Trial · NCT02352493
A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
NCT02352493, Phase 1, Phase 2, Completed, Alnylam Pharmaceuticals; results first posted 2020-03-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH. Trials, record NCT02352493. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02352493
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
- NCT number
- NCT02352493
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 62 participants, actual
- Lead sponsor
- Alnylam Pharmaceuticals
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- ALN-CC5, Sterile Normal Saline (0.9% NaCl)
- First posted
- 2015-02-02
- Last updated
- 2020-03-30
- Results first posted
- 2020-03-30
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events | 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) |
| Secondary | Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP) | Part A: through day 70; Part B: through day 140; Part C: through day 140 | 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. |
| Secondary | Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP) | Part A: through day 70; Part B: through day 140; Part C: through day 140 | 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. |
| Secondary | Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels | Part A: through day 70; Part B: through day 140; Part C: through day 140 | 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. |
| Secondary | Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer) | 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 | Maximum observed plasma concentration (Cmax) of ALN-CC5 (cemdisiran) 25-mer. |
| Secondary | Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer) | 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 | Maximum observed plasma concentration (Cmax) of ALN-CC5 (cemdisiran) 23-mer. |
| Secondary | Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer) | 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 | Time of maximum observed plasma concentration (T max) of ALN-CC5 (cemdisiran) 25-mer. |
| Secondary | Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer) | 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 | Time of maximum observed plasma concentration (T max) of ALN-CC5 (cemdisiran) 23-mer. |
| Secondary | Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer) | 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 | Area under the plasma concentration-time curve over the dosing interval zero to time (AUC 0-t) of ALN-CC5 (cemdisiran) 25-mer. |
| Secondary | Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer) | 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 | Area under the plasma concentration-time curve over the dosing interval zero to time (AUC 0-t) of ALN-CC5 (cemdisiran) 23-mer. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events | Part A: through day 658; Part B: through day 532; Part C: through day 280 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP) | Part A: through day 70; Part B: through day 140; Part C: through day 140 | percentage reduction | MEAN | |
| SECONDARY | Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP) | Part A: through day 70; Part B: through day 140; Part C: through day 140 | percentage reduction | MEAN | |
| SECONDARY | Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels | Part A: through day 70; Part B: through day 140; Part C: through day 140 | percentage reduction | MEAN | |
| SECONDARY | Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (25-mer) | 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 | All healthy volunteers/patients who received at least one dose of study drug, and who had evaluable plasma or urine PK concentration. For the ALN-CC5 Multiple Dose - Eculizumab Naive group, Day 84 plasma samples were not collected. | ng/mL | MEAN |
| SECONDARY | Pharmacokinetic (PK) Effect of ALN-CC5: Cmax (23-mer) | 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 | All healthy volunteers/patients who received at least one dose of study drug, and who had evaluable plasma or urine PK concentration. For the ALN-CC5 Multiple Dose - Eculizumab Naive group, Day 84 plasma samples were not collected. | ng/mL | MEAN |
| SECONDARY | Pharmacokinetic (PK) Effect of ALN-CC5: T Max (25-mer) | 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 | All healthy volunteers/patients who received at least one dose of study drug, and who had evaluable plasma or urine PK concentration. For the ALN-CC5 Multiple Dose - Eculizumab Naive group, Day 84 plasma samples were not collected. | hr | MEDIAN |
| SECONDARY | Pharmacokinetic (PK) Effect of ALN-CC5: T Max (23-mer) | 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 | All healthy volunteers/patients who received at least one dose of study drug, and who had evaluable plasma or urine PK concentration. For the ALN-CC5 Multiple Dose - Eculizumab Naive group, Day 84 plasma samples were not collected. | hr | MEDIAN |
| SECONDARY | Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (25-mer) | 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 | All healthy volunteers/patients who received at least one dose of study drug, and who had evaluable plasma or urine PK concentration. For the ALN-CC5 Multiple Dose - Eculizumab Naive group, Day 84 plasma samples were not collected. | h*ng/mL | MEAN |
| SECONDARY | Pharmacokinetic (PK) Effect of ALN-CC5: AUC 0-t (23-mer) | 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 | All healthy volunteers/patients who received at least one dose of study drug, and who had evaluable plasma or urine PK concentration. For the ALN-CC5 Multiple Dose - Eculizumab Naive group, Day 84 plasma samples were not collected. | h*ng/mL | MEAN |
Source ClinicalTrials.gov
Results by group198
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events | Placebo - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 6 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 50mg - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 0 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 50mg (Japanese) - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 3 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 200mg - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 2 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 200mg (Japanese) - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 2 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 400mg - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 3 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 600mg - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 3 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 600mg (Japanese) - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 2 | Participants | |
| Number of Participants With Adverse Events | ALN-CC5 900mg - Single Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 3 | Participants | |
| Number of Participants With Adverse Events | Placebo - Multiple Ascending Dose | At least 1 Treatment Emergent Adverse Event (TEAE) | 6 | Participants |
1 to 10 of 198Source ClinicalTrials.gov
See all 198 results by groupParticipant flow54
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo - Single Ascending Dose | 8 | |
| Overall Study | STARTED | ALN-CC5 50mg - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 50mg (Japanese) - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 200mg - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 200mg (Japanese) - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 400mg - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 600mg - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 600mg (Japanese) - Single Ascending Dose | 3 | |
| Overall Study | STARTED | ALN-CC5 900mg - Single Ascending Dose | 3 | |
| Overall Study | STARTED | Placebo - Multiple Ascending Dose | 6 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 participant flowAdverse events1,260
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Placebo - Single Ascending Dose | 0 | 8 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 50mg - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 50mg (Japanese) - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 200mg - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 200mg (Japanese) - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 400mg - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 600mg - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 600mg (Japanese) - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ALN-CC5 900mg - Single Ascending Dose | 0 | 3 | |
| other | Fibroadenoma of breast | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Placebo - Multiple Ascending Dose | 0 | 6 |
1 to 10 of 1,260Source ClinicalTrials.gov
See all 1,260 adverse eventsAdverse event totals54
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo - Single Ascending Dose | 0 | 8 |
| Total, all-cause mortality | ALN-CC5 50mg - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 50mg (Japanese) - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 200mg - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 200mg (Japanese) - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 400mg - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 600mg - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 600mg (Japanese) - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | ALN-CC5 900mg - Single Ascending Dose | 0 | 3 |
| Total, all-cause mortality | Placebo - Multiple Ascending Dose | 0 | 6 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02352493
GET https://conductscience.org/api/trials/v1/NCT02352493/results?page=2