8.3

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

Information not present in EudraCT

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

E.8.7

Trial has a data monitoring committee

Yes

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

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

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

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)

Yes

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.2

Adults (18-64 years)

Yes

F.1.3

Elderly (>=65 years)

Yes

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

children over 7 years age
Patients willing and able to provide assent

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

176

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

2007-07-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2007-07-11

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2008-06-12

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