Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

A Phase 1/2 study testing QR-010 in people with Cystic Fibrosis, run by ProQR Therapeutics at 27 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis. Read more about this study

Who is it for?: Ages 18 to 60 years
Conditions the study is for: Cystic Fibrosis
What is being tested?: QR-010
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 70 participants
Where: 27 sitesin 10 countries
Study dates: Jun 2015to 14 Sep 2017
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 60 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of > 60 mmol/L
Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation
Body mass index (BMI) ≥ 17 kg/m2
Non-smoking for a minimum of two years
FEV1 ≥70% of predicted normal for age, gender, and height, at Screening
Stable lung function
Adequate hepatic and renal function

Who cannot join

Breast-feeding or pregnant
Use of lumacaftor or ivacaftor
Use of any investigational drug or device
History of lung transplantation
Hemoptysis

Trial locations and contacts

27 sites in 10 countries.

Country

City, postcode or hospital

27 sites in 10 countries

United States12 sites

Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 66160
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Kansas Medical Center Research Institute
Kansas City, Kansas, 66160
University of Southern California USC - Keck School of Medicine
Los Angeles, California, 90033
Stanford University
Palo Alto, California, 940304
University of Washington Medical Center
Seattle, Washington, 98195-6522
Washington University School of Medicine
St Louis, Missouri, 63110-1032

Other trials in United States

Germany3 sites

Charité Universitätsmedizin Berlin
Berlin, 13353
Medizinische Hochschule Hannover
Hanover, 30625
Munich U. Hospital, Cystic Fibrosis Center for Adults
Munich, 80336

Other trials in Germany

United Kingdom3 sites

Celerion
Belfast, Northern Ireland, BT9 6AD
Royal Brompton Hospital
London, SW3 6NP
Southampton General Hospital
Southampton, SO16 6YD

Other trials in United Kingdom

Belgium2 sites

Universitair Ziekenhuis Brussel
Brussels, 01090
University of Leuven
Leuven, 03000

Other trials in Belgium

France2 sites

HGRL Chu Nantes
Nantes, 44300
Hopital Necker- Enfants Malades
Paris, 75743

Other trials in France

Canada1 site

University of Calgary (Health Sciences Centre)
Calgary, Alberta, T2N 4N1

Other trials in Canada

Czechia1 site

Motol University Hospital
Prague, 15006

Other trials in Czechia

Denmark1 site

Cystic Fibrosis Center Rigshospitalet
Copenhagen, 02100

Other trials in Denmark

Italy1 site

Azienda Ospedaliera Universitaria Integrata di Verona
Verona, 37134

Other trials in Italy

Spain1 site

Hospital Vall D'Hebron
Barcelona, 08035

Other trials in Spain

About this study

Official title: Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis

A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

Read the detailed description

The purpose of this study is to evaluate the safety, tolerability, and to determine the pharmacokinetics of QR-010 administered via inhalation in adult homozygous for ΔF508 Cystic Fibrosis.

Conditions: Cystic Fibrosis
Being tested: QR-010, Placebo
Keywords: cystic fibrosis, ΔF508, RNA therapies, antisense oligonucleotide, CFTR, F508del, CF, RNA therapy
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

QR-010Drug
Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
PlaceboDrug
Normal Saline

Study groups in NCT02532764
Study group	Role	What they receive
QR-010	Experimental	QR-010 administered via inhalation either as a single dose or three times weekly for four weeks. Receives: Drug: QR-010
Placebo	Placebo comparator	Placebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study
Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Assessment of severity of treatment emergent adverse events (TEAEs).
Severity is graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Modified for CF (CTCAE v4.03). For events not present in this listing the following grading was applied:
Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Moderate: Minimal, local, or noninvasive intervention indicated; discomfort sufficient to reduce or interfere with daily activities; Severe: Medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization may be indicated; disabling; limits self-care with significant interference with daily activities; incapacitating with inability to perform self care activities of daily living; Life-threatening: Urgent intervention indicated; immediate risk of death.
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.
DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Secondary outcomes (7)

Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
Number of subjects experiencing at least one abnormality for the categories laboratory parameters, vital signs, ECG, spirometry and physical findings that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely.
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Maximum Serum Concentration
Cmax: QR-010 maximum serum concentrations
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Time to Maximum Serum Concentration
Tmax: Time to Cmax of QR-010 serum concentrations.
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Terminal Half-life (T1/2)
The terminal elimination half-life will be estimated by non-linear regression analysis of the terminal elimination slope
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Area Under the Curve to Final Sample [AUC(0-last)]
Area under the curve to the final sample with a concentration greater than lower limit of quantification (LLQ) will be calculated using the linear trapezoidal method
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Area Under the Curve to Infinity [AUC(0-∞)]
AUC0-∞: Area under the curve to infinity will be calculated based on the last observed concentration Clast(obs) using formula: AUC0-∞=AUClast+Clast(obs)/λz
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Serum Clearance (CL)
CL: Serum clearance will be estimated using the formula: CL = Dose/AUC0-∞.
Measured: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 201514 Sep 2017

First posted26 Aug 2015 (estimated)
Study startJun 2015
Primary completion14 Sep 2017
Study completion14 Sep 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Single group
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 70 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02532764
Sponsor's protocol ID: PQ-010-001
Status last verified: Jan 2019
First submitted: 13 Aug 2015
Last updated: 6 Feb 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
PubMed 31215818

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Active, not recruitingObservational study
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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis
ClinicalTrials.gov ID: NCT02532764
Acronym / sponsor ID: PQ-010-001
Conditions: Cystic Fibrosis
Keywords: cystic fibrosis, ΔF508, RNA therapies, antisense oligonucleotide, CFTR, F508del, CF, RNA therapy
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 70 participants (actual)
Lead sponsor: ProQR TherapeuticsIndustry (pharma, biotech, devices)
Collaborators: European Commission
Study start: Jun 2015
Primary completion: 14 Sep 2017 (actual)
Study completion: 14 Sep 2017 (actual)
First posted: 26 Aug 2015 (estimated)
Results: Posted 6 Feb 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

Detailed description

The purpose of this study is to evaluate the safety, tolerability, and to determine the pharmacokinetics of QR-010 administered via inhalation in adult homozygous for ΔF508 Cystic Fibrosis.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Single group
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
QR-010	Experimental	Drug: QR-010QR-010 administered via inhalation either as a single dose or three times weekly for four weeks.
Placebo	Placebo comparator	Drug: PlaceboPlacebo (normal saline) administered via inhalation either as a single dose or three times weekly for four weeks.

Who may qualify

Ages: 18 to 60 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (7 listed)

Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of > 60 mmol/L
Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation
Body mass index (BMI) ≥ 17 kg/m2
Non-smoking for a minimum of two years
FEV1 ≥70% of predicted normal for age, gender, and height, at Screening

Key exclusion criteria (5 listed)

Breast-feeding or pregnant
Use of lumacaftor or ivacaftor
Use of any investigational drug or device
History of lung transplantation
Hemoptysis

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
QR-010Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton	Drug	QR-010
PlaceboNormal Saline	Drug	Placebo

Primary outcome

Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Assessment of severity of treatment emergent adverse events (TEAEs). Severity is graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Modified for CF (CTCAE v4.03). For events not present in this listing the following grading was applied: Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Moderate: Minimal, local, or noninvasive intervention indicated; discomfort sufficient to reduce or interfere with daily activities; Severe: Medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization may be indicated; disabling; limits self-care with significant interference with daily activities; incapacitating with inability to perform self care activities of daily living; Life-threatening: Urgent intervention indicated; immediate risk of death.
Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.

7 secondary outcomes

Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Maximum Serum Concentration
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Time to Maximum Serum Concentration
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Terminal Half-life (T1/2)
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Area Under the Curve to Final Sample [AUC(0-last)]
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Area Under the Curve to Infinity [AUC(0-∞)]
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Serum Clearance (CL)
Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Results and safety

Results posted to the registry on 6 Feb 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
QR-010 SAD 6.25mg	6	6
QR-010 SAD 12.5 mg	6	6
QR-010 SAD 25 mg	9	9
QR-010 SAD 50 mg	6	6
Placebo SAD	9	9
QR-010 MAD 6.25	0	0
QR-010 MAD 12.5 mg	0	0
QR-010 MAD 25 mg	0	0
QR-010 MAD 50 mg	0	0
Placebo MAD	0	0

Primary outcome: Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts. Count of participants. Unit: Participants

Measure	QR-010 SAD 6.25 mg n = 6	QR-010 SAD 12.5 mg n = 6	QR-010 SAD 25 mg n = 9	QR-010 SAD 50 mg n = 6	Placebo SAD n = 9	QR-010 MAD 6.25 mg n = 6	QR-010 MAD 12.5 mg n = 6	QR-010 MAD 25 mg n = 7	QR-010 MAD 50 mg n = 6	Placebo MAD n = 9
Value	3	2	7	4	2	5	5	6	5	9

Population analysed: All subjects who received either QR-010 or placebo

Primary outcome: Severity of Treatment Emergent Adverse Events From Baseline Through End of Study

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts. Count of participants. Unit: Participants

Measure	QR-010 SAD 6.25 mg n = 6	QR-010 SAD 12.5 mg n = 6	QR-010 SAD 25 mg n = 9	QR-010 SAD 50 mg n = 6	Placebo SAD n = 9	QR-010 MAD 6.25 mg n = 6	QR-010 MAD 12.5 mg n = 6	QR-010 MAD 25 mg n = 7	QR-010 MAD 50 mg n = 6	Placebo MAD n = 9
Mild	3	2	5	4	2	5	4	5	1	5
Moderate	0	0	2	0	0	0	1	0	4	3
Severe	0	0	0	0	0	0	0	1	0	1
Life-Threatening	0	0	0	0	0	0	0	0	0	0
Death	0	0	0	0	0	0	0	0	0	0

Population analysed: All subjects who received either QR-010 or placebo.

Primary outcome: Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts. Count of participants. Unit: Participants

Measure	QR-010 SAD 6.25 mg n = 6	QR-010 SAD 12.5 mg n = 6	QR-010 SAD 25 mg n = 9	QR-010 SAD 50 mg n = 6	Placebo SAD n = 9	QR-010 MAD 6.25 mg n = 6	QR-010 MAD 12.5 mg n = 6	QR-010 MAD 25 mg n = 7	QR-010 MAD 50 mg n = 6	Placebo MAD n = 9
Value	0	0	0	0	0	0	0	0	0	0

Population analysed: All subjects who received either QR-010 or placebo. There were no Dose Limiting Toxicities (DLTs) reported in the SAD or MAD cohorts.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
QR-010 SAD 6.25 mg	0 of 6 (0%)	3 of 6 (50%)	0 of 6
QR-010 SAD 12.5 mg	0 of 6 (0%)	2 of 6 (33.3%)	0 of 6
QR-010 SAD 25 mg	0 of 9 (0%)	7 of 9 (77.8%)	0 of 9
QR-010 SAD 50 mg	0 of 6 (0%)	4 of 6 (66.7%)	0 of 6
Placebo SAD	0 of 9 (0%)	2 of 9 (22.2%)	0 of 9
QR-010 MAD 6.25 mg	0 of 6 (0%)	5 of 6 (83.3%)	0 of 6
QR-010 MAD 12.5 mg	0 of 6 (0%)	5 of 6 (83.3%)	0 of 6
QR-010 MAD 25 mg	0 of 7 (0%)	6 of 7 (85.7%)	0 of 7
QR-010 MAD 50 mg	1 of 6 (16.7%)	5 of 6 (83.3%)	0 of 6
Placebo MAD	1 of 9 (11.1%)	9 of 9 (100%)	0 of 9

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 31215818Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Stuart Elborn, MD principal investigator

Trust and Queen's University Belfast

27 sites in 10 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing QR-010 in people with Cystic Fibrosis, run by ProQR Therapeutics at 27 sites in 10 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 6 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 27 sites in United States, Germany, United Kingdom, Belgium, France and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Feb 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study (time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02532764, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2019.