Paroxysmal Nocturnal Hemoglobinuria (PNH)Phase 1/2NCT02352493

A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH

A Phase 1/2 study testing ALN-CC5 and Sterile Normal Saline (0.9% NaCl) in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Alnylam Pharmaceuticals at 4 sites in 2 countries.

Completed

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

Official record

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Trial summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH Read more about this study

Who is it for?: Ages 18 to 45 yearsHealthy volunteers can take part
Conditions the study is for: Paroxysmal Nocturnal Hemoglobinuria (PNH)
What is being tested?: ALN-CC5 and Sterile Normal Saline (0.9% NaCl)
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 62 participants
Where: 4 sitesin 2 countries
Study dates: Jan 2015to Aug 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 45 years
All sexes
Healthy volunteers accepted
Adult

Who may be able to join

Adequate complete blood counts, liver and renal function
12-lead electrocardiogram (ECG) within normal limits
Female subjects of child bearing potential agreeing to use a protocol specified method of contraception
Male subjects agreeing to use protocol specified methods of contraception
Willing to provide written informed consent and willing to comply with study requirements

Who cannot join

Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
History of multiple drug allergies or intolerance to subcutaneous injection
Parts A and B of the study: Used prescription medications within 14 days or 7 half-lives of administration of the first dose of study drug.
History of meningococcal infection

Trial locations and contacts

4 sites in 2 countries.

Country

City, postcode or hospital

4 sites in 2 countries

United Kingdom3 sites

Clinical Trial Site
Leeds
Covance Clinical Research Unit
Leeds
Richmond Pharmacology
London

Other trials in United Kingdom

Spain1 site

Clinical Trial Site
Barcelona

Other trials in Spain

About this study

Official title: A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Being tested: ALN-CC5, Sterile Normal Saline (0.9% NaCl)
Keywords: PNH, RNAi therapeutic
Medical subject headings: Hemoglobinuria, Paroxysmal

What participants receive

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

ALN-CC5Drug
Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection
Sterile Normal Saline (0.9% NaCl)Drug
calculated volume to match active comparator

Study groups in NCT02352493
Study group	Role	What they receive
ALN-CC5	Active comparator	Not described Receives: Drug: ALN-CC5
Sterile Normal Saline (0.9% NaCl)	Placebo comparator	Not described Receives: Drug: Sterile Normal Saline (0.9% NaCl)

What is being measured

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

Number of Participants With Adverse Events
Adverse events were reported for single-ascending doses (SAD) or multiple ascending doses (MAD) of ALN-CC5 when administered to healthy adult subjects and of multiple doses (MD) in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Measured: Part A: through day 658; Part B: through day 532; Part C: through day 280

Secondary outcomes (9)

Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP)
Complement activity was measured in serum samples collected at timepoints throughout the study using the CAP ELISA assay. Percentage reduction was calculated relative to baseline levels. A positive value indicates a reduction in CAP from baseline.
Measured: Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP)
Complement activity was measured in serum samples collected at time points throughout the study using the CCP ELISA assay. Percentage reduction was calculated relative to baseline levels. A positive value indicates a reduction in CCP from baseline.
Measured: Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels
Total C5 protein levels were measured in serum samples collected at time points throughout the study using a mass spectrometry-based method. Percentage reduction was calculated relative to baseline levels. A positive value indicates a reduction in C5 protein level from baseline.
Measured: Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer)
Maximum observed plasma concentration (Cmax) of ALN-CC5 (cemdisiran) 25-mer.
Measured: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer)
Maximum observed plasma concentration (Cmax) of ALN-CC5 (cemdisiran) 23-mer.
Measured: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer)
Time of maximum observed plasma concentration (T max) of ALN-CC5 (cemdisiran) 25-mer.
Measured: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer)
Time of maximum observed plasma concentration (T max) of ALN-CC5 (cemdisiran) 23-mer.
Measured: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer)
Area under the plasma concentration-time curve over the dosing interval zero to time (AUC 0-t) of ALN-CC5 (cemdisiran) 25-mer.
Measured: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer)
Area under the plasma concentration-time curve over the dosing interval zero to time (AUC 0-t) of ALN-CC5 (cemdisiran) 23-mer.
Measured: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84

Timeline

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

Jan 2015Aug 2017

First posted2 Feb 2015 (estimated)
Study startJan 2015
Primary completionApr 2016
Study completionAug 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 (2)Papers the sponsor has linked to this study.
Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)Studies of the same condition, open and under way.
More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
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: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 62 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02352493
Sponsor's protocol ID: ALN-CC5-001
Status last verified: Mar 2020
First submitted: 23 Jan 2015
Last updated: 30 Mar 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Ishigooka H, Katsumata H, Saiga K, Tokita D, Motoi S, Matsui C, Suzuki Y, Tomimatsu A, Nakatani T, Kuboi Y, Yamakawa T, Ikeda T, Ishii R, Imai T, Takagi T, Tanabe K. Novel Complement C5 Small-interfering RNA Lipid Nanoparticle Prolongs Graft Survival in a Hypersensitized Rat Kidney Transplant Model. Transplantation. 2022 Dec 1;106(12):2338-2347. doi: 10.1097/TP.0000000000004207. Epub 2022 Nov 22.
PubMed 35749284
Badri P, Jiang X, Borodovsky A, Najafian N, Kim J, Clausen VA, Goel V, Habtemariam B, Robbie GJ. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021 Mar;60(3):365-378. doi: 10.1007/s40262-020-00940-9.
PubMed 33047216

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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RecruitingObservational study
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Not yet recruitingPhase 3
A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
Paroxysmal Nocturnal Hemoglobinuria, PNH
Not yet recruitingPhase 2
Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria
PNH - Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 1
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Paroxysmal Nocturnal Hemoglobinuria, PNH

All Paroxysmal Nocturnal Hemoglobinuria (PNH) trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria
ClinicalTrials.gov ID: NCT02352493
Acronym / sponsor ID: ALN-CC5-001
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Keywords: PNH, RNAi therapeutic
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 62 participants (actual)
Lead sponsor: Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Jan 2015
Primary completion: Apr 2016 (actual)
Study completion: Aug 2017 (actual)
First posted: 2 Feb 2015 (estimated)
Results: Posted 30 Mar 2020

Clinical summary

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

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
ALN-CC5	Active comparator	Drug: ALN-CC5
Sterile Normal Saline (0.9% NaCl)	Placebo comparator	Drug: Sterile Normal Saline (0.9% NaCl)

Who may qualify

Ages: 18 to 45 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult

Key inclusion criteria (5 listed)

Adequate complete blood counts, liver and renal function
12-lead electrocardiogram (ECG) within normal limits
Female subjects of child bearing potential agreeing to use a protocol specified method of contraception
Male subjects agreeing to use protocol specified methods of contraception
Willing to provide written informed consent and willing to comply with study requirements

Key exclusion criteria (5 listed)

Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
History of multiple drug allergies or intolerance to subcutaneous injection
Parts A and B of the study: Used prescription medications within 14 days or 7 half-lives of administration of the first dose of study drug.
History of meningococcal infection

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
ALN-CC5Single or multiple doses of ALN-CC5 by subcutaneous (sc) injection	Drug	ALN-CC5
Sterile Normal Saline (0.9% NaCl)calculated volume to match active comparator	Drug	Sterile Normal Saline (0.9% NaCl)

Primary outcome

Number of Participants With Adverse Events
Time frame: Part A: through day 658; Part B: through day 532; Part C: through day 280
Adverse events were reported for single-ascending doses (SAD) or multiple ascending doses (MAD) of ALN-CC5 when administered to healthy adult subjects and of multiple doses (MD) in patients with paroxysmal nocturnal hemoglobinuria (PNH)

9 secondary outcomes

Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP)
Time frame: Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP)
Time frame: Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels
Time frame: Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer)
Time frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer)
Time frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer)
Time frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer)
Time frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer)
Time frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84
Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer)
Time frame: Part A: 0-48 hrs, Day 0; Part B (weekly dosing cohorts): 0-48 hrs, Day 28; Part B (biweekly, weekly/monthly/biweekly/monthly cohorts): 0-48 hrs, Day 84; Part C: 0- 24 hrs, Day 84

Results and safety

Results posted to the registry on 30 Mar 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo - Single Ascending Dose	8	8
ALN-CC5 50mg - Single Ascending Dose	3	3
ALN-CC5 50mg (Japanese) - Single Ascending Dose	3	3
ALN-CC5 200mg - Single Ascending Dose	3	3
ALN-CC5 200mg (Japanese) - Single Ascending Dose	3	3
ALN-CC5 400mg - Single Ascending Dose	3	3
ALN-CC5 600mg - Single Ascending Dose	3	2
ALN-CC5 600mg (Japanese) - Single Ascending Dose	3	3
ALN-CC5 900mg - Single Ascending Dose	3	1
Placebo - Multiple Ascending Dose	6	5
ALN-CC5 100mg Weekly - Multiple Ascending Dose	3	3
ALN-CC5 200mg Weekly - Multiple Ascending Dose	3	3
ALN-CC5 400mg Weekly - Multiple Ascending Dose	3	2
ALN-CC5 600mg Biweekly - Multiple Ascending Dose	3	1
ALN-CC5 200mg Weekly/Biweekly - Multiple Ascending Dose	3	2
ALN-CC5 200mg Weekly/Monthly - Multiple Ascending Dose	3	3
ALN-CC5 Multiple Dose - Eculizumab Treated	3	3
ALN-CC5 Multiple Dose - Eculizumab Naive	3	3

Primary outcome: Number of Participants With Adverse Events

Time frame: Part A: through day 658; Part B: through day 532; Part C: through day 280. Count of participants. Unit: Participants

Measure	Placebo - Single Ascending Dose n = 8	ALN-CC5 50mg (Japanese) - Single Ascending Dose n = 3	ALN-CC5 200mg - Single Ascending Dose n = 3	ALN-CC5 200mg (Japanese) - Single Ascending Dose n = 3	ALN-CC5 400mg - Single Ascending Dose n = 3	ALN-CC5 600mg - Single Ascending Dose n = 3	ALN-CC5 600mg (Japanese) - Single Ascending Dose n = 3	ALN-CC5 900mg - Single Ascending Dose n = 3	Placebo - Multiple Ascending Dose n = 6	ALN-CC5 100mg Weekly - Multiple Ascending Dose n = 3	ALN-CC5 200mg Weekly - Multiple Ascending Dose n = 3	ALN-CC5 400mg Weekly - Multiple Ascending Dose n = 3	ALN-CC5 600mg Biweekly - Multiple Ascending Dose n = 3	ALN-CC5 200mg Weekly/Biweekly - Multiple Ascending Dose n = 3	ALN-CC5 200mg Weekly/Monthly - Multiple Ascending Dose n = 3	ALN-CC5 Multiple Dose - Eculizumab Treated n = 3	ALN-CC5 Multiple Dose - Eculizumab Naive n = 3
At least 1 Treatment Emergent Adverse Event (TEAE)	6	3	2	2	3	3	2	3	6	2	3	3	3	2	3	3	3
At least 1 Serious Adverse Event (SAE)	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
At least 1 TEAE leading to discontinuation	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: Part A: through day 658; Part B: through day 532; Part C: through day 280. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo - Single Ascending Dose	0 of 8 (0%)	6 of 8 (75%)	0 of 8
ALN-CC5 50mg - Single Ascending Dose	0 of 3 (0%)	0 of 3 (0%)	0 of 3
ALN-CC5 50mg (Japanese) - Single Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 200mg - Single Ascending Dose	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
ALN-CC5 200mg (Japanese) - Single Ascending Dose	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
ALN-CC5 400mg - Single Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 600mg - Single Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 600mg (Japanese) - Single Ascending Dose	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
ALN-CC5 900mg - Single Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Placebo - Multiple Ascending Dose	0 of 6 (0%)	6 of 6 (100%)	0 of 6
ALN-CC5 100mg Weekly - Multiple Ascending Dose	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
ALN-CC5 200mg Weekly - Multiple Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 400mg Weekly - Multiple Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 600mg Biweekly - Multiple Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 200mg Weekly/Biweekly - Multiple Ascending Dose	0 of 3 (0%)	2 of 3 (66.7%)	0 of 3
ALN-CC5 200mg Weekly/Monthly - Multiple Ascending Dose	0 of 3 (0%)	3 of 3 (100%)	0 of 3
ALN-CC5 Multiple Dose - Eculizumab Treated	0 of 3 (0%)	3 of 3 (100%)	0 of 3

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 35749284Ishigooka H, Katsumata H, Saiga K, Tokita D, Motoi S, Matsui C, Suzuki Y, Tomimatsu A, Nakatani T, Kuboi Y, Yamakawa T, Ikeda T, Ishii R, Imai T, Takagi T, Tanabe K. Novel Complement C5 Small-interfering RNA Lipid Nanoparticle Prolongs Graft Survival in a Hypersensitized Rat Kidney Transplant Model. Transplantation. 2022 Dec 1;106(12):2338-2347. doi: 10.1097/TP.0000000000004207. Epub 2022 Nov 22.
Derived from this study: PubMed 33047216Badri P, Jiang X, Borodovsky A, Najafian N, Kim J, Clausen VA, Goel V, Habtemariam B, Robbie GJ. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021 Mar;60(3):365-378. doi: 10.1007/s40262-020-00940-9.

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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.

Nader Najafian, MD study director

Alnylam Pharmaceuticals

4 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ALN-CC5 and Sterile Normal Saline (0.9% NaCl) in people with Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Alnylam Pharmaceuticals at 4 sites in 2 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 5 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 30 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United Kingdom, Spain. 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 30 Mar 2020). 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?

Number of Participants With Adverse Events (time frame: Part A: through day 658; Part B: through day 532; Part C: through day 280). 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 NCT02352493, which also lists 2 linked publications.

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 30 Mar 2020; AzTrials last refreshed its copy on 8 Nov 2026.