Clinical Trials Register

Summary
EudraCT Number:	2015-004794-33
Sponsor's Protocol Code Number:	PA101B-UP-02
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2016-01-13
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

Poland - Office for Medicinal Products

A.2

EudraCT number

2015-004794-33

A.3

Full title of the trial

Treatment of Uremic Pruritus with Inhaled PA101B in Patients with End-Stage Renal Disease Requiring Hemodialysis

A.3.1

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

A clinical trial of inhaled PA101B in treating uremic pruritus in hemodialysis patients with chronic kidney failure

A.4.1

Sponsor's protocol code number

PA101B-UP-02

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	Patara Pharma, LLC
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	Patara Pharma, LLC
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Patara Pharma, LLC
B.5.2	Functional name of contact point	Rod Saponjic
B.5.3	Address:
B.5.3.1	Street Address	11455 El Camino Real, Suite 460
B.5.3.2	Town/ city	San Diego
B.5.3.3	Post code	92130
B.5.3.4	Country	United States
B.5.4	Telephone number	+1858 436 1618
B.5.5	Fax number	+1858 436 1605
B.5.6	E-mail	rsaponjic@patarapharma.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	No
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.3.1	Product name	Disodium Cromoglycate
D.3.2	Product code	PA101B
D.3.4	Pharmaceutical form	Nebuliser solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	not available
D.3.9.1	CAS number	15826-37-6
D.3.9.2	Current sponsor code	PA101B
D.3.9.3	Other descriptive name	SODIUM CROMOGLICATE, CROMOLYN SODIUM
D.3.9.4	EV Substance Code	SUB12286MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
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.IMP: 2
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Disodium Cromoglycate
D.3.2	Product code	PA101B
D.3.4	Pharmaceutical form	Nebuliser solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	not available
D.3.9.1	CAS number	15826-37-6
D.3.9.2	Current sponsor code	PA101B
D.3.9.3	Other descriptive name	SODIUM CROMOGLICATE, CROMOLYN SODIUM
D.3.9.4	EV Substance Code	SUB12286MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
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.IMP: 3
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Disodium Cromoglycate
D.3.2	Product code	PA101B
D.3.4	Pharmaceutical form	Nebuliser solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Inhalation use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	not available
D.3.9.1	CAS number	15826-37-6
D.3.9.2	Current sponsor code	PA101B
D.3.9.3	Other descriptive name	SODIUM CROMOGLICATE, CROMOLYN SODIUM
D.3.9.4	EV Substance Code	SUB12286MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	40
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	Nebuliser solution
D.8.4	Route of administration of the placebo	Inhalation use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Uremic Pruritus in patients with end-stage renal disease requiring hemodialysis

E.1.1.1

Medical condition in easily understood language

Uremic pruritus in patients with renal disease requiring hemodialysis

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	18.1
E.1.2	Level	LLT
E.1.2	Classification code	10060884
E.1.2	Term	Uremic pruritus
E.1.2	System Organ Class	100000004858

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 assess the effectiveness of inhaled PA101B delivered via eFlow high efficiency nebulizer in treating uremic pruritus in patients with end-stage renal disease (ESRD) requiring hemodialysis. Improvements in pruritus will be assessed by measuring the change from baseline in worst itching intensity using a numerical rating scale (NRS) as a patient-reported outcome measure
• To evaluate the safety and tolerability of inhaled PA101B in patients with ESRD requiring hemodialysis
• To assess biomarker levels prior to and following PA101B administration

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female patients age 18 through 80 years, inclusive.
2. Diagnosis of end-stage renal disease (ESRD) receiving hemodialysis for at least 3 months prior to the Screening Period
3. Able to receive conventional hemodialysis (i.e., not hemofiltration or hemodiafiltration) during the study
4. Pruritus present for at least 6 weeks
5. Mean pruritus severity score on a numerical rating scale (NRS) > 4 from the Screening Period (based on a minimum of 8 scores)
6. Patient-Assessed Disease Severity Scale Type B or C at the Screening Period
7. If receiving H1 antihistamines for pruritus treatment, the drug and dose has been stable during the 2 weeks prior to Screening and is expected to remain stable throughout the study
8. Hemoglobin > 8 g/dL at the Screening Period
9. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <2 X the upper limit of normal (ULN) at the Screening Period
10. Documentation of a urea reduction ratio (URR) >65% or single-pooled Kt/V >1.4 during the Screening Period
11. Sufficient manual dexterity to use the hand-held nebulizer or a caregiver who can reliably assist with assembly and use of the nebulizer
12. Adequate venous access for blood drawing, unless the dialysis access will be used for this purpose
13. Willingness and ability to provide written informed consent

E.4

Principal exclusion criteria

1. Current or recent history of clinically significant medical condition, laboratory abnormality, or illness that could put the patient at risk or compromise the quality of the study data as determined by the investigator
2. Myocardial infarction within 6 months or unstable angina, acute coronary syndrome, or interventional coronary procedure within 2 months prior to the Screening Visit
3. An upper or lower respiratory tract infection (including sinus infection) within 4 weeks prior to the Screening Visit
4. Severely symptomatic cardiopulmonary disease defined by the use of home oxygen treatment, dyspnea at rest or with minimal exertion, uncontrolled arrhythmias (e.g. atrial fibrillation with inadequate rate control), or history of life-threatening arrhythmias (e.g. cardiac arrest or syncope related to arrhythmia)
5. Acute exacerbation of asthma or chronic obstructive pulmonary disease resulting in hospitalization or visit to an emergency department or urgent care clinic within 6 months of the Screening Visit
6. Hospitalization for any medical reason other than for a pre-planned procedure or dialysis access related procedure within the 2 weeks prior to the Baseline Visit
7. Known malignancy for which the patient is receiving active treatment with a systemic drug
8. Participation in any other investigation drug study within 4 weeks prior to the Screening Visit
9. Use of cromolyn sodium within 2 weeks of the Screening Visit
10. Current (i.e., within 2 weeks prior to Screening) or anticipated use of baclofen, gabapentin, pregabalin and nalbuphine for the treatment of pruritus during the study (Note: Use of these medications for indications other than pruritus is permitted if the dose has been stable during the 2 weeks prior to Screening and is anticipated to remain stable during the study)
11. Current (i.e., within 2 weeks prior to Screening) or anticipated use during the study of glucocorticoids administered intravenous, oral, or transdermally (Note: Use of inhaled, intranasal, intrarticular, otic, and ophthalmic glucocorticoids is permitted during the study)
12. Females who are pregnant or breastfeeding, or if of child-bearing potential unwilling to practice acceptable means of birth control or abstinence during the study (e.g., abstinence, combination barrier and spermicide, or hormonal)
13. History of hypersensitivity or intolerance to cromolyn sodium
14. Anticipated changes in the dialysis regimen or modality during the trial (e.g., change in type of filter, increase or decrease in prescribed dialysis duration by 1 hour, change or in prescribed blood flow by >100 mL/minute that is expected to significantly affect dialysis clearance as determined by the investigator) or any such changes during the 2 weeks prior to the Screening Period

E.5 End points

E.5.1

Primary end point(s)

The change from Baseline (i.e., mean NRS of all available scores from the Placebo Run-in Period) in severity of pruritus at Week 7 (i.e., mean of all available NRS scores during Weeks 6 – 7)

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 7

E.5.2

Secondary end point(s)

The change from Baseline (i.e., the Placebo Run-in Period) in itch-specific quality of life (the total Skindex-10) score at Week 7 (i.e., mean score during Weeks 6 – 7)

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 7

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

Yes

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

proof-of-concept

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

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

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

United States

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

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

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.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

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)

not different from the expected normal treatment of that condition

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

2016-03-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2016-01-19

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2016-06-22

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